Muscle tissue disorder

This phase 2 trial tests whether a single dose of LX2006 gene therapy can improve heart muscle thickening and damage in people with Friedreich ataxia, a genetic disorder that often leads to life-threatening cardiomyopathy. Participants aged 6 and older with confirmed heart enlarg…

This study is testing the meningococcal B vaccine (Bexsero) in children and young adults aged 2 to 25 who have autoimmune rheumatic diseases like juvenile arthritis or lupus. Researchers want to see if the vaccine triggers a strong immune response and if it is safe, especially fo…

This study tests the safety and immune response of the 20-valent pneumococcal vaccine (PCV20) in children, adolescents, and young adults aged 2-25 with autoimmune rheumatic diseases like juvenile arthritis. All 114 participants will receive one dose of PCV20, and researchers will…

This study tests a gene therapy called SAR446268 for people aged 10 to 55 with myotonic dystrophy type 1. The therapy is given once through an IV and aims to reduce harmful DMPK RNA and improve muscle function. The trial has two parts: first, finding the safest dose in a small gr…

This study looks at whether setting a pacemaker to a personalized rate of 75 beats per minute, instead of the usual 50-60, can improve heart function and blood flow to the brain in people with high blood pressure and a slow heart rate. About 70 patients aged 65 and older will be …

This study tests whether omega-3 fatty acid supplements from fish oil, combined with a healthy diet, can reduce disease activity in adults with dermatomyositis, a rare autoimmune disease causing muscle weakness and skin rashes. About 300 participants will take either fish oil cap…

This study follows 335 adults with severe eosinophilic asthma who are taking benralizumab. Researchers want to see if the drug helps patients achieve partial or complete clinical remission—meaning fewer symptoms, no need for steroid pills, and better lung function—over 12 to 24 m…

This early-phase trial is testing an injection called GT719 in 10 adults with moderate to severe autoimmune diseases that haven't responded to other treatments. The main goal is to check safety and find the right dose. Researchers will also look for signs that the therapy might h…

This study tests an oral drug called SAT-3247 in 51 boys aged 7 to 10 with Duchenne muscular dystrophy who can still walk. The goal is to find the best dose, check safety, and see if it helps muscle strength. Participants take the drug or a placebo for 12 weeks, and all continue …

This Phase 3 trial tests the drug benralizumab (Fasenra) in children aged 6 to 17 with two rare eosinophilic diseases: EGPA and HES. The study aims to see if the drug is safe, how it works in the body, and whether it can help control the disease. Fourteen children will receive in…

This trial tests an investigational medicine called ATR 1072 in adults with PRKAG2 syndrome, a genetic condition that causes abnormal heart muscle thickening and rhythm problems. The drug is designed to reduce the activity of the faulty PRKAG2 gene. Researchers are evaluating its…

This study tests a one-time treatment called CABA-201, made from a patient's own immune cells, for people with active inflammatory muscle diseases like dermatomyositis. The therapy aims to reset the immune system by targeting and removing certain immune cells. Researchers will ch…

This study tests a daily injection called elamipretide in 48 people with genetically confirmed Barth syndrome, a rare condition that causes muscle weakness and heart problems. Participants will receive either the drug or a placebo for 72 weeks. The main goal is to see if the drug…

This study tests a drug called ublituximab for people with a rare autoimmune disease that causes severe muscle weakness. The goal is to see if adding this drug to standard treatment improves muscle strength and function more than standard treatment alone. About 30 adults with ear…

Researchers are testing a combination of two drugs, doxecitine and doxribtimine, in adults with thymidine kinase 2 (TK2) deficiency, a rare genetic condition that causes muscle weakness and breathing problems. The study will enroll about 15 adults with moderate to severe disease.…

This phase 3 trial tests whether a new drug called ABP 938 works as well as the approved treatment Eylea HD for wet age-related macular degeneration, a leading cause of vision loss. About 304 people aged 50 and older with untreated wet AMD will receive injections of either drug i…

This study tests whether the drug nerandomilast can slow lung scarring in people with autoimmune diseases like rheumatoid arthritis or scleroderma. About 400 adults whose lung function hasn't improved with standard treatment will take either nerandomilast or a placebo twice daily…

This Phase 3 study tests a drug called nucresiran in 1250 people with ATTR amyloidosis, a rare disease where abnormal proteins damage the heart. Participants receive either nucresiran or a placebo every 6 months. The goal is to see if the drug reduces deaths and heart-related hos…

This Phase 3 trial tests a drug called dazukibart in people with active dermatomyositis or polymyositis, rare diseases that cause muscle weakness and inflammation. About 318 adults will receive either the drug or a placebo by IV every 4 weeks for 48 weeks. The study aims to see i…

This study is for people who have both an autoimmune disease (like rheumatoid arthritis, lupus, or multiple sclerosis) and an advanced cancer that has spread or cannot be removed by surgery. It tests the safety and effectiveness of the immunotherapy drug nivolumab, given alone or…

This study tests an experimental drug called AOC 1044 for boys with Duchenne muscular dystrophy (DMD) who have a specific genetic change (exon 44 skipping). About 70 boys aged 7 to 16 who can still walk will receive either the drug or a placebo. The main goal is to see if the dru…

This Phase 1 trial tests the safety and tolerability of a new drug called AZD5492 in 72 adults with lupus, myositis, or rheumatoid arthritis. Participants receive one or two doses under the skin and are monitored for side effects and how the drug moves through the body. The study…

This study tests whether injecting sodium thiosulfate directly into abnormal calcium deposits can shrink them and reduce pain. It includes people with three rare conditions: systemic sclerosis, dermatomyositis, and a genetic disorder called iPPSD2. Participants receive up to 11 i…

This study follows 500 adults with transthyretin amyloid cardiomyopathy (ATTR-CM), a progressive heart condition caused by abnormal protein buildup. Participants take acoramidis as part of their usual care, and researchers collect data from hospitals and a European registry. The …

This study tests a one-time gene therapy called delandistrogene moxeparvovec in 83 people with Duchenne muscular dystrophy. The goal is to see if it is safe and helps the body make dystrophin, a protein missing in Duchenne. The trial is now enrolling non-ambulatory participants (…

This trial compares a new self-locking biceps tenodesis technique against a standard method for repairing the biceps tendon during rotator cuff surgery. About 100 adults with full-thickness rotator cuff tears and biceps tendon issues will be randomly assigned to one of the two pr…

This study is for people with a rare, high-risk cancer called retroperitoneal sarcoma. It compares two approaches: getting chemotherapy before surgery versus surgery alone. The goal is to see if the chemo-first approach helps prevent the cancer from coming back. About 250 partici…

This study tests whether switching from tafamidis to acoramidis can raise blood levels of transthyretin (TTR) in adults with transthyretin amyloid cardiomyopathy (ATTR-CM). The drug acoramidis stabilizes TTR to prevent harmful amyloid clumps that stiffen the heart. Researchers wi…

This early-stage study tests a new treatment called ALLO-329 for people with lupus, myositis, or scleroderma. The therapy uses specially engineered immune cells from a donor to target and calm the overactive immune system. The main goals are to check safety and see if it can impr…

This study tests whether early heart screening and treatment for iron overload in people with sickle cell disease can prevent heart problems and reduce hospital stays. About 100 adults with sickle cell disease and high iron levels will receive regular heart imaging and follow-up …

This early-stage study tests a new drug called SAR448501 (DR-0201) in 62 adults with lupus or rheumatoid arthritis. The drug is a bispecific antibody designed to target the immune system. The main goal is to check safety and find the right dose, not yet to prove it works. Partici…

This phase 3 trial tests whether belimumab (Benlysta) can stabilize or improve lung function in adults with interstitial lung disease caused by connective tissue diseases like rheumatoid arthritis or lupus. About 440 participants will receive either belimumab injections or a plac…

This study tests whether giving patients electrical muscle stimulation and high-protein shakes soon after a type of stroke (aneurysmal subarachnoid hemorrhage) can prevent muscle loss and improve recovery. Thirty adults will receive the combo therapy or standard care. The main go…

This study tests a surgery called total pancreatectomy with islet autotransplantation (TPIAT) in 30 adults with certain pancreatic tumors who are at high risk for a dangerous leak after standard surgery. The procedure removes the entire pancreas and transplants the insulin-making…

This early-stage study tests a new medicine called AZD4063 in 31 adults with a specific genetic form of dilated cardiomyopathy (PLN R14del). The main goal is to check the drug's safety and how the body processes it. Participants receive the drug as an injection under the skin and…

This early-stage trial tests a combination of two drugs—peposertib and a low-dose form of the chemotherapy doxorubicin—in people with advanced sarcomas that have spread or can't be removed by surgery. The goal is to find the safest dose and see if the combo can shrink tumors or s…

This study is checking the long-term safety and how well a medical device called the Pitch-Patch works for people who have had surgery for a torn rotator cuff in the shoulder. The Pitch-Patch is a small polyester patch that helps strengthen the repair. About 37 people who already…

This study tests a drug called depemokimab in adults with hypereosinophilic syndrome (HES), a rare condition where too many eosinophils (a type of white blood cell) cause organ damage. The trial includes 123 people whose HES is not well controlled despite standard therapy. Partic…

This study tests whether gonadal tissue removed from children with disorders of sex development (DSD) can be frozen and stored to preserve fertility. The tissue is already being removed for medical reasons, such as preventing cancer. Researchers will check the tissue for viable g…

This Phase 3 trial tests a gene therapy called FLT201 for adults with Gaucher disease type 1. The goal is to see if a single dose can keep blood counts stable so patients can stop their regular enzyme replacement or substrate reduction therapy. The study will enroll 45 people who…

This early-stage trial tests UX016, a drug designed to help people with GNE myopathy, a rare genetic muscle-weakening disease. Researchers will give the drug or a placebo to 24 adults to see if it is safe and improves muscle strength. The study is not yet recruiting.

This Phase 3 trial tests whether DYNE-101 can improve muscle function and daily life in 150 adults with myotonic dystrophy type 1 (DM1). Participants receive either the drug or a placebo by IV every few weeks for 48 weeks. The study measures how quickly people can stand from a ch…

This early-phase trial tests a new gene therapy called ASP2957 for X-linked myotubular myopathy (XLMTM), a rare muscle disease present at birth that often requires a ventilator to breathe. The therapy delivers a healthy copy of the MTM1 gene using a modified virus. Nine boys up t…

This phase 3 trial compares a once-weekly growth hormone injection (lonapegsomatropin) to a daily one (somatropin) in 186 prepubertal children with growth failure due to Turner syndrome, SHOX deficiency, being small for gestational age, or idiopathic short stature. The goal is to…

This trial tests an investigational cell therapy called FT839 in people with active autoimmune diseases, including ANCA-associated vasculitis, inflammatory myositis, rheumatoid arthritis, lupus, and systemic sclerosis. Participants receive FT839 as two intravenous infusions, some…

This study tests a brain implant that records and stimulates brain areas to help adults with severe paralysis (from spinal cord injury, ALS, or stroke) control assistive devices like computers or wheelchairs. The main goal is to check safety, and researchers will also see if part…

This study tests the long-term safety and effectiveness of an investigational drug called danicamtiv in adults with symptomatic genetic or familial dilated cardiomyopathy (DCM), a condition where the heart muscle weakens and enlarges. Participants who completed a prior 24-week st…

This Phase 2 study tests a drug called apitegromab in 60 adults with facioscapulohumeral muscular dystrophy (FSHD), a genetic condition that causes muscle weakness. Participants will receive either the drug or a placebo every 4 weeks for a year. The main goal is to see if the dru…

This study is testing a shoulder replacement device in 300 people with shoulder arthritis, bone death, or rotator cuff tears. Participants receive the implant and are followed for 10 years to see how well it works and if it is safe. The goal is to improve pain and shoulder functi…

This trial tests a device called the FiberLocker System, which includes a patch that surgeons attach to a torn rotator cuff tendon during repair surgery. The patch is meant to reinforce the repair and help it hold. Researchers will follow about 50 adults aged 40 to 65 who need su…

This program provides access to an experimental drug, AOC 1044, for people with Duchenne muscular dystrophy (DMD) whose genetic mutation can be treated by skipping exon 44. The goal is to help control the disease and improve muscle function. Participants must be at least 2 years …

This early-stage trial tests a single intravenous dose of AFTX-201, a gene therapy designed to deliver a working copy of the BAG3 gene to heart cells. It enrolls 22 adults aged 18–55 with dilated cardiomyopathy and a BAG3 mutation. The study primarily checks safety and tolerabili…

This study tests a new surgical technique for people with massive rotator cuff tears. Instead of cutting the biceps tendon, surgeons move it to help support the repair. The study will follow 35 adults for one year to see if it improves shoulder function, quality of life, and slee…

This study tests a new injectable drug called depemokimab in 30 children aged 6 to 11 with asthma that involves a specific type of inflammation. The drug is given alongside their usual asthma medicines to see how the body processes it, how well it works, and if it is safe. The go…

This trial tests whether adding a collagen membrane called Regeneten during arthroscopic rotator cuff repair improves tendon healing and quality compared to standard surgery alone. Adults with a torn supraspinatus tendon, sometimes with other small tears, are randomly assigned to…

This study tests whether removing a small part of the shoulder bone (coracoid) during reverse total shoulder replacement helps patients rotate their arm inward better after surgery. Sixty adults needing shoulder replacement will be randomly assigned to get the standard surgery or…

This study gives ongoing access to benralizumab (Fasenra) for people with asthma, eosinophilic granulomatosis with polyangiitis (EGPA), or hypereosinophilic syndrome (HES) who completed a prior benralizumab trial and still benefit from the drug. About 230 participants will receiv…

This study tests an oral drug called KT-621 in 264 adults with moderate to severe eosinophilic asthma that is not well controlled by current treatments. Participants will take either KT-621 or a placebo daily for several weeks. Researchers will measure changes in lung function an…

This study tests an investigational drug called ENTR-601-44 in 24 boys and young men with Duchenne muscular dystrophy (DMD) whose genetic mutation can be treated by skipping a specific part of the gene (exon 44). The trial has two parts: first, to find the safest and most effecti…

This study tests an investigational drug called ENTR-601-45 in people with Duchenne muscular dystrophy (DMD) who have a specific genetic change (exon 45 skipping). The goal is to check the drug's safety, find the right dose, and see if it can help muscles work better. About 24 ma…

This trial investigates whether using real-time ultrasound to guide shock wave therapy improves outcomes for people with calcific tendinitis of the shoulder, a painful condition where calcium deposits form in the rotator cuff tendons. Participants aged 20 to 70 with confirmed dep…

This trial tests whether taking inulin, a type of dietary fiber, for two weeks can improve cardiovascular health, muscle function, and the balance of gut and vaginal bacteria in perimenopausal women who are overweight or have obesity. Participants will take either inulin or a pla…

This phase 3 trial is testing whether an investigational drug called INT230-6, which combines three anti-cancer agents and is injected directly into tumors, can help people with advanced soft tissue sarcoma live longer than those receiving standard care. The study enrolls adults …

This trial compares two surgical techniques for treating a painful jaw condition called disc displacement without reduction, where the jaw's cushioning disc slips out of place and limits mouth opening. Participants will receive either arthroscopic lysis and lavage, a procedure th…

This study tests a special collagen implant (REGENETEN) for people with partial-thickness rotator cuff tears. The goal is to see if it helps patients return to daily activities faster than the standard surgical repair. About 234 adults who have not improved after 3 months of othe…

This pilot trial compares two surgical options for people with massive, irreparable rotator cuff tears that haven't improved with non-surgical treatment. One group receives a partial repair, leaving some of the tear unrepaired, while the other gets a newer procedure called superi…

This study is tracking 245 hernia patients who receive the GORE ENFORM biomaterial mesh during surgery. The mesh is absorbable and used to reinforce the repair. Researchers will monitor hernia recurrence, infections, and other complications for up to two years. The goal is to see…

This study tests the drug acoramidis in people with transthyretin amyloid cardiomyopathy (ATTR-CM), a condition where abnormal proteins build up in the heart, weakening it. Over 36 months, researchers measure whether acoramidis improves heart function and structure using cardiac …

This study tests an investigational drug called PGN-EDODM1 in 24 adults with myotonic dystrophy type 1, a condition causing muscle stiffness and weakness. Participants receive multiple doses of the drug or a placebo by IV to check safety and how the body processes it. The goal is…

This study compares a newer pacing method (HIS or left bundle branch pacing) to the standard biventricular pacing in 1100 heart failure patients across Nordic countries. The goal is to see if the new approach reduces death or unplanned heart failure hospitalizations. Participants…

This early-stage trial tests a new treatment called AZD0120 for adults with three autoimmune diseases: systemic sclerosis, idiopathic inflammatory myopathies, or difficult-to-treat rheumatoid arthritis. AZD0120 is made from a patient's own immune cells, which are modified in a la…

This early study tests a new treatment called CD19/BCMA CAR-T for people with severe autoimmune diseases like lupus and Sjögren's syndrome that haven't improved with standard care. The treatment involves taking a patient's own immune cells, modifying them to target specific immun…

This phase 3 trial tests whether givinostat, an oral drug, can slow muscle decline in boys aged 9 to 18 with Duchenne muscular dystrophy who can no longer walk. Participants receive either givinostat or a placebo for 18 months. The main goal is to see if the drug helps maintain u…

This study tests whether adding a vein-injected alcohol treatment to the usual heart ablation procedure can better control dangerous heart rhythms in people with prior heart attacks. About 156 adults with an implanted defibrillator and recent episodes of rapid heartbeat will be r…

This trial tests a procedure called FETO (fetoscopic endoluminal tracheal occlusion) for babies diagnosed before birth with a severe form of congenital diaphragmatic hernia (CDH). In CDH, a hole in the diaphragm lets abdominal organs push into the chest, preventing the lungs from…

This study tests whether temporarily stopping methotrexate for 2 weeks after receiving a pneumonia vaccine (PCV20) helps the vaccine work better in adults with autoimmune rheumatic diseases like rheumatoid arthritis or lupus. About 192 participants with stable, low disease activi…

This study follows 250 boys (ages 2 and older) with Duchenne muscular dystrophy who are taking vamorolone (AGAMREE). Researchers will monitor side effects, growth, bone health, heart function, and quality of life over time. The goal is to better understand the long-term safety an…

This study tests a gene therapy designed to help older adults regain muscle mass and strength lost with age. The treatment uses a harmless virus to deliver a gene that promotes muscle growth, sometimes combined with another gene that improves blood flow. The trial involves 12 par…

This Phase 3 trial compares a low-dose combination of prednisone and methotrexate to standard high-dose prednisone for people with active cardiac sarcoidosis. Researchers hope the combo works just as well but causes fewer side effects and improves quality of life. The study plans…

This trial compares two surgical methods for treating long head of the biceps tendon or SLAP lesions in people without full-thickness rotator cuff tears. One technique uses a self-locking mechanism, the other a 360 suture anchor. The goal is to see which provides better shoulder …

This study compares two implantable heart devices—Cardiac Contractility Modulation (CCM) and Cardiac Resynchronization Therapy (CRT)—in people with Chagas heart disease and advanced heart failure. The goal is to see which device better improves quality of life and heart function.…

This trial compares two approaches for people with rotator cuff arthropathy, a condition where the shoulder joint wears down due to a torn rotator cuff. One group receives reverse total shoulder replacement surgery followed by standard rehab, while the other group completes a 12-…

This study tests whether injecting a person's own bone marrow stem cells into or near the eye can help treat various retinal and optic nerve diseases, including age-related macular degeneration, retinitis pigmentosa, and glaucoma. Participants receive stem cell injections via dif…

This phase 2 trial tests whether dapagliflozin, a diabetes drug, is safe and can lower high urine protein levels in people with renal AL amyloidosis—a rare condition where abnormal proteins damage the kidneys. About 20 participants will take the drug daily for 6 months and be mon…

This study tests two ways to treat a slow heart rate caused by overactive nerves, without using a permanent pacemaker. One method uses freezing (cryoablation) and the other uses burning (radiofrequency) to calm the nerves. Sixty adults aged 18 to 60 with symptoms like fainting or…

This early-stage study tests a new drug (GSK5926371) in people with lupus and similar autoimmune diseases. The drug is designed to target and calm specific immune cells that attack the body. The main goal is to check if the drug is safe and how the body handles it, with 54 adults…

This early-stage trial tests a brain-computer interface called BrainGate for people with paralysis from conditions like ALS or spinal cord injury. A small sensor is placed in the brain to interpret movement-related signals, allowing users to control a tablet computer just by thin…

This study tests whether Enavogliflozin, a drug used for diabetes, can help people with amyloid cardiomyopathy, a condition where abnormal proteins build up in the heart. About 68 adults with heart failure symptoms will take the drug or a placebo for 12 weeks, then switch. The ma…

This trial tests a procedure called FETO, where a small balloon is placed in the windpipe of a fetus with severe left congenital diaphragmatic hernia (CDH). The balloon blocks the airway temporarily, which may help the lungs grow larger before birth. The study includes pregnant w…

This study looks at how well the drug mavacamten works for Chinese adults with obstructive hypertrophic cardiomyopathy, a condition where the heart muscle is too thick. About 500 people will be followed in regular clinic settings. The main goal is to see if the drug improves bloo…

This trial investigates whether using a device that sends mild electrical pulses to the thigh muscles (neuromuscular electrical stimulation, or NMES) can prevent muscle loss and weakness after abdominal surgery for colorectal or gastric cancer. Some participants will also receive…

This study is testing whether an implantable defibrillator (ICD) helps people with a certain type of heart failure (non-ischemic cardiomyopathy) live longer. About 2,500 participants will be randomly assigned to receive an ICD or not, and their survival will be tracked for up to …

This study tests whether a 4-month structured telerehabilitation program, delivered via video calls and an app, can reduce deaths and hospital readmissions in heart patients who have Post-Intensive Care Syndrome (PICS). About 326 adults aged 30-75 who were in the ICU for at least…

This study tests a pre-set treatment plan for managing heart rhythm issues that can occur after transcatheter aortic valve replacement (TAVR). The goal is to reduce the number of patients who receive a permanent pacemaker while ensuring no one is put at risk of sudden cardiac dea…

This study tests whether JAK inhibitors work better than calcineurin inhibitors as the first treatment for lung scarring in people with antisynthetase syndrome. About 80 adults with active lung disease will be followed for 12 months to see which drug improves breathing and surviv…

This Phase 3 trial tests whether baricitinib, a JAK inhibitor pill, can improve symptoms of dermatomyositis (a rare autoimmune disease causing muscle weakness and skin rashes) while allowing patients to stop steroids. 62 adults with active disease will receive baricitinib or a pl…

This study tests the safety and feasibility of the BrainGate2 system, a brain-computer interface. Tiny sensors are placed in the brain area that controls movement, allowing people with tetraplegia (paralysis of all four limbs) to control a computer cursor or other assistive devic…

This study is tracking up to 400 people who are having arthroscopic rotator cuff repair with a device called the REGENETEN Bioinductive Implant. The implant is placed to support healing of the torn tendon. Researchers will measure shoulder function, pain, and quality of life for …

This study is testing Bearing nsPVA particles, used during a procedure called uterine artery embolization, to treat uterine fibroids in 100 women. The goal is to see if these particles safely block blood flow to fibroids, reducing symptoms like pain and heavy bleeding. Participan…

This study tests a new medicine called DYNE-251 in boys aged 4 to 18 with Duchenne muscular dystrophy (DMD) who can still walk. The goal is to see if it helps them move better and slows muscle damage. Participants will receive either the drug or a placebo every 4 weeks for about …

This study tests whether a carefully controlled high-fat diet can improve heart function in people with a specific type of heart failure (non-ischemic dilated cardiomyopathy). Researchers believe that switching the heart's fuel source back to fats may help it pump more effectivel…

This study looks at whether the heart medication Camzyos (Mavacamten) can improve blood flow to the heart muscle in people with hypertrophic obstructive cardiomyopathy (HOCM). Researchers will use special PET-CT scans to measure blood flow before and after 12 months of treatment.…

This study tests a simple change at birth: instead of clamping the umbilical cord right away, doctors will keep it intact while helping the baby breathe. The goal is to see if this improves heart and lung function in full-term infants with congenital diaphragmatic hernia (CDH), a…

This study looks at whether people with a type of heart disease called obstructive hypertrophic cardiomyopathy can safely switch from one medication (mavacamten) to another (aficamten). The goal is to keep the heart working better and reduce symptoms. About 40 adults who are alre…

This Phase 2 study tests whether a Traditional Chinese Medicine (TCM) herbal formula can improve muscle strength and bone health in pre-frail adults aged 65 and older, compared to the nutritional supplement HMB. Over 24 weeks, 90 participants will take either the TCM powder or HM…

This study tests whether adding empagliflozin (a diabetes drug) to standard carvedilol treatment can prevent liver decompensation events and death in 400 adults with cirrhosis and left ventricular diastolic dysfunction (stiff heart). Participants will receive either empagliflozin…

This study tests whether two dietary supplements, HMB and 2-HOBA, can improve muscle mass, physical function, and overall health in adults aged 65 and older. Over 90 days, 120 participants will take one or both supplements or a placebo. Researchers will measure changes in muscle,…

This study tests whether adding a patch made from donated human skin to standard rotator cuff repair surgery can lower the chance of the tendon tearing again. Forty adults aged 50 to 75 with large tears will be randomly assigned to get surgery with or without the patch. An MRI on…

This phase 2 trial is testing whether filgotinib, a drug already used for arthritis, can help people with three rare immune diseases: Behçet's disease, myositis, and IgG4-related disease. The goal is to see if it can control disease activity and reduce the need for long-term ster…

This study tests if the drug cyclosporine can reduce heart muscle damage in people with Takotsubo syndrome, also known as broken heart syndrome. About 204 adults at high risk for complications will receive either cyclosporine or a placebo shortly after diagnosis. Researchers will…

This large real-world study is testing whether platelet-rich plasma (PRP) injections can reduce pain and improve function in people with chronic tendinopathies, including patellar, Achilles, elbow, and plantar fasciitis. Researchers will follow 2000 participants for 12 months, tr…

This study compares two approaches for people with ischemic cardiomyopathy (weak heart muscle due to blocked arteries) and multiple blocked vessels. One group gets optimal medical therapy alone, while the other also gets a heart artery procedure (PCI) guided by imaging and pressu…

This study tests whether colchicine, a common anti-inflammatory drug, can help people with chronic heart muscle inflammation (inflammatory cardiomyopathy). About 80 adults with this condition will receive either colchicine or a placebo to see if it prevents worsening heart failur…

This study tests the drug nivolumab in people with advanced rare cancers that have a specific marker (PD-L1). The goal is to see if the drug can shrink or control the tumors when other treatments have stopped working. Up to 28 adults will receive treatment for up to 12 months, an…

This study looks at a blood-filtering procedure called immunoadsorption (IA) in 50 people with ME/CFS or Post-COVID syndrome who have certain autoantibodies. The goal is to see if removing these antibodies improves symptoms like severe fatigue and brain fog. Researchers will trac…

This phase II trial compares the chemotherapy drugs trabectedin and gemcitabine in 100 people with advanced leiomyosarcoma that has worsened after initial treatment. The study aims to see which drug better delays cancer progression. It also includes an observational group for tho…

This early-phase trial is testing a new treatment called KN5601, which uses a patient's own immune cells (NK cells) that have been engineered to target and destroy harmful B cells. The goal is to see if it is safe and can help children aged 5 and older with rheumatic diseases lik…

This study tests a new gene therapy called TN-201 in 30 adults with hypertrophic cardiomyopathy caused by a specific genetic mutation (MYBPC3). The therapy is given as a one-time IV infusion and aims to correct the underlying genetic defect. The main goals are to check safety and…

This study tests whether giving enzyme replacement therapy to fetuses with certain rare genetic diseases (like MPS I, Gaucher, or Pompe) before birth is safe and feasible. About 10 pregnant participants will receive the treatment through the umbilical vein. The goal is to see if …

This pilot study tests a procedure called FETO, where a tiny balloon is placed in the fetus's windpipe to encourage lung growth. It is for pregnancies where the baby has severe left congenital diaphragmatic hernia (CDH) and very small lungs. The study will enroll 20 participants …

This study tests a drug called tideglusib in 120 people with a genetic heart condition that can cause dangerous heart rhythms. The drug aims to reduce abnormal heartbeats and improve heart function. Participants will take either tideglusib or a placebo daily for about a year to s…

This study looks at whether drinking non-alcoholic red wine, which is rich in natural compounds called polyphenols, can help older adults keep their thinking sharp and muscles strong. Researchers will give 72 people aged 60-74 either non-alcoholic red wine or water, both as part …

This study tests a gene therapy called GS010 for people with Leber hereditary optic neuropathy (LHON), a genetic condition that causes rapid vision loss. Researchers will give the treatment as an injection into the eye at two different doses to see if it improves vision and mitoc…

This Phase 3 trial tests whether colchicine, an anti-inflammatory drug, can reduce heart scarring and prevent complications in 300 people with acute myocarditis (heart inflammation). Participants receive colchicine or a placebo for six months alongside standard care. The goal is …

This study tests a procedure called FETO for unborn babies with a severe form of congenital diaphragmatic hernia (CDH), where the diaphragm has a hole and the lungs don't grow well. Doctors place a tiny balloon in the baby's windpipe to block it, which may help the lungs expand a…

This phase 3 trial tests the drug AL3818 (anlotinib) in people with advanced alveolar soft part sarcoma, leiomyosarcoma, or synovial sarcoma. Currently, only patients with alveolar soft part sarcoma are being enrolled. Participants receive AL3818 pills or a standard chemotherapy …

This study tests a new surgical approach for people with large rotator cuff tears. Doctors will use a special skin tissue (donated and processed to remove cells) combined with the patient's own bone marrow and shoulder lining cells to strengthen the repair. The goal is to see if …

This study compares a single dose of targeted radiation (SBRT) to the standard catheter ablation procedure for reducing dangerous heart rhythms in people with advanced heart disease. Sixty participants will be randomly assigned to one of the two treatments. The main goal is to se…

This study tests whether a drug called ACM-001.1 can help obese people keep their muscle while taking semaglutide for weight loss, and help them regain muscle after stopping semaglutide. About 120 participants will receive either ACM-001.1 or a placebo alongside semaglutide, and …

This study tests whether the drug regorafenib can help prevent bone sarcomas from coming back after initial treatment. About 168 people aged 12 and older with various bone sarcomas will be randomly assigned to take regorafenib for up to 12 months or to receive standard monitoring…

This phase 2 trial tests whether the drug regorafenib can help keep high-grade bone sarcomas from growing in patients who still have some disease after standard treatment. About 36 people aged 12 and older will take regorafenib daily for up to 12 months. The main goal is to see h…

This early-phase study tests a new treatment called TI-0032-III for people with severe autoimmune diseases like lupus and scleroderma that haven't improved with standard care. The treatment uses a special injection to create CAR-T cells inside the body, which then target and dest…

This clinical trial is testing whether a 3-week course of ibuprofen can reduce heart scarring better than standard acetaminophen in people hospitalized with acute myocarditis (heart muscle inflammation). The study will enroll 150 adults with mild cases (normal heart pumping funct…

This early study tests whether sotagliflozin, a drug already used for heart failure, can help people with a type of hypertrophic cardiomyopathy that doesn't block blood flow. Twenty-six adults will take the drug or a placebo for 12 weeks, then switch, to see if it improves exerci…

This study compares a new type of heart pacing (His-bundle pacing) to standard pacing in 120 adults with heart failure and right bundle-branch block. The goal is to see if the new method better improves the heart's pumping ability over 6 months. Participants will receive one of t…

This phase 2 trial tests whether spironolactone, a potassium-sparing diuretic, can slow the deterioration of the right ventricle and reduce arrhythmias in people with arrhythmogenic right ventricular dysplasia (ARVD). About 120 participants will receive either spironolactone or a…

This study tests a new drug called ATX-01 in 56 adults with myotonic dystrophy type 1, a condition that causes muscle stiffness and weakness. The drug aims to block a molecule that may contribute to the disease. The trial will first give a single dose, then multiple doses, to che…

This study compares a new pacing method (left bundle branch pacing) with a standard pacemaker-defibrillator in 30 people who have hypertrophic cardiomyopathy and a specific heart block after myectomy surgery. The goal is to see if the new method better prevents heart failure wors…

This study tests whether tocilizumab, a drug that blocks inflammation, can improve symptoms and breathing in adults with anti-MDA5+ dermatomyositis, a rare autoimmune disease affecting muscles, skin, and lungs. About 110 participants will receive either tocilizumab or a placebo e…

This study tests a procedure called FETO for babies with a severe birth defect where the diaphragm has a hole, allowing organs to crowd the lungs. A tiny balloon is placed in the baby's windpipe while still in the womb to help the lungs grow. The study tracks how many babies surv…

This study tests a procedure called FETO in 10 pregnant women carrying babies with a severe form of congenital diaphragmatic hernia (CDH). The procedure places a small balloon in the baby's windpipe to block it, which helps the lungs grow larger before birth. The balloon is remov…

This pilot trial tests whether a patient's own stem cells can help the heart muscle recover in people with advanced chronic heart failure who already have a mechanical heart pump (LVAD). Ten patients will receive their own CD34+ stem cells injected into a heart artery. Researcher…

This study tests a new gene therapy called LY-M001 for adults with Gaucher disease type 1, a genetic disorder that causes organ damage and other health problems. The therapy uses a harmless virus to deliver a working copy of the GBA1 gene to liver cells, aiming to restore the mis…

This early-stage study tests a new treatment called RXIM002 for people with severe autoimmune diseases that haven't responded to standard therapies. The treatment uses a special RNA molecule to create immune cells that target and destroy faulty B cells, which are causing the dise…

This study tests a new combination of drugs (tislelizumab, liposomal doxorubicin, and ifosfamide) as a first treatment for people with advanced soft tissue sarcoma that cannot be removed by surgery or has spread. The goal is to see if this combo shrinks tumors and is safe. About …

This study tests a medication called acoramidis in 200 people with a rare heart condition called transthyretin cardiac amyloidosis. The goal is to see if the drug can slow down the disease and prevent worsening of heart function. Participants either are newly diagnosed or switch …

This study compares two pacemaker strategies for people with sick sinus syndrome and AV conduction delay. Participants will be randomly assigned to receive either left bundle branch area pacing (LBBAP) or minimized ventricular pacing (MVP). The goal is to see which method leads t…

This study tests a special pacing technique called high-intensity his bundle pacing in 34 adults with heart failure and a narrow QRS pattern on their ECG. Standard pacing doesn't help these patients, but this new approach may improve heart function and quality of life. Participan…

This study tests a new procedure that uses radiofrequency energy to reduce thickened heart muscle in people with obstructive hypertrophic cardiomyopathy. About 124 adults with symptoms like chest pain or shortness of breath will be randomly assigned to receive the active procedur…

This study tests a procedure called FETO for unborn babies with a severe diaphragmatic hernia, where organs move into the chest and harm lung growth. A tiny balloon is placed in the baby's windpipe around 27 weeks of pregnancy to help the lungs grow, then removed around 34 weeks.…

Researchers are testing whether beta-amyloid PET/CT scans can detect changes in heart amyloid deposits after one year of treatment for light chain cardiac amyloidosis. Sixty adults with newly diagnosed AL-CA receive standard anti-plasma cell or targeted therapy and undergo PET/CT…

Researchers at Turku University Hospital are testing a PET imaging tracer called Al[18F]F-NOTA-folate to see how well it detects two heart conditions: cardiac sarcoidosis, an inflammatory disease of the heart muscle, and infective endocarditis, an infection of the heart valves. T…

Researchers at Semmelweis University are building and testing artificial intelligence algorithms that read standardized transvaginal ultrasound images. The study includes women aged 18 to 45 who have suspected endometriosis or adenomyosis, a confirmed uterine mass scheduled for s…

Researchers at Johns Hopkins University are studying whether neuromuscular ultrasound and MRI can serve as reliable markers of swallowing muscle problems in people with inclusion body myositis (IBM). The study compares imaging and clinical findings in 30 participants, including p…

This study tests a special radioactive tracer called [68Ga]Ga-FAPI-46 for PET scans in 40 people with transthyretin cardiac amyloidosis (ATTR-CM), a condition where abnormal proteins build up in the heart. The goal is to see if this scan can measure scar-like activity in the hear…

This study is testing a new imaging agent called [18F]FT8 to see if it can help doctors better see and diagnose amyloidosis, a disease where abnormal proteins build up in organs like the heart. The study will involve 20 patients with amyloidosis and 5 healthy volunteers. Research…

This study tests whether a newer type of CT scan, called photon-counting CT, can accurately measure blood flow to the heart muscle and detect scarring or fibrosis. Researchers will compare the CT results with cardiac MRI and invasive coronary angiography in 100 patients with aort…

This study tests whether a new radioactive dye, 68Ga-FAPI, can spot active scarring in the heart muscle using PET-MR scans. Researchers will scan about 290 people with various heart muscle conditions, such as heart failure with preserved ejection fraction, hypertrophic cardiomyop…

This study aims to create a new scoring system called HASS to reliably diagnose long biceps tendon injuries before surgery. Currently, these injuries are hard to confirm without surgery, leading to uncertainty. Researchers will combine physical exam tests and imaging data from 20…

This study tests whether a quantitative analysis of bone scintigraphy (SUVmax) can better track amyloid burden in the hearts of patients with transthyretin cardiac amyloidosis who are being treated with tafamidis. The current Perugini score is limited in assessing disease severit…

This study tests whether a radioactive tracer called Florbetaben F18 can reliably detect amyloid deposits in the heart using PET scans. Researchers will scan 15 people with and without cardiac amyloidosis twice to see if the results are consistent. The goal is to improve diagnosi…

This study is testing a cloud-based AI platform called Willem that analyzes electrocardiogram (ECG) data to detect Transthyretin cardiac amyloidosis (ATTR-CA), a rare and serious heart condition. Researchers will compare ECGs from 2000 adults—some with confirmed ATTR-CA and some …

This study is testing a cloud-based artificial intelligence platform called Willem that analyzes ECG recordings to detect heart rhythm problems and abnormal patterns. Researchers will compare the AI's readings to those of board-certified cardiologists in over 5,300 high-risk card…

This study is testing whether a computer program (machine learning) can help doctors diagnose severe heart attacks in patients who have a heart condition called left bundle branch block (LBBB). LBBB can hide the usual signs of a heart attack on an ECG, making it hard to tell if a…

This study tests an artificial intelligence program that analyzes heart rhythm data from a small, wearable patch to estimate how well the heart pumps blood. Researchers will compare the AI's results to standard ultrasound in 2,000 adults. If accurate, this could offer a simpler, …

This pilot study explores whether data from the HeartMate 3 heart pump can be used to monitor heart function without invasive tests or frequent imaging. Researchers will compare high-resolution pump data with standard echocardiograms and ECGs in 60 patients with end-stage heart f…

This study looks at whether a special PET scan can find early signs of scarring in the heart muscle of people with ARVC, a rare inherited heart disease that can lead to dangerous heart rhythms. Fifteen adults with ARVC will get this scan along with standard tests. Researchers hop…

This study is testing a new blood test that can diagnose single-gene disorders in unborn babies using a sample from the mother. The test looks at fetal DNA found in the mother's blood, which is safer than traditional invasive methods that carry a small risk of miscarriage. Resear…

This study tests a new PET imaging method that uses a special tracer called XTR003 along with FDG to see which parts of the heart are still alive in people with heart failure from blocked arteries. About 40 to 60 participants will be randomly assigned to receive either the new fa…

This study tests whether a specific ultrasound measurement can reliably detect superficial endometriosis on the uterosacral ligaments. Researchers will compare ultrasound findings with laparoscopic surgery results in 64 women undergoing gynecological surgery. If a clear cutoff th…

This study explores a new blood test that looks for DNA fragments from damaged heart cells to detect active heart inflammation in people with sarcoidosis. Researchers will compare results from 120 participants, including healthy volunteers and those with known heart conditions. I…

This trial tests whether placing a special gel inside the uterus after fibroid removal surgery can prevent the formation of scar tissue (adhesions) that may harm fertility. Women aged 18 to 45 who are scheduled for laparoscopic or abdominal myomectomy and have no pre-existing ute…

This study tests whether taking green tea extract (EGCG) and vitamin D3 daily can prevent uterine fibroids from growing back after surgical removal. About 240 women who have had fibroid surgery will receive either the supplements or a placebo. The goal is to see if this simple co…

This study tests whether the drug dexrazoxane can prevent heart damage caused by chemotherapy in people with diffuse large B-cell lymphoma (DLBCL). About 324 patients will receive either standard treatment plus dexrazoxane or standard treatment alone. The goal is to see if dexraz…

This study tests whether dapagliflozin, a drug used for diabetes and heart failure, can prevent heart damage caused by a common type of chemotherapy (anthracyclines) in breast cancer patients. One hundred women will receive either dapagliflozin or a placebo daily during their che…

Researchers are testing whether a Lingzhi-containing dietary supplement called CP003 reduces fatigue in people with chronic fatigue syndrome or persistent fatigue after COVID-19. The study compares people who take the supplement for six weeks with those who wait to start treatmen…

This study compares a high-tech rehabilitation program—using robotic systems, virtual reality, and balance platforms—to standard physical therapy for people with acute neuromuscular diseases like Guillain-Barré syndrome. About 124 adults aged 18 to 80 will participate. The goal i…

Researchers are testing whether a 12-week program that combines movement representation techniques with sensorimotor training can reduce shoulder pain in adults aged 50 and older who have had rotator cuff related shoulder pain for more than three months. The program uses action o…

This study compares two types of anesthesia for rotator cuff repair surgery: general anesthesia combined with local anesthesia versus local anesthesia alone. The goal is to see which approach leads to faster recovery and readiness to leave the recovery room. About 34 adults with …

This study is testing a gel called OSTENIL TENDON to see if it safely reduces pain and improves movement in people with tendon problems. Researchers will enroll 84 adults with one of four types of tendinopathy. The main goal is to measure pain changes three months after treatment…

This pilot study tests whether focused shockwave therapy can reduce pain and improve function in people with chronic neck and shoulder muscle pain. Sixty adults will be randomly assigned to receive either three weekly shockwave sessions or a home exercise program. Researchers wil…

This study tests whether modafinil, a drug used for sleep disorders, can reduce severe fatigue in adults with inactive inflammatory bowel disease (IBD). Sixty participants will take either modafinil or a placebo for 8 weeks, starting at 100 mg daily with possible dose increases. …

Researchers are testing whether a pacing technique called left bundle branch area pacing, guided by detailed imaging and electrical data, can improve outcomes for people with heart failure and left bundle branch block. The trial enrolls 100 adults with non-ischemic cardiomyopathy…

Researchers are testing whether a quadratus lumborum nerve block reduces pain after laparoscopic myomectomy, a surgery to remove uterine fibroids while keeping the uterus. The block is a temporary anesthetic injection into a lower back muscle. In this double-blind, randomized tri…

Achilles tendinopathy is a painful condition of the tendon at the back of the ankle. Researchers are testing whether injecting a collagen-based medical device around the tendon, combined with a physiotherapy program of eccentric strengthening exercises, improves pain and function…

This study tests whether a supplement called geranylgeraniol (GG), derived from annatto seeds, can reduce muscle pain and weakness caused by statin medications. Researchers will give 95 adults either a low or high dose of GG or a placebo for three months. The goal is to see if GG…

Researchers are testing a targeted ablation method for people with structural heart disease who have ventricular tachycardia, a dangerous fast heart rhythm. The procedure maps conducting channels inside heart scar tissue by pacing many sites, then uses radiofrequency energy to bl…

This study is testing a mind-body program called DNRS for people with ME/CFS (chronic fatigue syndrome) who may also have Long COVID. The program uses brain retraining to help calm overactive nerves and improve symptoms like fatigue, pain, and mood. Researchers will track quality…

This study compares two common injection treatments for chronic mid-back pain: the erector spinae plane (ESP) block and trigger point injection (TPI). About 76 adults with pain lasting at least three months will be randomly assigned to one treatment. Researchers will track pain l…

This study tests whether breathing pure oxygen in a pressurized chamber (hyperbaric oxygen therapy) can help people with mild to moderate chronic fatigue syndrome. 74 participants will receive either real treatment or a sham (placebo) for 60 sessions over 3 months. Researchers wi…

This trial tests whether a program built around small, deliberate choices — called micro-choices — can help people with persistent fatigue, including those with long COVID. Participants attend a four-day group workshop and receive three weeks of digital support to build new habit…

This study compares two types of full can exercises—concentric and eccentric—for people with partial rotator cuff tears. Sixty participants will do one type of exercise plus standard physiotherapy for 6 weeks. Researchers will measure changes in shoulder muscle structure, pain, a…

This trial tests whether drinking hydrogen-rich water for 16 weeks can reduce fatigue and improve physical functioning in people with ME/CFS (chronic fatigue syndrome). Half of the 80 participants will receive the active supplement, while the other half gets a placebo. The study …

This trial tests whether a three-day low-residue diet and deeper muscle relaxation, alone or together, can improve the surgical view during vNOTES hysterectomy (a scarless procedure through the vagina). The study involves 160 women having hysterectomy for non-cancer conditions. R…

This study tests whether the drug letrozole can improve symptoms and shrink fibroids in women with uterine fibroids. 140 participants will receive either letrozole or a placebo daily for 12 weeks. The main goal is to see if letrozole reduces symptom severity and improves quality …

This trial tests a 12-session telecare program for children aged 7 to 17 with limb-girdle muscular dystrophy. The group-based intervention focuses on social cognition, emotional management, and coping strategies to improve quality of life and reduce symptoms. Participants attend …

This study tests whether adding manual (hands-on) or self-applied myofascial release (using balls and massage sticks) to standard care—heat, TENS, and exercise—improves pain and function in people with chronic non-specific neck pain. Participants aged 18 to 45 with neck pain last…

This study tests whether adding hyaluronic acid to a standard corticosteroid injection works better for shoulder tendonitis pain than the steroid alone. About 132 adults with confirmed supraspinatus tendinopathy will receive either the combo or a placebo injection. Pain levels ar…

This study investigates whether a technique called ischemic compression—where a therapist applies gradual pressure to tender spots in neck muscles—combined with a home exercise program can reduce pain and improve quality of life in people with chronic non-specific neck pain. Half…

This study compares two exercise programs for people with rotator cuff-related shoulder pain. One group gets a standard, fixed program, while the other gets the same exercises but with personalized touches like preferred music, choice of exercises, and motivational feedback. Rese…

This study tests different dry needling and electroacupuncture methods on healthy adults with hidden muscle knots in the forearm. 150 participants will be split into five groups to see which technique causes the least soreness and best improves grip strength and movement. Results…

This study compares two methods of pulsed radiofrequency treatment for the suprascapular nerve in people with chronic shoulder pain from rotator cuff problems. Thirty-two adults aged 40 to 80 will be randomly assigned to receive the treatment at either the suprascapular notch or …

This study tests whether starting an accelerated shoulder rehabilitation program immediately after reverse total shoulder replacement leads to better pain relief and function than the standard rehab approach. About 74 people with shoulder osteoarthritis will be randomly assigned …

This trial compares two standard anesthesia methods—inhaled sevoflurane versus intravenous propofol—during arthroscopic rotator cuff repair. The goal is to see which one gives surgeons a clearer view inside the shoulder joint, which is important for safety and success. About 110 …

This study tests whether a personalized mix of endurance and strength exercises, done at home with video check-ins, can improve mobility and muscle strength in people with mitochondrial myopathy. Fifteen adults with confirmed genetic mutations will follow the program for up to 12…

This study tests whether a personalized exercise program can improve balance and physical function in adults with rare neuromuscular disorders like Charcot-Marie-Tooth disease, facioscapulohumeral muscular dystrophy, and myotonic dystrophy type 1. Participants will receive a 12-d…

This study tests whether a special laser (MLS class IV) can reduce chronic pain and fatigue in people with hypermobile Ehlers-Danlos syndrome (hEDS), a condition that causes loose joints and widespread pain. Twenty-five adults will receive 10 laser sessions over 5 weeks, with pai…

This study tests three types of injections for people with chronic neck and shoulder muscle pain caused by trigger points. It compares platelet-rich plasma (PRP), a mix of PRP and steroid, and steroid alone to see which works best. 150 adults aged 18-65 with a single active trigg…

This study tests whether taking omega-3 fish oil capsules and doing simple resistance exercises with elastic bands can help cancer patients maintain muscle mass and strength. It involves 288 adults with various cancers who are at risk of muscle wasting. The goal is to improve phy…

This study tests whether injecting the local anesthetic bupivacaine into the small surgical cuts and the vaginal cuff at the end of a laparoscopic hysterectomy can lower postoperative pain. 120 women having the surgery for non-cancer reasons will be randomly assigned to receive t…

This study looks at whether giving children with hypertrophic cardiomyopathy (a condition where the heart muscle is thicker than normal) a personalized exercise plan is safe and helpful. Ten children aged 10 to 18 will receive an exercise prescription from a physiotherapist and b…

This study compares two electrical frequencies (high vs. low) used in percutaneous electrical nerve stimulation (PENS) for shoulder pain. Thirty adults with a trigger point in the infraspinatus muscle will receive a single session, and researchers will measure changes in muscle s…

This study compares three common treatments for long-lasting muscle pain: dry needling, shock wave therapy, and pulsed radiofrequency. Researchers will enroll 60 adults with myofascial pain syndrome who have not improved with standard care. The goal is to find which method reduce…

This study tests whether a device that gently stimulates a nerve in the ear (transcutaneous vagus nerve stimulation) can help people with chronic fatigue from fibromyalgia or long COVID get more benefit from exercise. 60 adults who still feel very tired after a standard exercise …

This study tests whether a small device that gently stimulates a nerve in the ear can help people with long-term back pain. 48 adults will either get the ear stimulation plus standard rehab, or rehab alone, for 3 weeks. The goal is to see if the device improves daily function and…

This study tests whether adding pain neuroscience education (PNE) to standard rehab improves pain, movement, and fear of movement in 45 adults aged 40-75 recovering from rotator cuff surgery. Participants are split into three groups: standard rehab alone, standard rehab plus PNE …

This study tests whether Epidiolex (a CBD medicine) can help control pain after shoulder surgery when added to standard painkillers. About 80 adults having rotator cuff repair will take either CBD or a placebo for two weeks after surgery. They will track their pain and medication…

This study looks at whether dry needling can reduce pain from trigger points (tight, tender spots) in the trapezius muscle, which runs from the neck to the shoulders. About 70 adults with myofascial pain will receive either dry needling or a placebo treatment. Researchers will us…

This study tests a minimally invasive procedure called transcatheter arterial embolization (TAE) combined with physical therapy for people with chronic shoulder pain from rotator cuff tendinopathy that hasn't improved with standard treatments. About 41 adults with moderate to sev…

This study tests whether a fun, game-based occupational therapy program can improve quality of life, daily skills, and emotional health in 20 teens with Duchenne muscular dystrophy. Participants will use a computer or tablet at home. Researchers will compare results to standard t…

This study tests whether a special cold compression machine works better than regular ice packs for pain and swelling after arthroscopic rotator cuff repair. About 100 adults having this shoulder surgery will be randomly assigned to one of the two cooling methods. The goal is to …

This study looks at two different exercise programs—using flexi bands or whole-body electrical stimulation—for people with cancer. The goal is to see which one better helps maintain muscle strength, reduce inflammation, and improve quality of life during treatment. About 100 part…

This study tests whether adding a long-acting painkiller (liposomal bupivacaine) to a standard steroid injection during shoulder surgery can improve recovery for people with rotator cuff tears and shoulder stiffness. About 70 adults will be randomly assigned to receive either the…

This study compares two ways of managing rotator cuff tendinopathy, a common cause of shoulder pain. One group gets monthly check-ups for three months with treatment adjustments as needed, while the other follows standard care. Researchers want to see if the tighter follow-up lea…

This study tests a new home-based program that uses a special treadmill and virtual reality to help children with conditions like cerebral palsy, muscle diseases, or obesity improve their walking. About 30 children aged 6 to 17 will try the program at home for at least 3 sessions…

This study tests whether a custom 3D-printed mouth splint can help burn patients who have developed microstomia (a tight, small mouth opening). Ten participants will use the splint and be measured for changes in mouth size and function using a special survey. The goal is to ease …

This phase 3 trial is testing whether metformin, a common diabetes drug, can improve muscle function in adults with myotonic dystrophy type 1 (Steinert's disease). The study will enroll 142 participants who will receive either metformin or a placebo for 6 months. Researchers will…

This study tests whether 24 weeks of moderate exercise (three 60-minute sessions per week) can improve symptoms and heart function in people with hypertrophic cardiomyopathy, a condition where the heart muscle is thickened. About 70 participants will be randomly assigned to eithe…

This study investigates whether certain environmental exposures—like infections, stress, or sun exposure—are more common in people with anti-synthetase syndrome, a rare autoimmune condition that causes muscle weakness and lung disease. Researchers will compare 580 participants, i…

This study looks at how athletes at risk for patellar tendinopathy (jumper's knee) move during jumps. Researchers will use a camera system to track knee, hip, and ankle angles in 60 male athletes over 12 months. The goal is to find early movement changes that could lead to better…

Laminopathies and emerinopathies are rare disorders caused by mutations in the LMNA or EMD genes, often affecting skeletal muscles and the heart. Researchers at French hospitals are building a registry of 800 patients with confirmed mutations to record detailed genetic, neurologi…

This study uses functional MRI to see how the brain processes unpleasant sensations like pain and physical effort in people with myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS) compared to healthy volunteers. Researchers will apply mild heat and ask participants to sq…

This study aims to better understand takotsubo cardiomyopathy, also known as broken heart syndrome, by using manganese-enhanced MRI scans. Researchers will test how different heart medications affect manganese uptake in the heart muscle. The study involves 100 adults who have had…

This observational study aims to better understand primary mitochondrial myopathy, a rare muscle disease. Researchers will follow 1300 patients and healthy controls, measuring muscle strength, balance, and daily function over time. The goal is to develop and validate tools to tra…

Researchers at Sahlgrenska University Hospital in Sweden are observing 200 adults hospitalized for a severe COPD flare-up to see how often the condition triggers a temporary heart muscle weakness called Takotsubo stunning. They will use echocardiograms to check heart function at …

This study is tracking 20,000 people who have had or will have shoulder replacement surgery to see how well the implants work over at least 10 years. Researchers will measure pain, daily function, and strength using standard shoulder scores. The goal is to better understand the l…

Researchers are building a global registry to collect patient-reported surveys, genetic test results, and medical records from people with Charcot-Marie-Tooth disease and related inherited neuropathies. The study is open to children and adults with a confirmed or suspected diagno…

This study uses ultrasound, a safe imaging technique that uses sound waves, to get detailed pictures and measurements of nerves and muscles. Researchers aim to collect normal values from healthy adults and compare them with data from people who have neuromuscular disorders. The g…

This study looks at 50 adults with AL amyloidosis who achieved complete remission after initial chemotherapy. It checks if tiny amounts of remaining disease (called minimal residual disease, or MRD) affect organ recovery and survival. Researchers also test whether a stem cell tra…

This study aims to learn more about heart and blood vessel diseases by observing people who have them or are at risk. Researchers will collect samples and perform tests like imaging and stress tests on up to 5,000 participants, including healthy volunteers and relatives of affect…

This study follows about 50 members of a family with a rare inherited condition called familial hypereosinophilia, where high levels of a type of white blood cell can damage the heart and nerves. Researchers will track participants over many years with yearly checkups, blood test…

This study follows people with Multiple Acyl-CoA Dehydrogenase Deficiency (MADD) over time to learn how the condition progresses and affects daily life. Researchers will collect health data and patient feedback to identify patterns and potential markers of the disease. The goal i…

This study aims to learn more about myositis, a group of diseases that cause muscle inflammation and damage. Researchers will observe up to 1,200 children and adults with myositis, along with healthy volunteers, to track changes in the immune system and other health problems over…

This study aims to learn more about rare inherited disorders that affect the brain, spinal cord, muscles, and nerves. Researchers will collect medical history, perform exams, and run genetic tests on up to 3,500 participants. No new treatments are tested; the goal is to better un…

This study aims to uncover the genetic causes of hereditary leiomyomatosis renal cell cancer (HLRCC), a condition that can lead to painful skin bumps, uterine fibroids, and kidney tumors. Researchers will study people with known or suspected HLRCC and their family members to iden…

This study follows 10 Chinese adults who have a specific gene mutation (BAG3) linked to dilated cardiomyopathy, a condition where the heart becomes enlarged and weak. Researchers will track changes in heart function, symptoms, and biomarkers over time to better understand how the…

This study aims to understand how and why eosinophils (a type of white blood cell) become active in conditions like allergies, asthma, and parasitic infections. Researchers will observe up to 800 people aged 1 to 100 with high eosinophil levels. No experimental treatments are giv…

This study tracks how quickly the diaphragm weakens in 15 ICU patients on mechanical ventilation. Researchers use advanced muscle tests and brain stimulation to measure changes in diaphragm function and control. The goal is to better understand ventilator-induced diaphragm dysfun…

Researchers are following 3,000 Chinese adults with hypertrophic cardiomyopathy, a condition that thickens the heart muscle and can disrupt blood flow. The study records how patients are treated in everyday hospitals and tracks health events such as stroke, heart failure hospital…

Researchers are studying people with arrhythmia-induced cardiomyopathy, a condition where a fast or irregular heartbeat weakens the heart muscle. The study checks whether these patients carry more harmful gene variants than people with other types of cardiomyopathy or the general…

This study aims to develop and test new MRI methods for imaging the heart and blood vessels to improve diagnosis and management of heart disease. It will enroll up to 3,400 adults, including both patients with heart conditions and healthy volunteers. Participants will undergo MRI…

This global study follows people with POLG-related disorders, a group of rare mitochondrial conditions, to understand how the disease progresses over time. By observing clinical changes in up to 300 participants of all ages, researchers aim to identify key milestones that could s…

This observational study monitors the long-term safety of eplontersen, a treatment for transthyretin amyloidosis, in patients who have had a liver transplant or have severe liver impairment. Researchers will track side effects, lab changes, and serious health events in about 320 …

This study collects blood and tissue samples from 2,000 people with or without heart rhythm problems. Researchers will analyze the samples to find genetic differences linked to arrhythmias. The goal is to better understand the causes of these conditions, not to test a new treatme…

This study gathers medical information from 200 adults with possible nerve, muscle, or autonomic nervous system disorders who are not already in other NIH studies. Participants undergo standard tests like nerve conduction studies, EMG, and physical exams. The data is stored anony…

This study will look at 40 people with both hypertrophic cardiomyopathy (HCM) and atrial fibrillation (AF) to understand why AF is more common and harder to treat in HCM. Researchers will measure electrical signals in the heart and use advanced imaging. The goal is to find clues …

This study is observing 4,500 people who need pacemakers or similar heart devices. The goal is to create a tool that predicts who is at risk of developing a weakened heart from the device itself. By identifying high-risk patients early, doctors can choose better pacing methods to…

This study follows 150 people aged 7 and older with RYR1-related muscle disorders for 3 to 5 years. Researchers will collect medical history, perform physical exams, and use questionnaires, activity trackers, and tests of heart, lung, and muscle function. The goal is to better un…

Immune checkpoint inhibitors help the body's immune system fight cancer, but in some patients they trigger dangerous heart inflammation and other cardiovascular problems. Researchers are combing through the World Health Organization's global drug safety database, two French natio…

This study will observe 253 adults with heart rhythm problems who are scheduled for procedures like pacemakers or ablation. Researchers will use advanced imaging and AI tools to understand why some patients respond well to treatment while others do not. The goal is to find better…

Takotsubo cardiomyopathy, often called broken heart syndrome, mimics a heart attack and is triggered by intense emotional or physical stress. Researchers at the University of Aberdeen are identifying every diagnosed case in Scotland since 2010 to measure death rates and hospitali…

This study will follow about 2000 adults with a heart condition called ATTR-CM, where abnormal proteins build up and weaken the heart. Researchers want to see how patients do on a drug called vutrisiran in everyday medical care, not just in a controlled trial. They will measure s…

Researchers at the University of Oregon are studying how a protein in skeletal muscle called myosin binding protein C, or MyBP-C, changes with fatigue and aging. The study compares healthy adults aged 18 to 35 with adults aged 65 to 80. Participants perform knee extension exercis…

This observational study will analyze medical records from over 52,000 people with ATTR amyloidosis, a rare disease where abnormal proteins build up in organs. Researchers want to learn about patients' symptoms, treatments, and outcomes in everyday medical practice. No new treatm…

This study aims to develop better MRI methods for imaging the heart and brain, and to describe heart diseases using these new techniques. It will also investigate whether gadolinium, a common MRI contrast agent, stays in the body long after use. Up to 5,000 healthy volunteers and…

This study is testing a new non-invasive device that uses laser speckle to measure blood flow and structure inside the eye. Researchers will compare these images with standard vision tests in 500 people with various retinal conditions. The goal is to see if this technology can be…

This study uses ultrasound to measure muscle, fat, and fluid changes in 121 critically ill pregnant or gynecological patients in the ICU. The goal is to see if these body composition changes are linked to serious outcomes like death, need for a breathing machine, or longer hospit…

This study follows 1,000 adults with myotonic dystrophy type 1 (DM1) over time to measure how the disease progresses. Researchers will track walking speed, hand muscle relaxation, grip strength, heart and lung function, and daily activity. The goal is to establish reliable biomar…

This study aims to find better ways to measure myofascial pain, a common type of muscle pain. Researchers will use MRI and other imaging tools to look at muscle tissue in 100 adults with neck or shoulder pain. The goal is to identify imaging markers that can track how well treatm…

This study follows 50 adults with facioscapulohumeral muscular dystrophy type 2 (FSHD2), a rare inherited muscle disease, for 18 months. Researchers will measure muscle function and severity using standard clinical tests and collect blood samples to look for markers that might pr…

This study follows 65 people with Friedreich ataxia who also have a thickened heart muscle (cardiomyopathy). Researchers will measure changes in heart structure over time using imaging. The goal is to better understand how heart disease progresses in this condition, which could h…

This registry study follows about 100 people with FOP, a rare disease where soft tissue turns to bone. Some participants take palovarotene (an approved treatment), while others do not. Researchers will track side effects, flare-ups, and daily function over time to see how well th…

This study is looking for early signs of heart amyloidosis in Black adults who carry a specific gene change (V122I TTR) that raises their risk. Researchers will use heart MRI scans and blood tests to detect protein buildup before symptoms appear. The goal is to find ways to diagn…

This study is testing a new paddle-shaped catheter designed to map the heart's electrical signals in people with various heart rhythm disorders like atrial fibrillation and ventricular tachycardia. The device has many sensors to create a detailed map of the heart's chambers. The …

This study follows 105 boys from birth to age 3 who have Duchenne muscular dystrophy (DMD), identified through newborn screening. Researchers will measure motor and cognitive skills over time to understand how the disease develops in early childhood. The goal is to gather informa…

This study will collect blood samples from 115 adults with cardiac sarcoidosis to look for tiny particles called exosomes that might show whether the disease is active or quiet. The goal is to develop a simple blood test that could help doctors monitor the condition without needi…

This study looks at whether liver function and blood clotting tests can help doctors better understand and monitor cardiac transthyretin amyloidosis (ATTR-CA), a condition where abnormal protein builds up in the heart. Researchers will compare these tests in people with ATTR-CA a…

This study will enroll 5,000 adults with chronic heart conditions like heart failure, high blood pressure, or aortic stenosis. Participants will wear a Prolaio digital health patch that monitors heart rate, breathing, and other vital signs. The goal is to observe how these condit…

This study follows children with congenital or childhood myotonic dystrophy over time to see how their symptoms change. Researchers will test a range of assessments, from walking speed and hand strength to language and quality of life, to find the best ways to measure the disease…

This study creates a global registry for people with mitochondrial disorders—rare diseases that affect energy production in cells. By collecting health data from 6,000 participants worldwide, researchers aim to understand how these diseases progress and identify the best ways to …

This trial tests whether sharing diaphragm ultrasound results with doctors helps them decide when to remove breathing tubes in ICU patients who have failed their first attempt. Participants are randomly assigned to have their ultrasound results shared or kept hidden from the care…

This study looks at whether levels of a protein called HMGB1 are different in people with LAMA2-related muscular dystrophy, a condition that causes severe muscle weakness. Researchers will measure HMGB1 in blood and muscle samples already collected from about 25 patients. The goa…

This study looks at how energy production inside cells (mitochondria) works in people with cystinosis, a rare disease that causes cystine buildup and early aging. Researchers will measure cell energy activity in 25 patients and compare it to healthy people. The goal is to underst…

This study is testing a comprehensive MRI scan to see if it can better diagnose different heart conditions. Researchers will scan 2,000 adults with suspected or confirmed heart disease. The goal is to create a standard imaging method that improves early detection and risk predict…

This study looks at 200 adults with sick sinus syndrome who need a pacemaker. Researchers will compare two places to put the pacing wire—Bachmann's bundle or the right atrial appendage—to see which one better prevents heart damage. The goal is to learn which site leads to healthi…

This study investigates whether a person's glycolytic power — how efficiently their body uses sugar for quick energy — can predict muscle growth from resistance training. Healthy adults who have not done structured weight lifting in the past year will take a 10-second all-out cyc…

This study investigates post-exertional malaise (PEM), the hallmark symptom of ME/CFS, where even minor activity triggers severe symptom worsening. Researchers will compare 50 ME/CFS patients with 50 healthy volunteers matched by age, sex, and activity level. Participants undergo…

This study looks at a heart condition called cirrhotic cardiomyopathy that can affect people with advanced liver disease (cirrhosis). Researchers want to learn how common it is, what increases the risk, and whether bile acids in the blood play a role. About 440 adults with decomp…

This study follows 400 people in Norway with multiple myeloma, plasma cell leukemia, or AL amyloidosis who are receiving newer immunotherapies like teclistamab, elranatamab, or CAR-T cell therapy outside of clinical trials. Researchers will track how well these treatments control…

This study is a national registry in Hungary that collects medical information from people diagnosed with systemic amyloidosis, a rare disease where abnormal proteins build up in organs. Researchers will look at patient records to understand how the disease starts, how it is diag…

This registry tracks 2,000 adults with congenital heart disease who are on the waiting list for a heart or heart-lung transplant. The goal is to understand why some patients get worse or die while waiting, while others improve enough to be removed from the list. By collecting dat…

This study tests a new PET scan technique to better track heart damage in people with a condition called ATTR-CM, a progressive heart muscle disease. Researchers will scan 140 participants to see if the imaging can show how well treatments are working. The goal is to improve moni…

This study is building a library of information and samples from 60 adults with suspected autoimmune myopathy, a rare muscle disease. Researchers will collect blood, urine, stool, and muscle tissue, along with medical data, to better understand the condition. The goal is to impro…

This study is tracking 1,500 people with cardiac sarcoidosis—a condition where inflammation affects the heart—to learn how doctors currently diagnose and treat it. Researchers will also test whether corticosteroid therapy improves outcomes. The goal is to create better guidelines…

This study is testing whether a special ultrafast ultrasound can better measure muscle changes in boys and men with Duchenne or Becker muscular dystrophy. Researchers will compare muscle stiffness, fat content, and blood flow in 60 participants (patients and healthy volunteers) o…

This study follows children and adults worldwide who have a rare genetic muscle disease called TNNT1 myopathy. Researchers aim to document how the disease progresses over time, including survival and motor milestones. The goal is to identify reliable measures that can be used in …

This study looks at how wearing a shoulder sling changes the way people move and keep their balance during everyday activities like walking or climbing stairs. Researchers will track 30 people—some with shoulder injuries and some healthy—using motion-capture cameras. The goal is …

This study aims to discover new genes that may cause certain cancers to run in families. Researchers will collect blood samples and health information from 1,500 people in families where multiple members have had cancer, especially childhood cancers. The goal is to build a regist…

This study will test whether acupuncture can improve pain and function in people with chronic low back pain caused by myofascial pain syndrome. Sixty-four adults will receive either real acupuncture or sham (fake) acupuncture for eight weeks. Researchers will use full-body PET/CT…

This study will track jaw muscle activity in 42 adults with chronic jaw pain and teeth grinding (bruxism) who use a custom mouth splint at night. Participants will wear a portable device for 24 hours to measure muscle activity before starting the splint, two weeks later, and afte…

This study is testing how to safely perform skin allergy tests in people receiving biotherapies for cancers and blood disorders. Researchers will find the highest concentration of each biotherapy that does not cause a skin reaction in 9 out of 10 patients. The goal is to establis…

This study looks at how to reduce the long and frustrating journey to a diagnosis for people with developmental abnormalities. Researchers will review past cases, collect new blood or skin samples, and use advanced genetic testing. The goal is to understand why some people remain…

This study looks at how emotional intelligence—the ability to understand and manage emotions—relates to chronic orofacial pain (pain in the face or mouth lasting over three months). Researchers will compare 88 adults with and without this pain using questionnaires about emotions,…

This study is collecting skin and blood samples from 830 people with chronic inflammatory skin diseases like psoriasis, eczema, and lupus. Researchers want to find biological markers that can predict how well a person will respond to treatment after one year. The goal is to move …

ATROPOS is an international study collecting data from 1,500 cancer patients who developed heart inflammation (myocarditis) after receiving immune checkpoint inhibitors. Researchers will analyze how different immunosuppressive treatments, such as steroids, affect survival rates a…

This study is for children up to 4 years old with congenital myotonic dystrophy type 1 (CDM). Researchers want to find better ways to measure how the disease affects motor skills and language, and to identify biological markers. The goal is to improve future clinical trials and c…

This study is testing whether a watch-like device worn on the ankle can measure how well toddlers with Duchenne muscular dystrophy (DMD) move during their daily activities. Researchers will ask 30 boys with DMD and 30 without (ages 1-3) to wear the device for three 28-day periods…

This study looks at how people with ME/CFS (chronic fatigue syndrome) and people with heart conditions feel after a bike exercise test. Researchers want to see if the type and timing of symptoms after exercise are different between the two groups. 80 adults will take part, and th…

This study aims to learn why myotonic dystrophy type 1 affects children differently than adults, and why symptoms vary even within the same family. Researchers will observe 100 children (ages 0-17) through video calls and simple at-home activities, and analyze their genes from a …

This study aims to learn how well heart scans (MRI, ultrasound, PET) can detect and predict outcomes in people with suspected or known inflammatory heart diseases. Researchers will follow 5,000 adults for one year to see how often heart involvement is confirmed and how it affects…

This early-stage trial is studying how the drug dobutamine affects the heart in 15 people with a rare condition called wild-type transthyretin amyloid cardiomyopathy (ATTRwt). Participants will receive increasing doses of dobutamine while doctors measure heart function using ultr…

This study looks at whether inflammation inside the heart contributes to arrhythmogenic right ventricular cardiomyopathy (ARVC), a rare heart condition. Researchers will measure inflammatory markers in blood and heart tissue from 80 participants, including people with ARVC and he…

This study is examining heart valve tissue from 100 people undergoing surgery for aortic stenosis to see how often ATTR amyloid deposits are present. Researchers will use mass spectrometry to identify the type of amyloid. The goal is to better understand the link between amyloido…

This early-stage study tests a brain implant called BrainGate in just 3 people with severe paralysis or speech loss from conditions like ALS or spinal cord injury. The goal is to see if the device is safe and can let users control a computer cursor or other tools by thought alone…

This study looks at why teenagers who had surgery for a diaphragmatic hernia right after birth may find it hard to take part in sports and physical activities. Researchers will interview 20 teens and their parents, and use questionnaires to identify what helps or hinders their pa…

This study looks at why some people with heart failure don't respond well to diuretics (water pills). Researchers will give 50 participants either ammonium chloride or a placebo, along with standard diuretics, to see how it affects kidney function. The goal is to understand the b…

This study looks at whether CD34+ stem cells, which help maintain tiny blood vessels in the heart, are linked to how well the heart recovers in people with Takotsubo syndrome (also known as broken heart syndrome). Researchers will measure heart function in 40 participants at the …

This registry collects information from people with inherited retinal diseases, like retinitis pigmentosa and Stargardt disease. Participants share their symptoms, family history, and genetic test results online. The goal is to help researchers understand these rare diseases and …

This study is testing whether a smartphone app can accurately measure how people walk, without needing special markers or equipment. Researchers will compare the app's measurements to a standard motion-capture system in 30 volunteers—some with neuromuscular diseases like SMA or m…

This study tests a new, less invasive way to check diaphragm function in patients on breathing machines. Researchers will use ultrasound to measure diaphragm movement during a spontaneous breathing trial. The goal is to see if this method can help doctors decide when patients are…

This study is a worldwide registry that follows 5,000 adults with breast cancer, blood cancers, or those on immune checkpoint inhibitors. Researchers collect medical records, lab results, and imaging to identify who is at risk for heart problems during or after cancer treatment. …

This study follows 450 people with FSHD for at least 3 years to measure how the disease affects walking, arm use, breathing, and other daily activities. The goal is to improve patient care and help researchers design better clinical trials by understanding what changes in movemen…

This study is building a registry of 1,211 people in Brazil who have inherited heart conditions like cardiomyopathy, arrhythmias, and Marfan syndrome. Researchers will collect DNA samples and medical information to discover which genes are most commonly affected and how often the…

This study looks at a specific gene (IGLV1-44) to understand why it causes two different diseases: AL amyloidosis and POEMS syndrome. Researchers will analyze blood and bone marrow samples from 100 adults with these conditions or multiple myeloma. The goal is to find genetic patt…

This study looks at people who have long-lasting health problems after getting the COVID-19 vaccine, a condition called Post-Acute COVID-19 Vaccination Syndrome (PACVS). Researchers will track symptoms over eight months and test blood and blood vessel function to find biological …

This study is collecting information from 30 patients with an extremely rare ovarian cancer called primary ovarian leiomyosarcoma. Researchers will look at past and new cases to find patterns in how the cancer is diagnosed and treated. The goal is to create better guidelines for …

This early study tests whether a brain implant can help people with severe paralysis from conditions like ALS, spinal cord injury, or stroke control devices using their thoughts. The implant records brain signals to decode text or synthesized speech. Only 3 adults will participat…

This study aims to find out if genetics play a role in who gets broken heart syndrome (Takotsubo cardiomyopathy), a condition that mimics a heart attack and is often triggered by intense stress. Researchers will collect blood samples from 700 people diagnosed with the condition i…

This study is following about 362 adults with a type of heart disease called obstructive hypertrophic cardiomyopathy who are taking the drug mavacamten. Researchers want to see how the drug works in real doctor's offices, not just in strict research settings. They will track thin…

This study looks at whether brain wave tests (EEG) done before and after heart surgery in babies under 1 year old can predict later learning or behavior problems, such as autism or ADHD. About 50 infants will be followed to age 2. The goal is to find early warning signs so that c…

This study aims to find better ways to detect lung scarring (interstitial lung disease) early in people with connective tissue diseases like scleroderma or rheumatoid arthritis. Researchers will analyze tiny particles in the blood called extracellular vesicles and use AI to exami…

This study looks back at the last 10 years in Denmark to understand how many people use breathing machines at home through a tube in their windpipe. Researchers will track which diseases lead to this need and how many patients survive one year after starting. The goal is to spot …

This study will use advanced heart MRI scans on up to 100,000 people with known or suspected heart disease. The goal is to learn how these scans can help doctors better understand, diagnose, and treat different heart conditions. Researchers will track participants' health over ti…

This study is looking at 80 people with a genetic heart condition called arrhythmogenic cardiomyopathy, which can cause sudden cardiac death. Researchers will use a special PET-MRI scan to see if there is inflammation in the heart muscle, which is hard to detect with standard tes…

This study is building a large registry of 5,000 people with AL amyloidosis, a rare disease where abnormal proteins build up in organs. Researchers will collect real-world data on how the disease progresses and how current treatments affect it. No new drug is being tested; the go…

This study is creating a large European registry of 400 newly diagnosed AL amyloidosis patients. Researchers will collect medical data and blood samples to study the disease using advanced technology. The goal is to better understand how AL amyloidosis develops and progresses, wh…

This study is creating a network of hospitals in Italy to find and treat AL amyloidosis earlier. Researchers will screen people with certain blood conditions (MGUS or smoldering myeloma) using special tests. The goal is to speed up diagnosis, connect patients to the right care, a…

This study follows 200 adults with ATTRwt amyloidosis who are taking tafamidis to see how often they develop heart rhythm problems that need a pacemaker or defibrillator. Researchers will also track changes in diuretic use. Participants are checked every six months as part of rou…

This study aims to create a tool that uses stress heart MRI and routine medical data to predict which patients with dilated cardiomyopathy (a weakened, enlarged heart) are at highest risk for serious problems like heart failure or sudden cardiac death. Researchers will follow 2,0…

This study tests whether special heart MRI scans can find early signs of heart damage in cancer patients getting anthracycline chemotherapy. Current tests often miss this damage until it's advanced. About 93 adults will get several MRI scans before, during, and after treatment. T…

This study follows 500 people who are already scheduled for routine shoulder or knee surgery for sports injuries. Researchers will track their recovery over two years using questionnaires about pain and function. The goal is to find out which factors—like injury type or surgical …

This study collects information from up to 250 patients with lysosomal storage diseases (like certain forms of MPS, Pompe, Gaucher, and Wolman disease) to understand how these conditions develop and respond to treatments given before birth. Researchers will track symptoms, lab re…

This study looks at whether a movement screening test can accurately detect changes in Achilles tendon pain in runners over 40. Forty senior runners with Achilles tendinopathy will be randomly assigned to two physiotherapy groups, one with extra stretching. The goal is to see if …

This study aims to understand why myotonic dystrophy type 1 affects people so differently. Researchers will remotely assess muscle strength, memory, and activity in 1,000 participants, and analyze their DNA from a blood sample. All activities are done from home using a mailed too…

This study collects information from people with dermatomyositis to better understand how the disease affects their skin and quality of life. Researchers will use a special scoring tool called CDASI to measure skin changes over time. The goal is to improve knowledge about the dis…

This study aims to improve how doctors predict the risk of sudden cardiac death or heart failure in people with inherited heart diseases. Researchers will use artificial intelligence to analyze data from 1,000 participants, including medical history, ECGs, imaging, genetic tests,…

This study is creating a database of up to 5,000 people in Ohio with plasma cell disorders like multiple myeloma and amyloidosis. Researchers will track treatments, outcomes, and quality of life, while also offering patients access to expert consultations and information about su…

This study aims to create a large collection of blood samples and medical information from 800 people with autoimmune or inflammatory skin diseases. Researchers will use these samples to find new biomarkers and better understand what causes these conditions. The goal is to help d…

This study aims to create a collection of blood and urine samples from 100 people with Noonan syndrome. Researchers will use these samples to study how the disease works and look for factors that predict how it will progress. The study does not test any treatment, but may help gu…

This study looks at how different amounts of supervised indoor cycling change gut bacteria and related substances in healthy men aged 18 to 45. Participants will be placed into one of three groups: no structured training, moderate cycling, or high-load cycling. Researchers will m…

This study aims to collect blood samples and medical data from 3000 people with autoimmune diseases like lupus, scleroderma, and myositis. Researchers will use these samples to find new biomarkers and better understand these conditions. The goal is to help develop new treatments …

This study investigates whether serious muscle weakness, known to affect ICU patients, also occurs in people hospitalized outside the ICU. Researchers will follow about 600 adults in Italian hospitals, half with organ failure and half without, to see how often weakness develops a…

This study aims to learn how Pompe disease affects the brain and nerves over the long term. Researchers will use brain scans, thinking and language tests, and muscle checks in 45 people with Pompe disease. Participants will be followed for 3 to 6 years. No new treatment is being …

This study aims to identify genetic factors that affect how severe muscle and heart problems become in people with LMNA gene mutations. Researchers will collect skin and muscle samples from 40 participants and use advanced DNA and RNA analysis to look for protective or aggravatin…

This study investigates how a sugar modification called N-glycosylation affects AL amyloidosis, a rare disease where abnormal proteins build up in organs. Researchers will analyze blood and bone marrow samples from 100 adults with related conditions to better understand the disea…

This study looks at how exercise training changes muscle cells in people with mitochondrial myopathy, a rare disease that affects energy production. Researchers will compare a trained leg to an untrained leg in the same person, and also compare results with healthy volunteers. Th…

This study looks at whether quick, easy walking tests can replace the current complex and time-consuming exam used to track walking problems in people with facioscapulohumeral muscular dystrophy (FSH). Researchers will compare results from simple tests like the 6-minute walk with…

This study looks at whether using a uterine manipulator during hysterectomy changes sexual function three months after surgery. Sixty women having a hysterectomy for non-cancer reasons will be randomly assigned to have the surgery with or without the tool. The main goal is to com…

This study is collecting tissue samples and medical data from 800 high-risk sarcoma patients to develop personalized treatment approaches. Researchers will analyze genetic and protein profiles to better predict which patients benefit from chemotherapy before surgery. The goal is …

This study is testing different ways to measure muscle strength and movement in children with a rare muscle disease called FSHD. The goal is to find the best tests to use in future studies of new treatments. About 80 children aged 5 to 17 with confirmed FSHD will take part. The r…

This study is looking at whether a molecule in the blood called miR-1 can help doctors understand how muscle diseases like Duchenne muscular dystrophy and myotonic dystrophy are progressing. Researchers will compare miR-1 levels in 104 people, including patients with different mu…

This study is collecting information from 600 people with chronically high levels of eosinophils (a type of white blood cell) or related organ damage. Researchers want to describe the different symptoms, disease patterns, and genetic factors involved. The goal is to better unders…

This study looks back at medical records of 100 children with a genetic condition called RASopathy that causes severe heart thickening and heart failure. Researchers want to collect detailed information on how the disease progressed in the past. This will help design future studi…

This observational study will follow 2000 children and adults with imprinting disorders—rare genetic conditions like Silver-Russell and Prader-Willi syndromes. Researchers aim to describe the natural history of these diseases and identify common metabolic profiles, risks for obes…

This study follows about 70 people who have already received KYV-101, a CAR T-cell therapy, for autoimmune diseases like lupus and multiple sclerosis. Researchers will monitor participants for up to 15 years to track any delayed side effects, new cancers, or other health problems…

This study looks at 1,000 women to see how ultrasound features of adenomyosis (a condition where the uterine lining grows into the muscle wall) match up with their symptoms like pain and heavy bleeding. Participants get one routine ultrasound and fill out a symptom questionnaire.…

This study uses advanced heart MRI scans to look at blood flow patterns in people with different types of hypertrophic cardiomyopathy (thick heart muscle), including rare forms like Anderson-Fabry disease and cardiac amyloidosis. Researchers will also study first-degree relatives…

This study aims to create a national registry for people with Myotonic Dystrophy Type 1 (DM1) in Spain. Researchers will collect clinical data, genetic information, and patient reports from up to 3,000 participants. The goal is to better understand the disease and identify people…

This study is creating a large registry and tissue bank for people with mitochondrial disorders. Researchers will collect medical information and samples from up to 1,000 participants, including those diagnosed with or suspected to have a mitochondrial disease. The goal is to gat…

This study asks 700 people with rare myositis diseases (and their caregivers) what research questions matter most to them. Through online surveys and forums, the goal is to create a patient-driven list of priorities to guide future studies and funding. No drugs or treatments are …

This study looks at people who developed heart inflammation (myocarditis or pericarditis) after getting a COVID-19 vaccine. Researchers will review medical records and follow up with 400 participants for up to 4 years to understand long-term health effects. The goal is to learn m…

This study is looking for 75 adults with myotonic dystrophy (a muscle disease) and healthy volunteers to test new muscle imaging techniques. The goal is to find better ways to measure muscle changes over time, which could help future treatment studies. Participants will undergo M…

This observational study uses 3D echocardiography to compare heart shape, blood flow, and function between 80 endurance athletes and 80 sedentary people. Researchers aim to understand how long-term exercise remodels the heart's structure and improves its performance. Participants…

This study focuses on people with ME/CFS or Long COVID who are too sick to leave home. Researchers will provide monthly in-home medical visits and create personalized care plans. The goal is to see if this approach improves patients' health and eases the burden on their caregiver…

This study follows 1500 people who already have an implantable cardioverter-defibrillator (ICD) to prevent sudden cardiac death. Researchers will track heart function, genetics, and blood markers to find better ways to predict who is at highest risk. The goal is to improve how do…

This study looks at whether simple measurements—like thigh circumference and muscle stiffness—can reliably track muscle health in people with COPD. Researchers will measure 30 patients before and after a pulmonary rehabilitation program. The goal is to find an easy way to monitor…

This study looks at fat tissue surrounding the heart arteries in 60 patients who had a heart transplant. Researchers want to see if the size of fat cells close to the artery differs from those farther away, and if that relates to artery disease. The goal is to better understand h…

This study aims to describe a rare condition called panniculitis (inflammation of the fat under the skin) in people with dermatomyositis. Researchers will examine 20 participants aged 1 year and older to identify unique clinical and microscopic features. The goal is to better dis…

This study looks at how often the diaphragm (the main breathing muscle) stops working properly after lung surgery done without a breathing tube. About 120 adults having this type of surgery will have their diaphragm checked with ultrasound right after surgery and again 24 hours l…

This study is testing whether artificial intelligence can help doctors better classify and predict the behavior of rare smooth muscle tumors of the uterus, including those that are hard to diagnose (called STUMP). Researchers will train AI models on digital images of tumor tissue…

This study is a registry that collects health information from people in Switzerland who have neuromuscular disorders like SMA, DMD, BMD, and others. It aims to track symptoms, treatments, and outcomes over time to help researchers and doctors improve care. No new treatments are …

This study aims to understand why the right side of the heart fails in people with heart failure and high blood pressure in the lungs. Researchers will look at heart tissue samples from 50 adults to find the best ways to diagnose and treat this condition. Participants must be bet…

This registry enrolls pregnant women or those planning pregnancy who have autoimmune diseases like lupus or rheumatoid arthritis. Researchers at Duke University will track health data to find ways to improve outcomes for mothers and babies. The goal is to better understand risk f…

This study is testing whether a detailed heart MRI (called T1 mapping) can better predict who will develop heart disease, heart failure, or long COVID complications. Researchers will scan 6000 adults without known heart disease and follow them over time. The goal is to see if sub…

This study follows 100 adults who needed ECMO support for severe heart or lung failure. Researchers will track muscle size, strength, and quality of life over time to understand why some patients develop lasting weakness. No new treatments are being tested; the goal is to gather …

This study looks at whether tiny particles released by cells into the blood, called extracellular vesicles, can help detect a type of heart disease called ATTR amyloidosis earlier. The disease often goes unnoticed until serious damage occurs. Researchers will compare these partic…

This pilot study is testing a new type of PET/CT scan that uses a radioactive tracer called 68Ga-FAPI to detect active scarring (fibrosis) in the hearts of 30 people with heart failure. The goal is to see if this scan can spot early or ongoing scarring better than current MRI met…

The GEIST registry is an international study collecting information from 500 people with Takotsubo syndrome, also known as 'broken heart syndrome.' Researchers are gathering data on symptoms, test results, treatments, and outcomes to better understand this temporary heart conditi…

This study is not a treatment trial. It aims to collect blood samples from 400 children and their relatives who have rare autoimmune or autoinflammatory diseases (like lupus or juvenile arthritis). Researchers will analyze the samples to find genetic mutations that cause these co…

This study is building a global database of 1000 people with homozygous familial hypercholesterolemia (HoFH), a rare inherited condition causing extremely high cholesterol from birth. Researchers are gathering de-identified medical data from clinics around the world to better und…

This study compares two ways to manage fluids in cirrhosis patients with severe sepsis or septic shock. One method uses a bedside heart ultrasound (POCUS) to guide fluid amounts, while the other follows standard sepsis guidelines. The goal is to see if ultrasound-guided care redu…

This study uses special wearable devices to monitor how babies and children with spinal muscular atrophy (SMA) or Duchenne muscular dystrophy (DMD) move at home. About 100 children will wear a sensor-filled jumpsuit or an ankle monitor to track their motor skills over up to 30 mo…

This study aims to identify genes that make a person more likely to develop congenital diaphragmatic hernia (CDH), a birth defect where the diaphragm doesn't form properly. Researchers will collect DNA samples from 3,000 people with CDH or a family history of it, along with their…

This study looks at why about one-third of people with a heart condition called cardiac amyloidosis continue to get worse even with medication. Researchers will use special heart scans (ultrasound, MRI, and PET) at the start and after one year to see what is happening inside the …

This study at Johns Hopkins University is looking at how aging cells (senescent cells) contribute to uterine fibroids, which are non-cancerous growths in the uterus. Researchers will analyze tissue samples from 150 women with fibroids to map these cells and their signals. The goa…

This study creates a registry of 5,000 adults aged 50 and older with blood cancers like multiple myeloma and lymphoma. Researchers will track frailty, muscle loss, and other age-related health issues to better understand this group's needs. The goal is to gather information that …

This study tracks 10 people in Guadeloupe with dermatomyositis, a rare autoimmune disease that affects skin and muscles. Researchers want to see how severe the disease is at diagnosis, including muscle weakness, organ problems, and cancer links. No new treatment is being tested —…

This study aims to find a way to predict which cancer patients will experience side effects from immunotherapy. These side effects happen when the immune system attacks healthy tissues, similar to autoimmune diseases. Researchers will collect blood samples from 300 cancer patient…

This study tests a brain implant that reads signals from the brain to help people who have lost the ability to speak fluently communicate through a computer just by trying to talk. It involves up to 2 participants with conditions like spinal cord injury or ALS. The main goal is t…

This study aims to find genetic differences that make some cancer patients more likely to develop a rare but life-threatening heart inflammation (myocarditis) from immune checkpoint inhibitors. Researchers will compare the DNA of 100 patients who had this side effect with 400 who…