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 Phase 2 trial tests a personalized cell therapy called Descartes-08 for people with dermatomyositis or antisynthetase syndrome, two rare autoimmune diseases that cause muscle weakness and inflammation. The therapy uses a patient's own immune cells, modified to target and red…

This early-stage trial is testing a new drug called DNL952 in 32 adults with late-onset Pompe disease. The main goal is to check if the drug is safe and how the body processes it. Participants will receive the drug through an IV infusion at different doses.

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 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 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 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 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 trial is testing whether a lower weekly dose of the corticosteroid prednisolone (5 mg/kg per week) can be as effective as the standard higher dose (10 mg/kg per week) in preserving motor function in young boys with Duchenne muscular dystrophy (DMD). The study will enroll boy…

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 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 study tests the drug acalabrutinib in people with chronic lymphocytic leukemia (CLL) who also have moderate to severe heart failure. Researchers want to see if acalabrutinib causes fewer heart-related side effects compared to other standard treatments. About 60 adults will t…

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 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 study tests a one-time gene therapy called SGT-003 in 80 boys with Duchenne muscular dystrophy who can still walk. The goal is to see if it helps them move better, like standing up faster and climbing stairs. Participants will receive either the therapy or a placebo first, t…

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 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 study is testing a pacemaker lead placed in a specific area of the heart called the left bundle branch area. The goal is to see if this placement is safe and helps the heart beat more naturally in people with slow heart rates. About 140 participants will get the lead and be …

This study follows 60 adults who receive a total shoulder replacement using the PRIMA humeral stem, either in an anatomic or reverse configuration. The goal is to measure changes in shoulder function, pain, and quality of life over 24 months, as well as how well the implant holds…

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 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 study tests a small device called C-MIC that is implanted without open-heart surgery to help people with heart failure. It includes 22 adults with weak heart pumping (ejection fraction between 25% and 50%) despite standard medications. The device delivers microcurrents to th…

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 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 tests a new drug called ARO-DM1 in 78 adults aged 18-65 with type 1 myotonic dystrophy, a genetic muscle disorder. The goal is to check safety and how the body processes the drug. Participants receive either ARO-DM1 or a placebo by IV or injection. It is an early-stage…

This study tests an experimental drug called ARO-DUX4 in people with facioscapulohumeral muscular dystrophy type 1 (FSHD1), a genetic condition that causes muscle weakness. The main goal is to check the drug's safety and how the body processes it. About 60 adults and teens will r…

This study tests whether bromocriptine, a drug that stops breastfeeding, can improve heart function in women newly diagnosed with peripartum cardiomyopathy (a type of heart failure after childbirth). 200 women will receive either bromocriptine or a placebo, plus standard heart fa…

This study tests a compact heart-lung machine called Cardiohelp in 50 children with severe heart failure. The device supports the heart for up to 30 days while kids recover or await a transplant. The trial also compares two blood thinners to see which causes fewer bleeding or clo…

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…

This early-stage trial tests a single infusion of CRISPR-edited immune cells (CTX112) from a healthy donor in 80 adults with severe lupus, scleroderma, or myositis that hasn't responded to standard treatments. The goal is to see if the cells are safe and can reduce disease activi…

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 study tests a new treatment called NKX019 for people with autoimmune diseases like systemic sclerosis, rheumatoid arthritis, and vasculitis. NKX019 uses specially engineered immune cells to target and calm overactive B cells that cause the disease. The goal is to see if it i…

This early-stage trial tests a single dose of a gene therapy called NVC-001 in 21 adults with a genetic form of dilated cardiomyopathy (LMNA mutation). The therapy uses a harmless virus to deliver a modified gene into heart cells, aiming to protect the heart muscle. The main goal…

This trial tests whether adding a single injection of the anti-inflammatory drug meloxicam to a standard jaw joint washout procedure (arthrocentesis) improves outcomes for people with painful jaw joint problems. Half of the 50 adult participants will receive the combined treatmen…

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 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 Phase 3 study tests whether the drug mavorixafor can reduce serious infections and increase neutrophil levels in people with chronic neutropenia—a condition where the body doesn't make enough infection-fighting white blood cells. About 176 participants will receive either ma…

This study looks at whether anti-inflammatory drugs (like anakinra or canakinumab) can reduce painful flare-ups and prevent extra bone growth in people with a rare genetic condition called fibrodysplasia ossificans progressiva (FOP). Eleven people with severe FOP will be tracked …

This phase 2 trial tests whether intravenous immunoglobulin (IVIG) can help people with a rare autoimmune muscle disease called anti-HMGCR immune-mediated necrotizing myopathy. Twelve participants will receive either IVIG or a placebo every 4 weeks for 12 weeks, then all may rece…

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 study is testing whether a procedure called ultrasonic tenotomy can help people with a partial tear of the supraspinatus tendon in the shoulder. The procedure uses ultrasound to guide a needle-like device that breaks up and removes damaged tendon tissue, potentially stimulat…

This study tests a small, refillable implant placed in the eye that slowly releases medication for wet age-related macular degeneration (nAMD). It aims to see if refilling the implant every 24 weeks works as well as getting a standard eye injection every 4 weeks. About 68 Chinese…

This study tests an experimental daily pill, danicamtiv, in 332 people with genetic or familial dilated cardiomyopathy (a weakened, enlarged heart). Participants take the drug or a placebo for about 6 months to see if it improves heart function and exercise ability. The goal is t…

This early-phase trial is testing a new biologic drug called XmAb657 in 60 people, including healthy volunteers and those with autoimmune diseases like scleroderma, Sjögren's disease, and idiopathic inflammatory myopathies. The main goal is to check safety and how the body handle…

This study tests whether combining two drugs, teclistamab and daratumumab, can help people newly diagnosed with both multiple myeloma and light chain amyloidosis. About 30 adults will receive the treatment for up to 24 cycles. The goal is to see if the combo improves survival wit…

This study is for children and young adults (up to age 30) with a rare type of soft tissue cancer called non-rhabdomyosarcoma. It tests whether adding the drugs pazopanib and selinexor to standard chemotherapy can improve outcomes. Participants receive treatment tailored to their…

This early-stage study tests a new treatment called RD06-05, a universal CAR-T cell therapy, in up to 84 adults with autoimmune diseases like lupus, vasculitis, and scleroderma. The therapy targets and removes faulty immune cells to help control the disease. The main goal is to c…

This early-stage trial is testing a new drug called REC-617 in 15 people with a rare type of sarcoma (leiomyosarcoma) that has spread or cannot be removed and has not responded to prior treatments. The drug targets a protein (CDK7) that helps cancer cells grow. The main goal is t…

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 phase 2 trial tests whether the drug letrozole can extend the time before uterine leiomyosarcoma returns after initial treatment. About 40 women with early-stage disease will be randomly assigned to take letrozole daily or to be observed without treatment. The main goal is t…

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 phase 3 trial tests whether injecting a concentrate of your own bone marrow (BMAC) into the jaw joint during arthroscopy reduces pain and damage better than a standard steroid shot. Twenty adults with long-lasting TMJ pain will be randomly assigned to one of the two treatmen…

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 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 early-stage trial is testing a drug called RAY121 in 144 people with one of six immune diseases, including antiphospholipid syndrome and bullous pemphigoid. The drug works by blocking a specific part of the immune system (the complement pathway) that can cause tissue damage.…

This study compares two methods to control bleeding during laparoscopic myomectomy (surgery to remove fibroids from the uterus). One method uses a temporary tourniquet to block blood flow, and the other injects a medicine called epinephrine into the uterus to narrow blood vessels…

This phase 1 trial tests a new cell therapy called ICG318 CAR-T in adults with refractory inflammatory myopathy, a condition where the immune system attacks muscles, causing weakness and inflammation. The therapy uses a patient's own immune cells, engineered to target specific ma…

This early-phase trial tests a new drug called STC-15, either alone or combined with an immunotherapy drug (toripalimab), in people with advanced solid tumors or certain sarcomas. The study aims to check safety and whether the drugs shrink tumors. About 107 participants will be e…

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 tests a medicine called surlorian in 28 adults with a rare genetic muscle disease (RYR1-related myopathy) that causes weakness. The goal is to see if surlorian improves muscle strength and function, like standing from a chair or walking. Participants will be randomly a…

This early-stage trial tests an experimental cell therapy called NEUK203-215 in adults aged 18-65 with severe autoimmune diseases like lupus or scleroderma that have not responded to other treatments. The therapy uses immune cells from a healthy donor, engineered to target and at…

This study tests whether injecting a mixture of hyaluronic acid and I-PRF (a concentrate of healing factors from your own blood) into the jaw joint, after a cleaning procedure called arthrocentesis, can reduce pain and improve jaw movement in people with a displaced disc. Partici…

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 trial compares standard rotator cuff repair surgery to the same surgery plus a BioEnthesis patch, designed to help tendon reattach to bone more strongly. About 100 adults aged 18–65 with reparable rotator cuff tears will take part. The main goal is to see if the patch reduce…

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 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 study compares two approaches during reverse shoulder replacement: repairing the subscapularis tendon (part of the rotator cuff) or leaving it unrepaired. The goal is to see which method leads to better shoulder function, strength, and lower risk of dislocation. Adults aged …

This study looks at whether people with rheumatic diseases (like rheumatoid arthritis or lupus) can safely stop their immunosuppressant medications for a shorter time before elective shoulder replacement surgery. The goal is to see if this approach lowers the chance of disease fl…

This trial investigates whether empagliflozin, a drug originally used for diabetes, can help children with heart failure. About 60 children aged 1 month to 18 years with confirmed heart failure will receive either standard care plus empagliflozin or standard care alone for 90 day…

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 tests whether adding resilience training—a mix of group therapy and relaxation exercises—to standard rehabilitation helps people recover from Takotsubo cardiomyopathy (broken heart syndrome) or a recent heart attack. Participants attend two group therapy sessions and t…

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 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 study tests a personalized approach for people with AL amyloidosis who have a specific genetic change called t(11;14). All 41 participants start with a standard three-drug combination. If their blood responds quickly within a week, they continue that treatment; if not, they …

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 early study tests whether a second infusion of a patient's own modified immune cells (CAR-DC) is safe and can improve heart function in 5 adults with end-stage dilated cardiomyopathy. Participants had already received one dose but saw their heart function worsen again. The g…

This early-stage trial tests a new cell therapy for people with heart failure caused by blocked arteries. The treatment uses the patient's own immune cells, engineered to target scar tissue in the heart. The study will check safety and whether it can improve heart function in 15 …

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 follows adults who have received a Tipmed shoulder replacement to see how well the implant works and if any problems arise over the first year. Participants have conditions like severe arthritis, rotator cuff damage, or a broken shoulder bone. Researchers track shoulde…

This early-phase trial is testing a new drug called MTS109, made with mRNA technology, for people with moderate to severe autoimmune diseases like lupus and scleroderma. Fifteen participants will receive several injections over a month to see if it is safe and tolerable. The goal…

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 study tests a single intravenous dose of a gene therapy called GNR-097 in boys aged 4 to 9 with Duchenne muscular dystrophy (DMD). The therapy uses a harmless virus to deliver a shortened version of the dystrophin gene, which is missing or faulty in DMD. The trial aims to se…

This trial investigates whether sulforaphane, a nutrient found in broccoli, can reduce heart damage caused by the chemotherapy drug doxorubicin in breast cancer patients. About 70 participants will receive either sulforaphane or a placebo alongside their chemotherapy. The study m…

This study tests a procedure called FETO, done during pregnancy, for babies with a severe congenital diaphragmatic hernia (CDH). The goal is to see if FETO helps more babies survive after birth and have fewer long-term health issues compared to standard care. The study will enrol…

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 is testing a drug called mavacamten in adults with a type of heart disease called obstructive hypertrophic cardiomyopathy (HOCM) in Japan. The goal is to see if mavacamten can improve heart function and symptoms when added to their current treatment. About 36 participa…

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 gene-editing medicine called PBGENE-DMD in 18 boys aged 2 to 7 with Duchenne muscular dystrophy. The treatment aims to correct the genetic mistake that causes the disease, potentially restoring muscle strength. Researchers are first checking if …

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 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 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 is testing a small, leadless pacemaker placed in the heart's upper chamber (atrium) for people with sinus node dysfunction, a condition where the heart's natural pacemaker doesn't work properly. Participants receive the Aveir AR pacemaker and a loop recorder to track h…

This early-phase trial is testing a new type of gene therapy called CAR 70-BCMA dual-target CAR-T for people with relapsed or refractory plasma cell neoplasms, including multiple myeloma. The therapy involves taking a patient's own immune cells, re-engineering them to recognize a…

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 early-phase trial tests a personalized cell therapy called BCMA-targeted CAR-T cells in 30 adults with relapsed or refractory light chain amyloidosis, a rare disease where abnormal proteins build up in organs. The treatment uses the patient's own immune cells, modified to at…

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 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 whether the drug Ivonescimab can safely shrink or control advanced leiomyosarcoma, a rare soft-tissue cancer. About 20 adults who have already tried 1-3 prior treatments will receive the drug. The goal is to see if it slows tumor growth with few side effects.

This early-stage study tests a new drug, GT801, in 22 adults with moderate to severe autoimmune diseases that haven't responded to standard treatments. The main goals are to check safety and find the right dose. Researchers will also look for signs that the drug helps control the…

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 a new device called SINEFIX to repair small rotator cuff tears (up to 2 cm) in the shoulder. About 60 adults who need surgery for this condition will receive the implant. The goal is to see if it improves shoulder function and reduces pain compared to before…

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 phase 2 trial is testing elacestrant, a pill that breaks down estrogen receptors, in 30 people with advanced uterine sarcomas that are fueled by estrogen. The goal is to see if the drug can shrink tumors or slow their growth. Participants must have tumors that strongly expre…

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 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 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 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 is testing whether a drug called tadalafil (often used for erectile dysfunction) combined with a home cycling program can help boys with Duchenne muscular dystrophy. The drug aims to improve blood flow to muscles, which is often poor in DMD, while exercise builds stren…

This study tests the long-term safety and effectiveness of dazukibart in people with dermatomyositis or polymyositis, rare diseases that cause muscle weakness and rashes. Participants who completed a previous study can join. Some will receive the drug via IV every 4 weeks for abo…

This study tests vosoritide, a protein that stimulates bone growth, in 30 children with Noonan syndrome who are not growing well despite growth hormone treatment. The goal is to see if vosoritide can safely increase their growth rate over six months. Participants will receive one…

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 early-phase trial is testing a new cell therapy called QH103 for people with severe autoimmune diseases like multiple sclerosis and myasthenia gravis that haven't responded to standard treatments. The therapy uses specially engineered immune cells to target and attack the fa…

This early-stage study tests a new treatment called HBI0101 CAR T for people with severe autoimmune diseases like lupus, multiple sclerosis, and rheumatoid arthritis that haven't improved with standard treatments. The main goal is to find a safe dose and check for side effects in…

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 study follows over 1,000 people who receive a shoulder replacement with FX Shoulder Solutions systems. Researchers will track how often the implant needs to be replaced (revision) and measure pain, movement, and quality of life for up to 10 years after surgery. The goal is t…

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 tests a new treatment called anti-CD19/BCMA universal CAR-T cells for people with severe autoimmune diseases like rheumatoid arthritis, lupus, and scleroderma. The therapy aims to reset the immune system by targeting and removing faulty B cells. About 72 adults will re…

This study looks at whether adding a small amount of a patient's own processed fat tissue during arthroscopic rotator cuff repair can improve healing and reduce the chance of the tendon tearing again. About 52 adults with full-thickness tears will be followed for at least 5 years…

This study tests whether stem cells from donated umbilical cords can safely help heal partial rotator cuff tears. 21 adults with shoulder pain for at least 3 months who haven't improved with other treatments will receive a single injection of stem cells into the tear. Researchers…

This study tests whether a special graft made from donated placental tissue, called ActiveMatrix, can help the shoulder tendon heal better after rotator cuff repair surgery. About 72 people with a full-thickness tear in 1 or 2 tendons will be randomly assigned to get either a low…

This study tests whether a three-drug combination (SGLT2 inhibitor, ARNI, and MRA) can improve heart structure, function, and symptoms in 50 people with advanced HFpEF, a type of heart failure where the heart is stiff but pumps normally. Participants will receive either the tripl…

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 early study is testing a new treatment called CD19 CAR-T therapy for people with severe autoimmune diseases like lupus and scleroderma that haven't improved with standard medications. The therapy involves taking a patient's own immune cells, reprogramming them to target and …

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 phase 2 trial tests whether dapagliflozin, a drug used for diabetes, can prevent heart damage caused by the chemotherapy drug doxorubicin in breast cancer patients. Researchers will enroll 46 women with early-stage breast cancer who are about to start chemotherapy. Participa…

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 early-stage trial tests a single intravenous dose of a gene therapy called RP-A601 in 9 adults with a genetic heart condition (PKP2-ACM) that causes dangerous heart rhythms and heart failure. The therapy delivers a working copy of the PKP2 gene to heart cells to restore prot…

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 2 trial tests whether a 4-week course of an essential amino acid supplement can help older adults (65+) rebuild muscle after a hip fracture. Sixty participants will be randomly assigned to receive either the supplement or standard care alone. Researchers will measure m…

This early-stage study tests an injection called GT719 in 30 people with moderate to severe autoimmune diseases like lupus that haven't responded to standard treatments. The main goal is to check safety and find the right dose, while also looking at whether it helps control disea…

This study is testing a new medical device called the lok™ suture anchor system for repairing torn tendons and labrum in the shoulder. About 216 adults with rotator cuff tears, shoulder instability, or partial tendon avulsions will receive the anchors during surgery. Researchers …

This early study tests a drug called coramitug in 14 people with ATTR-CM, a heart condition caused by abnormal protein buildup. The drug is designed to bind to and help remove these protein clumps from the heart. Researchers will use special scans to see where the drug goes and w…

This study compares two surgical techniques to reduce blood loss during open fibroid removal. Forty women with symptomatic uterine fibroids will be randomly assigned to either bilateral uterine artery ligation or a pericervical mechanical tourniquet. The goal is to see which meth…

This study tests an experimental drug called SIM0718 for people with asthma that is not well controlled by current inhalers. About 418 adults and teens aged 12 to 75 will receive either the drug or a placebo for one year. The main goal is to see if the drug reduces the number of …

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 drug called avalglucosidase alfa in 13 Chinese children with infantile-onset Pompe disease, a rare genetic disorder that causes severe muscle weakness and heart problems. Participants will receive the drug through an IV every two weeks for about a year. The goa…

This study looks at whether giving sex hormone therapy to men with low hormone levels can help their shoulder tendons heal better after surgery. About 58 men will receive either hormone pills or a placebo after rotator cuff repair. The goal is to see if the therapy improves tendo…

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 early-phase trial is testing a personalized cell therapy called CAR-T, designed to target and destroy faulty immune cells in people with active autoimmune diseases like lupus, scleroderma, and Sjogren's syndrome. Up to 20 participants will receive the treatment after a short…

This study tests a drug called elranatamab in people with AL amyloidosis that has come back or not responded to prior treatment. The goal is to find a safe dose and see if the drug can reduce abnormal protein levels. About 49 adults will take part in this early-phase trial.

This early-phase trial tests whether injecting lab-grown muscle cells (MyoPAXon) into the foot is safe for adults with Duchenne muscular dystrophy who can no longer walk. Eight participants will receive the cells along with an immunosuppressant drug to prevent rejection. The main…

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 early-stage trial is testing a new treatment called YTS109 for people with severe autoimmune diseases like lupus and Sjogren's syndrome that have come back or not responded to standard care. About 18 adults will receive a single infusion of these specially engineered immune …

This early-stage study tests a new cell treatment called YTS109 in 18 adults with hard-to-treat autoimmune diseases like lupus and scleroderma. Participants receive a single infusion of these specially designed cells. The main goal is to check if the treatment is safe, and also t…

This study compares standard rotator cuff surgery to surgery plus a bioinductive patch that aims to help the tendon heal better. About 78 adults with full-thickness tears will be randomly assigned to one of the two groups. The main goal is to see if the patch leads to fewer tendo…

This study is testing a new device that uses near-infrared light to measure pelvic floor muscle fitness in women with myofascial pelvic pain, a condition that affects up to 26% of women. The goal is to create a simple, non-invasive diagnostic tool that could replace the need for …

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 Phase 1 trial is testing a new radioactive tracer called 99mTc-p5+14 to see how it spreads through the body and how much radiation it delivers. The study includes healthy volunteers and people with AL or ATTR systemic amyloidosis, a condition where abnormal protein deposits …

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 study tests whether a smartphone app that analyzes voice patterns can help diagnose and monitor myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS). Researchers will compare voice samples from 40 people with ME/CFS and those without to find unique voice signatures. T…

This study is testing whether a special PET/CT scan using a tracer called PentixaFor can accurately detect acute inflammation in the heart. It focuses on three conditions: heart transplant rejection, cardiac sarcoidosis, and myocarditis caused by cancer drugs. Researchers will en…

This study explores whether a quick, painless cheek swab can spot protein changes linked to inherited heart conditions that cause sudden cardiac death in young people. Researchers will collect samples from 26 patients with arrhythmic disorders or family members at risk. If the te…

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 aims to see if a special MRI designed for teeth can diagnose jaw joint disorders, tooth infections, and help plan dental implants as well as or better than traditional X-rays. About 470 adults needing dental imaging will get both an MRI and standard X-rays, and the res…

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 tests whether a new MRI contrast agent called Elucirem can detect heart scarring as effectively as the standard agent, but at a much lower dose. Researchers will compare images from 25 adults who previously had a heart MRI with the standard agent. If Elucirem works wel…

This study is testing a handheld device called mScan that uses a tiny, painless electrical current to measure muscle health. Researchers want to see if it can give similar results to an MRI, but faster and more conveniently. The study involves 150 adults with and without muscle d…

This study tests a new radioactive dye called 68Ga-NI-FAPI for PET/CT scans. It aims to better detect diseases like tumors, heart problems, and lung scarring by targeting a protein linked to tissue damage. About 30 people, including healthy volunteers and patients, will take part…

This study is testing whether a simple, non-invasive sensor called photoplethysmography (PPG) — already found in many smartwatches — can accurately predict how well the heart pumps blood (ejection fraction) and other heart health measures. Researchers will have 500 adults wear a …

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 phase 3 trial tests whether acoramidis can prevent or delay transthyretin amyloidosis (ATTR) in 587 adults who carry a faulty gene but have no symptoms yet. ATTR causes sticky plaques to build up in the heart and nerves, leading to heart failure and nerve damage. Acoramidis …

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 compares a new injection of liquid phase concentrated growth factor (LPCGF) to standard arthrocentesis (joint washing) for people with painful jaw clicking and limited mouth opening due to a displaced disc. Thirty-two adults aged 18-45 with unilateral disc displacement…

This pilot study tests whether VM100, a nutritional supplement made from ten natural ingredients, can improve quality of life for people with Duchenne muscular dystrophy (DMD). About 20 participants aged 8 and older will take VM100 or a placebo for 10 weeks. Researchers will meas…

This study tests whether Acceptance and Commitment Therapy (ACT), delivered through a smartphone app, can help caregivers of children with RASopathies (like Neurofibromatosis type 1 and Noonan syndrome) cope with parenting stress. The trial is fully remote and involves 70 adult c…

This study compares two brain-training programs for adults with ME/CFS who experience brain fog. Both programs include computer games and a mild nerve stimulation device, but one focuses on processing speed and daily tasks while the other emphasizes relaxation and healthy habits.…

This trial tests whether adding a structured patient education program to standard physical therapy helps people with chronic low back pain, knee osteoarthritis, or shoulder pain stick to their prescribed home exercises. Participants will be randomly assigned to receive either st…

This study tests whether a non-invasive brain stimulation technique called rTMS can improve symptoms of major depression and chronic pain at the same time. Fifty-four adults with moderate depression and pain will receive 30 sessions of active rTMS over several weeks. Researchers …

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 compares two non-surgical treatments for jumper's knee, a common overuse injury causing knee pain. Half the participants will receive extracorporeal shockwave therapy, which uses sound waves to stimulate healing, while the other half will receive dry needling, which in…

This study tests whether adding dry needling to standard physical therapy helps people with jaw pain (TMJ dysfunction). One hundred adults will either get standard rehab alone or rehab plus dry needling. Researchers will measure pain, jaw movement, and quality of life to see if t…

This study tests whether cycling while using a virtual park app can slow the loss of motor function in children and teens with neuromuscular diseases like muscular dystrophy. Twenty-two participants will first receive standard care for 3 months, then use the virtual park bike 3 t…

This study tests whether combining electroacupuncture with vacuum cupping therapy works better than electroacupuncture alone for shoulder tendinitis. Fifty-four adults with shoulder pain and stiffness will receive either electroacupuncture alone or electroacupuncture plus cupping…

This trial tests whether dry needling and low-level laser microcurrent electrical stimulation can reduce pain and inflammation in people with acute myofascial pain syndrome in the upper trapezius muscle. Participants are randomly assigned to receive one of these treatments, a com…

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 study tests whether a game-based biofeedback therapy can improve quality of life and sleep in people with dyssynergic defecation, a condition where pelvic floor muscles don't relax properly during bowel movements. Thirty-six adults will either receive the game-based therapy …

This study tests whether tele-rehabilitation—guided video sessions with self-massage, stretches, and posture tips—can reduce pain and improve neck movement in people with rhomboid trigger points. Thirty-six adults with neck and upper back pain will be randomly assigned to either …

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 common treatments for trigger finger: shock wave therapy and a finger splint. Researchers will enroll 54 adults with early-stage trigger finger to see which approach better reduces pain and improves hand function. The goal is to find out if shock wave ther…

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 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 trial tests whether rotator cuff drills—like plyometric throws and wall dribbles—or progressive resistance band exercises better reduce pain and improve shoulder stability and performance in male overhead athletes with chronic rotator cuff tendinopathy. Thirty athletes aged …

This study tests whether a supplement called MyCondro can help people with knee osteoarthritis move better and feel less joint discomfort. Researchers will give two different doses to 240 adults aged 45 and older who have had knee problems for at least six months. The main goal i…

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 a mental exercise program called graded motor imagery can reduce pain and improve shoulder function after surgery for a massive rotator cuff tear. The program includes tasks like recognizing shoulder positions, imagining movements, and using a mirror to t…

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 compares two types of exercises—core stability and scapular stabilization—to see which better improves hand grip strength in people with chronic myofascial neck pain. Participants will be assigned to one of three groups: core exercises, scapular exercises, or standard …

This study tests whether injecting a simple sugar water solution (5% dextrose) into or around a partial rotator cuff tear can reduce shoulder pain and improve function. Adults with chronic shoulder pain and a confirmed partial tear receive three ultrasound-guided injections over …

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 non-drug treatments—dry dynamic cupping and instrument-assisted soft tissue mobilization (IASTM)—for people with chronic myofascial low back pain lasting more than three months. Over two weeks, participants receive three sessions per week of one therapy. R…

This study looks at whether a digital platform called Band Connect helps people recovering from shoulder surgery or rotator cuff repair do their home exercises more consistently. About 500 adults aged 18 to 89 will use the platform, and researchers will track how many exercises t…

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 shining low-level laser light on muscles can reduce fatigue and improve quality of life in 40 adults with chronic fatigue syndrome. Participants will receive either real laser therapy or a sham treatment three times a week for eight weeks. Researchers wil…

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 gentle brain stimulation technique called tDCS, applied before eccentric knee exercises, can improve pain and function more than exercise alone in people with patellar tendinopathy (jumper's knee). Forty-two adults with chronic knee pain will be randoml…

This study tests whether a testosterone drug (Depo-Testosterone) can reduce fatigue and improve sexual function, mood, and body composition in young men (ages 18-54) who have survived cancer and now have low testosterone. Participants must be in remission for at least one year an…

This early-stage study is testing whether using a special muscle-mapping device to guide Botox injections can better relieve chronic pelvic floor pain in women compared to the standard injection method. Researchers will enroll 30 women with long-term pelvic pain and give them two…

This study looks at whether adding cognitive behavioral therapy (CBT) to standard pelvic floor exercises can improve symptoms in people with dyssynergic defecation, a type of chronic constipation. Researchers will enroll 26 adults aged 18 to 55 who have had constipation for over …

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 a short, two-session telehealth program called Physical Self-Regulation for people with chronic masticatory muscle pain (jaw pain). Participants will be randomly assigned to either this program or a control session that teaches about jaw anatomy. The goal is to s…

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 joining a virtual support group can help reduce anxiety in people who have had Takotsubo syndrome, also known as 'broken heart syndrome.' Participants meet online every other week for group therapy and education. The goal is to see if this support lowers …

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 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 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 tests whether playing virtual reality games can help people with Duchenne muscular dystrophy improve their arm and hand function. 36 participants will either receive standard physiotherapy or the same therapy plus VR games using a Meta Quest 3 headset. The games are de…

This study tests whether a special laser device (Erchonia EVRL) can temporarily ease chronic jaw pain caused by TMJ disorder. Researchers will compare the laser to a placebo in 64 adults who have had constant jaw pain for at least 3 months. The main goal is to see if more people …

This study tests a powered leg brace called the Nomad in 36 people who have trouble walking due to conditions like stroke, multiple sclerosis, or spinal cord injury. Participants will use the brace at home for three months to see if it improves their walking speed, balance, and q…

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 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 looks at whether a custom mouthguard (occlusal splint) can reduce pain and improve muscle function in people with teeth grinding (bruxism) and related jaw and neck muscle pain. About 40 adults will be split into two groups: one gets the mouthguard right away, the other…

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 looks at 134 people with De Quervain's disease, a painful thumb tendon condition. All participants get a steroid injection, then either standard physiotherapy or a program of eccentric exercises. The goal is to see which approach better reduces pain and improves hand f…

This study tests whether a 5-minute educational video about uterine fibroids and their treatments can help patients feel less conflicted when deciding on a treatment plan. About 154 women with fibroids will be randomly assigned to watch the video before their appointment or recei…

This study looks at whether adding a simple nerve-stimulation technique (using deep breathing to activate the vagus nerve) to standard physical therapy can better reduce pain and improve jaw movement in people with jaw joint disorders. Sixty adults with jaw muscle pain will recei…

This study tests whether using Doppler ultrasound to detect inflammation around calcium deposits in the shoulder can improve the results of a guided injection. 100 adults with chronic shoulder pain and calcific tendinitis will receive an ultrasound-guided injection, with or witho…

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 a handheld device called Sonodyn for people with chronic back, neck, or tension headache pain that hasn't improved with other treatments. The device uses a mix of low-energy ultrasound, electrical, and magnetic pulses to target painful trigger points. About 126 a…

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 looks at whether motor imagery training — mentally practicing movements without actually moving — can reduce pain, improve shoulder function, and lessen fear of movement in people with partial rotator cuff tears. About 80 adults will be randomly assigned to either stan…

This study tests whether taking amino acid supplements, along with a special diet and exercise program, can help people with facioscapulohumeral muscular dystrophy (FSHD) gain muscle, lose fat, and improve physical abilities like walking and strength. About 48 adults with FSHD an…

This study tests a new exercise approach for people with long-term shoulder pain caused by rotator cuff problems. Instead of just doing standard shoulder exercises, participants will also perform movements that shift their focus away from the shoulder. The goal is to see if this …

This study tests whether spraying a low-dose lidocaine solution under the diaphragm during single-incision laparoscopic surgery can reduce shoulder pain afterward. About 100 women having surgery for benign gynecological conditions like ovarian cysts or uterine fibroids will take …

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 tests whether using cold and compression after rotator cuff repair surgery reduces pain better than standard care. About 90 adults with partial or full-thickness rotator cuff tears will be randomly assigned to either a cold/compression device or usual care. Pain levels…

This study tests whether injecting platelet-rich plasma (PRP) into acupuncture points can reduce chronic neck pain caused by muscle strain. 72 adults with neck pain lasting over 3 months will receive either PRP or a placebo (salt water) injection. Researchers will measure pain le…

This study looks at whether one session of dry needling can quickly reduce calf muscle pain and improve sprint speed and jumping ability in amateur football players aged 18 to 25. Sixty participants will receive either real or sham needling, and their pain, speed, and performance…

This study compares two treatments for chronic leg pain caused by compartment syndrome: physiotherapy (with a focus on changing running style) and surgery (fasciotomy). The goal is to see if physiotherapy works as well as surgery, which could help more people avoid an operation. …

This study compares High-Intensity Focused Ultrasound (HIFU), a non-invasive procedure using sound waves to destroy fibroids, to myomectomy, a surgical removal. About 112 women aged 18-50 with symptomatic fibroids will be randomly assigned to one treatment. Researchers will track…

This study tests whether focused shock wave therapy can reduce pain and improve jaw function in people with temporomandibular disorders (TMD) of muscular origin. One hundred adults with moderate to severe jaw pain for at least 3 months will receive either shock wave therapy or a …

This study compares three treatments—exercise, dry needling, and a newer injection called interfascial block—for people with long-lasting pain in the upper back muscle (trapezius). About 78 adults aged 18 to 55 with at least 3 months of pain will be assigned to one of the three g…

This study tests whether whole-body electrical muscle stimulation (WB-EMS) can help adults with neuromuscular diseases like ALS, SMA, and muscular dystrophy exercise safely. Because these conditions weaken the nerves that control muscles, traditional exercise is often too hard. W…

This study tests whether adding a gentle electrical acupoint stimulation (TEAS) to a standard three-drug anti-nausea regimen can further reduce vomiting after laparoscopic surgery. Researchers will compare active TEAS to a sham (fake) version in 780 women at high risk for postope…

This study is testing whether hyperbaric oxygen therapy (breathing pure oxygen in a pressurized chamber) can improve symptoms like fatigue and brain fog in people with post-COVID syndrome. About 40 adults with long COVID will receive the treatment. The main goal is to see if a la…

This study tests whether online or group physiotherapy can be as effective as individual in-person sessions for treating jaw muscle pain (TMD). About 90 adults with jaw muscle pain will be randomly assigned to one of three treatment formats. The goal is to find more accessible wa…

This study tests different physical therapy combinations for people with shoulder tendon pain (supraspinatus tendinitis). Researchers will measure pain, range of motion, and tendon thickness using ultrasound. 60 adults aged 40-60 with shoulder pain for up to 3 months will be assi…

This study tests a dietary supplement called lumbrokinase in 120 adults with Long Covid, post-treatment Lyme disease syndrome, or ME/CFS. Participants take the supplement daily for 6 weeks. The goal is to see if it improves overall health and quality of life, as measured by a sim…

This study looks at whether a quick session of electrical stimulation can lower pain and help people with Achilles tendon injuries move better. About 30 adults with Achilles tendinopathy or a past rupture will try two different stimulation methods. The goal is to see if this appr…

This study compares two exercise programs for people with long-term pain in the Achilles, patellar tendon, or plantar fascia. One group does standard strength training, while the other adds early jumping (plyometric) exercises. Researchers will measure jump function after 12 week…

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…

This study looks at whether the Alexander technique, a method that teaches better posture and movement habits, can help children aged 5 to 9 with Duchenne muscular dystrophy control their upper limbs and feel better day-to-day. About 33 kids will take part, and researchers will m…

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 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 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 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 aims to learn more about RASopathies, a group of genetic conditions that can cause developmental issues, birth defects, and increased cancer risk. Researchers will follow up to 500 people of any age who have or may have a RASopathy, along with their family members, for…

This observational study looks at people with rotator cuff disease to better understand what changes in shoulder function scores really mean. It aims to define the smallest meaningful improvement, substantial benefit, acceptable symptom state, and maximum improvement for several …

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 investigates whether people who had carpal tunnel surgery might have a higher chance of having ATTR amyloidosis, a condition where abnormal proteins build up in tissues, potentially affecting the heart. Researchers will biopsy tissue from about 180 patients who had car…

This study follows about 50 boys under 18 with X-linked myotubular myopathy (XLMTM), a rare genetic muscle condition, to see how often they develop liver problems like cholestasis. No drugs are given—researchers simply collect health data, including liver scans, over one year. Th…

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 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 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 is looking at whether advanced heart imaging, especially cardiac MRI, can help identify the cause of cardiomyopathy and predict risks like death or heart failure in a diverse group of patients. The research focuses on adults in the Bronx, including many from Hispanic a…

This study aims to learn more about rare disorders that affect how the body processes chemicals called pyrimidines and purines. These disorders can cause problems in the brain, blood, kidneys, and immune system, ranging from mild to life-threatening. Researchers will compare test…

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 explores whether 3D images and models made from routine prenatal scans can help families and surgeons better understand congenital diaphragmatic hernia (CDH), a condition where a hole in the diaphragm lets abdominal organs move into the chest. Researchers will use scan…

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 tests a special MRI method to see how muscles and connective tissues move and respond to pressure. Researchers will compare healthy people and those with myofascial pain, and see if hands-on therapy (Tuina) changes tissue movement. Participants with pain will be random…

This study is for breast cancer patients receiving certain chemotherapies that can harm the heart. Researchers want to see if a genetic marker called TRPC6 can predict who will develop heart problems. By analyzing blood samples, they hope to find early warning signs and ways to p…

This study investigates whether tightness in the pectoralis minor, a small chest muscle, is linked to partial rotator cuff tears in the shoulder. Researchers will compare 45 patients with tears to 45 without, matching them by age, sex, body size, and dominant side. Using simple m…

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 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 investigates whether the thickness of trunk muscles, along with balance and body awareness, relates to shoulder problems caused by rotator cuff injuries. Researchers will compare measurements from people with rotator cuff issues to those without shoulder pain. The goal…

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 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 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 early-stage study tests whether people with advanced solid tumors can safely follow a diet low in methionine, an amino acid found in many proteins. Researchers will measure how well participants stick to the diet, check for side effects, and track changes in blood methionine…

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 study aims to discover biological markers (biomarkers) that could help doctors diagnose and track neurological autoimmune diseases such as multiple sclerosis, myasthenia gravis, and Guillain-Barre syndrome. Researchers will analyze samples from 50,000 people, including patie…

This study aims to better understand heart disease in people with Friedreich's ataxia, a genetic disorder that affects coordination and often leads to life-threatening cardiomyopathy. Researchers will use cardiac MRI, echocardiograms, and exercise stress tests to measure heart fu…

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 aims to understand how heart problems develop in people with Duchenne and Becker muscular dystrophy, as well as in carriers. Researchers will collect cardiac MRI scans and clinical data from 1,000 participants to build a registry. Using advanced image analysis and deep…

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 observing 150 people who have a pacemaker with a special setting called Closed Loop Stimulation (CLS) combined with left bundle branch area pacing. The goal is to see if this setting reduces the occurrence of atrial fibrillation over two years. Participants have sin…

This study tests whether inflating a small balloon during biofeedback therapy helps people with chronic constipation coordinate their muscles better. Biofeedback therapy teaches patients how to relax and tighten the right muscles to have a bowel movement. The study compares the s…

This study looks at what influences treatment expectations in adults with myofascial pain syndrome (MPS), a common cause of muscle pain. Researchers measure pain intensity, pain duration, quality of life, and fatigue to see which factors best predict how much benefit patients exp…

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 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 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 investigates whether fragments of tumor DNA circulating in the blood can be used to detect and monitor soft tissue sarcoma. Researchers will collect blood and tumor tissue samples from 40 adults with high-risk sarcomas to see if this DNA can serve as a biomarker for ca…

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 aims to better understand how Pompe disease affects the nerves and muscles involved in breathing. Researchers will analyze nerve conduction and diaphragm activity in 20 adults with Pompe disease or unexplained respiratory failure. The goal is to identify patterns that …

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 uncover the genetic causes of cardiomyopathy in children by analyzing DNA from affected individuals and their families. Researchers hope to identify mutations that lead to different types of cardiomyopathy, which could improve genetic counseling and deepen unde…

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 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 …

This study is following 700 adults with myotonic dystrophy type 1 (DM1) over two years. Researchers are measuring walking speed, lung function, and muscle tissue changes to find reliable markers of disease progression. The goal is to improve future clinical trials by better under…

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 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 gathers medical information from people with MNGIE, a rare genetic disease that affects the nerves and digestive system. Researchers want to learn how the disease progresses and find better ways to measure it. Up to 50 patients worldwide can join, and no new treatments…

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 study will collect electronic health records from up to 2500 people with Duchenne or Becker muscular dystrophy, including female carriers, across U.S. clinics. The data will be combined with patient-reported information to give researchers a fuller picture of the diseases. T…

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 uses three wearable devices—a continuous glucose monitor, a heart monitor, and an activity tracker—to see if high blood sugar is linked to heart problems in people with Duchenne muscular dystrophy. Researchers will compare 80 participants with and without DMD. The goal…

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 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 compares four types of nerve blocks used during arthroscopic shoulder surgery to see which one causes the least temporary diaphragm paralysis and leads to better recovery. Researchers will measure breathing function, pain levels, opioid use, and overall recovery qualit…

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 looks at blood clotting in people with autoimmune skin diseases like bullous pemphigoid and lupus. Researchers will collect blood samples from 39 participants—some with active disease and some with mild or controlled disease—plus 15 healthy controls. They will measure …

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 investigates whether certain genetic variations make some people with atrial fibrillation more likely to develop a weakened heart muscle (cardiomyopathy). Researchers will compare the DNA of 299 participants, including those with AF-related cardiomyopathy and control g…

This study looks at whether respiratory physiotherapy can prevent or reduce thinning of the diaphragm—the main breathing muscle—in patients who are on mechanical ventilators in the ICU. Researchers will measure diaphragm thickness using ultrasound in 66 adults who have been on a …

This study is building a worldwide online registry for people with Duchenne or Becker muscular dystrophy, including female carriers. Participants share their health information to help researchers learn more about the disease and to match patients with clinical trials. The goal i…

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 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 is looking for genetic clues that might explain why a very small number of people develop rare side effects like Guillain-Barré syndrome, blood clots with low platelets, or heart inflammation after a COVID-19 vaccine. Researchers will compare the DNA of people who had …

This study explores whether a non-invasive brain-computer interface (BCI) can help people with motor disorders, such as spinal cord injury or stroke, control assistive devices using their thoughts. Researchers will record brain signals with EEG and use machine learning to interpr…

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 looking for new genes and blood markers linked to arrhythmogenic cardiomyopathy (AVC), a genetic heart condition that can lead to heart failure and sudden cardiac arrest. Researchers will enroll 1,000 people, including patients with AVC or unexplained cardiac arrest…

This study aims to find early signs of Parkinson's disease in people who carry a change in the GBA1 gene, which puts them at higher risk. Researchers will use simple, non-invasive tests to check for subtle changes in movement, thinking, sleep, and other functions that can appear …

This study creates a registry to collect real-world information on the safety and effectiveness of ambroxol, a common cough medicine, when used at higher doses for Gaucher disease or GBA-related Parkinson disease. Researchers aim to gather data from 300 patients worldwide who are…

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 looks at medical records of 1000 children with conditions like autism, epilepsy, and Down syndrome to find common biological patterns. Researchers want to understand what causes these disorders and how children respond to treatments. The goal is to improve diagnosis an…

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 global registry collects information from people with Fibrodysplasia Ossificans Progressiva (FOP), a rare condition where soft tissues turn into bone. Up to 800 patients worldwide can report their symptoms, flare-ups, and mobility changes through a secure online portal. The …

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 watches how children under 5 with LAMA2-related muscular dystrophy move and develop over two years. Researchers will test muscle function, swallowing, breathing, and collect blood samples. The goal is to learn more about the disease so future treatments can be tested e…

This study follows up to 400 children with Pompe disease to see how their immune system reacts to enzyme replacement therapy. Researchers will collect medical records from birth to age 18 to understand which children develop antibodies that block treatment. The goal is to improve…

This study aims to understand how different types of changes in the DMD gene are linked to heart problems in boys and young men with Duchenne or Becker muscular dystrophy. Researchers will use heart tests and blood samples to look for early signs of heart issues. The study involv…

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 is building a large online registry of 10,000 adults with cardiomyopathy or myocarditis, plus those with a strong family history. Researchers will track participants over time to see how many experience serious events like heart failure, dangerous heart rhythms, or dea…

This study looks at how different types and intensities of exercise affect heart rhythm in people with arrhythmogenic right ventricular cardiomyopathy (ARVC). Researchers will measure extra heartbeats and heart workload during activities like walking or biking. The goal is to cre…

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 research study at Boston Children's Hospital is looking at the genes and proteins involved in congenital myopathies—rare muscle diseases that are present from birth. Researchers will analyze DNA from up to 4,000 participants, including patients and their family members, to f…

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 follows 100 people in Spain with nemaline myopathy, a rare muscle disease, to see how their symptoms change over time. Researchers will use ultrasound, movement tests, and breathing checks to map the disease's natural course. The goal is to build a detailed patient dat…

This study uses ultrasound to track how soft tissues like ligaments, tendons, and muscles stretch and move in different groups: baseball pitchers, people with ankle tendon problems, and patients recovering from ACL knee surgery. Researchers want to see if these measurements can h…

This study is testing whether a non-invasive blood pressure monitor can accurately detect blockages in the heart's main pumping chamber in people with hypertrophic cardiomyopathy (HCM). Researchers will compare blood pressure readings with standard invasive measurements in 800 ad…

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 follows up on people with hypertrophic cardiomyopathy (HCM), a genetic condition that thickens the heart muscle. Researchers want to see how scarring in the heart (called late gadolinium enhancement, or LGE) changes over at least 7 years and whether it predicts serious…

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 uses ultrasound imaging and machine learning to better understand low back pain by looking at the thoracolumbar fascia, a layer of tissue in the back. Researchers will compare images from people with acute pain, chronic pain, and no pain. Some participants with chronic…

This study looks at cholesterol and blood sugar levels in 200 people with Noonan syndrome and related conditions, aged 2 to 35. Researchers want to see if these levels differ by age, gender, or genetic type. The goal is to improve long-term care by identifying who might be at hig…

This study uses magnetic resonance spectroscopy, a special MRI technique, to measure the heart's energy levels without needles or biopsies. Researchers will compare healthy volunteers, people with heart failure, and those with other heart conditions to see if low energy metabolis…

This study looks at whether a rapid drop in a blood marker called dFLC can predict a complete response in people with AL amyloidosis treated with daratumumab-based therapy. Researchers will follow 50 patients to see if early changes in dFLC levels match up with later treatment su…

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 sleep issues contribute to tiredness and thinking problems in people with multiple sclerosis (MS). About 382 MS patients and a control group without MS will have overnight sleep tests, thinking assessments, and questionnaires. The goal is to better underst…

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 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…