Muscle tissue disorder

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

This study tests whether the Butantan-Dengue vaccine is safe and works well in people aged 12–59 with autoimmune rheumatic diseases (like rheumatoid arthritis or lupus) who are stable on low or no immune-suppressing drugs. Participants receive one vaccine dose and are monitored f…

This study tests a new treatment called AZD0120 for people with AL amyloidosis, a rare disease where abnormal proteins build up in organs. The therapy uses a patient's own immune cells, modified to target and destroy harmful cells. The study aims to see if it is safe and effectiv…

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

This study follows about 300 people with Duchenne muscular dystrophy who are taking the drug givinostat as part of their normal care. Researchers will track safety issues like low platelet counts and bleeding, and see how patients' muscle function changes over up to 5 years. The …

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

This 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 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 study tests a one-time gene therapy called RGX-202 in boys with Duchenne muscular dystrophy (DMD). The therapy delivers a mini version of the missing dystrophin protein to muscle cells. Researchers will check safety and whether it improves muscle function, like standing and …

This early-stage trial tests a one-time gene therapy called RP-A701 in 8 adults with dilated cardiomyopathy caused by a faulty BAG3 gene. The therapy uses a harmless virus to deliver a working copy of the gene, aiming to improve heart function. The main goal is to check safety, b…

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

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

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

This study tests 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 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 a fetal surgery called FETO for severe congenital diaphragmatic hernia (CDH), a condition where a hole in the diaphragm allows organs to crowd the lungs. Researchers will place a tiny balloon in the fetal windpipe to encourage lung growth, then remove it before b…

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

This 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 early-stage trial tests whether infusions of stem cells from donated newborn tissue are safe for young people aged 4 to 39 with dilated cardiomyopathy, a condition where the heart becomes enlarged and weak. Up to 36 participants will receive three intravenous doses over 30 d…

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

This study is 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 study tests an experimental drug called NNC6019-0001 in people with transthyretin amyloid cardiomyopathy (ATTR-CM), a condition where abnormal proteins build up in the heart. About 1,280 adults will receive either the drug or a placebo by IV, alongside their usual heart trea…

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

This phase 3 trial tests an experimental drug called sepofarsen in 32 people with Leber congenital amaurosis (LCA), a rare genetic condition that causes severe vision loss from birth. The drug is injected into one eye, while the other eye gets a placebo, to see if it safely impro…

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 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 is testing a device called the Integrity Implant System to see if it helps people with torn rotator cuffs heal better after surgery. The implant is used to support the repair and reduce the chance of the tear coming back. About 150 adults will take part, and their prog…

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

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

This study follows 300 adults with severe eosinophilic asthma who are prescribed benralizumab as part of their normal care in Germany. Researchers will track changes in asthma control, lung function, and medication use over one year. The goal is to see how the drug performs outsi…

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 an experimental oral drug called S-606001 in 45 adults with late-onset Pompe disease. Participants take the drug or a placebo on top of their standard enzyme replacement therapy. The main goal is to see if the drug improves lung function and walking ability over …

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

This 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 a single dose of SGT-003, a gene therapy that delivers a working version of the dystrophin gene to muscle cells. About 60 boys with Duchenne muscular dystrophy, aged from infancy to under 18, will receive the treatment and be followed for 5 years. The goal is to …

This study tests an experimental drug called linvoseltamab in 220 people with AL amyloidosis that has returned or not improved after prior therapy. The drug aims to reduce abnormal proteins that damage organs like the heart and kidneys. The trial first finds the safest dose, then…

This study tests a new two-drug treatment for people with advanced soft tissue sarcomas (cancers of the muscles, fat, or other connective tissues). The drugs, abemaciclib and gemcitabine, work together to stop cancer cells from growing. The trial will compare this new combo to th…

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

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

This study tests whether injections of donated skin cells (TPX-115) can improve shoulder function and reduce pain in people with partial-thickness rotator cuff tears. About 60 adults whose symptoms have lasted over 3 months despite other treatments will receive either the cell th…

This study tests a combination of two drugs, venetoclax and dexamethasone, in people with a rare blood disorder called AL amyloidosis that has come back or not improved after treatment. The trial includes 53 adults with a specific genetic marker (t(11;14)). The goal is to find th…

This study tests a drug called zanzalintinib (XL092) in 29 people with advanced leiomyosarcoma that has spread or cannot be removed by surgery. The drug works by blocking signals that help cancer cells grow. The goal is to see if it can slow the cancer's progression longer than c…

This phase 2b trial tests whether adding ALXN2220 to eplontersen works better than eplontersen alone for adults with transthyretin amyloid cardiomyopathy (ATTR-CM), a condition where abnormal proteins build up in the heart. About 326 participants will receive either the combinati…

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

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

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

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

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

This 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 early-stage study tests a one-time gene therapy called ALXN2350 for adults with a specific genetic heart condition (BAG3-related dilated cardiomyopathy). The heart muscle becomes weak and enlarged, making it hard to pump blood. The treatment aims to fix the faulty gene to im…

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 tests whether daily weight monitoring at home can reduce hospitalizations for heart failure in people with cardiac amyloidosis, a condition where protein buildup stiffens the heart. 320 adults with ATTR cardiomyopathy will either use a connected scale that alerts docto…

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 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-phase study tests a new drug called OM336 in 39 adults with active Sjogren's disease or idiopathic inflammatory myopathy who have not improved with at least two prior treatments. OM336 is an engineered antibody designed to target and potentially reduce harmful immune c…

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 study tests an experimental drug called OKN4395, taken as a pill, alone or together with the immunotherapy pembrolizumab. The goal is to find a safe dose and see if the combination can shrink tumors in people with advanced solid cancers like lung, colorectal, sto…

This early-stage study tests a single injection of a gene therapy called INS1201, given into the spinal fluid of young boys (ages 2 to 5) with Duchenne muscular dystrophy who can still walk. The main goal is to check if the treatment is safe and to see how it spreads in the body.…

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 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 study tests whether using cryopreserved aortic patches (donated tissue) can safely repair defects in the windpipe or bronchial tubes. Ten people with severe tracheal narrowing or deformities will receive this surgical patch. The main goal is to see if it reduces death and co…

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

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

This study tests 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 early-stage trial tests the safety and tolerability of a new drug called VX-670 in 52 adults with myotonic dystrophy type 1 (DM1), a genetic condition that causes muscle weakness and other problems. Participants receive either VX-670 or a placebo, and researchers will monito…

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 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 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 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 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 early-phase study tests an experimental drug called JNJ-79635322, a trispecific antibody designed to attack cancer cells in people with relapsed or refractory multiple myeloma or AL amyloidosis. About 180 participants will receive the drug to find a safe dose and check for s…

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 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 early-stage trial is testing a new treatment called CC-97540, which uses a patient's own immune cells modified to target and destroy faulty immune cells. It is for people with severe forms of lupus, myositis, scleroderma, or rheumatoid arthritis that have not improved with s…

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

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

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

This study 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 study tests whether benralizumab, an injected drug, can reduce asthma attacks in children aged 6 to 17 with severe eosinophilic asthma. About 200 participants will receive either benralizumab or a placebo, and researchers will track how long it takes for their first asthma f…

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

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

This study 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 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 early-phase trial tests whether adding zanzalintinib to the chemotherapy drug eribulin can help control advanced liposarcoma and leiomyosarcoma better than eribulin alone. About 18 adults whose cancer has worsened after prior treatments will receive the combination. The main…

This study tests whether ruxolitinib, a drug that blocks a specific immune signal (interferon-γ), can help people with inclusion body myositis, a rare muscle disease that causes progressive weakness. About 80 adults aged 45 and older will receive either the drug or a placebo. The…

This phase 3 trial tests an experimental drug called AOC 1020 in 200 adults with facioscapulohumeral muscular dystrophy (FSHD), a genetic condition that causes progressive muscle weakness. Participants receive either the drug or a placebo by IV infusion. The study measures muscle…

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 anifrolumab, a weekly injection, for adults with moderate to severe polymyositis or dermatomyositis—rare diseases that cause muscle weakness and inflammation. About 240 participants will receive either anifrolumab or a placebo added to their usual care for 52 wee…

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

This 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 single intravenous dose of a gene therapy called AB-1009 in 12 adults with late-onset Pompe disease. Participants must have been on enzyme replacement therapy for at least 6 months. The main goal is to check safety and side effects, while also seeing if the tre…

This early-stage study tests a new treatment made from healthy donor immune cells (CAR T cells) for people with severe autoimmune diseases like lupus that haven't responded to standard therapies. The goal is to see if these cells can safely target and calm the overactive immune s…

This early-phase trial tests a new drug called GT801 in 22 people with moderate-to-severe autoimmune diseases that haven't responded to other treatments. The main goal is to check safety and find the right dose. Researchers will also look for signs that the drug helps control the…

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 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 early-stage study is testing a new cell therapy called FT819 for people with severe autoimmune diseases like lupus, vasculitis, and scleroderma. The treatment involves giving specially engineered immune cells along with chemotherapy drugs. The main goal is to see if it's saf…

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 adults with high-risk soft tissue sarcoma that can be removed by surgery. It tests whether adding an immunotherapy drug (atezolizumab) to standard chemotherapy, radiation, and surgery can delay the cancer from coming back. About 50 participants will receive the …

This early-stage study tests a single intravenous dose of a gene therapy called LY3884961 in 15 adults with Gaucher disease type 1. The goal is to see if it is safe and can reduce or replace the need for ongoing enzyme replacement or substrate reduction therapy. Participants must…

This phase 2 trial tests a therapy called AlloNK, made from donor cord blood immune cells, combined with the drug rituximab. It aims to see if this combination is safe and can help people with severe forms of rheumatoid arthritis, Sjögren's disease, myositis, or scleroderma that …

This Phase 2 trial tests an experimental drug called INCB000928 (zilurgisertib) in people with fibrodysplasia ossificans progressiva (FOP), a rare genetic condition where soft tissues turn into bone. About 98 participants aged 2 and older will receive either the drug or a placebo…

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

This 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 follows 21 adults who previously received an injection of stem cells from donated umbilical cords to treat rotator cuff disease. Researchers want to see if the treatment remains safe and helps with shoulder pain and function over the long term. Participants fill out qu…

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

This phase 3 trial is testing whether adding benralizumab (an injectable drug) to medium-dose inhalers can reduce asthma attacks in people with eosinophilic asthma whose symptoms are not well controlled. About 400 participants aged 12 to 75 will be randomly assigned to receive be…

This study tests an experimental drug called etentamig (ABBV-383) in adults with AL amyloidosis, a rare disease where abnormal proteins build up in organs. About 76 participants will receive the drug by infusion to see if it is safe and can control the disease. The trial has two …

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

This study tests if temporarily blocking the blood supply to the uterus and ovaries during minimally invasive fibroid surgery can reduce blood loss. About 120 women with large or numerous fibroids will be randomly assigned to receive the vessel-blocking technique or standard surg…

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

This study tests 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 whether adding dupilumab to standard treatment can help people with hypereosinophilic syndrome (HES) who still have symptoms despite current biologic therapy. About 30 adults will receive dupilumab injections for up to 48 weeks while continuing their usual medica…

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 a new medicine, DYNE-101, in 116 adults with myotonic dystrophy type 1 (DM1), a condition that causes muscle weakness and stiffness. The main goals are to check if the drug is safe and if it can reduce muscle stiffness and improve muscle function. Participants re…

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 study tests a drug called belantamab mafodotin in people with a rare blood disease called AL amyloidosis that has come back or not responded to other treatments. The goal is to find the safest and most effective dose. The study has two parts: first, different doses are teste…

This study tests a new drug called TQB2934 for people with systemic light chain amyloidosis, a rare disease where abnormal proteins damage organs. The drug is a bispecific antibody that helps the immune system target and destroy harmful cells. The trial will enroll 70 adults who …

This early-phase trial tests a gene therapy called SPOT-03 in 9 boys with Duchenne muscular dystrophy (DMD), aged 2 to 8. The main goal is to see if the treatment is safe and tolerable. Researchers will also check if it increases dystrophin protein levels in muscles, which could …

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 phase 3 trial tests whether adding a stem cell transplant to standard chemotherapy helps people with newly diagnosed AL amyloidosis. About 338 participants will receive either chemo alone or chemo followed by a stem cell transplant. The goal is to see which approach better d…

This study tests a gene therapy called RP-A501 in 14 males with Danon disease, a rare genetic condition that causes heart problems. The treatment uses a harmless virus to deliver a working copy of the LAMP2 gene, aiming to improve heart muscle function and reduce thickening. Part…

This study is testing a drug called deucravacitinib in 10 adults with idiopathic inflammatory myopathies, a group of rare diseases that cause muscle inflammation and weakness. Participants will take one pill daily for 6 months. Researchers will track symptom changes and lab resul…

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 tests whether stem cells from umbilical cord lining can safely reduce symptoms of dermatomyositis and polymyositis, two autoimmune diseases that cause muscle weakness and inflammation. About 40 adults will receive three doses of stem cells or a placebo, then switch. 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 early-phase trial is testing a new treatment called GC012F for people with AL amyloidosis that has come back or not responded to other therapies. GC012F is a type of immunotherapy that uses a patient's own immune cells, modified to attack two specific targets on the abnormal…

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

This study 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 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 study looks at a fetal surgery called FETO for babies with a severe diaphragmatic hernia, where organs move into the chest. The surgery places a balloon in the baby's windpipe to help the lungs grow, then removes it before birth. Researchers will compare 40 babies who get FE…

This study tests whether adding a BioBrace® implant during rotator cuff surgery helps the tendon heal better and reduces the chance of re-tearing. About 268 adults aged 40–70 with full-thickness tears will be randomly assigned to get surgery with or without the implant. The main …

This early-phase trial is testing EPI-321, a one-time gene therapy designed to silence the faulty gene that causes facioscapulohumeral muscular dystrophy (FSHD). The study will enroll 12 adults with FSHD Type 1 to see if the treatment is safe and tolerable, and whether it shows a…

This early-stage trial tests a new biologic drug called ARGX-119 in 16 adults with a rare genetic muscle weakness condition called DOK7-congenital myasthenic syndrome (CMS). The study aims to see if the drug is safe and how the body processes it. Participants will receive either …

This study tests a drug called teclistamab in 30 people with AL amyloidosis, a rare disease where abnormal proteins damage organs. Participants have already had other treatments. The goal is to see if teclistamab can reduce or eliminate the harmful proteins in the blood. The stud…

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 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 a one-time gene therapy called RGX-314 for wet age-related macular degeneration (AMD), a leading cause of vision loss. About 714 adults aged 50-89 who currently need regular eye injections will receive either the gene therapy or standard treatment. The goal is to…

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 whether a patch made from donated placental tissue can help rotator cuff tears heal better after surgery. Half of the 120 participants will get the patch along with standard repair, and the other half will get standard repair alone. An MRI one year later will che…

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 is testing a new biologic drug called CC312 in just 6 adults with lupus, myositis, or scleroderma that has not responded to standard treatments. The main goal is to check safety and find the right dose, not yet to prove it works. Participants receive multip…

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 a medicine called aficamten in children aged 12 to 18 with a heart condition that makes it hard for blood to pump out. The goal is to see if the drug can reduce pressure inside the heart and improve symptoms like shortness of breath. About 55 participants will re…

This program provides expanded access to triheptanoin for people with long-chain fatty acid oxidation disorders (LC-FAOD) who have few treatment options and cannot join a clinical trial. The goal is to help manage the disease by providing an alternative energy source for the body…

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 early-stage trial is testing Descartes-08, a cell therapy made from modified immune cells, in 50 young people aged 12 and older with four autoimmune diseases: childhood-onset lupus, ANCA-associated vasculitis, juvenile myasthenia gravis, and juvenile dermatomyositis. The stu…

This study tests a new device that uses extreme cold (cryoablation) to treat a fast, dangerous heart rhythm called sustained monomorphic ventricular tachycardia. About 206 adults with this condition will receive the freezing treatment. The goal is to see if it safely stops the ab…

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-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 phase 2 study tests a new radioactive imaging agent, 18F-mFBG, to measure nerve damage in the hearts of 20 people with stable heart failure and implantable defibrillators (ICDs). The goal is to see if the agent can reveal differences in nerve function between those who have …

This study enrolls about 4,000 adults aged 60 and older with heart failure who are at high risk for ATTR amyloidosis, a rare disease that stiffens the heart. Doctors receive special training on how to spot and diagnose the condition using standard tests. The goal is to see if thi…

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

This study looks at tissue removed during back surgery to find early signs of a heart condition called ATTR cardiac amyloidosis. Researchers will test spine samples from 1,663 older adults for abnormal protein deposits. If found, participants will get further heart tests to confi…

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

This study 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 offers expanded access to a PET scan using a radioactive tracer called 18F-DOPA to locate small, insulin-producing lesions in the pancreas. It is for patients with congenital hyperinsulinism, Beckwith-Wiedemann syndrome, or insulinoma who have low blood sugar. The goal…

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

This study tests whether artificial intelligence can help doctors interpret echocardiograms (heart ultrasounds) more accurately and quickly. Researchers at Mayo Clinic will deploy AI algorithms to flag signs of genetic cardiomyopathy, ischemic heart disease, and cardiac amyloidos…

This study tests a radioactive imaging agent called Iodine-124 evuzumitide (AT-01) in 150 people with systemic amyloidosis. The goal is to see how well it detects amyloid deposits in the heart and other organs using PET/MRI or PET/CT scans. If accurate, it could become a new tool…

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

This 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 tests whether the blood thinner rivaroxaban can prevent strokes, heart attacks, and death in people with hypertrophic cardiomyopathy (HCM) who have early signs of atrial damage but normal heart rhythm. About 532 adults aged 40-80 with HCM and reduced left atrial strain…

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 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 the drug sonlicromanol can reduce fatigue and improve physical abilities like balance and leg strength in adults with a specific genetic form of mitochondrial disease. About 220 participants will take either the drug or a placebo twice daily for 52 weeks.…

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 Phase 2 trial tests whether KL1333 can reduce fatigue and improve leg strength in adults with primary mitochondrial disease, a genetic condition that affects energy production. About 180 participants will receive either KL1333 or a placebo twice daily for 48 weeks. The study…

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 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 tests whether a numbing injection called Twin Block can reduce jaw muscle pain and the need for opioid painkillers after wisdom tooth removal. About 48 healthy adults who have jaw pain the day after surgery will receive either the Twin Block or a placebo. Researchers w…

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

This study tests a hands-on therapy called Mobilization with Movement (MWM) for people with rotator cuff tears. 75 adults with shoulder pain for at least 3 months will be split into three groups: MWM, standard physical therapy, or a control group. Researchers will measure pain, f…

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 giving misoprostol (a drug often used for stomach ulcers and labor) before hysteroscopic fibroid removal can reduce the amount of fluid absorbed by the body during surgery. Too much fluid can be dangerous. Forty women with small fibroids (1-3 cm) will rec…

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 Phase 3 study tests whether the drug sotagliflozin can improve symptoms and daily function in 500 adults with hypertrophic cardiomyopathy (HCM), a condition where the heart muscle thickens. Participants take either the drug or a placebo pill once daily for 26 weeks. The main…

This study tests a drug called ninerafaxstat in people with a type of heart muscle disease that makes the heart stiff and causes symptoms like shortness of breath and fatigue. The trial involves 165 participants who will receive either the drug or a placebo. The main goal is to s…

This study looks at whether adding core stabilization exercises to standard physical therapy can reduce pain and improve shoulder function in people aged 40-60 with partial rotator cuff tears. About 69 participants will be assigned to either core exercises or traditional trunk an…

This study looks at whether doing breathing exercises before orthopedic surgery (like knee, hip, or fracture repair) can help your lungs work better after the operation. It involves 36 adults who have used tobacco and are at risk for breathing problems after surgery. Participants…

This study tests a home monitoring program called LiverWatch for people with advanced liver disease (cirrhosis). Participants get a Fitbit, personalized nutrition advice, and weekly check-ins to boost activity and manage symptoms. The goal is to see if this approach improves qual…

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

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

This study 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 injecting sodium thiosulfate directly into painful calcium deposits (calcinosis) can shrink them and ease symptoms. Twenty adults with scleroderma, dermatomyositis, or mixed connective tissue disease will receive ultrasound-guided injections. The main goa…

This study looks at whether using a blood flow restriction cuff during exercise can help reduce pain in people with rotator cuff-related shoulder pain. 30 adults will try four different exercise routines with varying cuff pressures to see which works best for pain relief. The goa…

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 Phase 3 trial tests whether the experimental drug KLH-2109 can reduce heavy menstrual bleeding in people with uterine fibroids. About 254 participants will take either KLH-2109 or a standard treatment for 24 weeks, tracking their bleeding and visiting the clinic for safety c…

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 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 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 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 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 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 doing mental tasks while performing shoulder exercises can improve pain and function better than standard exercises alone. 46 adults with chronic shoulder pain from rotator cuff issues will participate in a 6-week program. Researchers will measure pain, s…

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 a physical therapist-guided exercise program can improve strength and walking ability in adults with myotonic dystrophy type 2 (DM2). Twenty-four participants will be split into three groups, each doing the same exercises but with different levels of ther…

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 tests whether a device that delivers gentle electrical stimulation to the pelvic floor can reduce chronic pelvic pain in women. Sixty women with myofascial pelvic pain will either receive the device treatment or standard care (education and exercises). The goal is to s…

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 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 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 tests a virtual reality program called SurviVRSE that teaches breast cancer survivors about heart health. Thirty women currently receiving certain cancer treatments will use the VR headset during one infusion session. Researchers will measure how much they learn, how e…

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

This study looks at whether a blood test for tumor DNA (ctDNA) and detailed scans can help predict how well chemotherapy works in people with a rare soft tissue cancer called leiomyosarcoma. About 40 participants will give blood samples and have scans before, during, and after tr…

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

This study 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 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 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 will test how the heart drug empagliflozin is absorbed and processed in 10 children with Duchenne muscular dystrophy, ages 8 to 18. The goal is to find the best dose for protecting the heart in this rare disease. Participants will take the drug by mouth and have blood …

This study screens males with Duchenne muscular dystrophy (ages 0 to under 25) for antibodies against AAV8, a virus used in some gene therapies. The goal is to find out how many have these antibodies and to identify who might be eligible for future gene therapy trials. No treatme…

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

This study aims to 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 early-stage study will check if two different tablet forms of the drug HRS-1893 are processed the same way in the body. Thirty-six healthy adults will take both versions and have blood tests to measure drug levels. The goal is to see if the two formulations are equivalent, n…

This study is testing a new leg exoskeleton designed by the NIH to help children and young adults with muscle weakness from conditions like cerebral palsy or spinal cord injury. Researchers want to see how well the device works in different real-world settings, like walking on a …

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 aims to collect medical information and tissue samples from 500 people with suspected or confirmed inflammatory muscle diseases, as well as healthy volunteers. Researchers will use blood, muscle, and skin samples to study what causes these diseases and how they progres…

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 will follow about 1,850 people with ATTR amyloidosis, a rare disease that affects the heart and nerves. Researchers will collect information on patients' health, treatments, and outcomes over time to better understand the disease and how current therapies work in every…

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 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 is collecting blood and tumor samples from 200 people with metastatic leiomyosarcoma who are starting chemotherapy. Researchers will analyze circulating tumor DNA (ctDNA) and genetic changes to see if they can predict how well the chemotherapy works. Participants also …

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

This global registry enrolls people with Pompe disease to track how the condition changes over time, whether they receive treatment or not. By collecting data from up to 2,000 participants, researchers hope to better understand the disease's progression and improve patient care. …

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

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

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

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

This study looks at 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 looks at whether it is safe and possible to do a special exercise test (MRI combined with a bike workout) in 20 people who already have a specific type of pacemaker. The goal is to see how different pacemaker settings affect the heart's pumping ability during light exe…

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

This study is for people with a rare genetic muscle condition called congenital myasthenic syndrome (CMS). It does not test any new drug or treatment. Instead, researchers will collect information from up to 100 participants over 4 visits to better understand how the disease affe…

This study looks at two ways to manage pain in children having the Nuss procedure for pectus excavatum (a sunken chest). One group received standard nerve freezing (cryoanalgesia) alone, while the other also had a nerve block near the diaphragm. Researchers will compare pain scor…

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

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

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

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

This study is a large registry that will follow about 1,700 people with obstructive hypertrophic cardiomyopathy in the US and Europe. Researchers will track how patients respond to mavacamten or other standard treatments in real-world settings, focusing on safety and effectivenes…

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

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

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

This 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 translates and tests several health questionnaires—covering pain, arm and leg function, mood, and daily activities—for use with Albanian-speaking people. Researchers will check if the translated versions are reliable and accurate by giving them to about 300 participant…

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 tests a new tool called the PRISM Dashboard, which gives physiotherapists feedback on how their decisions compare to their peers. The goal is to see if this feedback helps reduce differences in care for people with muscle and joint pain. About 60 physiotherapists and t…

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 study follows up to 5,000 people with autoinflammatory diseases (like NOMID, CANDLE, and juvenile dermatomyositis) and their healthy relatives. Researchers will collect medical history, blood samples, and imaging over 2-5 day visits to learn how these diseases work and find …

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 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 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 uses a special PET scan to measure brain inflammation in people with fibromyalgia, chronic fatigue syndrome, multiple sclerosis, and healthy volunteers. Researchers want to see if those with pain and fatigue have higher levels of brain inflammation. The study does not …

This study looks at whether a longer fasting period before an upper endoscopy can lower the risk of stomach contents entering the lungs (aspiration) in people taking GLP-1 receptor agonists (like Ozempic or Wegovy). Researchers at Mayo Clinic will check the stomachs of 150 adults…

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

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

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

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

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

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

This study tests whether adding servo-assisted bed cycling to standard physical therapy can reduce muscle breakdown in critically ill patients with sepsis. Researchers will measure inflammation and muscle damage markers in 24 participants over 7 days. The goal is to understand if…

This study follows 60 people with Danon disease, a rare genetic heart condition, to learn how it progresses over time. Researchers will monitor heart function and symptoms in males aged 8 and older and females aged 8 to 50. The goal is to gather natural history data that could su…

This study is a national registry in the UK that collects health information from 2500 people with hypertrophic cardiomyopathy (HCM), a common inherited heart condition. Researchers will gather data from medical records, heart scans, blood tests, and genetics to better understand…

This study looks at whether a doctor's computer tool (MedSafer) and patient brochures (EMPOWER) can help reduce the use of potentially harmful medications in adults aged 60 and older with rheumatic diseases who take five or more regular drugs. Researchers will follow 100 particip…

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 looks at whether Ryeqo, a newer hormone treatment for uterine fibroids and endometriosis, increases the risk of dangerous blood clots. Researchers will take blood samples from 30 women before they start Ryeqo and again after 3 months of treatment. The goal is to measur…

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 testing a special PET scan that uses a tracer called 68Ga-FAPI to look for inflammation and scarring in the hearts of people with a specific type of heart failure (HFpEF). About 35 adults with this condition will get the scan to see how well it works. The goal is to…

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

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

This study looks 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 follows pregnant women with Pompe disease to see how the condition and its treatments affect pregnancy and infant growth. Researchers will track complications and monitor babies for up to three years after birth. No new drugs are being tested; participants receive thei…

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 is testing whether special insoles worn inside shoes can measure how well children with Duchenne muscular dystrophy (DMD) or spinal muscular atrophy (SMA) walk. About 106 participants, including healthy children, will wear the insoles during walking tests and in daily …

This study is testing whether a protein called titin, found in urine, can serve as a reliable marker of muscle injury in people with Duchenne or Becker muscular dystrophy. Researchers will measure titin levels before and after activities like walking down stairs, and track daily …

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 measures how hard children with conditions like neuromuscular disease, scoliosis, or heart problems work to breathe. Researchers use a thin tube placed through the nose into the esophagus to measure breathing muscle strength and effort. The goal is to better understand…