Muscle tissue disorder

This study tests a new treatment called cipaglucosidase alfa/miglustat in children (0 to under 18 years) with late-onset Pompe disease. The goal is to see if it is safe and how well it works. Participants either have never had enzyme therapy before or have been on it for at least…

This early-phase study tests a single injection of a gene therapy called V001-BCMA for people with autoimmune diseases like lupus that have not responded to standard treatments. The therapy targets and modifies specific immune cells to reduce disease activity. The main goal is to…

This study looks at the long-term safety and effects of a drug called EDG-5506 (sevasemten) in people with Becker muscular dystrophy, a muscle-weakening disease. About 260 adults and teens who were in earlier EDG-5506 studies will take the drug and be monitored for side effects a…

This study tests whether tranexamic acid, a drug that helps blood clot, can reduce blood loss during planned hip replacement surgery. About 1000 adults having elective hip replacement will receive the drug directly into the joint during surgery. The goal is to see if it lowers th…

This study tests whether a cancer-killing virus (T-VEC) combined with radiation can shrink soft tissue sarcomas before surgery. About 40 adults with newly diagnosed, removable sarcomas will receive the virus injected into the tumor plus radiation. The main goals are to see how ma…

This phase 4 study tests the drug mavacamten in 63 adults with a type of heart muscle disease called obstructive hypertrophic cardiomyopathy. The goal is to see if the drug can improve heart structure, measured by MRI, and ease symptoms like shortness of breath. Participants take…

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 tests an experimental drug called NS-089/NCNP-02 (Brogidirsen) in 20 boys with Duchenne muscular dystrophy whose genetic mutation can be fixed by skipping exon 44. The drug is given as a weekly IV infusion and aims to help the body produce a working version of the dyst…

This study tracks people with severe eosinophilic asthma who already take Fasenra (benralizumab) every 8 weeks. Participants use electronic monitors on their regular inhalers and a study-provided rescue inhaler (AirSupra) to see if asthma control stays steady throughout the dosin…

This Phase 2 trial tests a drug called RO7204239 in 51 adults with facioscapulohumeral muscular dystrophy (FSHD), a genetic condition that causes progressive muscle weakness. The drug is an antibody that blocks myostatin, a protein that normally limits muscle growth, to help pres…

This study tests a drug called mavacamten in 132 people with hypertrophic cardiomyopathy (thick heart muscle) that blocks blood flow in the middle or tip of the heart's main pumping chamber. The goal is to see if mavacamten can reduce the pressure difference across the blockage a…

This early-stage trial tests a single dose of a gene therapy called JWK007 in 3 boys aged 5-10 with Duchenne muscular dystrophy. The main goal is to check safety and tolerability, while also looking at whether it helps muscle function. It is a very small, first-in-human study, so…

This study tests a drug called BBP-418 (Ribitol) in 81 people aged 12 to 60 with limb girdle muscular dystrophy type 2I (LGMD2I), a genetic condition that causes progressive muscle weakness. Participants receive either the drug or a placebo for 36 months to see if it slows the di…

This study follows 17 people with Pompe disease who have already taken part in earlier trials of avalglucosidase alfa (Nexviadyme). It aims to check the drug's long-term safety and effectiveness by giving it every two weeks as an IV infusion. The study will continue until the dru…

This study tests a gene therapy called delandistrogene moxeparvovec in 13 children under age 4 with Duchenne muscular dystrophy. The goal is to see if it is safe and can help produce a key muscle protein. The children will be followed for about 5 years.

This study tests a device called a motor neuroprosthesis, an implantable brain computer interface designed to help people with severe paralysis control digital devices like computers or tablets. The device aims to bypass damaged motor pathways and transmit brain signals directly …

This study tests satralizumab, a drug that calms inflammation, in 30 children aged 8 to 17 with Duchenne muscular dystrophy. The goal is to see if it improves bone density and muscle function. Participants receive injections for several months while continuing standard steroid th…

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

This trial tested a gene therapy called AT132 for X-linked myotubular myopathy (XLMTM), a rare muscle disease present at birth that causes severe breathing problems. The therapy aimed to deliver a healthy copy of the MTM1 gene to improve muscle function. However, the study was st…

This phase 2 trial tests whether a patient's own fat-derived cells (stromal vascular fraction) can improve healing after surgical repair of a chronic rotator cuff tear. The study enrolls 56 adults aged 45-65 with full-thickness tears who have not improved with physical therapy. T…

This phase 2 trial tests whether saracatinib, an oral drug originally developed for cancer, can prevent new abnormal bone growth in adults with fibrodysplasia ossificans progressiva (FOP), a rare genetic condition where muscle and soft tissue turn into bone. Twenty adults with ac…

This early-stage study tests a gene therapy called AT845 for adults with late-onset Pompe disease, a condition that weakens muscles. The treatment aims to deliver a working copy of the GAA gene to muscle cells. Eleven participants who have been on standard enzyme replacement ther…

This phase 2 trial is testing the drug ruxolitinib in people with hypereosinophilic syndrome, a rare condition where the body produces too many eosinophils (a type of white blood cell), causing organ damage. The study aims to see if ruxolitinib can normalize blood cell counts and…

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 trial tests the long-term safety and effectiveness of apitegromab, an investigational antibody that blocks myostatin, a protein that limits muscle growth. People with type 2 or type 3 spinal muscular atrophy who completed earlier apitegromab studies can join. Researchers wil…

This study tests whether the drug sacubitril/valsartan can reduce scarring in the heart muscle of people with a type of heart failure called HFpEF (stiff heart). The trial will enroll 36 adults aged 50 and older with heart failure and a normal pumping function. Participants will …

This study follows 200 adults who receive the Shoulder iD™ implant to treat severe rotator cuff tears or shoulder arthritis. Researchers measure how well the shoulder works and how often the implant needs to be fixed or replaced over 10 years. Participants attend regular check-up…

This phase 2 trial tests a drug combination including daratumumab for people with AL amyloidosis, a rare disease where abnormal proteins damage organs, especially the heart. The study has two groups: one gets the full combo right away, the other starts some drugs later. Researche…

This Phase 3 trial tests a cell therapy called deramiocel (CAP-1002) in 106 boys and young men with Duchenne muscular dystrophy. Participants receive either the cell therapy or a placebo every 3 months for a year, then all can receive the therapy for another year. The goal is to …

This study tests a combination of two drugs, lurbinectedin and doxorubicin, in people with advanced soft-tissue sarcoma or leiomyosarcoma that cannot be cured with standard treatments. The first part finds a safe dose, and the second part compares the combination to lurbinectedin…

This trial tests whether injections of PRP lysate, a product made from a patient's own blood platelets, can improve shoulder pain, strength, and tendon tear size in adults with partial rotator cuff tears. Participants receive either PRP lysate or hypertonic dextrose prolotherapy,…

This study tests a single dose of a gene therapy called AB-1003 in 10 adults with a rare genetic muscle disease (LGMD2I/R9). The goal is to see if it is safe and can help improve muscle function. Participants must be able to walk or run 10 meters in under 30 seconds.

This study tests whether benralizumab can improve lung structure and function in people with severe eosinophilic asthma who still have symptoms despite standard treatments. About 75 adults aged 18-70 will receive either the drug or a placebo for 48 weeks, followed by a 4-week che…

This trial tests whether an experimental drug called HRS-1893 can improve symptoms and reduce heart-related events in people with obstructive hypertrophic cardiomyopathy, a condition where the heart muscle thickens and blocks blood flow. About 228 adults aged 18 to 85 with modera…

This early-phase trial tests whether injecting special donor cells (E-CEL UVEC) into the shoulder during rotator cuff surgery is safe and might improve healing. Twenty adults aged 45-70 with full-thickness tears will receive the cells at the repair site. The main goal is to check…

This study tests whether a low-caffeine green tea extract (EGCG) can shrink uterine fibroids and improve pregnancy and live birth rates in women aged 18-40 who have been trying to conceive for at least six months. Participants take the extract and are monitored for changes in fib…

This trial is testing whether a person's genetic makeup can predict how well they respond to three common pain treatments: low-level laser therapy, therapeutic ultrasound, and prolotherapy (a dextrose injection). Researchers will recruit 80 people with myofascial pain and 80 with…

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 phase 3 study is testing the long-term safety and effectiveness of an experimental drug called AOC 1001 (del-desiran) for people with myotonic dystrophy type 1 (DM1), a genetic muscle disorder. The trial enrolls 230 adults who have completed a prior AOC 1001 study and will r…

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 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 study follows 80 babies with severe congenital diaphragmatic hernia (CDH) who had a special surgery called FETO while still in the womb. Researchers want to see if these babies survive to leave the hospital more often and have fewer long-term health issues compared to babies…

This study tests whether a piece of donated aorta (the body's largest artery) can be used as a patch to repair severe windpipe or bronchial defects in children. The surgery is for kids who cannot have standard windpipe repair. Only 5 children will be enrolled, and the main goal i…

This study tests whether stem cells from donated umbilical cords can help boys with Duchenne muscular dystrophy (DMD). The cells are given through a vein and are meant to reduce inflammation and improve muscle function. The trial includes boys aged 5 to 10 who can still walk and …

This study tests a new growth hormone medicine called somapacitan, given once a week, in 47 children who are very short due to being born small for gestational age, or having Turner syndrome, Noonan syndrome, or idiopathic short stature. The main goal is to see if it is safe and …

This trial is testing an investigational drug called DYNE-251 in boys with Duchenne muscular dystrophy (DMD) who have a specific genetic mutation. The goal is to see if the drug can safely help their muscles produce dystrophin, a protein that is missing in DMD. Participants recei…

This study tests a single dose of SGT-001 gene therapy in 12 boys (children and teens) with Duchenne muscular dystrophy. The main goal is to check safety and how well the body tolerates the treatment. Participants will be followed for about 5 years to monitor side effects and any…

This Phase 3 trial tests an experimental drug called ALXN2220 in about 1,180 adults with transthyretin amyloid cardiomyopathy (ATTR-CM), a condition where abnormal protein builds up in the heart, causing heart failure. The drug is designed to remove those protein deposits. Partic…

This study tests the long-term safety and effectiveness of an experimental drug called VX-670 in adults with myotonic dystrophy type 1 (DM1). Participants who completed a previous VX-670 study can join. The drug is given through a vein, and researchers will monitor side effects a…

This early-phase trial tests a single intravenous dose of LY-M001 gene therapy in 9 children (ages 6 to 17) with type 1 Gaucher disease. The goal is to see if it is safe and can improve key symptoms like liver size and blood markers. Researchers will monitor participants for side…

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 compares two standard surgeries for people aged 60 and older with a severe three-tendon tear in the shoulder: reverse total shoulder replacement versus rotator cuff repair. Researchers will measure shoulder function, pain, and satisfaction one year after surgery to see…

This phase 2 trial tests whether daratumumab, an antibody that targets cancer cells, can improve outcomes for people with multiple myeloma after they receive a stem cell transplant. The study involves 31 participants and is currently active but not recruiting. The main goal is to…

This early-phase study tested a new gene editing treatment called HG302 in 4 boys with Duchenne muscular dystrophy (DMD), a severe muscle-wasting disease. The therapy uses CRISPR technology to fix the genetic error and help muscles produce dystrophin, a protein essential for musc…

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 early-stage trial is testing whether stem cells from a donor can help control heart failure in people whose hearts were weakened by a type of chemotherapy called anthracyclines. About 72 participants will either receive standard care alone or standard care plus stem cells gi…

This study tests an experimental cell therapy called tabelecleucel in 190 people with diseases caused by the Epstein-Barr virus (EBV), including certain lymphomas and post-transplant complications. The treatment uses specially prepared immune cells to fight the virus. The main go…

This phase 2 trial tests an experimental drug called AOC 1044 in people with Duchenne muscular dystrophy whose genetic mutation makes them candidates for exon 44 skipping therapy. The drug is given by intravenous infusion. Researchers are evaluating long-term safety and whether i…

This study tests a new drug called encaleret against usual treatments for people with a rare genetic condition called autosomal dominant hypocalcemia type 1 (ADH1), which causes low blood calcium and high urine calcium. About 67 participants will receive either encaleret or stand…

This study compares a newer pacemaker method, left bundle branch area pacing (LBBAP), to the standard right ventricular pacing in 200 patients with heart block and normal heart function. The goal is to see if LBBAP better preserves the heart's pumping ability and reduces complica…

This phase 2 trial is testing whether combining daratumumab, venetoclax, and dexamethasone can produce a deep and rapid response in people newly diagnosed with light-chain (AL) amyloidosis who carry a specific genetic marker called t(11;14). The study enrolls 36 participants and …

This trial tests whether an 8-week course of the immunosuppressive drug prednisone can reduce episodes of ventricular tachycardia (a potentially fatal fast heart rhythm) and improve heart function in people with non-ischemic cardiomyopathy and confirmed heart inflammation. Partic…

This study is for people with Duchenne muscular dystrophy (DMD) who have already taken part in a previous trial of BMN 351. It tests the long-term safety of weekly intravenous infusions of BMN 351 and whether it helps maintain physical function. Only 18 participants will be enrol…

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 gene therapy called PF-06939926 in 114 boys with Duchenne muscular dystrophy (DMD), a severe muscle-wasting disease. Two-thirds receive the gene therapy, while one-third get a placebo, but can switch to the real treatment after one year. The main goal is to see…

This phase II trial tests whether simvastatin, a widely used cholesterol medication, can reduce the size of uterine fibroids and ease related symptoms. Researchers will give 60 women either simvastatin or a placebo daily for 12 weeks, measuring fibroid volume by ultrasound and tr…

This study tests the long-term safety of a drug called efgartigimod PH20 SC in adults with active idiopathic inflammatory myopathy, a group of rare muscle diseases. Participants who completed a previous trial will receive the drug as a shot under the skin for up to 51 months. The…

This study tests an experimental drug, BBP-418, in people with limb girdle muscular dystrophy type 2I (LGMD2I), a rare genetic condition that causes progressive muscle weakness. The drug aims to fix a molecular defect by helping a faulty enzyme work better. The trial includes bot…

This study is a long-term extension of an earlier trial, testing the safety of a drug called RAY121 in people with six different immune system disorders, including antiphospholipid syndrome and immune thrombocytopenia. Participants have already completed four doses of RAY121 and …

This phase 2 study is testing whether combining two drugs, rucaparib and nivolumab, can treat advanced or metastatic leiomyosarcoma (LMS), a rare soft-tissue cancer. The 20 participants take rucaparib pills twice daily and get nivolumab infusions every four weeks. The main goal i…

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 an experimental drug, garetosmab, in 63 adults with fibrodysplasia ossificans progressiva (FOP), a rare condition where soft tissues turn into bone. The goal is to see if the drug safely reduces new bone growth and painful flare-ups. Participants receive either t…

This early-phase trial tests a combination of three drugs—azacitidine, venetoclax, and pevonedistat—in adults newly diagnosed with a challenging form of acute myeloid leukemia (AML). The goal is to find the best dose and see if the combo can improve remission rates. About 40 part…

This early-phase trial tests whether adding the drug isatuximab to standard chemotherapy can safely treat people with high-risk AL amyloidosis, a rare disease where abnormal proteins damage organs. Eleven participants will receive the combination to see if it reduces toxicity and…

This study tests a new treatment called NXC-201 CAR-T 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 the cells producing these proteins. The trial is open to …

This study is for people with FSHD, a genetic disease that causes muscle weakness. It tests the long-term safety and how well the body tolerates a drug called AOC 1020, given through a vein. About 84 adults who completed a previous study will take part. The main goal is to check …

This trial tests whether a custom-made plastic implant (ultra-high molecular weight polyethylene) works better than a standard titanium implant for people with chronic jaw dislocation. Participants have a jaw that repeatedly dislocates and has not improved with conservative treat…

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 trial investigates whether adding dry needling to manual therapy helps people with myofascial headache. Participants receive either manual therapy alone or manual therapy plus dry needling over four sessions. The study measures changes in headache-related disability, neck fu…

This study tests a program called PIXI that coaches parents of infants diagnosed with rare neurogenetic disorders (such as Fragile X, Angelman, or Down syndrome) during the first year of life. The program combines education about the disorder, guided parent-child interaction acti…

This Phase 3 study tests whether benralizumab, a biologic injection given every 4 weeks, can reduce flares in people with hypereosinophilic syndrome (HES), a rare condition where too many eosinophils cause organ damage. About 134 patients aged 12 and older will receive either ben…

This phase 2 trial tests an oral drug called venglustat, given alongside the standard enzyme therapy Cerezyme, in adults with Gaucher disease type 3 (a rare genetic disorder affecting the body and brain). The study has four parts: first, it checks spinal fluid biomarkers to disti…

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 follows 175 people with spinal muscular atrophy who previously received the gene therapy Zolgensma in clinical trials. Researchers will monitor them for 5 years to track serious side effects and developmental milestones. The goal is to understand how safe and effective…

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 trial investigates whether intravenous infusions of tiny particles (extracellular vesicles) from heart progenitor cells can safely improve heart function in people with severe dilated cardiomyopathy and heart failure. Participants receive three infusions and are monitored fo…

This study is testing the long-term safety of a daily oral drug called zagociguat in 44 adults with MELAS, a rare genetic disease that affects energy production in cells. All participants previously completed a lead-in study of the same drug. Researchers will monitor side effects…

This study tests the long-term safety and effectiveness of an experimental drug called BBP-418 (ribitol) in people with limb-girdle muscular dystrophy type 2I/R9, a rare genetic muscle-weakening disease. Participants who completed a previous study will take BBP-418 orally twice d…

This phase 3 study tests Coagadex, a human clotting factor X, in 15 adults with acquired factor X deficiency caused by AL amyloidosis. The goal is to see if it safely stops active bleeding and helps manage bleeding during surgery. Participants receive the drug on demand for bleed…

This phase 3 trial is testing a drug called aficamten in 36 Japanese adults with symptomatic obstructive hypertrophic cardiomyopathy, a condition where the heart muscle thickens and makes it hard to pump blood. The drug aims to reduce over-contraction of the heart muscle. Researc…

This early-stage trial is testing a drug called BMN 351 in 18 boys aged 4 to 10 with Duchenne muscular dystrophy who have a specific genetic change. The drug is designed to help the body produce a shorter but still useful version of the muscle protein dystrophin. The main goal is…

This study follows 16 infants aged 6 months or younger with infantile-onset Pompe disease, a rare genetic disorder that weakens muscles and breathing. All receive alglucosidase alfa (Myozyme) as part of their routine care. Researchers track how many survive without needing a brea…

This Phase 2 trial tests whether N-Acetylcysteine (NAC), an antioxidant, can help people with ME/CFS (chronic fatigue syndrome). Researchers will give 95 participants different doses of NAC or a placebo for four weeks, then measure brain glutathione levels and markers of oxidativ…

This phase 3 trial tests whether adding lurbinectedin to standard doxorubicin chemotherapy helps people with metastatic leiomyosarcoma live longer without their cancer growing. About 469 adults who have not had prior treatment for advanced disease are being studied. The main goal…

This study tests whether switching to avalglucosidase alfa is safe and more effective for people with late-onset Pompe disease whose condition has worsened on the standard treatment, alglucosidase alfa. Participants receive biweekly infusions of the new drug and are monitored for…

This program allows eligible patients to receive RGX-202, a gene therapy, on a single-patient basis outside of a clinical trial. It is designed for those with serious conditions who have no other treatment options. Currently, the program is temporarily not available.

This early-stage trial is testing a new treatment called KITE-363 for people with severe autoimmune diseases like lupus and scleroderma that haven't improved with standard treatments. KITE-363 is a type of cell therapy where a patient's own immune cells are modified in a lab to t…

This study tests if a new growth hormone medicine (somapacitan) given once a week works as well as the standard daily growth hormone (Norditropin) for children who are very short due to being born small, or having Turner syndrome, Noonan syndrome, or unknown causes. About 412 chi…

This study tests a drug called eplontersen in Chinese adults with a heart condition caused by abnormal protein buildup. The goal is to see if the drug lowers that protein and is safe over time. Participants will be randomly assigned to receive the drug or a placebo for 24 weeks, …

This study aimed to see if taking Myfembree (a combination pill) daily after fibroid removal surgery could delay the return of fibroids and improve quality of life. It was designed for premenopausal women with uterine fibroids who had surgery. However, the trial was withdrawn bef…

This early-phase trial tests a new cell therapy called BEN301 for several autoimmune diseases, including scleroderma and rheumatoid arthritis. The therapy uses specially engineered immune cells (CAR-Treg cells) to calm the overactive immune system. The study involves 24 adults an…

This phase 2 trial tests whether nipocalimab, given intravenously, can improve symptoms of active idiopathic inflammatory myopathies (myositis) better than a placebo. About 36 adults with myositis will receive either nipocalimab or a placebo, along with standard steroid treatment…

This early study tests a new device called SINEFIX to repair small rotator cuff tears (up to 2 cm) in 9 adults. The implant is placed during keyhole surgery to reattach the torn tendon to the bone. Researchers will use MRI scans to check if the tendon heals properly and if there …

This study is testing a new robotic system, the Medtronic Hugo™, for performing hysterectomies. It includes 70 patients with conditions like uterine fibroids, endometriosis, or certain cancers. The goal is to see if the robot is safe and can complete the surgery without switching…

This study tests a device called AccuCinch that is placed around the heart to help it pump more effectively. It is for people with heart failure and a weakened heart muscle. The goal is to see if the device improves quality of life, exercise ability, and reduces serious heart eve…

This study aimed to test a new type of cell therapy (called CAR T-cells) in people with severe lupus or inflammatory myopathy that did not get better with standard treatments. The therapy uses donor cells to target and destroy faulty immune cells. However, the study was withdrawn…

This study tests a new device that combines two heart therapies: one to strengthen heartbeats and another to stop dangerous rhythms. About 300 adults with heart failure and a weak heart pump will get the device. Researchers will check if it safely converts life-threatening heart …

This Phase 3 trial tests a gene therapy called SRP-9001 for Duchenne muscular dystrophy (DMD), a severe muscle-wasting disease. It includes 148 males who can and cannot walk. Participants receive a one-time IV infusion of the therapy or a placebo, and are followed for about 128 w…

This study is testing a special implant (Tapestry) that helps repair a key shoulder tendon (subscapularis) during shoulder replacement surgery. About 100 adults with shoulder arthritis or injury will be followed for 2 years to see if the tendon heals better with the implant. The …

This phase 2 trial tests two oral drugs, ribociclib and everolimus, in people with advanced dedifferentiated liposarcoma or leiomyosarcoma who have had at least one prior treatment. The goal is to see if the combination can stop or slow tumor growth. About 48 participants will ta…

This study tests whether a special heart pacing technique (left bundle branch pacing) combined with standard heart failure drugs can improve heart function better than drugs alone. It includes 50 adults with non-ischemic heart failure and a specific heart block. The main goal is …

This study is for people with systemic amyloidosis, a disease where abnormal proteins build up in organs. It tests the long-term safety of an experimental drug called AT-02. About 120 adults will receive the drug and be monitored for side effects and how the drug behaves in the b…

This study follows 500 people with Duchenne muscular dystrophy to see how well a gene therapy (ELEVIDYS) works compared to standard steroid treatment over time. Researchers will measure movement abilities, breathing, and safety, including liver problems. Participants must already…

This study is testing whether a special pacemaker (cardiac resynchronization therapy, or CRT) can improve heart function and symptoms in people with mild heart failure and a specific type of heart block called left bundle-branch block. About 21 participants will receive a CRT dev…

This phase II trial tests whether combining cabozantinib (a targeted therapy) with temozolomide (a chemotherapy drug) can slow tumor growth in people with advanced leiomyosarcoma or other soft tissue sarcomas that cannot be surgically removed or have spread. About 96 participants…

This study tests a one-time gene therapy called RGX-314 for wet age-related macular degeneration (AMD), a leading cause of vision loss. The treatment aims to help the eye produce its own medicine to stop leaky blood vessels, potentially replacing the need for repeated eye injecti…

This trial aimed to test an experimental drug called CM-336 for people with AL amyloidosis that has returned, not responded to treatment, or only partially responded after initial therapy. The drug works by helping the immune system attack and destroy harmful cells. However, the …

This pilot study tests a device called Travagus One, which stimulates a nerve in the ear to reduce inflammation in boys with Duchenne muscular dystrophy (DMD). Twenty boys aged 5-17 will use the headset at home for 5 minutes twice a day for one week. Researchers will measure infl…

This study follows about 80 boys with Duchenne muscular dystrophy who have already taken vamorolone in earlier studies. Researchers want to see how safe the drug is over a longer time, especially its effect on bone health, like spine fractures. The boys continue taking vamorolone…

This study tests whether combining two drugs, lenvatinib and pembrolizumab, can safely shrink or control advanced soft tissue sarcomas that have spread or can't be removed by surgery. About 52 adults with specific sarcoma types will receive the combination. The goal is to measure…

This study looks at the long-term safety and effects of a drug called SAT-3247 in 10 people with Duchenne muscular dystrophy who were in a previous trial. Participants take the drug by mouth five days a week for about 11 months. Researchers will check for side effects and measure…

This study is checking the long-term safety of a drug called eplontersen in people with ATTR-CM, a heart condition caused by abnormal protein buildup. About 1400 adults who completed earlier studies will receive eplontersen injections. Researchers will monitor side effects, kidne…

This study tests whether adding stem cells from a patient's own fat tissue to standard rotator cuff surgery can improve healing and reduce recovery time. About 30 adults with shoulder tendon tears will receive the stem cell injection during surgery. The goal is to see if this sim…

This study tests whether a person's own stem cells can be safely injected into the spinal fluid to treat multiple system atrophy (MSA), a rare and serious brain disease. About 30 adults aged 30-80 with MSA will receive the treatment. The main goal is to check for side effects, an…

This study tests a new enzyme replacement therapy called avalglucosidase alfa in babies with infantile-onset Pompe disease, a rare genetic disorder that causes severe muscle weakness and breathing problems. The treatment is given through an IV every other week for up to 4 years. …

This phase 2 trial is testing whether a patient's own bone marrow stem cells can improve heart function and ease symptoms in people with dilated cardiomyopathy and heart failure. Twenty participants will have stem cells collected from their hip and then infused into their heart a…

This early-stage trial tests a daily injection of ASA-001 in two adults with a rare genetic muscle disease (ADSS1 deficient myopathy). The main goals are to see if the drug is safe and if it can slow the disease. Participants will take the drug for 8 months and have clinic visits…

This study is following 110 people who received a Tornier HRS shoulder replacement to see how well it improves pain and function. Participants have conditions like arthritis, fractures, or rotator cuff tears. The goal is to collect long-term safety and performance data for regula…

This early study tests a new freezing treatment for people who recently had a heart attack or unstable chest pain. The treatment uses a special balloon to freeze high-risk plaque in the heart's arteries that could rupture and cause another heart attack. The goal is to stabilize t…

This study follows 66 boys with Duchenne muscular dystrophy who previously received RGX-202 gene therapy. Researchers will monitor side effects and measure muscle function over time, such as how fast they can stand, walk, or climb. The goal is to see if the treatment remains safe…

This study tests a new treatment for cancers linked to the Epstein-Barr virus (EBV), such as Hodgkin lymphoma and nasopharyngeal carcinoma. The treatment uses immune cells from healthy donors that are specially trained to attack EBV-infected cells. These cells are stored in a ban…

This study tests a special body suit that uses gentle electric pulses to stimulate muscles, helping astronauts exercise more efficiently in space. Twelve astronauts on the International Space Station will wear the suit during their workouts. Researchers will measure muscle stiffn…

This study tests whether adding the drug sodium valproate to a standard jaw-washing procedure can lower inflammation in people with a painful jaw joint condition. Ten female participants with jaw clicking and pain will receive the drug intravenously during the procedure. Research…

This study tests a new surgical technique to remove large uteri through the vagina using a sealed bag and electric fragmentation. The goal is to avoid abdominal scars and prevent the spread of tissue fragments. Twenty-five women aged 38 to 52 with large uteri are participating. T…

This study tests whether a special fish-oil emulsion given through an IV can improve outcomes for newborns with a diaphragm defect that causes severe lung pressure. About 40 babies will receive either the fish-oil emulsion or a standard soy-oil one. Researchers will measure lung …

This study tests a drug called vosoritide in 56 children with short stature caused by certain genetic conditions. The drug targets the growth plate to help children grow faster. Participants are observed for 6 months, then treated with daily injections for 12 months to check safe…

This study tests a procedure called FETO, where a tiny balloon is placed in the windpipe of unborn babies with severe congenital diaphragmatic hernia (CDH). The balloon helps the lungs grow before birth and is later removed. Researchers want to see if this improves survival and r…

This early-phase study tests a new treatment called Prula-cel for people with autoimmune diseases like lupus, scleroderma, and vasculitis. Prula-cel uses donor immune cells engineered to target and destroy faulty immune cells that attack the body. The goal is to find a safe dose …

This trial tests whether a procedure called catheter ablation works better than anti-arrhythmic drugs for people with structural heart disease who have had a dangerous fast heart rhythm (ventricular tachycardia). About 162 participants will be randomly assigned to either ablation…

This study tests a fully enclosed protective bag used during laparoscopic myomectomy to remove uterine fibroids. The bag is meant to keep fibroid tissue completely isolated, preventing any leakage or spread during the procedure. Twenty women aged 28-46 with fibroids 6-10 cm will …

This early-phase study tests the safety of combining two immunotherapy drugs—nivolumab and BO-112—given before surgery for soft tissue sarcoma that can be removed. About 14 adults with certain sarcoma types will receive these drugs to see if they help the immune system fight the …

This study was designed to test a new treatment for people with autoimmune diseases like lupus, scleroderma, and myositis. It combined a specially engineered immune cell product (CLBR001) with a targeted antibody drug (SWI019), with or without a preparatory chemotherapy step. How…

This study looks at the long-term safety of an experimental drug called WVE-N531 in people with Duchenne muscular dystrophy (DMD) who have already taken it in a previous study. About 175 participants will receive the drug and be monitored for side effects, heart and lung function…

This study tests a single dose of SRP-9003 gene therapy in 17 people with limb girdle muscular dystrophy 2E/R4, a genetic muscle-weakening disease. The goal is to restore a missing protein in muscle cells and improve muscle function. Both walkers and non-walkers can join, and the…

This expanded access program gave a single patient with Leber Hereditary Optic Neuropathy (a genetic cause of vision loss) an experimental gene therapy called GS010. The treatment was injected into both eyes to test safety. Only one person was involved, so the results are very li…

This Phase 3 trial tests whether higher doses of eteplirsen (Exondys 51) can safely improve muscle function in boys with Duchenne muscular dystrophy whose genetic mutation allows exon 51 skipping. About 160 ambulatory boys will receive one of three doses (30, 100, or 200 mg/kg) t…

This study tests whether adding intravenous immunoglobulin (IVIg) to standard steroid treatment early in myositis helps patients improve faster. Myositis is a group of rare diseases causing muscle inflammation and weakness. The trial includes 44 adults diagnosed within the past y…

This study is for people with a rare heart condition called ATTR amyloidosis with cardiomyopathy, where abnormal protein builds up in the heart. It tests the long-term safety and effectiveness of a drug called vutrisiran, given as an injection every three months. About 700 adults…

This study aimed to test a monthly injection called ION-682884 in people with TTR amyloid cardiomyopathy, a condition where abnormal protein deposits damage the heart. Only patients who had already completed a 2-year study of a similar drug (inotersen) were eligible. The goal was…

This early-phase trial tests a single-dose gene therapy called SRP-9003 in 6 people with limb girdle muscular dystrophy type 2E/R4, a rare genetic muscle-weakening disease. The main goals are to check safety and see if the therapy can produce the missing beta-sarcoglycan protein …

This early-stage trial is testing a new treatment called KYV-101 for people with severe autoimmune diseases like lupus, scleroderma, and vasculitis. The treatment involves taking a patient's own immune cells, modifying them to target and destroy faulty B cells, and giving them ba…

This phase 3 trial tests a gene therapy called NR082 for Leber's hereditary optic neuropathy (LHON), a genetic condition that causes rapid vision loss. About 95 people aged 12 to 75 with a specific ND4 mutation will receive a single injection of the therapy or a sham procedure. T…

This study aims to see if growing a patient's tumor cells in the lab (called organoids) can help doctors choose the most effective first-line chemotherapy for advanced leiomyosarcoma, a rare cancer of smooth muscle. About 50 adults aged 18-75 who have not yet received systemic tr…

This study tests whether injecting atelocollagen and hyaluronic acid during rotator cuff surgery improves healing. 124 people aged 40-75 with confirmed tears are enrolled. They receive one or both injections during arthroscopic repair and are followed for 2 years with MRI scans a…

This study looks at how children aged 1 month to 15 years with a late-diagnosed congenital diaphragmatic hernia (a hole in the muscle that separates the chest from the belly) do after surgery. Researchers reviewed 10 cases treated at one hospital with either keyhole or open surge…

This study compares two common injections—platelet-rich plasma (PRP) and corticosteroids—given during arthroscopic rotator cuff repair. 387 adults with full-thickness supraspinatus tears are randomly assigned to receive PRP, corticosteroid, or a placebo injection during surgery. …

This early-phase trial is testing a CAR-T cell therapy called RD06-04 in 12 people with active autoimmune diseases like lupus, scleroderma, and vasculitis. The treatment involves reprogramming a patient's own immune cells to target and destroy faulty B cells. The main goal is to …

This early-phase trial planned to test a one-time infusion of genetically modified immune cells (CAR-T cells) targeting CD19 in people with several autoimmune diseases, including lupus and multiple sclerosis. The goal was to see if it was safe and could reduce harmful antibodies.…

This phase 2 trial tests a new enzyme replacement therapy called avalglucosidase alfa (Nexviazyme) in 22 children with infantile-onset Pompe disease who are not responding well to standard treatment. The study aims to see if the new drug is safe and can improve outcomes. Particip…

This study tests a modified plan to prevent and treat cytomegalovirus (CMV) infection in people with blood cancers who have had a donor stem cell transplant. CMV is a common virus that can cause serious problems after transplant. The study involves 153 participants and looks at w…

This study tests whether nicotinamide riboside, a form of vitamin B3, can improve walking distance and muscle function in adults with mitochondrial myopathy, a rare muscle disorder. Thirty-four participants receive either the supplement or a placebo for several months. The main g…

This study follows 900 people with hypertrophic cardiomyopathy who have already taken aficamten in earlier trials. Researchers want to see if the drug is safe and tolerable over a longer period. Participants take aficamten tablets daily, with regular clinic visits to monitor for …

This early-phase trial tests a new drug called RAG-18 in 12 boys aged 4-15 with Duchenne muscular dystrophy. The drug is a small activating RNA given through an IV, designed to help muscles work better. The main goal is to check safety and how the body handles the drug, with earl…

This phase 3 trial tests whether sirolimus, a drug used in organ transplants, can slow disease progression in people with inclusion body myositis (IBM), a rare muscle-weakening condition. The study involves 140 adults aged 45 and older who can walk at least 200 meters. Over 84 we…

This early-stage study is testing a new treatment called CNTY-101 for people with severe autoimmune diseases like lupus, scleroderma, and inflammatory myopathies that haven't improved with standard therapies. CNTY-101 uses specially engineered immune cells to target and destroy f…

This study tests a stem cell transplant method for people with inherited metabolic disorders and severe osteopetrosis. The goal is to get the donor cells to take hold while keeping side effects low. Participants receive chemotherapy drugs before the transplant to prepare their bo…

This study tests whether a structured diet, exercise, and lifestyle program can help prevent uterine fibroids from returning after surgery. Researchers will follow 100 women aged 18-50 who recently had fibroids removed and are currently fibroid-free. The goal is to see if the pro…

This study compares two ways to recover after reverse total shoulder replacement: using a smartphone app (mymobility) for home exercises versus standard in-person physical therapy. Researchers will enroll 100 adults to see if the app works just as well. The main goal is to measur…

This program offers garetosmab to adults with fibrodysplasia ossificans progressiva (FOP) who finished the main OPTIMA study. The goal is to provide the drug before it is officially approved. Participants must meet specific health criteria to join.

This early-stage trial tests a gene therapy called LX2006 in 8 people with Friedreich's ataxia who also have heart muscle disease. The therapy delivers a healthy copy of the FXN gene to heart cells using a modified virus. The main goal is to check safety over 5 years, while also …

This study tests whether colchicine, a drug used for gout, can improve blood vessel stiffness and heart function in people with high blood pressure. About 150 adults with hypertension and additional risks like diabetes or high cholesterol will take colchicine or a placebo daily f…

This study tests a drug called mavacamten in 44 teenagers aged 12 to 18 who have a condition where the heart muscle is too thick and blocks blood flow. The goal is to see if the drug can reduce the blockage and improve symptoms. Participants are randomly assigned to receive eithe…

This study tests whether a combination of anti-inflammatory and heart-protecting drugs can reduce symptoms and heart damage in people with Long COVID who have ongoing heart inflammation. About 279 adults with heart inflammation seen on MRI will receive either the drug combo or a …

This early-phase trial tests whether metformin, a widely used diabetes drug, can prevent muscle loss and insulin resistance in healthy older adults during five days of bed rest. Researchers will measure changes in muscle size and insulin sensitivity. The study involves 64 partici…

This study tests a one-time gene therapy called AB-1002, given directly into the heart arteries, for people with advanced heart failure (NYHA Class III). The goal is to see if it can improve heart function, reduce symptoms, and lower the risk of death or hospitalization. About 15…

This phase 2 trial is testing whether the oral chemotherapy drug ixazomib can help control AL amyloidosis after initial treatment. Seventeen adults with the disease will take ixazomib along with low-dose dexamethasone to see if it delays organ progression or the need for more the…

This study is testing a shoulder replacement device called the Medacta Shoulder System in 100 people with shoulder arthritis or rotator cuff tears. The goal is to see how well the implant works after 2 years, focusing on shoulder function, safety, and patient satisfaction. Partic…

This study tests the long-term safety of an experimental drug called NNC6019-0001 in people with transthyretin amyloid cardiomyopathy, a rare heart disease that can lead to heart failure. All 80 participants have already completed an earlier study and will receive the drug every …

This study tests a special implant called TAPESTRY that is placed during shoulder surgery to support healing of torn rotator cuff tendons. About 170 adults with partial or full-thickness tears will receive the implant and be followed for 6 months. The main goal is to see if the i…

This study follows 10 people who already received LX2020 gene therapy for arrhythmogenic cardiomyopathy caused by a PKP2 gene mutation. Researchers will monitor them for years to see if the treatment remains safe and continues to help control the disease. The goal is to understan…

This early-stage trial tests a gene therapy called LX2020 for people with a heart condition called arrhythmogenic cardiomyopathy, caused by a change in the PKP2 gene. The study involves 10 adults who already have a defibrillator implanted. The main goal is to see if the treatment…

This study tests an experimental drug called sevasemten in 76 children aged 4-9 with Duchenne muscular dystrophy. The goal is to check the drug's safety, how the body processes it, and its effect on muscle health markers. Participants are randomly assigned to receive the drug or …

This study tests an experimental drug called EDG-5506 (sevasemten) in 43 children and teens aged 6 to 17 with Duchenne muscular dystrophy who have already received gene therapy. The goal is to see if the drug is safe and how it affects muscle damage markers. Participants are rand…

This study tests the long-term safety and effectiveness of a cell therapy called deramiocel (CAP-1002) in people with Duchenne muscular dystrophy who completed the earlier HOPE-2 trial. Participants receive an infusion of deramiocel every 3 months for about 5 years, with the opti…

This study follows 75 people with Gaucher disease type 1 who previously received FLT201 gene therapy. Researchers will monitor them for 5 years to see if the treatment remains safe and if its effects last. The goal is to understand whether a single dose can provide long-term dise…

This pilot study tests a procedure called FETO in 14 unborn babies with severe left congenital diaphragmatic hernia (CDH), a birth defect where abdominal organs push into the chest and stunt lung growth. Doctors place a tiny balloon in the baby's windpipe via fetoscopy to trap lu…

This pilot study is testing the drug tirzepatide (Zepbound) in 40 older adults with obesity to see how it affects body composition, bone density, and physical function over nine months. Participants take weekly injections, track their diet and activity, and attend regular check-i…

This study tests an experimental drug called andecaliximab in 92 children and adults with fibrodysplasia ossificans progressiva (FOP), a rare genetic condition where soft tissues turn into bone. The goal is to see if the drug can safely reduce new bone growth and flare-ups. Parti…

This study is checking the long-term safety of a drug called acoramidis in people with a rare heart condition called transthyretin amyloid cardiomyopathy. About 389 participants who finished a previous 30-month trial will take acoramidis twice daily for up to 5 years. Researchers…

This study tests a procedure called FETO, where a tiny balloon is placed in the windpipe of a fetus with severe left congenital diaphragmatic hernia (CDH). The balloon blocks fluid from leaving the lungs, causing them to grow larger before birth. The goal is to improve survival f…

This early-stage trial tests a gene therapy called ATA-100 for people with LGMDR9, a rare genetic muscle disease that causes progressive weakness. Six adults receive a single intravenous infusion of the therapy, which delivers a working copy of the FKRP gene. The main goal is to …

This study tests a drug called empasiprubart in adults with dermatomyositis, a rare disease causing muscle weakness and skin rash. Three participants receive either the drug or a placebo for 25 weeks, then are followed for 65 more weeks. The goal is to see if the drug is safe and…

This study tests the long-term safety and effectiveness of an experimental drug called DS-5141b (Renadirsen) in 8 people with Duchenne muscular dystrophy who have already completed a previous study. Participants receive a weekly injection under the skin. Researchers will monitor …

This study tests whether waiting to clamp the umbilical cord until after the baby's lungs have filled with air can reduce dangerous lung blood pressure (pulmonary hypertension) in newborns with congenital diaphragmatic hernia (CDH). About 140 infants with left-sided CDH will be r…

This study compares two types of breathing muscle training in people who have had a lung transplant. One group does standard constant-resistance exercises, while the other adds short bursts of harder effort. The goal is to see which method better improves breathing strength and d…

This Phase 3 trial tests dexpramipexole, an oral tablet, as an add-on treatment for people with eosinophilic asthma that is not well controlled. About 600 participants aged 12 and older will receive either the drug or a placebo for 24 weeks. The study measures improvements in lun…

Functional somatic disorders cause persistent physical symptoms that doctors cannot explain by other diseases. The DISTRESS trial evaluates a new clinic where internal medicine doctors, trained to recognize these disorders, examine patients and provide guidance to their general p…

Researchers at Beijing Anzhen Hospital are studying whether a computer model that combines two types of heart scans can predict 12-month survival in people with light-chain cardiac amyloidosis, a condition where abnormal proteins stiffen the heart. The study enrolls 49 patients w…

Researchers are testing whether artificial intelligence can combine ultrasound images with routine health data to track changes in a person's physiology over time. The study enrolls adults with heart, lung, kidney, or blood vessel conditions, as well as healthy volunteers. The go…

This study will test if artificial intelligence (AI) can spot a rare heart condition called ATTR-CM in people with heart failure. Researchers will use AI to analyze electrocardiograms (ECGs) and echocardiograms (Echo) from 150 adults. The AI's findings will be checked against a s…

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 whether artificial intelligence can detect transthyretin amyloid cardiomyopathy (ATTR-CM), a serious and often underdiagnosed heart condition, earlier than usual. Researchers will use AI algorithms to analyze routine ECGs and heart ultrasounds from up to 1.5…

This study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…

This study looks at whether adding just 3 minutes to a cardiac MRI can help doctors find heart inflammation (myocarditis) early in cancer patients on immunotherapy. Myocarditis is a rare but often deadly side effect of these drugs. The researchers will scan 200 patients who alrea…

This study tests a new, non-invasive ultrasound method called electromechanical wave imaging (EWI) to see if it can more accurately locate and diagnose heart rhythm problems compared to standard tests. About 322 adults already scheduled for a heart procedure will get an EWI scan …

This study tests a new injection called NEMO-103 to see if it makes shoulder MRI pictures clearer for diagnosing problems like torn tendons or cartilage. About 85 adults with known or suspected shoulder issues will get both a regular MRI and one with the injection. The goal is to…

This study tests whether an artificial intelligence (AI) program can detect hypertrophic cardiomyopathy (HCM), a serious heart condition, using ECGs from standard machines, the Apple Watch, and the KardiaMobile device. Researchers will compare the AI's accuracy across these devic…

This study was designed to see if a special MRI technique (BOLD) could measure how well fetal lungs work in babies with diaphragmatic hernia or abdominal wall defects. Researchers planned to scan the lungs while the mother breathed normal air and then oxygen, looking for changes …

This study is testing a way to find cardiac amyloidosis—a rare but serious heart condition—earlier in people aged 65 and older. Researchers in Turkey will screen 800 patients at family medicine clinics using simple blood tests and medical history. The goal is to see if this appro…

This study tests whether a new, more powerful 7 Tesla MRI can better detect scarring and swelling in the heart muscle of people with cardiomyopathy. Researchers will scan 13 adults aged 20-70 to see if the higher-resolution images improve diagnosis. The goal is to see if this adv…

This observational study tests three non-invasive technologies—ultrasound, electrical impedance, and nerve excitability tests—to see if they can reliably diagnose myofascial pain syndrome. Researchers will compare results from 90 people with and without the condition. The goal is…

This study looks at whether a special biopsy done with ultrasound guidance before surgery can correctly tell if a uterine tumor is cancerous or not. It involves 19 women aged 35 and older who have suspicious uterine tumors. The goal is to avoid risky surgical procedures if the tu…

This study aims to create a simple test battery to determine fall risk in people with neuromuscular disorders, such as muscular dystrophy or ALS. Researchers will assess 108 participants using several physical tests like walking, standing, and rising from a chair. The goal is to …

This study investigates whether a painless fluorescent light exam can help find early warning signs of oral cancer in adults. Participants complete a health questionnaire and receive a standard oral exam plus a fluorescence scan. The goal is to see if this simple, non-invasive to…

This study tests the Shingrix vaccine in over 2,000 adults with autoimmune rheumatic diseases like rheumatoid arthritis and lupus, who are at higher risk for shingles. Researchers will compare vaccinated patients to unvaccinated ones to see if the vaccine is safe and triggers a s…

Researchers test whether a short prehabilitation program before rotator cuff repair helps people over 50 recover better. The program includes two 60-minute sessions one week before surgery, covering postoperative stages, pain management, sling use, daily activity strategies, and …

This study compares two 12-week exercise programs—eccentric training and heavy-slow resistance training—in 30 adults aged 18–50 with patellar tendinopathy (knee pain lasting over 3 months). Researchers measure changes in tendon thickness, stiffness, strength, and pain to see whic…

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

This trial tests whether shockwave therapy can improve knee function, balance, and fear of movement in adolescents with patellar tendinopathy, a common overuse injury causing knee pain. Fifty teens with pain in one knee will receive low-energy shockwaves to the tendon and thigh m…

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 compares biofeedback training to lifestyle changes (like special positions and massage) for women with dyssynergic defecation, a condition where pelvic floor muscles don't relax properly during bowel movements. About 58 women aged 35-65 with chronic constipation will p…

This study looks at whether a procedure called microwave ablation can safely and effectively treat symptomatic uterine fibroids in 100 premenopausal women. The procedure uses ultrasound guidance to deliver heat directly to the fibroids, aiming to shrink them and reduce symptoms l…

This trial compares two gentle manual therapies—the Emmett Technique and Bowen Therapy—for people with upper trapezius myofascial pain syndrome, a common cause of neck pain and stiffness. About 46 adults aged 18 to 45 with chronic neck pain will be randomly assigned to receive on…

This trial compares two pain medications, tramadol and tapentadol, to see which provides better pain relief after arthroscopic rotator cuff repair surgery. Adults aged 18-80 undergoing this elective shoulder surgery will receive one of the two drugs as part of a multimodal pain m…

This study compares two manual therapy techniques—Positional Release Technique and Post Isometric Relaxation—to see which better reduces pain, improves neck movement, and eases daily disability in people with tight levator scapulae muscles. About 44 adults aged 25 to 40 with neck…

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 phase 3 trial tests whether aficamten, an oral drug, can improve quality of life and exercise capacity in 500 adults with symptomatic non-obstructive hypertrophic cardiomyopathy, a heart condition that makes the heart muscle thick and stiff. Participants receive either afica…

This trial tests whether a small device that stimulates the vagus nerve through the ear can improve quality of life for people with long COVID who also meet the criteria for ME/CFS (chronic fatigue syndrome). Participants will use the device daily for six weeks, and researchers w…

This trial is testing whether watching educational videos before minimally invasive gynecologic surgery can lower patient anxiety. Participants are randomly assigned to either watch the videos or receive standard counseling alone. The study measures anxiety and preparedness throu…

This trial tests whether a rehabilitation program that exercises the entire leg — from foot to hip — can improve balance, reduce pain, and restore function better than standard tendon-focused exercises in people with chronic Achilles tendinopathy. Fifty adults aged 18 to 45 with …

This study explores whether weekly music and movement sessions are practical and enjoyable for children aged 6 to 18 with myotonic dystrophy type 1 (DM1). Over 10 weeks, participants attend 45-minute music classes, undergo physical and cognitive tests, and provide biological samp…

This study tests a new combination of nerve blocks for elderly patients (65 and older) having surgery for a broken shoulder. The goal is to see if the new approach provides better pain relief during surgery and lowers the risk of temporary diaphragm paralysis, which can cause bre…

This pilot study tests the drug ruxolitinib in people with advanced non-small cell lung cancer who are experiencing cancer cachexia—unintended weight loss and muscle wasting. The main goal is to check for side effects and see how the drug affects body signals related to appetite …

This study compares lifestyle counseling alone versus lifestyle counseling combined with clinical Pilates in 62 women with temporomandibular disorders (TMD). Participants will be randomly assigned to one of two groups for 8 weeks. Researchers will measure pain, jaw function, post…

This study compares three non-surgical treatments—exercise therapy, shockwave therapy, and ultrasound-guided irrigation—against a wait-and-see approach for people with painful calcium deposits in the shoulder. About 116 participants with confirmed calcific tendinopathy will be ra…

This study tests a modified surgical technique for people with massive, irreparable rotator cuff tears. The procedure moves the lower trapezius tendon to help restore shoulder function. Twenty-five patients will be followed to see if pain decreases and shoulder ability improves.

This study tests whether giving people with rotator cuff shoulder pain regular updates on their progress (like pain scores and strength) helps them recover better. 56 adults aged 18–35 will either get standard rehab or standard rehab plus these progress updates. The goal is to se…

This study tests whether applying an ice pack to the lower abdomen after vNOTES hysterectomy can lower pain and reduce the need for painkillers. 122 women having surgery for benign conditions will be randomly assigned to receive either standard care or standard care plus ice pack…

This study tests whether a 6-week, caregiver-assisted breathing program done at home with remote coaching is practical and helpful for children with Duchenne muscular dystrophy. Twelve children will do breathing exercises, airway clearance, and relaxation techniques at least 5 ti…

This study explores whether a supervised, circuit-based resistance training program can help glioblastoma patients maintain muscle mass and perform daily tasks more easily. Researchers will enroll 24 adults with glioblastoma who are currently on treatment. Participants will exerc…

This study tests whether a specific type of exercise called eccentric contraction (lengthening muscles under tension) can help people with shoulder pain syndrome. Sixty adults aged 18-70 with rotator cuff tendinitis will do either eccentric exercises plus standard therapy or stan…

This study tests whether adding yellow or green light therapy to standard care (ice and exercises) can reduce pain and improve knee function in 60 athletes with jumper's knee. Participants will receive either yellow light, green light, or standard care alone. The goal is to see i…

This study looks at whether low-dose rapamycin can reduce symptoms and improve quality of life in people with ME/CFS or Long-COVID. About 150 participants will take the drug as prescribed by their doctor and complete surveys and blood tests over time. The goal is to see if the dr…

This study looks at whether kinesio taping can quickly reduce pain, improve how far you can move your shoulder, and lessen fear of movement in people with rotator cuff syndrome. About 52 adults aged 18 to 65 with shoulder pain for at least 3 months will take part. Researchers wil…

This small study tests a procedure called radiofrequency ablation for people with chronic shoulder pain that hasn't improved with standard treatments like physical therapy or injections. The treatment uses heat to calm the nerves that send pain signals from the shoulder joint. Te…

This study looks at whether different levels of exercise can improve heart health in young adults who survived childhood cancer and were treated with a type of chemotherapy called anthracyclines, which can weaken the heart. Twenty survivors aged 18 to 39 will do either high-inten…

This study tests whether adding perturbation-based exercises (unexpected movements that challenge balance) to standard physiotherapy can improve pain, strength, and shoulder function in people with partial rotator cuff tears. Sixty adults aged 18–65 will be randomly assigned to e…

This study compares two ways to help people with shoulder pain activate important muscles: using real-time ultrasound images versus standard verbal or visual feedback. About 26 adults with shoulder pain will do exercises in a lab, and researchers will measure muscle activity and …

This study tests whether empagliflozin, a drug typically used for diabetes, can help people with hypertrophic cardiomyopathy (a thickened heart muscle) who do not have diabetes. About 250 adults will receive either the drug or a placebo daily for 12 months. The main goals are to …

This study is testing whether a drink containing CBD and CBN can reduce facial pain and headaches caused by muscle tension. 42 adults with pain lasting over 3 months will be randomly assigned to receive either the CBD/CBN drink or a placebo. Researchers will measure pain and musc…

This study tests whether wearing a custom mouth splint and getting a granisetron injection into the jaw muscle reduces pain from temporomandibular disorders (TMD) better than the splint alone or with lidocaine. About 48 adults with jaw muscle pain will be followed for 6 months. T…

This study tests whether a device that guides slow breathing can improve heart rate variability and quality of life in people who have had takotsubo syndrome (also known as broken heart syndrome). Ten patients with a history of takotsubo and ten healthy volunteers will use the RE…

This study aimed to see if a lidocaine injection or a lidocaine patch works better for muscle pain in the neck and back. It was designed for adults visiting the emergency department. However, the study was withdrawn before any participants were enrolled.

This study compares two recovery programs after rotator cuff repair surgery. One group follows standard care—wearing a sling for 6 weeks with limited movement. The other group starts moving sooner, guided by comfort rather than strict restrictions. Researchers will measure should…

This study tests whether a dual-task training program—combining physical exercises like walking with mental tasks like counting—can improve both motor and cognitive function in 16 boys aged 6–12 with Duchenne muscular dystrophy. Participants will be randomly assigned to either st…

This study tests a program called FACE-Rare, designed to support family caregivers of children with rare, life-limiting diseases. The program includes three sessions to help families prepare for future medical decisions and improve their quality of life. Researchers will compare …

This study tests whether the drug vericiguat, already approved for heart failure, can improve physical function and reduce fatigue in people aged 18-50 with post-COVID syndrome. About 104 participants will receive either vericiguat or a placebo for 10 weeks, then be followed for …

This study is testing the long-term safety and effectiveness of a drug called mexiletine PR for people with myotonic dystrophy types 1 and 2. The drug is taken once daily as a liquid to help reduce muscle stiffness (myotonia). The study includes 176 adults and teens who have alre…

This pilot study tests whether adding blood flow restriction therapy to standard physical therapy helps elderly patients (65 and older) recover muscle strength and size after hip fracture surgery. Twenty participants will have their thigh and leg measurements, muscle strength, pa…

This study tests whether injecting collagen into the neck can reduce pain and inflammation in people with long-term neck muscle pain. Forty adults with chronic neck pain will receive either collagen or a standard painkiller injection over five weekly sessions. Researchers will me…

This study tests whether giving vitamin C through an IV before and during a hysterectomy can reduce blood loss. Forty women with fibroids or adenomyosis will receive either vitamin C or a placebo. Researchers will measure blood loss during surgery and check if vitamin C helps mai…

This study looks at how many times a needle should be passed through a trigger point to best relieve myofascial pain. 300 adults with chronic muscle pain will be randomly assigned to receive 2, 10, or 20 needle passes during their injection. Researchers will measure pain relief, …

This study tests whether a traditional Chinese herbal paste called Yiqi Fuyuan Paste can reduce fatigue in adults with Chronic Fatigue Syndrome (CFS). Eighty participants will take either the paste or a placebo daily for two months. Researchers will measure fatigue levels and che…

This study tests a computer-guided needle technique combined with a platelet-rich plasma (PRP) injection for people with a painful jaw joint condition called temporomandibular disorder (TMD). The goal is to see if the computer-guided method improves jaw function and reduces pain …

This pilot study aimed to compare a single injection of connective tissue matrix (CTM) against a standard corticosteroid shot for treating rotator cuff tendinopathy. Participants would have been randomly assigned to one of the two injections and followed for six months with quest…

This study tests whether a combination of exercise, fruit, and vitamin supplements can reduce frailty in older adults aged 65 to 80. About 934 participants with low physical activity and low fruit intake will be split into two groups: one receives the combined intervention three …

This study looks at whether adding a steroid to a nerve block can better control pain after a procedure that treats uterine fibroids or adenomyosis. About 28 women aged 30 to 60 will receive either a standard numbing nerve block or one with added steroids. Researchers will measur…

This study tests a new, team-based rehabilitation program for 125 working-age adults with persistent physical symptoms and chronic fatigue. The program includes doctors, therapists, and counselors who work together to help patients manage their symptoms and improve daily function…

This phase 3 trial tests whether inhaled salbutamol, a drug used for asthma, can help newborns with transient tachypnoea (rapid breathing after birth). About 608 babies born between 32 and 42 weeks will receive either salbutamol or a placebo. The goal is to see if the drug reduce…

This study tests whether a drug called siltuximab can reduce symptoms like weakness, fatigue, nausea, and pain after a stem cell transplant in patients aged 60-75 with multiple myeloma or AL amyloidosis. The drug blocks a protein linked to inflammation, which may help patients re…

This study tests whether a gentle electrical brain stimulation device, used at home under remote supervision, can help reduce fatigue in people with myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS). Twenty participants will use the device for 20 minutes each weekday ov…

This study looks at people with late-onset Pompe disease to measure antibodies against a virus used in gene therapy and against the standard enzyme replacement therapy. It also checks for biomarkers in blood and urine. About 119 teenagers and adults will give samples over 2 years…

This study involves 324 adults with facioscapulohumeral muscular dystrophy (FSHD) across 14 international sites. Researchers are testing two new measurement tools—a functional test called FSHD-COM and a muscle-impedance device—to better track disease progression. The goal is to i…

This pilot study looks at biomarkers in the blood of people with fragile sarcolemmal muscular dystrophy, a condition that makes muscle membranes weak. Researchers will collect blood samples at rest and after exercise during four 5-day hospital stays. The goal is to better underst…

This study looks at how the position and angle of surgical anchors used in rotator cuff repair affect cyst formation and tendon healing. Researchers will use MRI scans to measure cyst volume and assess tendon recovery in 70 adults aged 18–70 undergoing primary arthroscopic repair…

This long-term study looks at children and adults with inherited nerve and muscle disorders that start early in life, like muscular dystrophy. Researchers will track symptoms over time and collect genetic samples from affected individuals, their family members, and healthy volunt…

This study monitors heart rate variability in 150 adults with type 2 diabetes who also have heart failure, chronic kidney disease, or both. Participants will wear a 24-hour heart monitor before and after starting a standard SGLT2 inhibitor medication. The goal is to understand ho…

This 24-month observational study will follow 80 people with Becker muscular dystrophy (BMD) to better understand how the disease progresses. Researchers will measure muscle strength, walking speed, breathing, and heart function using standard tests. The goal is to identify which…

This study follows 20 newborns and children diagnosed with late-onset Pompe disease through newborn screening. Researchers will track their health for up to 4.5 years to document early muscle and joint symptoms, biomarkers, and quality of life. The goal is to learn when to start …

This study gathers health information from children under 18 who have cardiomyopathy linked to mutations in the MYBPC3 gene. Researchers aim to map the disease's natural course, identify risk factors, and measure how it affects quality of life. By reviewing past and future medica…

Researchers are testing a muscle biopsy method that uses mild electrical stimulation to sample the junction where nerves meet muscle. The goal is to see if this technique helps distinguish between inclusion body myositis and motor neuron disease, which can look alike. The study i…

This study is a disease registry that collects information from routine medical care of about 350 adults diagnosed with transthyretin amyloid cardiomyopathy (ATTR-CM) in Taiwan, Hong Kong, and Malaysia. Researchers will look at patient characteristics, treatments received, and ho…

This study follows 50 adults who had surgery for a broken shinbone to see if changes in the kneecap tendon's stiffness relate to knee pain. Researchers use a special ultrasound before and up to 6 months after surgery. The goal is to better understand why some people develop front…

This study will use a special blood test called mass spectrometry to look at immune cells in 500 people with various autoimmune diseases and healthy volunteers. The goal is to find patterns that could help doctors diagnose these conditions sooner. No treatments are being tested; …

This study aims to better understand fatigue by comparing people who have felt tired for more than a month with those who are not fatigued. Researchers will use questionnaires, physical tests, and optional brain scans to identify different types of fatigue. The goal is to gather …

This study uses anonymous health insurance records from Germany to learn more about a rare heart condition called ATTR-CM, where abnormal protein builds up in the heart. Researchers will look at survival rates, hospital visits, and how common the disease is. No new treatments are…

This study is developing a digital twin—a virtual model of the heart and blood vessels—to better understand and manage hypertrophic cardiomyopathy (HCM), a condition where the heart muscle thickens. Researchers will gather detailed health data from 2500 people with HCM or the gen…

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 follows adults with pathogenic variants in the anoctamin 5 gene, which causes a form of limb-girdle muscular dystrophy. Over three years, researchers will use whole-body MRI to measure changes in muscle fat content, and questionnaires to track fatigue and quality of li…

This study aims to describe the pattern of muscle involvement in people with limb girdle muscular dystrophy caused by mutations in the anoctamin 5 gene. Researchers will collect and analyze MRI scans from 200 participants to see which muscles are affected and how much fat replace…

This study tests two different drug combinations in people with advanced colorectal, pancreatic, or leiomyosarcoma cancers that have not responded to standard treatments. Participants receive either olaparib plus durvalumab or cediranib plus durvalumab. The main goal is to unders…

This study is testing a new physical therapy protocol for people over 50 who have had arthroscopic rotator cuff surgery. Researchers will track shoulder function, pain levels, and sleep quality over the recovery period. The goal is to gather better evidence on what helps patients…

This study looks at whether sensors inside implanted heart devices can warn of worsening heart failure. Researchers will track 500 adults with heart failure or cardiomyopathy who have a Boston Scientific ICD or CRT-D equipped with HeartLogic, Sleep Incline, and Apnea Scan functio…

This study is looking at how magnetic resonance imaging (MRI) and spectroscopy can be used to detect and track changes in the muscles of people with facioscapulohumeral muscular dystrophy (FSHD). The goal is to identify reliable biomarkers—measurable indicators of disease activit…

This study follows people who previously received NTLA-2001, an experimental gene-editing therapy, for a type of amyloidosis that can affect nerves or the heart. The goal is to monitor long-term safety and see if the therapy's effects on disease markers persist over time. Partici…

This study examines genetic test results from over 600 people who had acute myocarditis (heart muscle inflammation) confirmed by MRI. Researchers want to find out how common certain gene variants are in this group and whether they are linked to worse outcomes like heart failure o…

This study looks at how myotonic dystrophy types 1 and 2 affect the brain. About 100 adults aged 30-65 will have MRI scans, thinking and movement tests, and blood draws. Some will also have a spinal tap. The goal is to find brain changes that could be used as markers in future tr…

This study looks at tiny particles called exosomes, taken from a patient's own platelet-rich plasma, to see if they might help repair jaw joints. Researchers will compare these exosomes with other blood products in lab tests on cells. The goal is to understand their content and p…

This study looks at whether detailed analysis of heart MRI images (called radiomics) can better predict life-threatening heart rhythms in people with hypertrophic cardiomyopathy (HCM), a condition where the heart muscle thickens. Researchers will also see if the drug mavacamten, …

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 tested whether a special PET imaging probe called [68Ga]CBP8 can measure scarring (fibrosis) in the hearts of people with cardiac amyloidosis, a rare condition where abnormal proteins build up in the heart. Researchers compared 18 participants with cardiac amyloidosis …

This study tests whether wearable sensors worn on the wrist and as a pendant can accurately track changes in arm and leg function in people with inclusion body myositis (IBM), a rare muscle disease that causes progressive weakness. Researchers aim to see if these sensors can prov…

This study looks at how inflammation and oxidation (chemical reactions in the body) might damage the heart and lead to heart failure. Researchers are tracking over 1,200 healthy adults aged 40+ (or 18+ with a family history of heart failure) to see if waste products in blood or u…

This study looks at people with ALS and similar diseases like frontotemporal dementia and hereditary spastic paraplegia. Researchers want to understand how genes affect the disease and find biological markers (biomarkers) to help develop future treatments. About 708 participants,…

This 24-month observational study follows 100 people aged 12–50 with Limb Girdle Muscular Dystrophy type R1 (LGMD R1). Researchers will test whether a motor function scale called NSAD and muscle fat measurements from MRI can reliably track disease progression. The goal is to vali…

This study tests new radio frequency coils for MRI scanners to see if they can produce better images in children, including those with Duchenne muscular dystrophy. Researchers will check for side effects like heating and discomfort, and measure image quality. The goal is to impro…

This study follows about 35 people with dilated cardiomyopathy caused by a BAG3 gene mutation, a condition that weakens the heart muscle. Over three years, researchers will monitor heart function, symptoms, and quality of life using tests like imaging and blood work. The goal is …

This study aims to create a tool that predicts which patients with inflammatory rheumatic diseases (like rheumatoid arthritis, vasculitis, or polymyalgia rheumatica) will develop serious side effects from long-term steroid use. Researchers will collect routine medical data—such a…

This Johns Hopkins study looks at whether a leaky blood-brain barrier and immune changes contribute to cognitive problems in people with ME/CFS (chronic fatigue syndrome), both those who got sick before the pandemic and those who developed it after COVID-19. Researchers will use …

This study looks at how rotator cuff injuries—common causes of shoulder pain—might affect posture, trunk rotation, and balance. Researchers will compare 40 people with rotator cuff problems to 40 healthy volunteers, measuring things like spine curvature and balance using smartpho…

This study looks at how middle-aged men and women lose muscle after a week of not using one leg, and how they recover with exercise. Researchers will measure muscle strength, size, and genetic activity to find sex-specific differences. The goal is to learn more so future treatmen…

This study is for heart failure patients who do not have a specific heart rhythm pattern called left bundle branch block (non-LBBB). These patients often get a device called CRT-D, which combines a pacemaker and a defibrillator, but not everyone responds well. Researchers will fo…

This study tests a new imaging method to see if white blood cells enter the brain in people with multiple sclerosis, fibromyalgia, or chronic fatigue syndrome. Participants receive their own white blood cells tagged with a radioactive tracer, then undergo a brain PET/MRI scan. Th…

This study follows 1000 Chinese patients with myotonic dystrophy type 1 (DM1) to understand how the disease affects multiple body systems and leads to disability. Researchers will collect health data and blood samples every 3 to 6 months for several years. The goal is to identify…

This study will collect and analyze genetic data from 1000 people with suspected inherited metabolic diseases, including conditions like epilepsy and mitochondrial disorders. Researchers at Karolinska University Hospital aim to improve diagnosis by using advanced genetic testing …

This study follows about 700 women for up to 12 years after they had a uterine-sparing fibroid treatment, such as myomectomy or uterine artery embolization. Researchers want to see how long these treatments last before another procedure is needed, and how women's symptoms and qua…

This study looks at whether a tool called the Chelsea Critical Care Physical Assessment tool (CPAx) can accurately track recovery in 120 adults who developed muscle weakness after being on a breathing machine in the ICU. The goal is to find the smallest meaningful improvement tha…

This study follows 150 adults with sporadic inclusion body myositis (sIBM) for two years to see how the disease progresses. Participants visit the clinic every six months for tests like muscle strength, breathing, and swallowing. No treatments are given—the goal is to learn more …

This study looks at whether diabetes or blood sugar problems can worsen thinking and memory issues in people with myotonic dystrophy type 1. Researchers will use MRI brain scans and thinking tests over 4 years to track changes. The goal is to understand why some patients have mor…

This study aims to make genetic testing for inherited heart muscle disease available directly in heart failure clinics, instead of requiring a separate referral to a genetics specialist that can take years. Researchers will train heart doctors to order a simple blood or saliva te…

This study looks at how the repaired rotator cuff tendon moves after surgery, which may affect recovery. 125 people getting rotator cuff repair will have small markers placed on the tendon during surgery. CT scans and MRIs over 5 years will track tendon position and healing, and …

This study is testing a virtual reality game designed specifically for people with Duchenne muscular dystrophy (DMD). Six participants aged 7 and older will play the game once using a VR headset and hand tracking. Researchers will measure how easy and enjoyable the game is, and w…

This study looks at whether rotator cuff disease runs in families. Researchers will collect DNA, medical history, and family history from 400 people to understand genetic risk factors. The goal is to build a resource for future studies, not to test a new treatment.

This study follows 331 adults with hypertrophic cardiomyopathy (HCM) to see how they are treated in everyday medical practice, especially with the drug mavacamten. Researchers will collect data on diagnosis, symptoms, treatments, and outcomes over time. The goal is to understand …

This study examines blood samples from 50 people with dermatomyositis, an autoimmune disease that raises the risk of cancer. Researchers are looking for new antibodies and immune molecules that might signal which patients are more likely to develop cancer. The goal is to improve …

This study looks at over 1,200 adults with type 2 diabetes and heart muscle problems to see if diabetic cardiomyopathy is a unique condition. Researchers will use detailed medical tests, imaging, and biological data to find patterns. The goal is to better understand and diagnose …

This study watches 40 adults who need a leadless pacemaker to see if where the device sits inside the heart changes heart rhythm or pumping strength. Researchers will use CT scans and ECGs to check heart function and valve leakage. The goal is to find the best spot for the pacema…

This study is testing a new way to care for people with rare diseases. It will use advanced genetic testing and a team of specialists to help diagnose patients faster and coordinate their care better. The study involves 136 participants with certain rare diseases and aims to redu…

This study is for people with autosomal dominant hypocalcemia types 1 or 2, rare genetic conditions that cause low blood calcium. Researchers will collect past and future health data from 95 participants to learn how the disease changes over time. The goal is to better understand…

This nationwide registry follows 578 women with uterine fibroids who choose either radiofrequency ablation (Acessa) or myomectomy. Researchers track symptom changes, quality of life, and need for repeat treatment over three years. The goal is to understand which procedure provide…

This pilot study aims to learn more about a procedure called FETO for unborn babies with a severe form of congenital diaphragmatic hernia (CDH). The researchers want to see if placing a balloon in the baby's windpipe before birth can help the lungs grow and improve survival. The …

This study looks at 600 adults with non-compaction cardiomyopathy, a rare heart muscle condition that can lead to heart failure, stroke, or dangerous heart rhythms. Researchers will use heart scans, genetic tests, and health records to find better ways to predict who is at highes…

This study looks at whether mepolizumab, a drug already used to reduce asthma attacks, can also improve physical activity and exercise tolerance in people with severe asthma. Researchers will track daily steps and activity levels in 62 adults over six months. The goal is to see i…

This study looks at 57 people who received a COVID-19 vaccine to understand why some develop heart injury or myocarditis. Researchers use advanced heart scans (PET/MRI) and blood tests to find early warning signs. The goal is to better identify those at risk and improve follow-up…

This study was designed to compare two standard surgical techniques for bicep tenodesis—using one anchor or two mini anchors—to see which keeps the tendon in place better. It planned to track tendon movement with X-rays right after surgery, at 2 weeks, and at 6 months. However, t…

This study uses advanced heart MRI scans to look for the earliest signs of dilated cardiomyopathy (a weakened heart muscle) in close relatives of people already diagnosed. Researchers will follow 650 family members over time to see how genetic factors relate to heart changes. The…

This study looks at whether a smartwatch ECG can predict heart muscle disease (cardiomyopathy) in adult survivors of childhood cancer as well as a standard clinic ECG does. About 1,300 survivors will have both a standard 12-lead ECG and a 30-second Apple Smartwatch ECG. The goal …

This study explores whether the characteristics of bone marrow in the upper arm bone can predict how well a torn rotator cuff heals after surgery. Researchers will enroll 50 adults aged 18-75 with full-thickness tears and use MRI and CT scans to measure tendon healing. The goal i…

This study tracks 40 people with nasal polyps and severe asthma who are taking mepolizumab, a drug that reduces inflammation. Researchers collect nose, blood, and sputum samples before treatment and at 3, 6, and 12 months to see how the drug affects symptoms, nose bacteria, and g…

This study looks at whether a new type of heart medication, called a cardiac myosin inhibitor, can improve blood flow in the small blood vessels of the heart in people with hypertrophic cardiomyopathy (HCM). Researchers will use special ultrasound imaging to measure blood flow be…

This study looks at 200 people with dermatomyositis, a rare muscle disease that often affects the lungs. Researchers are using special PET/CT scans and machine learning to create a model that can better predict if a patient has interstitial lung disease. The goal is to improve di…

This study follows 45 people with nemaline myopathy in the UK to learn how the disease naturally progresses. Researchers collect medical exams, quality-of-life surveys, and physical assessments over time. The goal is to identify the best ways to measure the disease in future clin…

This study looks at ways to get patients to complete follow-up surveys after shoulder surgery. About 400 adults having shoulder arthroscopy will either receive education about the importance of surveys or get Amazon gift cards for completing them. The goal is to see which method …

This study tests whether using an ultrasound measure of heart muscle coordination (mechanical dyssynchrony) can better select heart failure patients for cardiac resynchronization therapy (CRT) than current guidelines. About 500 patients will receive a CRT device, but pacing will …

This study follows 50 athletes who had acute myocarditis (heart muscle inflammation) to see how often they develop dangerous heart rhythms. Participants wear a portable ECG monitor and do a treadmill stress test at 3 months and 1 year after their illness. The goal is to learn mor…

This pilot study looks at whether light, self-paced swimming is a tolerable activity for people with ME/CFS and related conditions like Long COVID and fibromyalgia. Participants will swim for 15-30 minutes at their own pace and track symptoms and heart rate variability using an a…

This study was designed to follow patients with full-thickness rotator cuff tears over 10 years to see how their shoulder function and quality of life changed. It aimed to identify factors linked to improvement. However, the study was withdrawn before enrolling any participants, …

This study was designed to track pregnancies in women with rare autoimmune and systemic diseases to understand complications and improve care. It planned to enroll women with conditions like lupus, rheumatoid arthritis, and scleroderma. However, the study was withdrawn before any…

This study follows 2000 people with dilated cardiomyopathy (a weakened, enlarged heart) over several years to learn how genetics and heart scarring affect the disease. Participants give blood for genetic testing and have heart MRI scans. The goal is to improve diagnosis and pave …

This study measures how often people with myotonic dystrophy type 1 fall and what factors affect their balance. Researchers will also assess fear of falling and leg muscle strength. The goal is to better understand fall risk in this condition.

This study looks at how sepsis, a severe infection, can cause heart problems in intensive care patients. Researchers will collect data, blood samples, and heart ultrasound images from 330 adults with septic shock. The goal is to find patterns that help doctors better diagnose and…

This study watches a single gynecology resident as they learn to perform a hysterectomy through the vagina using a special camera, leaving no belly scars. The goal is to see how quickly the resident improves, measuring surgery time and any complications. Nineteen women with benig…

This study is investigating whether bruxism (teeth grinding or clenching) is associated with pelvic pain, erectile dysfunction, and anxiety in young men aged 18-30. Researchers will use questionnaires and physical exams to assess 100 participants. The goal is to better understand…

This study looks at whether a substance called YKL-40 in the blood can help predict how well heart failure patients will respond to cardiac resynchronization therapy (CRT), a special pacemaker. About 51 adults with severe heart failure will have blood samples taken during their r…

This study uses a special total-body PET/CT scanner to take detailed images of muscles and tissues in people with chronic low back pain caused by myofascial pain. The goal is to find new ways to measure pain-related changes in the body, such as blood flow and tissue health. Fifty…

This study explores whether bruxism (teeth grinding or clenching) is related to pelvic floor dysfunction in young women aged 18-30. Researchers will use questionnaires and physical exams to assess jaw function, pelvic floor symptoms, and anxiety in 100 participants. The goal is t…

This study looks back at the medical records of about 4 million people who had physical or occupational therapy for muscle and joint problems. Researchers want to see if different ways of giving therapy lead to different results. No new treatments are tested—the goal is to learn …

This study looks at whether a special PET scan can help doctors better predict sudden cardiac arrest in people with heart failure and blocked arteries. About 300 participants who already have a defibrillator will be followed to see if heart nerve damage, heart size, or blood mark…

This study explores whether the levels of certain receptors (CB1, CB2, TRPV-1) in the blood are linked to chronic orofacial pain from jaw muscles. Researchers will compare 102 adults with and without this pain, using blood tests and clinical exams. The goal is to understand how t…

This study looks at how the muscles in the arms and hands change in people with Duchenne muscular dystrophy (DMD). Researchers will measure muscle tone, stiffness, and elasticity using a device called MyotonPRO. They will also test hand function and muscle strength. The goal is t…

This study looks at how thinking skills (called executive functions) relate to everyday activities like self-care and play in children with Duchenne muscular dystrophy (DMD). Researchers will compare 38 children with DMD to healthy children using surveys and functional tests. The…

This study aims to uncover the genetic roots of aortic aneurysms and valve disease by analyzing tissue and blood samples from 3,000 participants. Researchers will look for new disease-causing genes and factors that affect disease severity. The goal is to build a biorepository to …

This study follows about 400 people with Duchenne muscular dystrophy who have already received the gene therapy SRP-9001 in an earlier study. The goal is to monitor their safety and muscle function over time. No new treatment is given in this study.

This study looks at heart rhythm problems in people with hypertrophic cardiomyopathy, a condition where the heart muscle is thicker than normal. Researchers want to see how often new rhythm issues like atrial fibrillation happen, and whether sleep apnea plays a role. About 260 ad…

This study tests a digital tool called AsthmaOptimiser that helps doctors assess asthma control and find ways to improve treatment during a single visit. About 746 adults with asthma who have had recent flare-ups will take part. The goal is to see if the tool can identify better …

This study planned to test holistic medicine—like counseling and group talks—for unmarried women with uterine fibroids living on Tribal Lands or in Alaska. It aimed to see if these non-clinical approaches could help, but the trial was withdrawn before any participants joined. No …

This study looks at why over half of ICU patients develop severe muscle weakness that can last for months or years. Researchers will use muscle biopsies, ultrasound, and strength tests on 50 critically ill adults to find better ways to diagnose and track this condition. The goal …

This observational study will enroll 10,000 adults with hypertrophic cardiomyopathy (HCM), a condition where the heart muscle thickens. Researchers will combine genetic tests, advanced heart imaging, and blood markers to create better tools for early diagnosis and to predict risk…

This study is testing whether an artificial intelligence (AI) model can automatically write reports for cardiac MRI scans as accurately as experienced radiologists. Researchers will compare AI-generated reports with those written by experts using 20,000 patient scans. The goal is…

This study looks at whether surgically removing uterine fibroids (myomectomy) changes blood flow in the main uterine artery. Researchers will use Doppler ultrasound to measure blood flow before and about 40 days after surgery in 70 premenopausal women. The goal is to provide new …

This study looks at whether a special training program can help family doctors use focused heart ultrasound (FoCUS) to accurately detect common heart conditions like heart failure and valve disease. About 500 patients will get both a family doctor's FoCUS and a cardiologist's ful…

This study follows 25 people with limb-girdle muscular dystrophy type 2A (LGMD2A), a rare genetic disease that causes progressive muscle weakness. Researchers will measure how muscle strength changes over time and how it affects quality of life. The goal is to better understand t…

This study aims to find biological markers in blood, saliva, and sputum that can predict how well patients with EGPA (a rare inflammatory disease) and severe asthma respond to the drug mepolizumab. Researchers will analyze proteins in these samples before and after treatment to u…

This study collects blood or skin samples from 100 adults with inherited heart rhythm disorders (like Long QT Syndrome or Brugada Syndrome) and healthy volunteers. Researchers will turn these samples into stem cells and then into heart cells to study how these diseases work and t…

This study will use advanced cardiac MRI scans to look at different patterns of heart inflammation in 5,000 people with myocarditis or inflammatory cardiomyopathy. The goal is to see if certain MRI findings can better predict who will have serious heart problems later, like heart…

This study is a nationwide registry in Taiwan that will follow 300 people who have or will receive a leadless pacemaker. The goal is to track how well the device works and any complications that may occur during or after the procedure. The study does not test a new treatment but …

This study creates a database of 476 patients in the Bronx who were tested for transthyretin amyloid cardiomyopathy (ATTR-CM), a serious and often missed heart condition. Researchers collected health records, imaging results, and outcomes like hospitalizations and deaths. The goa…

This observational study looks at 165 people with two different types of heart muscle thickening: syndromic/metabolic cardiomyopathy (SMCs) and hypertrophic cardiomyopathy (HCM). Researchers want to see if SMCs patients have worse heart outcomes like death or heart failure, and w…

This study uses advanced MRI techniques to measure changes in heart muscle tissue in 8,000 adults with heart failure, heart muscle disease, inflammation, coronary artery disease, or chronic kidney disease. The goal is to see if these measurements can help doctors better diagnose …

This study looks at people with rare adrenal tumors (pheochromocytomas and paragangliomas) that do not produce excess hormones. Researchers want to see if these 'silent' tumors still cause heart problems, blood pressure changes, or metabolic issues like diabetes and high choleste…

This study aims to discover new genetic mutations that cause mitochondrial disorders by analyzing tissue samples from up to 6,900 participants. It includes people with suspected or known mitochondrial diseases, such as MELAS or Leigh's Disease, who lack a genetic diagnosis. The r…

This study looks at 100 people who spent at least a week in the ICU and had kidney injury. Researchers will measure kidney function, muscle loss, and bone health to understand long-term effects. The goal is to improve follow-up care for ICU survivors.

This study aimed to find out how many people with trigeminal neuralgia (a severe facial nerve pain condition) also have temporomandibular disorder (TMD), which causes jaw pain. Researchers planned to follow patients from three clinics and test a questionnaire to better diagnose b…

This study looks at how often muscle loss (sarcopenia) happens in adults who need a breathing machine in the ICU for more than 48 hours. Researchers will measure muscle health using simple tests and ultrasound. The goal is to understand how common this problem is and how it chang…

This study looks at 50 people with TTR amyloid cardiomyopathy, a condition where abnormal proteins build up in the heart. Researchers want to see if a special SPECT scan can measure blood flow in the heart's tiny vessels, which may be impaired even without blocked arteries. Parti…

This study is creating a large database of 1400 adults and children with hypertrophic cardiomyopathy (HCM) in Russia. Researchers will track their health over time to learn how the disease progresses and affects daily life. The goal is to improve diagnosis and care for HCM patien…

This study is a large international registry that collects information from patients who have a heart ablation procedure for ventricular tachycardia or premature ventricular contractions. The goal is to track how well the procedure works and any complications, helping doctors imp…

This study is for people with Long COVID, Lyme disease, or ME/CFS (chronic fatigue syndrome). Researchers want to see if a special blood test can find problems with T-cells, which are part of the immune system. Participants will give blood samples and fill out symptom questionnai…

This study is working to create better tools for measuring muscle function in people with facioscapulohumeral muscular dystrophy (FSHD). Researchers will follow 100 adults with FSHD for up to 24 months, testing new ways to track disease progression. The goal is to make future cli…

This pilot study is testing a radioactive antibody called 124I-Humanized 3F8 to see how it travels through the body and attaches to tumors in people with melanoma, neuroblastoma, or sarcoma. Seven participants will receive the antibody and then have PET/CT scans at several time p…

This study was designed to track heart muscle changes in people with Duchenne muscular dystrophy using two cardiac MRIs taken two years apart, along with blood tests for heart failure markers. It planned to enroll participants aged 6 and older with a confirmed genetic diagnosis. …

This study tests a new 3D imaging method to measure how much air the lungs can hold in people with suspected diaphragm weakness. About 100 adults will have two quick 3D scans while holding their breath. The goal is to see if this technique gives reliable results compared to stand…

This study compares two types of radiation therapy for left-sided breast cancer: proton beam therapy and conventional photon therapy. The goal is to see which one causes fewer changes to the heart, measured by MRI scans. About 60 adults with non-metastatic breast cancer will part…

This study follows 205 people with four types of limb-girdle muscular dystrophy (LGMD) to understand how the disease changes over time. Participants will have their muscle strength, movement, and breathing tested regularly for up to 5 years. No treatment is given; the goal is to …

This study looks at immune cells from people with autoimmune diseases (like lupus), people with infections, and healthy volunteers. Researchers want to understand how these cells differ and what makes them attack the body in autoimmune conditions. The study involves blood and bon…

This study looks at a possible side effect of a heart treatment called PFA for atrial fibrillation: injury to the phrenic nerve, which can paralyze part of the diaphragm and affect breathing. Patients get a special X-ray before and after the procedure to check diaphragm movement.…

This study aims to find better ways to measure disease progression in people with advanced facioscapulohumeral muscular dystrophy (FSHD) who use wheelchairs. Researchers will test various muscle strength and function tests over two years in 30 adults. The goal is to develop relia…

This study was designed to track hair loss in women with uterine fibroids who are taking the medication Oriahnn. Researchers planned to follow about 1,600 women for up to 24 months, using questionnaires and scalp photos to see how common hair loss is, when it starts, and if it go…

This observational study will follow 300 people with Duchenne muscular dystrophy who are already taking exon-skipping therapies (eteplirsen, golodirsen, or casimersen) as part of their routine care. Researchers will collect data on movement, lung function, and heart function over…

This observational study will enroll 40 adults with facioscapulohumeral muscular dystrophy (FSHD) to understand how muscle degeneration affects movement. Researchers will use MRI to assess muscle health and 3D motion capture to analyze walking and posture. The goal is to create a…