Muscle tissue disorder

This phase 3 trial tested whether adding bevacizumab (Avastin) to standard chemotherapy (docetaxel and gemcitabine) helps people with advanced or recurrent uterine leiomyosarcoma. The study enrolled 107 participants but was terminated early. Researchers measured how long patients…

This phase 3 trial tested whether adding ADI-PEG 20 to standard chemotherapy (gemcitabine and docetaxel) could help people with advanced leiomyosarcoma, a rare soft tissue cancer. The study planned to enroll 218 participants whose cancer had progressed after prior treatment. Unfo…

This early study tested a new cell therapy called LUCAR-G79 in just 3 adults with severe autoimmune diseases that did not respond to standard treatments. The therapy uses specially engineered immune cells to target and potentially calm the overactive immune system. The main goals…

This study tests whether an additional dose of a COVID-19 vaccine can improve immune protection in adults and children with autoimmune diseases like rheumatoid arthritis, lupus, multiple sclerosis, and others who are taking immunosuppressive medications. Participants who had a we…

This study looked at a shoulder implant used to treat pain and joint problems from conditions like arthritis, fractures, and rotator cuff tears. It involved 67 adults who received the implant and tracked how long it lasted and any side effects over 10 years. The goal was to confi…

This early-phase trial tested a new oral drug called BBI-355, alone or with other targeted therapies, in people with advanced solid tumors that had extra DNA (oncogene amplifications). The study aimed to find the safest dose and check for side effects. It was terminated early, so…

This study tested an experimental drug called MYK-224 in 18 adults with a type of heart disease that makes the heart muscle too thick and blocks blood flow. The goal was to see if the drug is safe and helps reduce symptoms. The trial was stopped early, so results are limited.

This study tested an experimental drug called CRD-4730 in people with a rare inherited heart rhythm disorder (CPVT) that can cause dangerous fast heartbeats. Seven adults received two different doses of the drug and a placebo to check safety and how the body processes it. The stu…

This early-stage study tested a new drug called SAR445514, which helps the body's natural killer (NK) cells attack cancer cells in people with multiple myeloma or light-chain amyloidosis that had returned or stopped responding to other treatments. The study was terminated early, …

This study tested whether taking the steroid oxandrolone for 12 weeks after rotator cuff repair could improve tendon healing and muscle mass. Only 4 people participated before the trial was stopped early. Because it was so small, we cannot draw any reliable conclusions about whet…

This phase 2 trial tested two doses of an oral drug called fidrisertib (IPN60130) in children and adults with fibrodysplasia ossificans progressiva (FOP), a rare condition where soft tissues turn into bone. The goal was to see if the drug could reduce new abnormal bone growth com…

This early-stage trial tested an oral drug called TNO155, alone or with another drug (nazartinib), in 227 adults with advanced solid tumors like lung cancer, melanoma, and head/neck cancer. The main goal was to check safety and find the right dose. The study was terminated early,…

This study tested a drug called XmAb23104 in people with advanced sarcoma, a rare cancer. The goal was to see if the drug could shrink tumors and if it was safe. Only 6 people took part before the study was ended early.

This study tested the safety of golodirsen (Vyondys 53) in boys and men with Duchenne muscular dystrophy who can no longer walk. Only 2 people took part before the trial was stopped early. Participants received weekly IV infusions for up to 96 weeks, with extra follow-up. The goa…

This study tested a drug called vatiquinone in 68 people with mitochondrial disease and epilepsy that doesn't respond to standard treatments. Participants were randomly assigned to receive either vatiquinone or a placebo for 24 weeks to see if the drug could reduce the number of …

This study tested a drug called OMS721 in 31 people with certain kidney diseases, including IgA nephropathy and lupus nephritis. The main goal was to check if the drug is safe and if it reduces protein in the urine, a sign of kidney damage. The study was stopped early, so results…

This study looked at two types of implantable cardioverter defibrillator (ICD) leads in children and patients with congenital heart disease. The goal was to see which lead type lasts longer without breaking and is easier to remove if needed. The trial was terminated early, so res…

This study tested a drug called inotersen in 31 people with a rare condition where a faulty protein builds up in the heart, causing damage. The drug is designed to lower levels of that protein. The trial was stopped early, so it's unclear if it works, but researchers hoped to see…

This early-phase trial tested a single infusion of IMPT-514, a personalized CAR T cell therapy, in 3 adults with severe lupus, vasculitis, or inflammatory myopathy that did not respond to standard treatments. The goal was to check safety and see if the therapy could reset the imm…

This early-stage trial tested the safety of a new drug called AZD0233 in 84 healthy adults aged 18 to 50. The drug is being developed for dilated cardiomyopathy, a condition where the heart becomes enlarged and weak. Participants received either the drug or a placebo, and researc…

This study tested a gene therapy called SRP-9003 for people with limb-girdle muscular dystrophy type 2E (LGMD2E), a rare genetic disease that causes muscle weakness. The treatment aimed to deliver a working gene to muscle cells to help them produce a missing protein. Only 6 peopl…

This early study tested a new procedure called SESAME that uses a wire threaded through blood vessels to slice away excess heart muscle in people with hypertrophic cardiomyopathy. Only one adult participated before the study was terminated. The goal was to see if the technique co…

This study looked at the long-term safety of dexpramipexole in adults with severe eosinophilic asthma. Participants had already completed earlier phase 3 trials. The goal was to track side effects and asthma control over time. However, the study was terminated early, which may li…

This study tested a daily pill called dexpramipexole in 654 people with severe eosinophilic asthma, a type of asthma linked to high levels of certain white blood cells. The goal was to see if the drug could reduce severe asthma attacks over a year. However, the trial was terminat…

This phase 3 study tested an experimental drug called birtamimab in 208 people with advanced AL amyloidosis, a rare disease where abnormal proteins damage organs like the heart. Participants received either birtamimab or a placebo, plus standard chemotherapy. The goal was to see …

This Phase 3 trial tested dexpramipexole, an oral drug, in over 1,000 people with severe eosinophilic asthma that was not well controlled by standard treatments. The goal was to see if it could reduce severe asthma attacks over a year. However, the study was terminated early, so …

This study tested a medicine called Hizentra (IgPro20) in 134 adults with dermatomyositis, a rare disease causing muscle weakness and skin rashes. The goal was to see if weekly shots under the skin could improve symptoms better than a placebo. The study was stopped early, but res…

This study tested an experimental drug called losmapimod in 14 adults with FSHD1, a rare genetic condition that causes progressive muscle weakness. The main goal was to check safety and tolerability, and to see if the drug affects certain biological markers. The trial was termina…

This phase 2 study tested a drug called PGN-EDO51 in 7 people with Duchenne muscular dystrophy whose genetic mutation can be corrected by skipping exon 51. The drug was given by IV infusion to see if it is safe and tolerable. The trial was terminated, so results are limited.

This study tested a gene therapy (delandistrogene moxeparvovec) combined with a drug called imlifidase to see if it could safely deliver the therapy to boys with Duchenne muscular dystrophy who had antibodies that might block the treatment. Only 5 participants were planned, but t…

This study tested a drug called losmapimod for people with a rare muscle-weakening disease called FSHD. The goal was to see if the drug could slow muscle loss and improve arm function over 48 weeks. About 260 adults with FSHD were randomly assigned to receive either losmapimod or…

This study tested whether cutting a specific tendon (the conjoint tendon) during reverse shoulder replacement surgery helps patients move their hand behind their back better. The trial planned to enroll 26 adults having their first reverse shoulder replacement, but it was termina…

This Phase 2 trial tested a single dose of gene therapy (fordadistrogene movaparvovec) in 10 boys with early-stage Duchenne muscular dystrophy. The goal was to check safety and whether the therapy could help muscles produce a mini-dystrophin protein. The study was terminated earl…

This study follows 7 people with Duchenne muscular dystrophy who previously received an experimental gene therapy called fordadistrogene movaparvovec. Researchers will monitor them for 10 years to check for side effects and see if the treatment continues to help with movement. Th…

This phase 2 trial tested the drug losmapimod in 76 adults with FSHD, a genetic condition that causes progressive muscle weakness. Participants took either losmapimod or a placebo for 48 weeks to see if the drug was safe and could help control the disease. The study was terminate…

This early-stage trial tested a single infusion of gene therapy (PF-06939926) in 23 people with Duchenne muscular dystrophy, both those who could still walk and those who could not. The main goal was to check safety and tolerability, while also measuring dystrophin protein levels…

This study tested an oral drug called duvoglustat in adults with Pompe disease, a rare genetic disorder that causes muscle weakness. Only 3 people took part, and the study was stopped early. Researchers looked at safety and whether the drug could improve walking distance and othe…

This early study tested whether a blood-cleaning procedure called plasmapheresis could allow boys with Duchenne muscular dystrophy who have antibodies against the gene therapy carrier to still receive the treatment. Only 3 boys were enrolled before the study was stopped early. Th…

This was a very early (Phase 1) study testing a gene therapy called SRP-6004 for people with limb girdle muscular dystrophy type 2B/R2, a rare muscle-weakening disease. The goal was to see if a single IV infusion of the therapy is safe and can help the body produce a missing prot…

This early-stage trial tested a gene therapy called SRP-9004 in just 4 people with limb girdle muscular dystrophy type 2D/R3, a rare muscle-weakening disease. The main goal was to check safety, not effectiveness. The study was terminated early, so results are limited.

This study tested the drug nintedanib (Ofev) in 11 people with myositis-related interstitial lung disease, a condition where lung scarring occurs alongside muscle inflammation. The goal was to see if the drug is safe and tolerable when added to standard immune-suppressing treatme…

This study tested a drug called ixazomib plus dexamethasone against other standard treatments for people with relapsed AL amyloidosis, a rare disease where abnormal proteins build up in organs. The trial aimed to see if the combination improved blood markers and slowed heart or k…

This Phase II study tested a radioactive tracer called 68GaNOTA-Anti-MMR-VHH2 to see if it can light up certain immune cells (macrophages) on PET scans. The trial included 29 people with head and neck cancer, lymphoma, atherosclerosis, or sarcoidosis. The goal was to see if the t…

This pilot study tested whether a special PET scan (FDG-PET/CT) can detect hidden heart inflammation in cancer patients receiving immune checkpoint inhibitors (ICIs). The goal was to catch this inflammation early, before it becomes severe. Only 2 participants were enrolled, and t…

This early-phase study tested a new radioactive imaging drug called 11C-MC1 to see if it can help detect inflammation in people with rheumatoid arthritis or idiopathic inflammatory myopathy, as well as in healthy volunteers. Participants received PET/CT scans after an injection o…

This study tests whether the drug pregabalin, alone or with a rehabilitation program, can reduce chronic fatigue in people with post-COVID syndrome. Participants are adults aged 18 to 65 who had COVID-19 at least six months ago and still feel very tired. The trial compares four g…

This study tested a medicated patch containing ketoprofen (a pain reliever) for people with tendonitis or bursitis pain in the shoulder, elbow, or knee. About 381 adults with these conditions were randomly assigned to receive either the active patch or a placebo patch. The goal w…

This study tested whether a personalized exercise program could reduce fatigue in people with chronic fatigue syndrome. About 101 participants were planned, but the trial was terminated early. The approach involved tailoring aerobic and resistance exercises based on weekly fatigu…

This study looked at whether a brief, noninvasive brain stimulation technique called tDCS could immediately reduce pain and improve function in people with fibromyalgia or chronic fatigue syndrome. About 20 participants received one session, and researchers measured changes in pa…

This study looked at whether using a lower fluid pressure (60 mmHg) during hysteroscopy, a procedure to remove fibroids, works as well as the standard pressure (80 mmHg). The goal was to see if it improves surgery time, fluid use, and surgeon's view. The study was stopped early a…

This study tested whether the drug secukinumab could reduce pain and improve function in adults with moderate to severe rotator cuff tendinopathy, a common shoulder condition. Participants received either the drug or a placebo for 24 weeks. The trial was terminated early, and res…

This study looked at whether adding blood flow restriction training to standard physical therapy helps people regain knee strength and muscle size after meniscus repair surgery. About 15 participants were split into two groups: one did standard rehab, the other added the special …

This study tested whether a personalized pacing algorithm (myPACE+) could improve quality of life and exercise capacity in people with non-obstructive hypertrophic cardiomyopathy who already have an implantable defibrillator. Only one person was enrolled before the trial was term…

This study looked at whether cutting a small nerve in the wrist during surgery to remove a ganglion cyst helps reduce pain and improve hand function more than just removing the cyst alone. Only 3 adults with painful wrist cysts took part before the study was stopped early. The re…

This study tested the drug secukinumab against a placebo in 60 adults with moderate to severe rotator cuff tendinopathy, a painful shoulder condition. The goal was to see if secukinumab could improve symptoms and shoulder function over 24 weeks. However, the trial was terminated …

This study tested whether using a blood flow restriction (BFR) cuff during physical therapy could help people with shoulder injuries regain strength and return to activity faster. Only 3 people took part before the study was stopped early. The approach uses a special tourniquet t…

This study collects small skin samples from children aged 2 to 17 with Costello syndrome or a related condition. Researchers will analyze the cells to understand how a mutation in the HRAS gene affects energy use and mitochondria. The goal is to learn more about the disease's und…

This study followed 167 adults with mental health issues like anxiety, depression, or sleep problems who visited their primary care doctor. Researchers used health conversations and a blood test to track lifestyle changes and heart disease risks over up to 20 years. The goal was …

This study tested whether two gut hormones, GIP and GLP-2, could reduce bone breakdown in children with spinal muscular atrophy, cerebral palsy, or Duchenne muscular dystrophy who use wheelchairs. Participants received a liquid meal and then either a hormone injection or a placeb…

This study looked at whether healthcare workers without ultrasound training could use AI software to perform heart scans on people with hypertrophic obstructive cardiomyopathy (HOCM) who are eligible for the drug mavacamten. Participants had two scans on the same day: one by a no…

This study looked at whether muscle stem cells (mesoangioblasts) from people with muscle loss due to cancer or aging are healthy enough to be used for future cell therapy. Researchers took small muscle samples and blood from 7 participants and measured how well the cells grew and…

This study looked at two different ways surgeons place the top part of a reverse shoulder replacement: sitting on top of the bone (onlay) or set into the bone (inlay). The goal was to see if one method leads to better arm movement after surgery. The study enrolled 19 people with …

This study compared three different rehabilitation programs after arthroscopic rotator cuff repair. Participants were divided into groups with varying periods of immobilization and passive motion before starting active rehab. The goal was to see which program leads to the best sh…

This study looked at whether proteins that control our body's daily rhythms (clock proteins) can predict how severe asthma will get. Researchers monitored these proteins in the blood and sputum of 50 adults with severe eosinophilic asthma before and during treatment with mepolizu…

This study collected bone, cartilage, skin, and fat tissue from 50 patients undergoing surgery at Children's National Medical Center. The goal was to grow cells from these tissues in the lab and analyze them to better understand bone and connective tissue diseases. The study was …

This study aims to find out how common transthyretin amyloidosis cardiomyopathy (ATTR-CM) is in Russian patients with a certain type of heart failure. Researchers will review medical records and then invite some patients for extra heart tests to confirm or rule out ATTR-CM. The g…

This study looked at one person with facioscapulohumeral muscular dystrophy (FSHD) to better understand the disease. Researchers examined muscle tissue and checked for specific biomarkers. The goal was to learn more about how FSHD affects the body, not to test a treatment.

This registry aimed to track pregnancy outcomes in women who were exposed to elagolix (brand names Orilissa and Oriahnn) for endometriosis or uterine fibroids. The study planned to enroll 584 pregnant women, half with elagolix exposure and half without, and follow them through pr…