Hypertrophic cardiomyopathy

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 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 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 study tests a new drug called EDG-7500 in 100 adults with hypertrophic cardiomyopathy, a condition where the heart muscle is abnormally thick. The goal is to check the drug's safety and how it affects heart function. Participants receive single or multiple doses, and researc…

This early-stage trial tests a combination of three drugs—venetoclax, ixazomib, and dexamethasone—in 24 patients with light chain amyloidosis that has returned or not responded to prior treatment. The study focuses on finding the safest dose and checking side effects. All partici…

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

This early-phase trial tests 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 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 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 study tests an experimental drug called CAEL-101 in 125 people with a rare disease called AL amyloidosis, where abnormal proteins build up in organs like the heart and kidneys. The drug is designed to remove those protein deposits. The goal is to see if it helps people live …

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 phase 3 trial tests whether CAEL-101, an antibody that removes abnormal protein deposits from organs, can help people with AL amyloidosis live longer and avoid heart-related hospital stays. About 281 participants who have not yet received treatment for their plasma cell diso…

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 new drug called DAY101 (tovorafenib) against standard chemotherapy for children and young adults with a type of brain tumor called low-grade glioma that has a specific gene change (RAF alteration). The goal is to see if the new drug works better at shrinking tu…

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 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 study tests the drug mavacamten in 50 adults in India who have a condition where the heart muscle is too thick and blocks blood flow, causing symptoms like shortness of breath and chest pain. The goal is to see if the drug is safe and works well. Participants will take the d…

This early-phase study tests a new drug called ERAS-601, alone or with other cancer treatments, in adults with advanced solid tumors that have not responded to standard therapies. The main goals are to find safe doses and check for side effects. About 90 participants will take pa…

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 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 whether adding daratumumab back to pomalidomide and dexamethasone can improve complete remission rates in 15 adults with relapsed or refractory AL amyloidosis who have already received daratumumab. Participants receive the three-drug combination to control the di…

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 phase 3 trial tests an experimental drug called AOC 1001 in 159 adults with myotonic dystrophy type 1, a genetic condition that causes muscle weakness and stiffness. Participants receive either the drug or a placebo by intravenous infusion. The main goal is to see if the dru…

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 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 a new surgery done on a beating heart to help people with a type of thick heart muscle disease that causes symptoms like chest pain and shortness of breath. The surgery aims to remove a small part of the thickened heart muscle to improve blood flow and heart func…

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 tests a new, more stable version of the chemotherapy drug melphalan in people with multiple myeloma or AL amyloidosis who are getting a stem cell transplant. The goal is to find the best dose that reaches a target level in the body, while possibly causing fewer side ef…

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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 registry that will follow up to 221 children with Noonan Syndrome who are already taking or starting Norditropin® for short stature. Researchers will collect information on growth, side effects, and quality of life over time. No new treatment is being tested—the g…