Hypertrophic cardiomyopathy

This trial is testing an experimental treatment called FKC289 for people with primary AL amyloidosis that has relapsed or not responded to standard care. FKC289 uses a patient's own immune cells, engineered to recognize and attack the abnormal plasma cells that produce the harmfu…

This study tests a minimally invasive procedure called SESAME for people with thickened heart muscle that blocks blood flow from the heart. The procedure uses a catheter to make a controlled slice in the muscle, aiming to relieve symptoms like shortness of breath. Researchers wil…

Researchers are testing whether trametinib, an FDA-approved cancer drug, can help infants with RASopathies who have a severe, life-threatening heart condition called hypertrophic cardiomyopathy. The trial enrolls about 25 babies with a confirmed genetic diagnosis. Participants re…

Researchers are testing whether adults with obstructive hypertrophic cardiomyopathy can safely taper off beta-blockers after their heart condition has stabilized on mavacamten. The trial enrolls 286 people aged 18 to 75 who have been on a stable dose of mavacamten and one beta-bl…

This phase 3 trial is testing whether an experimental drug called BHB-1893 works better than the standard beta-blocker metoprolol for adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM), a condition where the heart muscle thickens and blocks blood flow. The stu…

This study tests whether adjusting the dose of mavacamten based on heart pumping strength can further reduce the blockage of blood flow in people with obstructive hypertrophic cardiomyopathy. Participants are adults who have been on a stable mavacamten dose for at least 12 weeks …

This phase 3 trial compares an investigational drug called etentamig against a standard combination therapy (daratumumab, cyclophosphamide, bortezomib, and dexamethasone) in people newly diagnosed with AL amyloidosis, a rare disease where abnormal proteins build up in organs. Abo…

This early study tests whether the drug CAEL-101/anselamimab can bind to and shrink abnormal protein deposits (amyloid) in people with AL amyloidoma. Five adults with measurable amyloid deposits will receive the drug and be monitored for up to 72 weeks. The goal is to see if the …

This phase II trial is testing whether a combination of selinexor, daratumumab, and dexamethasone (called XDd) can help people with advanced light-chain cardiac amyloidosis, a condition where abnormal proteins build up in the heart. The study aims to see if the regimen can achiev…

This phase 2 trial is testing an investigational drug called QLS4131 in people newly diagnosed with AL amyloidosis, a condition where abnormal proteins build up in organs and cause damage. QLS4131 is a trispecific antibody designed to target and eliminate the plasma cells produci…

This study tests an experimental drug called elranatamab in people newly diagnosed with AL amyloidosis who have not yet had treatment. The drug helps the body's immune cells find and destroy the abnormal cells causing the disease. Researchers want to see if it can produce deep re…

This trial tests whether a mechanical, spring-driven device called FreedomEDGE® can safely and effectively deliver daratumumab, a standard treatment for multiple myeloma and amyloidosis. Currently, nurses give these injections by hand, which can be physically demanding. About 100…

This phase 3 trial compares an experimental CAR-T cell therapy, NXC-201, with a standard drug combination (daratumumab plus CyBorD) in people newly diagnosed with AL amyloidosis, a rare condition where abnormal proteins damage organs. Participants receive either a single infusion…

This study tests whether pelvic floor muscle training (PFMT) with a handheld biofeedback device can improve erectile function and sexual quality of life in men with functional hypogonadism (low testosterone) and erectile dysfunction. Participants are randomly assigned to PFMT wit…

This study tests two chemotherapy-free treatments for people newly diagnosed with AL amyloidosis, a rare blood disorder. The choice of treatment depends on whether the patient has a specific genetic change called t(11;14). Those with t(11;14) receive a daily pill called sonrotocl…

This phase 2 trial tests an oral drug called Aom0304 in adults with hypertrophic cardiomyopathy, a condition where the heart muscle is abnormally thick. The study includes people with both obstructive and non-obstructive forms. Participants receive different doses of Aom0304 for …

This Phase 2 trial tests a new drug called QLS4131, given under the skin, in combination with other medications for people with malignant plasma cell neoplasms like multiple myeloma. The study will compare different drug combinations to see which works best at shrinking tumors an…

This early-phase study tests a new treatment called CAR19-BCMA CAR-T for people with certain blood cancers (plasma cell neoplasms or lymphomas/leukemias) that have returned or not responded to at least three prior treatments. The therapy uses a patient's own immune cells, modifie…

This study tests a new treatment for people with a rare disease called light-chain cardiac amyloidosis that has come back or not responded to other treatments. The treatment uses specially engineered immune cells (CAR-NK cells) from donated umbilical cord blood to target and atta…

This early-phase trial tests a new type of immune cell therapy called GPRC5D CAR-T in 18 people with relapsed or treatment-resistant plasma cell disorders, such as multiple myeloma. The therapy involves giving patients a single infusion of their own modified immune cells designed…

This study tests whether henagliflozin, a drug currently used for diabetes, can reduce scarring in the heart muscle of people with a type of hypertrophic cardiomyopathy (non-obstructive HCM). The trial will enroll 150 adults and use special PET scans to measure changes in scar ti…

This study explores whether a PET/MR scan using a radioactive tracer called 18F-Florbetaben can detect amyloid deposits—abnormal protein clumps that can damage organs—in people with certain blood disorders. Participants receive a single injection of the tracer followed by a whole…

This early-stage trial tests amlodipine, a calcium channel blocker used for high blood pressure, in 20 adults with myotonic dystrophy type 1. The goal is to see if the drug is safe and can improve muscle strength, reduce stiffness, and help with daily function. All participants w…

This study screens up to 1,000 people with personal or family histories of certain cancers to see if they qualify for ongoing genetics research at the National Cancer Institute. Participants fill out a 15-20 minute online survey about their health and family history. No treatment…

Researchers at University Health Network in Toronto are reviewing the records of 150 adults who had septal myectomy for hypertrophic obstructive cardiomyopathy. They compare intraoperative echocardiograms with predischarge echocardiograms to see how often moderate or worse mitral…

This pilot study will test a new method to see how two pain medicines, naproxen and pregabalin, work in healthy adults. Up to 25 participants will receive each medicine and a placebo in random order. They will undergo several short, controlled pain tests, like a cold water hand b…

This study follows 100 adults with myotonic dystrophy (types 1 or 2) for two years to understand how muscle stiffness, daily function, and heart health change over time. Researchers will look back at up to 18 months of past medical records and then track participants with clinic …

This study looks at people with obstructive hypertrophic cardiomyopathy — a condition where the heart muscle is abnormally thick and blocks blood flow. Researchers want to see if certain abnormalities in the mitral valve or its supporting structures affect how well the drug mavac…

This study will collect health information from 3000 people with hypertrophic cardiomyopathy (HCM) across multiple hospitals in China. The goal is to build a comprehensive database to understand how the disease is currently diagnosed and treated, and to identify factors that affe…

This study will check children under 18 with chronic liver disease for hidden heart problems. Doctors will use simple tests like ECG and ultrasound to look for issues such as abnormal heart rhythms or weakened heart muscle. The goal is to better understand how liver disease affec…

This study aims to better understand a condition called multilocus imprinting disorder (MLID), where multiple genes are affected by abnormal chemical marks. Researchers will test a new technique to detect these marks in 96 people, including those with known imprinting disorders a…

This study looks at how a person's genes affect their response to the heart drug mavacamten for obstructive hypertrophic cardiomyopathy. Researchers will collect DNA samples from 300 patients to see which genetic types respond best. The goal is to improve dosing safety and effect…

This study gathers information from 5,000 adults with hypertrophic cardiomyopathy, a condition where the heart muscle is too thick. Researchers will use heart MRI scans to look for scarring and build a better way to predict who is at risk of dying from the disease. The goal is to…

This study aims to find out why the heart medication mavacamten works better for some people with hypertrophic cardiomyopathy (a condition where the heart muscle is too thick) than for others. Researchers will look at participants' genes, including the specific gene causing their…

This study looks at whether a new medication (mavacamten) can improve the energy levels of the heart muscle in people with obstructive hypertrophic cardiomyopathy, a condition where the heart wall thickens and blocks blood flow. Researchers will use a special MRI scan to measure …

This study looks at why people with Noonan syndrome often bleed easily, especially from the skin, mouth, or nose. Researchers will compare a simple questionnaire about bleeding history with blood tests in 100 patients. The goal is to find better ways to predict serious bleeding, …