Restrictive 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 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 study tests the long-term safety and tolerability of acoramidis, an oral medication that stabilizes the transthyretin protein, in people newly diagnosed with transthyretin amyloid cardiomyopathy (ATTR-CM), a condition that can lead to heart failure. Participants who complete…

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 the long-term safety and effectiveness of an experimental oral drug called S-606001 in adults with late-onset Pompe disease, a genetic condition that causes progressive muscle weakness. Participants who completed a prior S-606001 study can join, and they will rec…

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 explores whether starting transdermal estradiol (a skin gel or patch) soon after menopause can protect the heart and brain in women who have hot flashes and at least one cardiovascular risk factor. One hundred women will be randomly assigned to receive the hormone ther…

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 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 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 study tests a new injectable drug called depemokimab in 30 children aged 6 to 11 with asthma that involves a specific type of inflammation. The drug is given alongside their usual asthma medicines to see how the body processes it, how well it works, and if it is safe. The go…

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 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 study tests whether a 12-month digital program with health coaching and a wearable device can reduce scarring in the heart muscle caused by high blood pressure. Researchers will enroll 200 adults aged 40-70 with early signs of heart thickening. The goal is to see if lifestyl…

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 gives ongoing access to benralizumab (Fasenra) for people with asthma, eosinophilic granulomatosis with polyangiitis (EGPA), or hypereosinophilic syndrome (HES) who completed a prior benralizumab trial and still benefit from the drug. About 230 participants will receiv…

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 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 early trial tests whether baricitinib, an immune-calming drug already used for arthritis, can reduce heart inflammation in people with cardiac sarcoidosis. Ten adults will take the drug for 16 weeks alongside their usual steroid-sparing medication. Researchers will use PET s…

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 Phase 2 trial tests AD17002, a nasal spray that aims to calm the immune system in people with moderate to severe eosinophilic asthma that is not well controlled by current treatments. About 126 adults will receive either the spray or a placebo for 3 months. Researchers will …

This study explores whether specialized eye imaging can identify people with a rare heart condition called transthyretin amyloid cardiomyopathy (ATTR-CM). Researchers will use machine learning to analyze eye scans from 500 adults—some with ATTR-CM and others with heart failure bu…

This study tests a special radioactive tracer called [68Ga]Ga-FAPI-46 for PET scans in 40 people with transthyretin cardiac amyloidosis (ATTR-CM), a condition where abnormal proteins build up in the heart. The goal is to see if this scan can measure scar-like activity in the hear…

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 study will test whether a faster type of bone scan (CZT-SPECT) can accurately diagnose transthyretin cardiac amyloidosis, a serious heart condition. Researchers will compare the new scan with the standard method in 200 patients. If it works, it could make diagnosis quicker a…

This study tests whether a computer model can find signs of a heart condition called cardiac amyloidosis in bone scans that were done for other reasons. Researchers will look back at 57 patients' scans to see if the model can correctly identify those with the disease. If it works…

This study tests whether a special PET-CT scan using a drug called evuzamitide can detect signs of cardiac amyloidosis in people whose standard heart scans were unclear but who have high levels of a blood marker called TAD1. About 25 adults with heart failure or a genetic risk fo…

This study tests whether a structured 5-week cardiac rehabilitation program can improve exercise ability and quality of life in 40 adults with cardiac amyloidosis. Participants will undergo supervised aerobic and strength training five days a week, with tests before and after the…

This study tests whether increasing the pacemaker's lower rate from 60 to 80 beats per minute improves quality of life and daily function in people with ATTR cardiac amyloidosis. The heart in this condition is stiff and depends on a faster heart rate to pump blood. Thirty-four pa…

This study tests whether a home-based exercise program combined with nutritional supplements can improve walking ability and muscle strength in people aged 70 and older with transthyretin cardiac amyloidosis, a heart condition that causes weakness and frailty. About 102 participa…

This study looks at whether a digital tool called Luscii is practical and acceptable for people with a rare heart condition called ATTR-CM. About 60 adults in Austria, Germany, and Italy will use a smartphone app and home devices to share health information with their care team. …

This study looks at whether biologic therapy can reverse long-term lung damage in adults with severe asthma. Researchers will follow 150 patients and 50 healthy volunteers for two years, using advanced imaging and lung function tests. The goal is to see if treatment leads to 'cli…

This observational study monitors the long-term safety of eplontersen, a treatment for transthyretin amyloidosis, in patients who have had a liver transplant or have severe liver impairment. Researchers will track side effects, lab changes, and serious health events in about 320 …

This study will follow 200 people with suspected or confirmed cardiac amyloidosis, a condition where abnormal proteins build up in the heart and cause heart failure. Over two years, participants will undergo heart MRI scans, exercise tests, and blood work at regular visits. The g…

This study will observe 143 older adults with severe aortic stenosis who are scheduled for a minimally invasive valve replacement procedure called TAVR. Researchers want to find out how many also have cardiac amyloidosis, a condition where abnormal proteins build up in the heart.…

This study looks at 50 adults with AL amyloidosis who achieved complete remission after initial chemotherapy. It checks if tiny amounts of remaining disease (called minimal residual disease, or MRD) affect organ recovery and survival. Researchers also test whether a stem cell tra…