Intrinsic cardiomyopathy

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 a drug called CardiolRx in 90 hospitalized COVID-19 patients who also had heart disease or risk factors like diabetes or high blood pressure. The goal was to see if the drug could lower the chance of death, needing intensive care, or having heart problems. The t…

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 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-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 device called Efferon LPS that filters blood during heart surgery when a heart-lung machine is used. The goal was to see if it could prevent multiple organ failure, especially kidney damage. Only 14 people took part before the study was stopped, so the results…

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-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 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 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 letting patients manage their own recovery precautions after heart surgery leads to a better quality of life compared to following standard sternal precautions. Ten adults who had heart surgery were followed for up to a year. Researchers checked pain,…

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 looked at how two common pain relievers—celecoxib (a Coxib) and naproxen (an NSAID)—affect blood clotting in people with rheumatoid arthritis who also have heart disease or are at high risk for it. All participants took low-dose aspirin daily. The goal was to see if on…

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 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…