Clinical Trials

Are you between the ages of 18 and 50 and diagnosed with Friedreich's ataxia? In this study, researchers are testing a new medication, gene therapy, to treat cardiomyopathy associated with Friedreich's ataxia, and you may be able to participate if you are not taking any corticosteroids or immunosuppressive medications to treat Friedreich's ataxia. This study is taking place in New York City, New York.

Do you have heart failure? In this clinical trial, researchers are using images of the heart and blood tests to learn more about Black and Hispanic patients with heart failure. You may be able to participate if you are at least 60 years old; of Black, Hispanic, or Caribbean origin; and have been diagnosed with heart failure. The study is taking place in locations in Connecticut, Massachusetts, and New York.

Have you been diagnosed with peripartum cardiomyopathy and are within 5 months of your due date? This study is evaluating the effectiveness of a medicine called bromocriptine to treat peripartum cardiomyopathy, or weakness of the heart muscle that occurs during or shortly after pregnancy. To participate in this study, you must be at least 18 years old, diagnosed with peripartum cardiomyopathy, and within 5 months of your delivery date. This study takes place in Pittsburgh, Pennsylvania.

22q11.2 deletion syndrome is a genetic disorder that can cause heart defects, facial abnormalities, and developmental and learning disabilities. The severity of the disorder can vary widely among people. This study will analyze DNA from people with 22q11.2 deletion syndrome to identify genetic variations that may affect the severity of the disorder.

Does your child have Down Syndrome? Did they have a complete atrioventricular septal defect (CAVSD) repair within the first year of their life? Researchers in this study are investigating how the
brain develops in children with Down Syndrome who have congenital heart disease. To participate, your child must be between 5 and 12 years old and have Down Syndrome. The study will enroll children who have congenital heart disease as well as children who do not have congenital heart disease. The study is taking place in locations in Delaware, Georgia, Indiana, Massachusetts, Michigan, Missouri, New York, Ohio, Pennsylvania, South Carolina, Texas, Utah, Washington, D.C., and Toronto, Canada

Do you or one of your children have a congenital heart defect? This study will find both common genetic causes of congenital heart disease and ways that genes influence results of medical treatment. To participate in this study, you or your child must have congenital heart disease. This study is located in Los Angeles, Palo Alto, and San Francisco, California; New Haven, Connecticut; Boston, Massachusetts; New York and Rochester, New York; Philadelphia, Pennsylvania; and Salt Lake City, Utah.

Are you a greater Boston resident aged 24 to 30? This study aims to bridge the gap in knowledge between known executive function deficits in childhood congenital heart disease and adult well-being. The study is Recruitinghealthy volunteers. To participate in this study, you must be between the ages of 24 and 30, live in the greater Boston area, read fluently in English, and not have metal implants, a pacemaker, or orthodontia that would prevent MRI. This study is located in Boston, Massachusetts.

Are you an adult female with LAM? If so, you may be able to participate in this clinical study where researchers are observing patients who are taking or have taken a certain kind of medication, mammalian target of rapamycin (mTOR), to treat lymphangioleiomyomatosis, also known as LAM. You must be at least 18 years old to take part. The study is taking place at locations in California, Colorado, Florida, Georgia, Illinois, Massachusetts, Michigan, Minnesota, Missouri, New York, Ohio, Oregon, Pennsylvania, South Carolina, Tennessee, Texas, Utah, and Washington.

Do you or your child have a platelet, bleeding, or white blood cell disorder? This study is looking at people who have platelet or other blood disorders. The researchers will use a blood sample to look for problems with platelets and a possible genetic cause in the participant’s DNA or RNA. To participate in this study, you must be a child or adult who either is healthy or has a platelet disorder, coagulation disorder, or white blood cell disorder. This study is located in New York, New York.

Does your infant have a low platelet count? This study is investigating the safety and efficacy of platelet transfusion as a treatment for infants with thrombocytopenia. Researchers are looking at the positive effects of platelet transfusion against the negative effects, such as the release of inflammatory molecules and the formation of blood clots. To participate in this study, your child must be younger than 6 months old and have a low platelet count (less than 100 × 109/L). This study takes place in Boston, Massachusetts.