Clinical Trials

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.

Does your infant have a low platelet count? This study investigates immature platelet counts as a marker for bleeding risk in newborns with thrombocytopenia, compared with platelet counts alone. Immature platelets are the most recently produced platelets and may be a better marker of platelet production. To participate in this study, you must have a newborn who is less than 32 weeks’ gestational age, has a birth weight greater than 500 grams, and has a platelet count less than 100 × 109/L. This study takes place in multiple locations in the United States, the Netherlands, and Sweden.

Are you an adult with polycythemia vera or thrombocytosis? This study aims to find a new treatment or cure for polycythemia vera and thrombocytosis by locating genes and their changes, or mutations, that may contribute to these disorders. To participate in this study, you must have polycythemia vera with elevated hemoglobin (higher than 18 in men and 16 in women) or thrombocytosis with an elevated platelet count (higher than 450,000). This study takes place in Salt Lake City, Utah.

Have you been diagnosed with LAM? This study is trying to understand the genes and proteins that are responsible for causing lung damage in patients with LAM. To participate in this study, you must be between 16 and 99 years old and diagnosed with LAM. This study is located in Bethesda, Maryland.

Are you an adult female who has been diagnosed with LAM? This study is examining which delivery method of albuterol—inhaled or nebulizer—is better at improving lung function in adult women who have LAM. This study is located in Bethesda, Maryland.

Do you have LAM and would be willing to help research? This study aims to identify the right dose of sirolimus to treat people who have LAM. To participate in this study, you must be female, between 18 and 90 years old, and diagnosed with LAM. This study is located in Bethesda, Maryland.

This study is being done to test the comparative effectiveness of participating in a Pulmonary Rehabilitation Program at home using new technology and health coaching following dismissal from the hospital compared to the referral to a center based pulmonary rehabilitation that may include the choice of center based or telehealth.

This project will compare the effectiveness of two novel strategies - one involving video narratives of other patients telling their story of how they overcame challenges and completed PR, the other involving telephonic peer coaching with an individual with lived experience - to enhanced usual care, and to each other, at increasing patient participation in PR after an exacerbation.

Pulmonary rehabilitation (PR) is a structured program of exercise and self-management support that has been proven to relieve shortness of breath and increase quality of life when initiated after an exacerbation, but unfortunately, few eligible patients participate. This project will compare the effectiveness of two novel strategies - one involving video narratives of other patients telling their story of how they overcame challenges and completed PR, the other involving telephonic peer coaching with an individual with lived experience - to enhanced usual care, and to each other, at increasing patient participation in PR after an exacerbation.