Clinical Trials

Are you or your child at least 13 years old and have sickle cell disease? In this stidy, researchers are trying to find out if a new medication improves the success of a blood stem cell transplant. People 13 years old or older who are eligible for a blood stem cell transplant to treat sickle cell disease may be able to participate. Healthy family members over age 13 who are matched to transplant recipients are also needed to donate blood for the study. This study is taking place at the National Institutes of Health Clinical Center in Bethesda, Maryland.

The goal of this clinical trial is to compare different types of blood transfusions for critically injured patients who require large-volume blood transfusions. Participants must be at least 15 years old and need a massive blood transfusion. The study is taking place in locations in Alabama, California, Louisiana, Maryland, Missouri, North Carolina, Ohio, Oregon, Pennsylvania, Texas, Washington, and Wisconsin.

The investigators' central hypothesis is that early combination therapy with two PAH-specific oral therapies that have been shown to be well tolerated in the pediatric population, sildenafil and bosentan, will result in better World Health Organization (WHO) functional class at 12 months after initiation of PAH treatment than therapy with sildenafil alone.

This study is a two-part, prospective non-controlled, non-randomized Phase I/II clinical trial to assess the safety, feasibility and efficacy of cellular gene therapy in patients with chronic granulomatous disease using transplantation of autologous bone marrow CD34+ cells transduced ex vivo by the G1XCGD lentiviral vector containing the human CGD gene.

Have you or your child been diagnosed with a blood disorder? If so, you (or they) may be able to participate in a clinical study where researchers are studying a type of infusion using cord blood. Participants can be of any age but must have a blood disorder diagnosed by their doctor. The study is taking place in locations in Alabama, Arizona, California, Colorado, Delaware, Florida, Georgia, Illinois, Indiana, Kentucky, Maryland, Massachusetts, Michigan, Minnesota, Mississippi, Missouri, Nebraska, New York, Ohio, Oklahoma, Oregon, Pennsylvania, South Carolina, Tennessee, Texas, Virginia, Washington, Washington, D.C., West Virginia, and Wisconsin.

The primary goal of this study is to determine whether curative therapies for individuals with SCD will result in improved or worsening heart, lung, and kidney damage when compared to individuals with SCD receiving standard therapy. The investigators will also explore whether certain genes are associated with a good or bad outcome after curative therapy for SCD.

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.

Do you or does your child have a lipid disorder and want to improve future diagnosis and treatment? This study is exploring how different diagnostic tests can help us understand how lipid disorders, including high blood cholesterol, affect the body. Information from this study may help improve the way lipid disorders are diagnosed or treated in the future. To participate, you or your child must be at least 2 years old. The study is being conducted in Bethesda, Maryland.

Do you or your child have a lung disease? This study will investigate the genes involved in the breathing process and in the development of lung diseases such as asthma or sarcoidosis to improve understanding of the role they play. To participate in this study, you or your child must be between 2 and 90 years old. This study is located in Bethesda, Maryland, at the NIH Clinical Center.

Do you or your child have sickle cell disease and have a stem cell donor? This study is exploring how to improve bone marrow transplant procedures so the body better accepts donor stem cells. Participants in this study must be at least 4 years old. This study is located in Bethesda, Maryland.