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Spotlight Clinical Trials

Clinical research is at the heart of all medical advances, identifying new ways to prevent, detect or treat disease. If you have a bone marrow failure disease, you may want to consider taking part in a clinical trial, also called a research study.

Sirolimus (Rapamune ) for Relapse Prevention in People With Severe Aplastic Anemia Responsive to Immunosuppressive Therapy

Status(es): Recruiting
Study Date(s): Monday, December 19, 2016 to Thursday, August 31, 2023
Disease(s): Aplastic Anemia
Age Group: 2 years and older
People with severe aplastic anemia (SAA) do not make enough red and white blood cells, and/or platelets. Their body's immune system stops the bone marrow from making these cells. The treatment cyclosporine leads to better blood counts. But when this treatment is stopped, the disease may return in 1 in 3 people. The drug sirolimus may help by suppressing the immune system.

The National Myelodysplastic Syndromes Natural History Study

Status(es): Recruiting
Study Date(s): Friday, April 1, 2016 to Monday, March 31, 2025
Disease(s): Myelodysplastic Syndromes (MDS)
Age Group: 18 years and older
Multi-center study enrolling patients suspected or newly diagnosed with myelodysplastic syndromes (MDS), myelodysplastic syndromes/myeloproliferative neoplasms (MDS/MPN) overlap disorder, or idiopathic cytopenia of undetermined significance (ICUS).  Participants will be followed long term.  Clinical data, blood, and tissue samples will be collected to establish a biorepository to facilitate the study of the natural history of MDS.