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About the Study

Who runs this study?

This study is being conducted by the Sickle Cell Branch of the National Heart, Lung, and Blood Institute (NHLBI). The NHLBI is dedicated to improving the care of individuals with sickle cell disease: https://www.nhlbi.nih.gov/health-topics/education-and-awareness/sickle-cell

The Principal Investigator of the study is Dr. Julia Xu, MD, MScGH, a hematologist specializing in sickle cell disease (SCD) and global health. The Medically Responsible Investigator is Dr. Swee Lay Thein, MD, PhD, a Senior Investigator at the NHLBI, Chief of the Sickle Cell Branch, and hematologist specializing in SCD, thalassemia, and genetics.

What is this study about?

Participants in this study will have already taken part in our short-term study of the safety of mitapivat. Our goal for this study is to treat participants with SCD with a stable dose of mitapivat for a longer duration (1-2 years) and evaluate the long-term safety and clinical effects of mitapivat treatment for SCD. We will use a number of laboratory and clinical tests to assess the potential benefits of mitapivat. In addition, we will use quality of life questionnaires to help us understand the effect of mitapivat on pain, fatigue, and other symptoms that individuals with SCD often experience.

What kind of commitment is involved?

Participants interested in this study will first be screened for eligibility. If eligible, participants will begin taking the study drug, mitapivat, and return to the NIH for clinic visits and blood draws at regular intervals throughout the duration of the study. All participants will be asked to attend at least 10 clinic visits during the first 48-week (~1 year) period of the study. Participants benefiting from treatment may have an option to continue mitapivat treatment for an additional 48 weeks and attend 4 additional clinic visits. Participants will also be asked to take part in a few clinical tests every 6 months and to complete quality of life questionnaires every 3 months. In addition, participants may return to the NIH for unscheduled clinic visits if they or the research team have any concerns about the study drug.

How can I ask questions or ask for help?

Email us at ingrid.frey@nih.gov (or click on Contact). We will respond within 2 business days.