Research

Sickle Cell Disease Treatment with BMT and Conditioning (STRIDE) – Phase II

A Study of Hematopoietic Stem Cell Therapy for Young Adults with Severe Sickle Cell Disease

Protocol Description

Sickle Cell Transplantation to Prevent Disease Exacerbation (STRIDE) is a study focused on young adults with severe sickle cell disease (SCD) to better understand the safety and feasibility of bone marrow transplantation (BMT) preceded by a special medication regimen called Reduced Intensity Conditioning (RIC). The use of RIC prior to BMT, a form of stem cell therapy, has been shown to cure SCD, but has been performed almost exclusively in children with matched sibling donors. This study is a first step to help to determine if BMT using RIC can be successful in older patients, and possibly those without matched family donors.
Study Level: Phase II

Eligibility Criteria

Subject to certain exclusion criteria, male and female candidates between the ages of 16 and 40 with severe SCD are eligible for this study. The candidates’ doctors and study team will determine if the patient meets the requirements of severe SCD.
Males: Ages 16 to 40
Females: Ages 16 to 40

Requirements
Prior to the transplant, patients must undergo numerous tests, including blood, lungs, kidneys, liver and brain to help study doctors determine eligibility. In addition, each participant’s doctor will search for a bone marrow donor, either a sibling whose bone marrow matches or an unrelated donor identified through the National Marrow Donor Program. Participants will receive an 8-day chemotherapy RIC regimen prior to transplant. In addition to care from their regular doctors, participants will be followed by the study team for at least a year after the transplant.
Visits: Requires hospitalization
Duration: Minimum of 14 months, inclusive of a year of post-transplant follow-up

Status: Open for Enrollment

Source(s) of Support
National Heart, Lung, and Blood Institute, National Institutes of Health

Additional Resources
Study Description at National Institutes of Health
National Marrow Donor Program