Study of Stem Cell Transplant for Aggressive Sickle Cell Disease
This study is testing a stem cell transplant approach for people with aggressive sickle cell disease. You might be eligible if you are between 16 and 60 years old and have complications like stroke or frequent pain crises. The treatment involves several medications, including ATG, fludarabine, and cyclophosphamide, along with low-dose total body irradiation (TBI) before receiving stem cells. The main goal is to see how many patients successfully engraft (when the new stem cells start making healthy blood cells) by day 60 after the transplant. The current recruitment status is unclear.
- Study design
- This is a Phase II interventional study aiming to enroll 50 participants.
- What's involved
- Not specified in the trial record.
- Compensation
- Not stated in the trial record.
- Follow-up
- The primary outcome is measured up to Day +60 after the stem cell infusion.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
Study of HLA-Haploidentical Stem Cell Transplantation to Treat Clinically Aggressive Sickle Cell Disease
At a glance
Conditions
Where it's being run
1 sites across 1 statesStudy leadership
- Damiano Rondelli, MD · PRINCIPAL_INVESTIGATOR · University of Illinois at Chicago
Who to contact
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What this trial measures
- Estimate the number of patients who engraft by Day +60Up to Day +60
Patients who achieve \< 5% peripheral blood donor chimerism by Day +30 and do not have a Day +60 measure will be regarded as failing to achieve full donor chimerism by Day +60; patients who achieve \> 5% donor chimerism by Day +30 but do not have a Day +60 measure will be considered nonevaluable for the primary endpoint.