drugset / Trial / NCT07599176

Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors

NCT07599176

Phase 1/2 Recruiting 90 enrolled National Heart, Lung, and Blood Institute (NHLBI)
Non-randomizedSequentialOpen-labelTreatment

Summary

This is a non-ablative (partial) stem cell transplant for patients with severe sickle cell disease or beta-thalassemia requiring red cell transfusions. The intensity of the transplant is slightly increased from our previous transplant regimens. The goal is to aim for higher percentage of donor cells to stably remain in the recipients long term.

Timeline

Start
2026-07-08
Primary completion
2032-06-30
Completion
2035-06-30

Drugs

EvaluationDrugModalityDoseRoute
Subject Abatacept Other / unclassified 10 mg/kg
Subject Alemtuzumab Monoclonal antibody 1 mg/kg