T-Haplo for SCD

“A Phase II Stratified Trial to Assess Haploidentical T-depleted Stem Cell Transplantation in Patients With Sickle Cell Disease With no Available Sibling Donor”

This study investigates a specific type of stem cell transplant for children with sickle cell disease who do not have a compatible healthy sibling donor. The procedure involves using a semi-compatible (haploidentical) donor from the family—such as a parent—whose stem cells are prepared in the laboratory (“T-depleted”) in a way that reduces the risk of severe rejection reactions. The goal of this Phase II study is to assess how safe and effective this transplantation procedure is—that is, whether it can permanently control or even cure sickle cell disease without causing an unacceptable number of severe side effects.

This type of study is of particular interest to affected families because it offers an option for children who, until now, were not eligible for a stem cell transplant due to the lack of a suitable sibling donor.
 

Patient enrollment in Heidelberg is possible since July 2021.