Clinical trials
Full profile accessFollow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
This nonmyeloablative allogeneic peripheral blood stem cell transplantation (HSCT) protocol was developed and evaluated by the University of Texas Southwestern Medical Center for the treatment of severe congenital anemias, specifically sickle cell disease and thalassemia. The procedure utilizes a nonmyeloablative conditioning regimen consisting of low-dose total body irradiation, alemtuzumab (Campath) for in vivo T-cell depletion, and sirolimus for post-transplant immunosuppression. By using unmanipulated peripheral blood stem cells from an HLA-matched donor, the protocol aims to establish stable donor chimerism and restore normal hematopoietic function without the high morbidity and mortality associated with traditional myeloablative conditioning. This approach is designed to minimize the risk of graft-versus-host disease (GVHD) while ensuring successful engraftment in adult patients with high-risk congenital anemias.
Beyond the preview
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Follow clinical development from study design and recruitment through results.
Explore development by indication, patient population, and geography.
Trace asset ownership, licensing agreements, and commercial partnerships.
Explore the patent landscape and regulatory exclusivity around an asset.
Compare development programs by target, modality, and indication.
Connect source evidence and development news to your research questions.
See how Gosset can support your research on allogeneic peripheral blood stem cell transplantation.