This study is looking at a new way to prepare patients for a stem cell transplant using a "reduced-intensity conditioning" (RIC) regimen. This means using lower doses of chemotherapy drugs like Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, and Thiotepa, compared to standard treatments. This approach is for people with non-cancerous conditions such as primary immunodeficiency, bone marrow failure, inherited metabolic disorders, hereditary anemias, and inflammatory conditions. The study aims to see how well patients do after transplant, specifically looking at transplant-related deaths, brain development, and how well their immune system recovers, all measured one year after the transplant. You could be eligible if you are between 2 months and 55 years old and have a suitable stem cell donor.

Study design

This is an interventional study planning to enroll 100 participants. It is not specified if it is randomized or blinded.

What's involved

Not specified in the trial record.

Compensation

Not stated in the trial record.

Follow-up

Participants will be followed for late effects and ongoing graft success after transplant, with primary outcomes measured at 1 year post-transplant.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.