This study is testing a new approach for preparing patients with severe sickle cell disease (SCD) for a bone marrow transplant. It uses a drug called 131I-apamistamab, which combines an antibody with a radioactive substance, instead of the usual conditioning treatment. The goal is to find the lowest effective dose of 131I-apamistamab. This is the first time 131I-apamistamab is being used for advanced SCD before a stem cell transplant. The study aims to see if this new method can be effective while potentially reducing long-term side effects associated with standard radiation. You may be able to join if you are 12-50 years old with severe sickle cell anemia and have an HLA-matched sibling donor, especially if you've had a stroke or other neurological event. The main goal is to see how many patients experience graft failure (when the transplanted cells don't take) 42 days after the transplant.

Study design

This is a Phase I, single-center study aiming to find the minimum effective dose of 131I-apamistamab, with a planned enrollment of 24 participants.

What's involved

Before starting therapy, you will undergo a red blood cell exchange transfusion. You will receive 131I-apamistamab, Sirolimus (an immunosuppressant taken orally), and Campath (an antibody), along with Total Body Irradiation.

Compensation

Not stated in the trial record.

Follow-up

The primary endpoint for measuring success is graft failure rate, which is assessed 42 days after the blood stem cell 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.