Repeat Stem Cell Transplant for Sickle Cell Disease
This study is for people with sickle cell disease or beta thalassemia who have already had a stem cell transplant, but their disease has returned. The goal is to give a second transplant using a special method called CliniMACS CD34 Reagent, which helps prepare your body to accept more donor cells. This treatment aims to make more room in your bone marrow for healthy donor cells to grow, hoping to cure your sickle cell disease. The study is looking for 32 participants, and success will be measured by whether your body accepts the new cells without rejection for up to five years. The study is currently unclear on its recruitment status.
- Study design
- This is an interventional study with a planned enrollment of 32 participants. It is not specified if it is randomized or blinded.
- What's involved
- You will undergo screening with a medical history, physical exam, and blood tests. Recipients will also have heart and breathing tests, x-rays, a bone marrow sample, and teeth and eye exams.
- Compensation
- Not stated in the trial record.
- Follow-up
- The primary goal is to see if your body accepts the new cells without rejection, which will be measured at 5 years.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
Repeat Peripheral Blood Stem Cell Transplantation for Patients With Sickle Cell Disease and Falling Donor Myeloid Chimerism Levels
At a glance
Conditions
Where it's being run
1 sites across 1 statesStudy leadership
- Courtney F Joseph, M.D. · PRINCIPAL_INVESTIGATOR · National Heart, Lung, and Blood Institute (NHLBI)
Who to contact
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What this trial measures
- dichotomous positive/negative outcome where a positive response is defined by absence of graft rejection5 years
The primary endpoint for each individual is a dichotomous positive/negative outcome where a positive response is defined by absence of graft rejection, and absence of severe acute GVHD (grade 3 and higher), or moderate to severe chronic GVHD evaluated 100 days post-transplant.