A Study Using Immune Suppression Prior to Stem Cell Transplant for Children and Adults with Sickle Cell Disease or Beta-Thalassemia


Full Title

Pre-Transplant Immune Suppression with Hematopoietic Cell Transplantation from Haploidentical Donors for Adults and Children with Sickle Cell Disease or ß-Thalassemia (Haplo PTCy)


Sickle cell disease and beta-thalassemia are blood diseases caused by a genetic change (mutation) in hemoglobin, the protein in red blood cells that carries oxygen throughout the body. People with these diseases may be offered a stem cell transplant. Stem cell transplantation involves receiving healthy blood-forming cells (stem cells) from a donor to replace the diseased or damaged cells in the bone marrow.

In this study, researchers want to determine if giving medications to quiet down the immune system before standard conditioning therapy and stem cell transplantation can reduce the risk of serious side effects of transplant. Participants will receive fludarabine and dexamethasone (medications that lower the activity of the immune system) before standard conditioning therapy and stem cell transplantation. Depending on how the body responds to fludarabine and dexamethasone, the study doctor may decide a patient should receive another medication, called cyclophosphamide, instead of fludarabine. In addition, depending on the results of routine blood tests, participants may receive the drugs bortezomib and rituximab, which also help with immune suppression.

Who Can Join

To be eligible for this study, patients must meet several requirements, including:

  • Participants must have sickle cell disease or beta-thalassemia and be candidates for a donated stem cell transplant.
  • Prior stem cell transplantation from a donor is not permitted.
  • Patients must be physically well enough that they are able to be mobile, take care of themselves, and engage in all but physically strenuous activities. For example, they must be well enough that they could carry out office work or light housework.
  • This study is for people ages 2-50.


For more information about this study and to ask about eligibility, please contact 1-833-MSK-KIDS.




Phase II (phase 2)

Disease Status

Newly Diagnosed & Relapsed/Refractory



ClinicalTrials.gov ID