Stem Cell Transplant with TCRab Depletion for Sickle Cell Disease and Beta Thalassemia

Recruiting in Palo Alto (17 mi)

Overseen byTimothy Olson, MD, PhD

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Academic

Waitlist Available

Sponsor: Children's Hospital of Philadelphia

No Placebo Group

Trial Summary

What is the purpose of this trial?This is a single arm pilot study of peripheral stem cell transplantation (PSCT) with ex vivo t-cell receptor alpha beta+(TCRαβ+) T cell and cluster of differentiation 19+ beta (CD19+ B) cell depletion of unrelated donor (URD) grafts using the CliniMACS device in patients with sickle cell disease (SCD) and beta thalassemia major (BTM).

Eligibility Criteria

This trial is for patients with severe forms of sickle cell disease or beta thalassemia major who have had strokes, frequent pain episodes, or require regular blood transfusions. It's not open to those who've had a previous stem cell transplant, are pregnant, or can't receive blood transfusions due to severe allergies.

Inclusion Criteria

I have had a stroke or lasting neurological issues for more than a day.

My spleen is enlarged and my hemoglobin levels have dropped.

My genetic test confirms I have Beta Thalassemia.

+11 more

Exclusion Criteria

I have had a stem cell transplant before.

I have stopped any clinical trial treatments before starting transplant therapy.

I cannot receive blood transfusions due to severe reactions to them.

+3 more

Participant Groups

The study tests a peripheral stem cell transplantation technique using the CliniMACS device to remove certain T cells and B cells from donor grafts in patients with sickle cell disease and beta thalassemia major.

2Treatment groups

Experimental Treatment

Group I: Sickle Cell DiseaseExperimental Treatment1 Intervention

Patients with Sickle Cell Disease (SCD) will be given previously established, disease-specific chemotherapy based conditioning regimens prior to hematopoietic stem cell transplantation using TCRalpha/beta and B cell depleted peripheral blood stem cells from closely matched unrelated donors.

Group II: Beta Thalassemias MajorExperimental Treatment1 Intervention

Patients with Beta Thalassemias Major (BTM) will be given previously established, disease-specific chemotherapy based conditioning regimens prior to hematopoietic stem cell transplantation using TCRalpha/beta and B cell depleted peripheral blood stem cells from closely matched unrelated donors.