A Blood Stem Cell Transplant for Sickle Cell Disease

Running, not enrolling · Phase 1

Conditions studied: Sickle Cell Disease, Sickle Cell Disorder, Hemoglobinopathies, Thalassemia, Anemia, Sickle Cell

In brief

Blood stem cells can produce red blood cells (which carry oxygen), white blood cells of the immune system (which fight infections) and platelets (which help the blood clot). Patients with sickle cell disease produce abnormal red blood cells. A blood stem cell transplant from a donor is a treatment option for patients with severe sickle cell disease. The donor can be healthy or have the sickle cell trait. The blood stem cell transplant will be given to the patient as an intravenous infusion (IV). The donor blood stem cells will then make normal red blood cells - as well as other types of blood cells - in the patient. When blood cells from two people co-exist in the patient, this is called mixed chimerism. Most children are successfully treated with blood stem cells from a sibling (brother/sister) who completely shares their tissue type (full-matched donor). However, transplant is not an option for patients who (1) have serious medical problems, and/or (2) do not have a full-matched donor. Most patients will have a relative who shares half of their tissue type (e.g. parent, child, and brother/sister) and can be a donor (half-matched or haploidentical donor). Adult patients with severe sickle cell disease were successfully treated with a half-matched transplant in a clinical study. Researchers would like to make half-matched transplant an option for more patients by (1) improving transplant success and (2) reducing transplanted-related complications. This research transplant is being tested in this Pilot study for the first time. It is different from a standard transplant because: 1. Half-matched related donors will be used, and 2. A new combination of drugs (chemotherapy) that does not completely wipe out the bone marrow cells (non-myeloablative treatment) will be used to prepare the patient for transplant, and 3. Most of the donor CD4+ T cells (a type of immune cells) will be removed (depleted) before giving the blood stem cell transplant to the patient to improve transplant outcomes. It is hoped that the research transplant: 1. Will reverse sickle cell disease and improve patient quality of life, 2. Will reduce side effects and help the patient recover faster from the transplant, 3. Help the patient keep the transplant longer and 4. Reduce serious transplant-related complications.

Key facts

Study ID

NCT03249831

Run by

City of Hope Medical Center

People needed

3

Starts

2019-01-04

Expected to finish

2027-01-25

Last updated by the study team

2026-03-05

Who can join

Age: 18 and older, up to 45. Sex: any. Healthy volunteers: not accepted.

You may not qualify if…

• Prior stem cell transplant
• Prior bone marrow transplant
• Concurrent other investigational agents, chemotherapy, biological therapy or radiation therapy
• Planned use of moderate and strong CYP3A4 inhibitors
• Active infection
• Major surgery within the last 30 days
• Clinically significant liver fibrosis or cirrhosis if on chronic transfusion therapy > 6 months
• Active malignancy (other than non-melanoma skin cancers)
• History of allergic reactions attributed to compounds of similar chemical or biologic composition to any in the pre- or post-transplant regimen.
• Women of childbearing potential: pregnant or breastfeeding

Where it is running

• City of Hope Medical Center — Duarte, California, United States

Full record on ClinicalTrials.gov

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