Pilot Study to Evaluate the Safety and Feasibility of Induction of Mixed Chimerism in Sickle Cell Disease Patients With COH-MC-17: a Non-Myeloablative, Conditioning Regimen and CD4+ T-cell-depleted Haploidentical Hematopoietic Transplant

About the study

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:

Half-matched related donors will be used, and
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
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:

Will reverse sickle cell disease and improve patient quality of life,
Will reduce side effects and help the patient recover faster from the transplant,
Help the patient keep the transplant longer and
Reduce serious transplant-related complications.

Study point of contact

Lisa Pullens, RN
626-218-1231
LPullens@coh.org
Joseph Rosenthal, MD
626-218-8442
jrosenthal@coh.org

Locations

1 United States site

Age

18 Years - 45 Years

Phase

Phase 1

Study type

Interventional

Gender

All

Interventions

Drug

Biological

participation restrictions

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

Locations

  • Duarte, California, United States, City of Hope Medical Center, 91010 [Recruiting]
Last updated 2022-07-06