NCT03421756

Stem Cell Transplant in Patients With Severe Sickle Cell Disease

A Pilot Study Evaluating the Efficacy of Non-Myeloablative Matched Related Donor Peripheral Blood Stem Cell Transplant in Patients With Severe Sickle Cell Disease

• Status: Terminated
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 1 (actual)
• Sponsor: Kathleen Dorritie · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This is a prospective pilot study of matched-related donor allogeneic stem cell transplantation in adults with severe sickle cell disease using a matched-sibling PBSC graft with a non-myeloablative conditioning regimen (Alemtuzumab).

Detailed description

Stem cell transplantation recipients will be given Alemtuzumab, which is a non-myeloablative pre-transplant conditioning regimen. This non-myeloablative therapy uses doses of chemotherapy and radiation to weaken (but not destroy) the patients bone marrow and immune system, while still allowing their body to accept the donor's stem cells. Alemtuzumab will be given 7 days prior to stem cell infusion at 0.03 mg/kg IV, 6 days prior to stem cell infusion at 0.1 mg/kg IV, and 5 thru 3 days prior to stem cell infusion at 0.3 mg/kg IV.

Conditions

• Sickle Cell Disease

Interventions

Type	Name	Description
DRUG	Alemtuzumab	Alemtuzumab is a non-myeloablative pre-transplant conditioning regimen. Non-myeloablative therapy uses doses of chemotherapy and radiation to weaken (but not destroy) the patients bone marrow and immune system, while still allowing their body will accept the donor's stem cells. Alemtuzumab will be given 7 days prior to stem cell infusion at 0.03 mg/kg IV, 6 days prior to stem cell infusion at 0.1 mg/kg IV, and 5 thru 3 days prior to stem cell infusion at 0.3 mg/kg IV.
RADIATION	Total Body Irradiation	300 cGy will be administered in a single fraction on Day - 2. TBI is used commonly as part of pre-transplant conditioning in a variety of settings.
DRUG	Sirolimus	Sirolimus will be used for the prevention of graft-verus-host disease and will begin on Day - 1.

Timeline

• Start date: 2018-03-29
• Primary completion: 2018-09-15
• Completion: 2022-05-15
• First posted: 2018-02-05
• Last updated: 2022-06-28

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study