NCT03214354

Nonmyeloablative Stem Cell Transplant in Children with Sickle Cell Disease and a Major ABO-Incompatible Matched Sibling Donor

A Phase II Pilot Study of Nonmyeloablative Conditioning Hematopoietic Stem Cell Transplantation in Children with Sickle Cell Disease Who Have a Matched Related Major ABO-Incompatible Donor (Sickle-AID)

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 12 (estimated)
• Sponsor: University of Calgary · Academic / Other
• Sex: All
• Age: 1 Year – 19 Years
• Healthy volunteers: Not accepted

Summary

The aim of this study to evaluate the safety and efficacy of a nonmyeloablative conditioning regimen for allogeneic hematopoietic stem cell transplantation (HSCT) in pediatric patients with sickle cell disease (SCD) who have a matched related major ABO-incompatible donor. The nonmyeloablative regimen will use alemtuzumab, total body irradiation (TBI) and sirolimus for immune suppression. This study will expand the access of HSCT for patients with SCD who are currently not eligible because of donor restrictions.

Detailed description

Sickle cell disease (SCD) is a debilitating chronic blood disorder with multi-system end-organ damage that leads to morbidity and early mortality. The only cure for SCD is hematopoietic stem cell transplantation (HSCT), which given the risks with unrelated HSCT, is only an option for a minority of patients who have a matched sibling donor. In the field of HSCT, blood group ABO incompatibility between donor and recipient is not a contraindication and several studies do not show compromised outcomes. However, in the context of nonmyeloablative (NMA) conditioning and major ABO-incompatibility, when the recipient has existing antibodies to donor red blood cells, pure red cell aplasia (PRCA) may occur. This phase II pilot study will enroll SCD patients with a matched related major ABO-incompatible donor to determine the safety and efficacy of NMA-HSCT. Biological studies will include a plan to study and monitor red cell engraftment in this population to facilitate early detection and interventional measures to prevent and treat PRCA.

Conditions

• Sickle Cell Disease
• Stem Cell Transplant Complications
• Red Blood Cell Disorder
• Pure Red Cell Aplasia

Interventions

Type	Name	Description
DRUG	Alemtuzumab	Alemtuzumab, Day -7 to -3. Dose: 0.2mg/kg/dose SC once daily x 5 days
RADIATION	Total Body Irradiation	TBI 300 cGy on Day -2
DRUG	Sirolimus	Sirolimus is used for GVHD prophylaxis

Timeline

• Start date: 2017-07-05
• Primary completion: 2028-07-01
• Completion: 2028-07-01
• First posted: 2017-07-11
• Last updated: 2024-12-10

Locations

1 site across 1 country: Canada