RecruitingPhase 2NCT03214354

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)

Sponsor

University of Calgary

Enrollment

12 participants

Start Date

Jul 5, 2017

Study Type

INTERVENTIONAL

Conditions

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

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.

Eligibility

Min Age: 1 YearMax Age: 19 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called Alemtuzumab, a drug called Sirolimus, and others for people with pure red cell aplasia, red blood cell disorder, and other related conditions. The study is currently recruiting participants at 1 location. People eligible for this study include aged 1 Year to 19 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGAlemtuzumab

Alemtuzumab, Day -7 to -3. Dose: 0.2mg/kg/dose SC once daily x 5 days

RADIATIONTotal Body Irradiation

TBI 300 cGy on Day -2

DRUGSirolimus

Sirolimus is used for GVHD prophylaxis

Locations(1)

Alberta Children's Hospital

Calgary, Alberta, Canada

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NCT03214354

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