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

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 study tests a non-myeloablative (reduced-intensity) stem cell transplant approach for children and teenagers with sickle cell disease (SCD) who need a transplant but whose sibling donor has an incompatible blood type (ABO mismatch). Sickle cell disease causes red blood cells to become misshapen and block blood flow, leading to pain crises, strokes, and organ damage. This protocol aims to achieve a successful transplant with fewer side effects than full-intensity conditioning. You may be eligible if: - You are between 12 months and 19 years old - You have sickle cell disease (HbSS, HbSC, HbS/β+, or HbS/β0) - You meet standard criteria for stem cell transplantation, including a history of strokes, repeated pain crises, acute chest syndrome, priapism, pulmonary hypertension, or other serious complications - You have a matched sibling donor available You may NOT be eligible if: - You are unable to comply with the study protocol - You have a known allergy or sensitivity to sirolimus (the anti-rejection medication used) Talk to your doctor to see if this trial is right for you.

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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