NCT03214354 Nonmyeloablative Stem Cell Transplant in Children With Sickle Cell Disease and a Major ABO-Incompatible Matched Sibling Donor
| NCT ID | NCT03214354 |
| Status | Recruiting |
| Phase | Phase 2 |
| Sponsor | University of Calgary |
| Condition | Sickle Cell Disease |
| Study Type | INTERVENTIONAL |
| Enrollment | 12 participants |
| Start Date | 2017-07-05 |
| Primary Completion | 2028-07 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
In Phase 2, researchers evaluate early signs of effectiveness. You may be randomized to receive the active treatment or a comparator. Monitoring continues closely.
This trial targets 12 participants in total. It began in 2017-07-05 with a primary completion date of 2028-07.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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 Criteria
Inclusion Criteria: * Patients must be ≥ 12 months and \< 19 years of age at the time of study enrollment. * Patients must have sickle cell disease as defined by hemoglobin electropheresis, as follows: * homozygous Hb S disease (HbSS), * sickle-Hb C disease (HbSC), * sickle beta-plus-thalassemia (HbS/β+), or * sickle beta-null-thalassemia (HbS/βo) * Patients must meet standard eligibility criteria to undergo HSCT, including but not limited to one or more of the following: * history of repeated (more than 1) bony (vaso-occlusive) crisis * history of stroke * elevated transcranial Doppler velocity not eligible for hydroxyurea, as per TWiTCH trial (ie. severe vasculopathy) * history of acute chest crisis or splenic sequestration crisis * history of priapism in males * history of osteonecrosis * pulmonary hypertension as documented by tricuspid regurgitation jet velocity (TRV) \> 2.5 m/s on echocardiogram * red cell allo-immunization (≥ 2 antibodies) during long term transfusion therapy * Sickle complications should be present despite the use of hydroxyurea, but this is not an absolute requirement, if the treating team considers the patient to be at high risk for further crisis episodes. Exclusion Criteria: * Patients who are unable to comply with or follow the study protocol. * Patients with known hypersensitivity to sirolimus, its derivatives or to any of its components.
Contact & Investigator
Frequently Asked Questions
Who can join the NCT03214354 clinical trial?
This trial is open to participants of all sexes, aged 1 Year or older, up to 19 Years, studying Sickle Cell Disease. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT03214354 trial and what does that mean for participants?
Phase 2 trials evaluate whether the treatment shows signs of effectiveness while continuing to monitor safety. More participants are enrolled than in Phase 1 to help refine the treatment protocol.
Is NCT03214354 currently recruiting?
Yes, NCT03214354 is actively recruiting participants. Contact the research team at tony.truong@ahs.ca for enrollment information.
Where is the NCT03214354 trial being conducted?
This trial is being conducted at Calgary, Canada.
Who is sponsoring the NCT03214354 clinical trial?
NCT03214354 is sponsored by University of Calgary. The trial plans to enroll 12 participants.
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