NCT03214354RecruitingPhase 2
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)
Source: ClinicalTrials.gov · Last updated 1 May 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Phase (registry)
- Phase 2
- Study type
- Interventional
- Enrollment
- 12 (estimated)
- First submitted
- 5 Jul 2017
- Start date
- 5 Jul 2017
- Primary completion date
- 31 Jul 2028
- Lead sponsor
- University of Calgary
- Diseases
- Sickle cell disease
- NCT number
- NCT03214354
What you commit to
- Study type: Interventional · Phase 2
- Planned participants: 12
- From 5 Jul 2017 to the primary completion date, 31 Jul 2028
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other illness
- Patients with known hypersensitivity to sirolimus, its derivatives or to any of its components.
Other
- Patients who are unable to comply with or follow the study protocol.
Who the study is looking for
- 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.
Age in the registry: 1 year to 19 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Alberta Children's Hospital Coordinator of this site? Confirm its status | Calgary | Canada | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.