Recruiting
Phase 2

Stem Cell Transplant

Sponsor:

University of Calgary

Code:

NCT03214354

Conditions

Sickle Cell Disease

Stem Cell Transplant Complications

Red Blood Cell Disorder

Pure Red Cell Aplasia

Eligibility Criteria

Sex: All

Age: 1 - 19

Healthy Volunteers: Not accepted

Interventions

Alemtuzumab

Total Body Irradiation

Sirolimus

Study Details

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.

Conditions

Sickle Cell Disease

Stem Cell Transplant Complications

Red Blood Cell Disorder

Pure Red Cell Aplasia

Study ID

NCT03214354

Start date

Jul 5, 2017

Status verified date

Apr, 2026

Completion date

Jul, 2028

Anticipated

Primary completion date

Jul, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 1 - 19

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

12 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Non-myeloablative conditioning

Non-myeloablative conditioning

Interventions

Alemtuzumab

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

Total Body Irradiation

TBI 300 cGy on Day -2

Sirolimus

Sirolimus is used for GVHD prophylaxis

Primary outcome measure

  • Incidence of pure red cell aplasia (PRCA) [ Time Frame: 6 months from enrollment ]

Central Contacts and Locations

Central contacts

Tony Truong, MD, MPH

403-955-7272tony.truong@ahs.ca

Greg Guilcher, MD

403-955-7272

Locations

Alberta Children's Hospital

Recruiting

Calgary, Alberta, Canada, T3B 6A8

Contacts

Tony Truong, MD, MPH

403-955-7272tony.truong@ahs.ca

Greg Guilcher, MD

403-955-7272

Principal Investigator:

Tony Truong, MD, MPH

More Information

Sponsor

University of Calgary

Last update posted

May 1, 2026

Last verified

Apr, 2026

Keywords

  • sickle cell disease
  • stem cell transplant
  • red blood cell engraftment
  • nonmyeloablative
  • pure red cell aplasia

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by University of Calgary on 2026-05-01.