Testing a gentler stem cell transplant for children with sickle cell disease
Tests a lower-toxicity transplant using a sibling donor in children with sickle cell disease.
Plain-language title generated from the registry record
Registry title: Minimizing Toxicity in HLA-identical Sibling Donor Transplantation for Children With Sickle Cell Disease
Key facts
- Where
- Calgary and Toronto, CanadaSee the 2 sites
- Phase
- Phase 2
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 2 and 25 years old?
Source: NCT03587272, who can take part
Where in Canada
- Alberta Children's HospitalCalgary, CanadaTaking people
- The Hospital for Sick ChildrenToronto, CanadaTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Robert Nickel
- Trial code
- NCT03587272
- Record updated
- 10 March 2026
- Original description
This multisite prospective study seeks to determine if HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus (Sickle transplant Using a Nonmyeloablative approach, "SUN") can decrease the toxicity of transplant while achieving a high cure rate for children with sickle cell disease (SCD).Read full original descriptionShow less
Minimizing Toxicity in HLA-identical Sibling Donor Transplantation for Children With Sickle Cell Disease
This multisite prospective study seeks to determine if HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus (Sickle transplant Using a Nonmyeloablative approach, "SUN") can decrease the toxicity of transplant while achieving a high cure rate for children with sickle cell disease (SCD).
Inclusion: Patients with genotypes hemoglobin SS and Sβ0 thalassemia must have at least one of the following:; History of an abnormal transcranial Doppler measurement defined as TCD velocity ≥200 cm/sec by the non-imaging technique (or ≥185 cm/sec by the imaging technique) measured at a minimum of two separate occasions; History of cerebral infarction on brain MRI (overt stroke, or silent stroke if ≥3 mm in one dimension, visible in two planes on fluid-attenuated inversion recovery T2-weighted images); History of two or more episodes of acute chest syndrome (ACS) in lifetime; History of three or more SCD pain events requiring treatment with an opiate or IV pain medication (inpatient or outpatient) in lifetime; History of any hospitalization for SCD pain or ACS while receiving hydroxyurea treatment; History of two or more episodes of priapism (erection lasting ≥4 hours or requiring emergent medical care); Administration of regular RBC transfusions (≥8 transfusions in the previous 12 months); At least two episodes of splenic sequestration requiring red blood cell transfusion or splenectomy after at least one episode of splenic sequestration; Patients with all other sickle genotypes (hemoglobin SC, Sβ+ thalassemia) must have at least one of the following:; Clinically significant neurologic event (overt stroke); History of two or more episodes of ACS in the 2-years period preceding enrollment; History of three or more SCD pain events requiring treatment with an opiate or IV pain medication (inpatient or outpatient) in the 1-year period preceding enrollment; History of any hospitalization for SCD pain or ACS while receiving hydroxyurea treatment; History of two or more episodes of priapism (erection lasting ≥4 hours or requiring emergent medical care); Administration of regular RBC transfusions (≥8 transfusions in the previous 12 months); At least two episodes of splenic sequestration requiring red blood cell transfusion or splenectomy after at least one episode of splenic sequestration.
Exclusion: Infection Disease: Uncontrolled bacterial, viral or fungal infections (undergoing appropriate treatment and with progression of clinical symptoms) within 1 month prior to conditioning. Patients with febrile illness or suspected minor infection should await clinical resolution prior to starting conditioning. Patients with confirmed seropositivity for HIV and patients with active Hepatitis B or C determined by serology and/or NAAT are excluded; Heme: History of RBC alloantibodies against donor RBC antigens (even if current antibody screen is negative). Major ABO incompatibility with donor; Liver: Direct (conjugated) bilirubin > 1.5 mg/dL, transaminases >5x upper limit of normal for age; Cardiac: Left ventricular shortening fraction <25% or ejection fraction <50% by ECHO; Kidney: Estimated creatinine clearance less than 60 mL/min/1.73m2; Pulmonary function: Diffusion capacity of carbon monoxide (DLCO) <35% (adjusted for hemoglobin). Baseline oxygen saturation <85% or PaO2 <70; General: Life expectancy less than 6 months. Pregnant or breastfeeding patients.