Comparing transplant and standard treatment for children with sickle cell disease
Compares stem cell transplant with ongoing disease-modifying medicines in children with sickle cell disease.
Plain-language title generated from the registry record
Registry title: Comparing the Effectiveness of Matched Related Donor Hematopoietic Stem Cell Transplantation to Disease Modifying Therapy in Pediatric Patients With Sickle Cell Disease
Key facts
- Where
- Calgary, Montreal, Toronto, Vancouver, and Winnipeg, CanadaSee the 6 sites
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 3 and 20 years old?
Source: NCT06941389, who can take part
Where in Canada
- Alberta Children's Hospital (MRD-HCT)Calgary, CanadaTaking people
- Alberta Children's Hospital (NT-DMT)Calgary, CanadaNot yet recruiting
- Universite de Montreal / Ste Justine (MRD HCT)Montreal, CanadaNot yet recruiting
- The Hospital for Sick Children (MRD-HCT)Toronto, CanadaNot yet recruiting
- British Columbia Children's Hosptial (MRD HCT)Vancouver, CanadaNot yet recruiting
- HSC Winnipeg Children's Hospital/University of Manitoba (MRD-HCT)Winnipeg, 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
- University of Rochester
- Trial code
- NCT06941389
- Record updated
- 4 June 2026
- Original description
The WeDecide study is a large observational study comparing the long-term effects of matched related donor hematopoietic stem cell transplantation (MRD HCT) and non-transplant disease-modifying therapies (NT-DMT) for pediatric patients with sickle cell disease (SCD). The study aims to assess health-related quality of life (HRQoL), cognitive function, risks, and benefits of both treatments, including survival rates, chronic complications, and organ damage prevention. With 160 children in the MRD HCT group and 320 in the NT-DMT group, aged 3-20.9 years, the study will follow participants for three years, examining factors like disease severity, treatment history, and social determinants of health. By providing a comprehensive comparison, the study seeks to inform clinical decisions and improve understanding of SCD treatment outcomes, ultimately supporting families and healthcare providers in choosing the best treatment options.Read full original descriptionShow less
Comparing the Effectiveness of Matched Related Donor Hematopoietic Stem Cell Transplantation to Disease Modifying Therapy in Pediatric Patients With Sickle Cell Disease.
The WeDecide study is a large observational study comparing the long-term effects of matched related donor hematopoietic stem cell transplantation (MRD HCT) and non-transplant disease-modifying therapies (NT-DMT) for pediatric patients with sickle cell disease (SCD). The study aims to assess health-related quality of life (HRQoL), cognitive function, risks, and benefits of both treatments, including survival rates, chronic complications, and organ damage prevention. With 160 children in the MRD HCT group and 320 in the NT-DMT group, aged 3-20.9 years, the study will follow participants for three years, examining factors like disease severity, treatment history, and social determinants of health. By providing a comprehensive comparison, the study seeks to inform clinical decisions and improve understanding of SCD treatment outcomes, ultimately supporting families and healthcare providers in choosing the best treatment options.
Inclusion: Pediatric patients aged between 3 and 20.9 years; Children diagnosed with sickle cell Anemia (HB SS or HBSB0 Thalassemia); For the MRD HCT group, children who are candidates for matched related donor hematopoietic stem cell transplantation (MRD HCT); For the NT-DMT group, children who are receiving non-transplant disease-modifying therapies (NT-DMT) for SCD; Participants (or their guardians) must provide informed consent to be part of the study; Participants must be willing to undergo the necessary assessments and follow-up visits over the 3-year study period.
Exclusion: For the MRD HCT group, children who are not eligible for the transplant or do not have a matched related donor; Children who do not have sickle cell anemia or related conditions; Children younger than 3 years or older than 20.9 years; Children who are currently enrolled in other clinical trials that might interfere with the WeDecide study; Children who are unable to adhere to the study protocol or follow-up requirements.