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Comparing the Effectiveness of Matched Related Donor Hematopoietic Stem Cell Transplantation to Disease Modifying Therapy in Pediatric Patients With Sickle Cell Disease

Comparing the Effectiveness of Matched Related Donor Hematopoietic Stem Cell Transplantation to Disease Modifying Therapy in Pediatric Patients With Sickle Cell Disease.

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
3 Years to 20 Years
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

  • Time periods mentioned: 3 years

Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.

Show the study's own words
  • "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."
  • "Participants, aged 3-20.9 years, are being followed for three years."
  • "The NT-DMT group will be assessed at the start of the study and then annually for three years."
Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

The WeDecide study compares stem cell transplants from a matched family donor with treatments that do not use a transplant for sickle cell disease. It is for children and young adults ages 3 to 20.9 with sickle cell anemia. Researchers will follow them for 3 years to compare quality of life, thinking skills, and long-term health risks and benefits.

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.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Be 3 to 20.9 years old.
  • Have sickle cell anemia of the Hb SS or HbS beta-zero thalassemia type.
  • For the transplant group, be able to receive a stem cell transplant from a matched family donor.
  • For the non-transplant group, be receiving treatment for sickle cell disease that does not use a transplant.
  • You or your guardian must give informed consent, meaning agreement after learning about the study.
  • Be willing to complete study tests and follow-up visits for 3 years.

Who can't join

  • Be younger than 3 or older than 20.9 years.
  • Not have sickle cell anemia or a related condition.
  • For the transplant group, be unable to receive a transplant or not have a matched family donor.
  • Be in another clinical trial that could interfere with this study.
  • Be unable to follow the study rules or complete follow-up visits.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility criteria

Inclusion Criteria: * 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 Criteria: * Children younger than 3 years or older than 20.9 years. * Children who do not have sickle cell anemia or related conditions. * For the MRD HCT group, children who are not eligible for the transplant or do not have a matched related donor. * 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.

Study sites (31)

AL

DC

DE

GA

IL

IN

MA

MO

NC

NJ

NY

OH

OK

PA

TN

TX