Comparing stem cell transplantation to disease-modifying therapy for children with sickle cell disease

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

The WeDecide study is an observational study that compares the long-term effects of matched related donor hematopoietic stem cell transplantation (MRD HCT) and non-transplant disease-modifying therapies (NT-DMT) in pediatric patients with sickle cell disease (SCD). It aims to assess health-related quality of life, cognitive function, and the risks and benefits of both treatments over a three-year period. The study will involve 160 children receiving MRD HCT and 320 children receiving NT-DMT, with evaluations conducted at multiple time points to track health outcomes and treatment impacts. Additionally, it will consider factors such as disease severity and social determinants of health to provide a comprehensive understanding of treatment effects.

Why it matters

Who can join: the full rules

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.

Where it's running

• Children's of Alabama (MRD-HCT)
  Birmingham, Alabama, United States Recruiting
• Nemours Children's Hospital, Delaware (MRD-HCT)
  Wilmington, Delaware, United States Recruiting
• Children's National Hospital (MRD-HCT)
  Washington D.C., District of Columbia, United States Recruiting
• Children's Healthcare of Atlanta (MRD-HCT)
  Atlanta, Georgia, United States Recruiting
• Comer Children's Hospital (MRD-HCT)
  Chicago, Illinois, United States Recruiting
• Riley Children's Hospital (MRD-HCT)
  Indianapolis, Indiana, United States Recruiting

Study contacts

• Study coordinator: John Horan, MD, MPH
• Email: John_Horan@URMC.Rochester.edu
• Phone: 5852742345

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Point-of-care newborn SCD screening and early care in Côte d'Ivoire

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• Sickle Cell Kidney Sample Bank

  This project will collect blood and urine from adults with sickle cell disease or sickle cell trait to see if linked samples and medical records can help researchers learn how kidney disease starts and progresses.

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• Examining megakaryocytes in patients with sickle cell disease

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• Depo-Provera (depot medroxyprogesterone acetate) for pain and biomarker changes in women with sickle cell disease

  This study will test whether an injectable birth control (Depo-Provera) can reduce pain in adult women with sickle cell disease.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.