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

June 2, 2026 updated by: John Horan, University of Rochester

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.

Study Overview

Status

Recruiting

Conditions

Detailed Description

The WeDecide study is a large observational study comparing the long-term effects of two treatment options for pediatric patients with sickle cell disease (SCD): matched related donor hematopoietic stem cell transplantation (MRD HCT) and non-transplant disease-modifying therapies (NT-DMT). The main goal is to understand how these treatments affect health-related quality of life (HRQoL) and cognitive function, using standard tools to measure both physical and mental health. The study also looks at risks and benefits of MRD HCT, such as the potential for chronic complications, improved survival, and prevention of organ damage.

The study includes two groups: 160 children receiving MRD HCT and 320 children receiving NT-DMT. Participants, aged 3-20.9 years, are being followed for three years. The MRD HCT group will be assessed before the transplant and then at several points post-transplant. The NT-DMT group will be assessed at the start of the study and then annually for three years.

The research also considers factors like disease severity, treatment history, and social determinants of health (such as family finances and caregiver health literacy) to better understand how these elements might influence treatment outcomes. The study tracks the use of disease-modifying therapies, as well as hospital visits and other care events, throughout the three years. It will also monitor survival rates and other important health outcomes.

This study is significant because it is the first large-scale research comparing these two treatment options for SCD in children. The results will provide essential insights into how these treatments impact long-term health and help guide clinical decisions and treatment recommendations. The goal is to help families and healthcare providers make informed decisions about the best treatment options for SCD.

The study uses advanced methods to ensure fair comparisons between the two groups by accounting for differences in their characteristics. It will also adjust for any factors that could influence the results, helping to identify meaningful differences in health outcomes between the two treatments. Ultimately, the WeDecide study aims to improve our understanding of sickle cell disease treatment and provide a foundation for future research into new therapies.

Study Type

Observational

Enrollment (Estimated)

480

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • Canada
    • Alberta
      • Calgary, Alberta, Canada, T3B 6A8
        • Not yet recruiting
        • Alberta Children's Hospital (NT-DMT)
        • Contact:
        • Principal Investigator:
          • Gurpreet Singh
      • Calgary, Alberta, Canada, T3B6A8
    • British Columbia
      • Vancouver, British Columbia, Canada
        • Not yet recruiting
        • British Columbia Children's Hosptial (MRD HCT)
        • Contact:
        • Principal Investigator:
          • Meera Rayar
    • Manitoba
      • Winnipeg, Manitoba, Canada, R3E 0V9
        • Recruiting
        • HSC Winnipeg Children's Hospital/University of Manitoba (MRD-HCT)
        • Principal Investigator:
          • Ashley Chopek
        • Contact:
    • Ontario
      • Toronto, Ontario, Canada, ON M5G 1E8
        • Not yet recruiting
        • The Hospital for Sick Children (MRD-HCT)
        • Principal Investigator:
          • Muhammad Ali
        • Contact:
    • Quebec
      • Montreal, Quebec, Canada
        • Not yet recruiting
        • Universite de Montreal / Ste Justine (MRD HCT)
        • Contact:
        • Principal Investigator:
          • Michael Duval
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35294
        • Recruiting
        • Children's of Alabama (MRD-HCT)
        • Contact:
        • Principal Investigator:
          • Hilary Haines
      • Birmingham, Alabama, United States, 35294
        • Not yet recruiting
        • Children's of Alabama (NT-DMT)
        • Principal Investigator:
          • Jeff Lebensburger
        • Contact:
    • Delaware
      • Wilmington, Delaware, United States, 19803
        • Recruiting
        • Nemours Children's Hospital, Delaware (MRD-HCT)
        • Principal Investigator:
          • Emi Caywood
        • Contact:
    • District of Columbia
      • Washington D.C., District of Columbia, United States, 20010
        • Recruiting
        • Children's National Hospital (MRD-HCT)
        • Principal Investigator:
          • Robert Nickel
        • Contact:
    • Georgia
      • Atlanta, Georgia, United States, 30329
        • Recruiting
        • Children's Healthcare of Atlanta (MRD-HCT)
        • Principal Investigator:
          • Ann Haight
        • Contact:
    • Illinois
      • Chicago, Illinois, United States, 60637
        • Recruiting
        • Comer Children's Hospital (MRD-HCT)
        • Principal Investigator:
          • James LaBelle
        • Contact:
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Recruiting
        • Riley Children's Hospital (MRD-HCT)
        • Principal Investigator:
          • Jodi Skiles
        • Contact:
      • Indianapolis, Indiana, United States, 46202
        • Not yet recruiting
        • Riley's Children Hospital (NT-DMT)
        • Principal Investigator:
          • Seethal Jacobs
        • Contact:
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Not yet recruiting
        • Boston Children's Hospital (NT-DMT)
        • Contact:
        • Principal Investigator:
          • Natasha Archer
      • Boston, Massachusetts, United States, 02115
        • Recruiting
        • Boston Children's Hospital (MRD-HCT)
        • Contact:
        • Principal Investigator:
          • Lev Gorfinkel
    • Missouri
      • St Louis, Missouri, United States, 63130
        • Not yet recruiting
        • Wahington Univ in St Louis (NT-DMT)
        • Principal Investigator:
          • Melanie Fields
        • Contact:
      • St Louis, Missouri, United States, 63130
        • Not yet recruiting
        • Washington University in St Louis (MRD-HCT)
        • Principal Investigator:
          • Shalini Shenoy
        • Contact:
    • New Jersey
      • Hackensack, New Jersey, United States, 07601
        • Recruiting
        • Hackensack University Hospital (MRD-HCT)
        • Principal Investigator:
          • Christine Camacho-Bydume
        • Contact:
    • New York
      • Buffalo, New York, United States, 14203
        • Recruiting
        • Roswell Park (MRD-HCT)
        • Principal Investigator:
          • Kanwaldeep Mallhi
        • Contact:
      • New York, New York, United States, 10032
        • Not yet recruiting
        • Columbia Presbytarian (NT-DMT)
        • Principal Investigator:
          • Nancy Green
        • Principal Investigator:
          • Margaret Lee
        • Contact:
      • Queens, New York, United States, 11040
        • Not yet recruiting
        • Cohen's Children Hospital (NT-DMT)
        • Principal Investigator:
          • Banu Aygun
        • Contact:
      • Rochester, New York, United States, 14642
        • Not yet recruiting
        • University of Rochester (MRD-HCT)
        • Principal Investigator:
          • John Horan
        • Contact:
      • Rochester, New York, United States, 14642
        • Not yet recruiting
        • University of Rochester (NT-DMT)
        • Principal Investigator:
          • Suzie Noronha
        • Contact:
      • The Bronx, New York, United States, 10467
        • Not yet recruiting
        • Children's Hospital at Montefiore (MRD HCT)
        • Contact:
        • Principal Investigator:
          • Mahvish Rahim
      • The Bronx, New York, United States, 10467
        • Recruiting
        • Children's Hospital at Montefiore (NT-DMT)
        • Contact:
        • Principal Investigator:
          • Kaitlin Strumph
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27514
        • Not yet recruiting
        • UNC Children's Hospital (MRD HCT)
        • Contact:
        • Principal Investigator:
          • Kim Kasow
      • Chapel Hill, North Carolina, United States, 27599.
        • Not yet recruiting
        • UNC Children's Hospital (NT-DMT)
        • Contact:
        • Principal Investigator:
          • Maria Boucher
      • Charlotte, North Carolina, United States, 28203
        • Recruiting
        • Atrium Health (MRD-HCT)
        • Principal Investigator:
          • Michael Kent
        • Contact:
    • Ohio
      • Columbus, Ohio, United States, 43205
        • Not yet recruiting
        • Nationwide Children's Hospital (NT-DMT)
        • Principal Investigator:
          • Anthony Villela
        • Contact:
    • Oklahoma
      • Oklahoma City, Oklahoma, United States, 73104
        • Not yet recruiting
        • Oklahoma Children's Hospital (MRD-HCT)
        • Principal Investigator:
          • Laura Rooms
        • Contact:
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Recruiting
        • Children's Hospital of Philadelphia (MRD-HCT)
        • Principal Investigator:
          • Tim Olson
        • Contact:
    • Tennessee
      • Memphis, Tennessee, United States, 38105
        • Not yet recruiting
        • St Jude Children Hospital (NT-DMT)
        • Principal Investigator:
          • Cliff Takemoto
        • Contact:
      • Memphis, Tennessee, United States, 38105
        • Recruiting
        • St. Jude Children's Research Hospital (MRD-HCT)
        • Principal Investigator:
          • Akshay Sharma
        • Contact:
    • Texas
      • Houston, Texas, United States, 77030
        • Not yet recruiting
        • Texas Children's Hopsital (NT-DMT)
        • Principal Investigator:
          • Venee Tubman
        • Contact:
      • Houston, Texas, United States, 77030
        • Recruiting
        • Texas Children's Hospital (MRD-HCT)
        • Principal Investigator:
          • Khaled Yassine
        • Contact:
      • San Antonio, Texas, United States, 78229
        • Not yet recruiting
        • UT San Antonio (NT-DMT)
        • Contact:
        • Principal Investigator:
          • Melissa Frei Jones

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

The WeDecide study includes 480 pediatric patients with sickle cell disease (SCD) from two sub-studies: Project Sickle Cure (PSC), focusing on patients receiving transplant through the Sickle Cell Transplant Advocacy Research Alliance (STAR), and the Globin Research Network for Data and Discovery (GRNDaD), a registry of patients on non-transplant disease-modifying therapies (NT-DMT) run by the National Association of Sickle Cell Centers (NASCC).

MRD HCT Group: 160 children receiving matched related donor hematopoietic stem cell transplantation (MRD HCT) will be assessed before and at multiple post-transplant time points to evaluate long-term effects.

NT-DMT Group: 320 children receiving non-transplant disease-modifying therapies (NT-DMT) will be assessed at study start and annually for three years to track treatment effects.

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
NT-DMT Cohort
This group includes 320 participants from U.S. and Canadian sites receiving non-transplant disease-modifying therapies, monitored annually over three years to assess health-related quality of life, cognitive function, disease progression, and healthcare utilization.
MRD HCT Cohort
This group includes 160 participants from U.S. and Canadian sites receiving non-transplant disease-modifying therapies, monitored annually over three years to assess health-related quality of life, cognitive function, disease progression, and healthcare utilization.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Health-Related Quality of Life as Measured by the Pediatric Quality of Life Inventory (PedsQL) Generic Core Scale (Parent-Proxy Report)
Time Frame: Baseline, 1-year, 2-year, 3-year follow-up
Measures physical, emotional, social, and school functioning as reported by parents.
Baseline, 1-year, 2-year, 3-year follow-up
Cognitive Function as Measured by the NIH Toolbox Cognitive Battery
Time Frame: Baseline, 1-year, 2-year, 3-year follow-up
Assesses the following cognitive domains: attention, executive function, memory, language, processing speed, and working memory.
Baseline, 1-year, 2-year, 3-year follow-up

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Health-Related Quality of Life as Measured by the Pediatric Quality of Life Inventory (PedsQL) Generic Core Scale (Patient Reported)
Time Frame: Baseline, 1-year, 2-year, 3-year follow-up
Description: Assesses self-reported physical, emotional, social, and school functioning (for age-eligible participants).
Baseline, 1-year, 2-year, 3-year follow-up
Health Related Quality of Life Using the PedsQL Sickle Cell Disease Module (Patient and Parent-Proxy Reported)
Time Frame: Baseline, 1-year, 2-year, 3-year follow-up
Description: The Peds QL Sickle Cell Module assesses pain and hurt, pain impact, pain management and control, worry, emotions, treatment and communication.
Baseline, 1-year, 2-year, 3-year follow-up
Peds QL Family Impact Module (Parent Reported)
Time Frame: Baseline, 1-, 2- and 3-year follow-up
Assess the burden a parent experiences. It assesses: Physical, Emotional, Social and Cognitive functioning; communication, worry, family daily activities, and family relationships.
Baseline, 1-, 2- and 3-year follow-up
Health Quality of Life as Measured by the Patient Reported Outcomes Measurement Information System (PROMIS, Patient and Parent-Proxy Reports)
Time Frame: Baseline, 1-year, 2-year, 3-year follow-up
Assesses physical, mental and social health
Baseline, 1-year, 2-year, 3-year follow-up
The Patient Reported Outcomes Measure System (PROMIS) Pain Behavior subscale (Patient and Parent-Proxy Reports)
Time Frame: Baseline, 1-year, 2-year and 3-year follow-up
Baseline, 1-year, 2-year and 3-year follow-up
Number of Acute Care Visits (ED, Hospitalizations, Infusion/Clinic)
Time Frame: Time Points: Year 1, Year 2, Year 3
Unit of Measure: Number of Visits per Year Description: Annual count of emergency, inpatient, or infusion center visits for acute care.
Time Points: Year 1, Year 2, Year 3
Incidence and Severity of Chronic Graft-Versus-Host Disease
Time Frame: Day 100, 6 months, Year 1, Year 2, Year 3
Unit of Measure: Number of Participants with GVHD by Severity Description: Reports the GVHD occurrence in transplant recipients with severity grading.
Day 100, 6 months, Year 1, Year 2, Year 3

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University of Rochester

Collaborators

Patient-Centered Outcomes Research Institute

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 1, 2024

Primary Completion (Estimated)

October 1, 2030

Study Completion (Estimated)

October 1, 2030

Study Registration Dates

First Submitted

April 15, 2025

First Submitted That Met QC Criteria

April 15, 2025

First Posted (Actual)

April 23, 2025

Study Record Updates

Last Update Posted (Actual)

June 4, 2026

Last Update Submitted That Met QC Criteria

June 2, 2026

Last Verified

June 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • BPS-2023C1-31041 (Other Grant/Funding Number: PCORI)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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