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Sickle Cell Disease (SCD)WeDecideNCT06941389

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

An observational study of Sickle Cell Disease (SCD), run by University of Rochester at 37 sites in 2 countries.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial summary

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). Read more about this study

Who is it for?
Ages 3 to 20 years
Conditions the study is for
Sickle Cell Disease (SCD)
How many people
480 participants
Where
37 sitesin 2 countries
Study dates
1 Jun 2024to 1 Oct 2030
Phase
Observational
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 3 to 20 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

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.

Who may be able to join

  • 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.

Who cannot join

  • 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.

Trial locations and contacts

37 sites in 2 countries. Contact the study team or a site near you to ask about taking part.

John Horan, MD, MPH contact

5852742345

John_Horan@URMC.Rochester.edu

Savannah Pangrazio, BS contact

5852750932

Savannah_Pangrazio@URMC.Rochester.edu

37 sites in 2 countries

United States31 sites, 31 recruiting

  • Children's Healthcare of Atlanta (MRD-HCT)

    Atlanta, Georgia, 30329

    Judith Talib, 4047854075, judith.talib@choa.org

    Ann Haight

    Recruiting

  • Children's of Alabama (MRD-HCT)

    Birmingham, Alabama, 35294

    Jana Hilburn, (205) 6389324, jiqal@uabmc.edu

    Hilary Haines

    Recruiting

  • Children's of Alabama (NT-DMT)

    Birmingham, Alabama, 35294

    Jeanine Dumas, (205) 638-9100, jdumas@uabmc.edu

    Jeff Lebensburger

    Not yet recruiting

  • Boston Children's Hospital (MRD-HCT)

    Boston, Massachusetts, 02115

    Lydia L Osuagwu, 617-355-6000, Lydial_Osuagwu@dfci.harvard.edu

    Lev Gorfinkel

    Recruiting

  • Boston Children's Hospital (NT-DMT)

    Boston, Massachusetts, 02115

    Nadia Tarhini, 617-355-6000, Nadia.Tarhini@childrens.harvard.edu

    Natasha Archer

    Not yet recruiting

  • Roswell Park (MRD-HCT)

    Buffalo, New York, 14203

    Stephanie Guilford, 716-845-1300 ext. 1898, stephanie.guilford@roswellpark.org

    Kanwaldeep Mallhi

    Recruiting

  • UNC Children's Hospital (MRD HCT)

    Chapel Hill, North Carolina, 27514

    Juanita Cufee, 919-966-1178, juanita_cuffee@med.unc.edu

    Kim Kasow

    Not yet recruiting

  • UNC Children's Hospital (NT-DMT)

    Chapel Hill, North Carolina, 27599.

    Lindsey Akpuogu, (919) 962-2211, lakpuog@email.unc.edu

    Maria Boucher

    Not yet recruiting

  • Atrium Health (MRD-HCT)

    Charlotte, North Carolina, 28203

    Marithza Amaya, 9804422309, Marithza.Amaya@atriumhealth.org

    Michael Kent

    Recruiting

  • Comer Children's Hospital (MRD-HCT)

    Chicago, Illinois, 60637

    Christopher Omahen, 7737028137, comahen@bsd.uchicago.edu

    James LaBelle

    Recruiting

  • Nationwide Children's Hospital (NT-DMT)

    Columbus, Ohio, 43205

    Anthony Villela, (614) 722-2000, anthony.villella@nationwidechildrens.org

    Anthony Villela

    Not yet recruiting

  • Hackensack University Hospital (MRD-HCT)

    Hackensack, New Jersey, 07601

    Elana Smilow, (551) 996-5673, elana.smilow@hmhn.org

    Christine Camacho-Bydume

    Recruiting

  • Texas Children's Hopsital (NT-DMT)

    Houston, Texas, 77030

    Daniela Westerhold, (832) 824-1000, diwester@texaschildrens.org

    Venee Tubman

    Not yet recruiting

  • Texas Children's Hospital (MRD-HCT)

    Houston, Texas, 77030

    Helton D. Cruz Rivas, 832-824-1798, hdcruz@texaschildrens.org

    Khaled Yassine

    Recruiting

  • Riley Children's Hospital (MRD-HCT)

    Indianapolis, Indiana, 46202

    Courtney Spiegel, 3172783105, clorch@iu.edu

    Jodi Skiles

    Recruiting

  • Riley's Children Hospital (NT-DMT)

    Indianapolis, Indiana, 46202

    Jillian Bouck, (317) 944-5000, jrbouck@iu.edu

    Seethal Jacobs

    Not yet recruiting

  • St Jude Children Hospital (NT-DMT)

    Memphis, Tennessee, 38105

    Jason Hodges, 866 278-5833, Jason.Hodges@STJUDE.ORG

    Cliff Takemoto

    Not yet recruiting

  • St. Jude Children's Research Hospital (MRD-HCT)

    Memphis, Tennessee, 38105

    Stacy High, 9012336555, stacy.high@stjude.org

    Akshay Sharma

    Recruiting

  • Columbia Presbytarian (NT-DMT)

    New York, New York, 10032

    Nikhil Punwani, (212) 305-5437, np2927@cumc.columbia.edu

    Nancy Green

    Not yet recruiting

  • Oklahoma Children's Hospital (MRD-HCT)

    Oklahoma City, Oklahoma, 73104

    Christina Gonzalez, (405) 271-4412 ext. 42510, Christina-Gonzalez@ouhsc.edu

    Laura Rooms

    Not yet recruiting

  • Children's Hospital of Philadelphia (MRD-HCT)

    Philadelphia, Pennsylvania, 19104

    Peter Nicholas, 2674269376, nicholasp@chop.edu

    Tim Olson

    Recruiting

  • Cohen's Children Hospital (NT-DMT)

    Queens, New York, 11040

    Ferdausi Shifath, (833) 462-6436, fshifath@northwell.edu

    Banu Aygun

    Not yet recruiting

  • University of Rochester (MRD-HCT)

    Rochester, New York, 14642

    Savannah Pangrazio, 585-275-0932, Savannah_Pangrazio@URMC.Rochester.edu

    John Horan

    Not yet recruiting

  • University of Rochester (NT-DMT)

    Rochester, New York, 14642

    Savannah Pangrazio, 585-275-0932, Savannah_Pangrazio@URMC.Rochester.edu

    Suzie Noronha

    Not yet recruiting

  • UT San Antonio (NT-DMT)

    San Antonio, Texas, 78229

    Sophia Isabel Salinas, 210-358-4000, salinass9@uthscsa.edu

    Melissa Frei Jones

    Not yet recruiting

  • Wahington Univ in St Louis (NT-DMT)

    St Louis, Missouri, 63130

    Bethany Kellogg, (314) 935-5000, bethany.kellogg@wustl.edu

    Melanie Fields

    Not yet recruiting

  • Washington University in St Louis (MRD-HCT)

    St Louis, Missouri, 63130

    Ian Snyder, 314-273-5953, ian.s@wustl.edu

    Shalini Shenoy

    Not yet recruiting

  • Children's Hospital at Montefiore (MRD HCT)

    The Bronx, New York, 10467

    Elaine Chung, 7187412396, elchung@montefiore.org

    Mahvish Rahim

    Not yet recruiting

  • Children's Hospital at Montefiore (NT-DMT)

    The Bronx, New York, 10467

    Karen Ireland, (718) 741-2426, KIRELAND@montefiore.org

    Kaitlin Strumph

    Recruiting

  • Children's National Hospital (MRD-HCT)

    Washington D.C., District of Columbia, 20010

    Julia Malloy, 2024764225, JMALLOY@childrensnational.org

    Robert Nickel

    Recruiting

  • Nemours Children's Hospital, Delaware (MRD-HCT)

    Wilmington, Delaware, 19803

    Megan Petrick, (302) 651-5584, Megan.Petrick@nemours.org

    Emi Caywood

    Recruiting

Other trials in United States

Canada6 sites, 6 recruiting

  • Alberta Children's Hospital (NT-DMT)

    Calgary, Alberta, T3B 6A8

    Alex Fidanova, 403-955-7211, Alex.Fidanova@albertahealthservices.ca

    Gurpreet Singh

    Not yet recruiting

  • Alberta Children's Hospital (MRD-HCT)

    Calgary, Alberta, T3B6A8

    Alex Fidanova, 403-955-7211, Alex.Fidanova@albertahealthservices.ca

    Greg Guilcher

    Recruiting

  • Universite de Montreal / Ste Justine (MRD HCT)

    Montreal, Quebec

    Karine Leveille, 514 345-4830, karine.leveille.hsj@ssss.gouv.qc.ca

    Michael Duval

    Not yet recruiting

  • The Hospital for Sick Children (MRD-HCT)

    Toronto, Ontario, ON M5G 1E8

    Erilda Kapllani, 416-813-7654 ext. 424714, trixia.luzon@sickkids.ca

    Muhammad Ali

    Not yet recruiting

  • British Columbia Children's Hosptial (MRD HCT)

    Vancouver, British Columbia

    Peter Subrt, 604-875-2161, psubrt@cw.bc.ca

    Meera Rayar

    Not yet recruiting

  • HSC Winnipeg Children's Hospital/University of Manitoba (MRD-HCT)

    Winnipeg, Manitoba, R3E 0V9

    Kim Shore, (204) 787-4850, kshore@cancercare.mb.ca

    Ashley Chopek

    Recruiting

Other trials in Canada

About this study

Official title: 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.

Read the 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.

Keywords
SCD, MRD HCT, NT DMT
Medical subject headings
Anemia, Sickle Cell

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Study groups in NCT06941389
Study groupRoleWhat they receive
NT-DMT CohortThis 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 CohortThis 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 being measured

The primary outcome is the main question the study is designed to answer.

  1. Health-Related Quality of Life as Measured by the Pediatric Quality of Life Inventory (PedsQL) Generic Core Scale (Parent-Proxy Report)

    Measures physical, emotional, social, and school functioning as reported by parents.

    Measured: Baseline, 1-year, 2-year, 3-year follow-up

  2. Cognitive Function as Measured by the NIH Toolbox Cognitive Battery

    Assesses the following cognitive domains: attention, executive function, memory, language, processing speed, and working memory.

    Measured: Baseline, 1-year, 2-year, 3-year follow-up

Secondary outcomes (7)

  1. Health-Related Quality of Life as Measured by the Pediatric Quality of Life Inventory (PedsQL) Generic Core Scale (Patient Reported)

    Description: Assesses self-reported physical, emotional, social, and school functioning (for age-eligible participants).

    Measured: Baseline, 1-year, 2-year, 3-year follow-up

  2. Health Related Quality of Life Using the PedsQL Sickle Cell Disease Module (Patient and Parent-Proxy Reported)

    Description: The Peds QL Sickle Cell Module assesses pain and hurt, pain impact, pain management and control, worry, emotions, treatment and communication.

    Measured: Baseline, 1-year, 2-year, 3-year follow-up

  3. Peds QL Family Impact Module (Parent Reported)

    Assess the burden a parent experiences. It assesses: Physical, Emotional, Social and Cognitive functioning; communication, worry, family daily activities, and family relationships.

    Measured: Baseline, 1-, 2- and 3-year follow-up

  4. Health Quality of Life as Measured by the Patient Reported Outcomes Measurement Information System (PROMIS, Patient and Parent-Proxy Reports)

    Assesses physical, mental and social health

    Measured: Baseline, 1-year, 2-year, 3-year follow-up

  5. The Patient Reported Outcomes Measure System (PROMIS) Pain Behavior subscale (Patient and Parent-Proxy Reports)

    Measured: Baseline, 1-year, 2-year and 3-year follow-up

  6. Number of Acute Care Visits (ED, Hospitalizations, Infusion/Clinic)

    Unit of Measure: Number of Visits per Year Description: Annual count of emergency, inpatient, or infusion center visits for acute care.

    Measured: Time Points: Year 1, Year 2, Year 3

  7. Incidence and Severity of Chronic Graft-Versus-Host Disease

    Unit of Measure: Number of Participants with GVHD by Severity Description: Reports the GVHD occurrence in transplant recipients with severity grading.

    Measured: Day 100, 6 months, Year 1, Year 2, Year 3

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted23 Apr 2025
  2. Study start1 Jun 2024
  3. Primary completion1 Oct 2030 (estimated)
  4. Study completion1 Oct 2030 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Observational
Observational model
Cohort
Time perspective
Prospective
Enrolment
480 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06941389
Sponsor's protocol ID
BPS-2023C1-31041
Other IDs
BPS-2023C1-31041
Acronym
WeDecide
Status last verified
Jun 2026
First submitted
15 Apr 2025
Last updated
4 Jun 2026
Results posted
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

An observational study of Sickle Cell Disease (SCD), run by University of Rochester at 37 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 4 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 37 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact John Horan, MD, MPH (5852742345, John_Horan@URMC.Rochester.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06941389.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Jun 2026.

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