Recruiting

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

Sponsor:

University of Rochester

Code:

NCT06941389

Conditions

Sickle Cell Disease (SCD)

Eligibility Criteria

Sex: All

Age: 3 - 20

Healthy Volunteers: Not accepted

Study Details

Brief 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). 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.

Conditions

Sickle Cell Disease (SCD)

Study ID

NCT06941389

Start date

Jun 1, 2024

Status verified date

Jun, 2026

Completion date

Oct 1, 2030

Anticipated

Primary completion date

Oct 1, 2030

Anticipated

Eligibility Criteria

Sex: All

Age: 3 - 20

Healthy Volunteers: Not accepted

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 Design

Enrollment

480 participants

Anticipated

Interventions and Outcome Measures

Arms

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.

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 ]
Cognitive Function as Measured by the NIH Toolbox Cognitive Battery [ Time Frame: Baseline, 1-year, 2-year, 3-year follow-up ]

Central Contacts and Locations

Central contacts

John Horan, MD, MPH

5852742345 John_Horan@URMC.Rochester.edu

Savannah Pangrazio, BS

5852750932 Savannah_Pangrazio@URMC.Rochester.edu

Locations

Children's of Alabama (MRD-HCT)

Recruiting

Birmingham, Alabama, United States, 35294

Contacts

Jana Hilburn

(205) 6389324 jiqal@uabmc.edu

Principal Investigator:

Hilary Haines

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

Recruiting

Wilmington, Delaware, United States, 19803

Contacts

Megan Petrick

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

Principal Investigator:

Emi Caywood

Children's National Hospital (MRD-HCT)

Recruiting

Washington D.C., District of Columbia, United States, 20010

Contacts

Julia Malloy

2024764225 JMALLOY@childrensnational.org

Principal Investigator:

Robert Nickel

Children's Healthcare of Atlanta (MRD-HCT)

Recruiting

Atlanta, Georgia, United States, 30329

Contacts

Judith Talib

4047854075 judith.talib@choa.org

Principal Investigator:

Ann Haight

Comer Children's Hospital (MRD-HCT)

Recruiting

Chicago, Illinois, United States, 60637

Contacts

Christopher Omahen

7737028137 comahen@bsd.uchicago.edu

Principal Investigator:

James LaBelle

Riley Children's Hospital (MRD-HCT)

Recruiting

Indianapolis, Indiana, United States, 46202

Contacts

Courtney Spiegel

3172783105 clorch@iu.edu

Principal Investigator:

Jodi Skiles

Boston Children's Hospital (MRD-HCT)

Recruiting

Boston, Massachusetts, United States, 02115

Contacts

Lydia L Osuagwu

617-355-6000 Lydial_Osuagwu@dfci.harvard.edu

Principal Investigator:

Lev Gorfinkel

Hackensack University Hospital (MRD-HCT)

Recruiting

Hackensack, New Jersey, United States, 07601

Contacts

Elana Smilow

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

Principal Investigator:

Christine Camacho-Bydume

Roswell Park (MRD-HCT)

Recruiting

Buffalo, New York, United States, 14203

Contacts

Stephanie Guilford

716-845-1300 stephanie.guilford@roswellpark.org

Principal Investigator:

Kanwaldeep Mallhi

Children's Hospital at Montefiore (NT-DMT)

Recruiting

The Bronx, New York, United States, 10467

Contacts

Karen Ireland

(718) 741-2426 KIRELAND@montefiore.org

Principal Investigator:

Kaitlin Strumph

Atrium Health (MRD-HCT)

Recruiting

Charlotte, North Carolina, United States, 28203

Contacts

Marithza Amaya

9804422309 Marithza.Amaya@atriumhealth.org

Principal Investigator:

Michael Kent

Children's Hospital of Philadelphia (MRD-HCT)

Recruiting

Philadelphia, Pennsylvania, United States, 19104

Contacts

Peter Nicholas

2674269376 nicholasp@chop.edu

Principal Investigator:

Tim Olson

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

Recruiting

Memphis, Tennessee, United States, 38105

Contacts

Stacy High

9012336555 stacy.high@stjude.org

Principal Investigator:

Akshay Sharma

Texas Children's Hospital (MRD-HCT)

Recruiting

Houston, Texas, United States, 77030

Contacts

Helton D. Cruz Rivas

832-824-1798 hdcruz@texaschildrens.org

Principal Investigator:

Khaled Yassine

Alberta Children's Hospital (MRD-HCT)

Recruiting

Calgary, Alberta, Canada, T3B6A8

Contacts

Alex Fidanova

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

Principal Investigator:

Greg Guilcher

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

Recruiting

Winnipeg, Manitoba, Canada, R3E 0V9

Contacts

Kim Shore

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

Principal Investigator:

Ashley Chopek

More Information

Sponsor

University of Rochester

Last update posted

Jun 4, 2026

Last verified

Jun, 2026

Keywords

SCD
MRD HCT
NT DMT

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by University of Rochester on 2026-06-04.