Clinical trial NCT06941389: Comparing the Effectiveness of Matched Related Donor Hematopoietic Stem Cell Transplantation to Disease Modifying Therapy in Pediatric Patients With Sickle Cell Disease
Official title: Comparing the Effectiveness of Matched Related Donor Hematopoietic Stem Cell Transplantation to Disease Modifying Therapy in Pediatric Patients With Sickle Cell Disease.
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Sickle Cell Disease (SCD)
Keywords: SCD
MRD HCT
NT DMT
Lead sponsor: University of Rochester
Sponsor class: OTHER
Collaborators: name: Patient-Centered Outcomes Research Institute
First posted: 2025-04-23
Enrollment: 480
Sex: ALL
Minimum age (years): 3
Maximum age (years): 20
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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.

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.

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.

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 with significant comorbidities or other health conditions that would interfere with the study's outcomes.
* 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.

Primary outcomes:
measure: Health-Related Quality of Life as measured by the Pediatric Quality of Life Inventory (PedsQL) Generic Core Scale at Baseline and Follow-Up Time Points
timeFrame: Baseline, 1-year, 2-year, 3-year follow-up
measure: Health-Related Quality of Life as Measured by PROMIS Pediatric Profile 25 (Self-Report and Parent-Proxy Report) at Baseline and Follow-Up Time Points
timeFrame: Baseline, 1-year, 2-year, 3-year follow-up
measure: Cognitive Function as Measured by the NIH Toolbox Cognitive Battery at Baseline and Follow-Up Time Points
timeFrame: Baseline, 1-year, 2-year, 3-year follow-up
Secondary outcomes:
measure: Perceived Discrimination as Measured by the Everyday Discrimination Scale at Baseline and Follow-Up Time Points
timeFrame: Baseline, 1-year, 2-year, 3-year follow-up
measure: Financial Wellness as Measured by the Personal Financial Wellness Survey at Baseline and Follow-Up Time Points
timeFrame: Baseline, 1-year, 2-year, 3-year follow-up
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama (MRD-HCT)
state: Alabama
city: Birmingham
country: United States
facility: University of Alabama (NT-DMT)
state: Alabama
city: Wilmington
country: United States
facility: Nemours Children's Hospital (MRD-HCT)
state: Delaware
city: Washington
country: United States
facility: Children's National Hospital (MRD-HCT)
state: District of Columbia
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta (MRD-HCT)
state: Georgia
city: Chicago
country: United States
facility: Comer Children's Hospital (MRD-HCT)
state: Illinois
city: Indianapolis
country: United States
facility: Riley Children's Hospital (MRD-HCT)
state: Indiana
city: Indianapolis
country: United States
facility: Riley's Children Hospital (NT-DMT)
state: Indiana
city: Boston
country: United States
facility: Boston Children's Hospital (MRD-HCT)
state: Massachusetts
city: Boston
country: United States
facility: Boston Children's Hospital (NT-DMT)
state: Massachusetts
city: Saint Louis
country: United States
facility: Wahington Univ in St Louis (NT-DMT)
state: Missouri
city: Hackensack
country: United States
facility: Hackensack University Hospital (MRD-HCT)
state: New Jersey
city: Bronx
country: United States
facility: Children's Hospital at Montefiore (MRD HCT)
state: New York
city: Bronx
country: United States
facility: Children's Hospital at Montefiore (NT-DMT)
state: New York
city: Buffalo
country: United States
facility: Roswell Park (MRD-HCT)
state: New York
city: New York
country: United States
facility: Columbia Presbytarian (NT-DMT)
state: New York
city: Queens
country: United States
facility: Cohen's Children Hospital (NT-DMT)
state: New York
city: Rochester
country: United States
facility: University of Rochester (MRD-HCT)
state: New York
city: Rochester
country: United States
facility: University of Rochester (NT-DMT)
state: New York
city: Chapel Hill
country: United States
facility: UNC Chapel Hill (NT-DMT)
state: North Carolina
city: Charlotte
country: United States
facility: Atrium Health (MRD-HCT)
state: North Carolina
city: Columbus
country: United States
facility: Nationwide Children's Hospital (NT-DMT)
state: Ohio
city: Oklahoma City
country: United States
facility: Oklahoma Children's Hospital (MRD-HCT)
state: Oklahoma
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia (MRD-HCT)
state: Pennsylvania
city: Memphis
country: United States
facility: St Jude Children Hospital (NT-DMT)
state: Tennessee
city: Memphis
country: United States
facility: St. Jude Children's Research Hospital (MRD-HCT)
state: Tennessee
city: Houston
country: United States
facility: Texas Children's Hopsital (NT-DMT)
state: Texas
city: Houston
country: United States
facility: Texas Children's Hospital (MRD-HCT)
state: Texas
city: San Antonio
country: United States
facility: UT San Antonio (NT-DMT)
state: Texas
city: Calgary
country: Canada
facility: Alberta Children's Hospital (NT-DMT)
state: Alberta
city: Calgary
country: Canada
facility: Alberta Children's Hospital (MRD-HCT)
state: Alberta
city: Winnipeg
country: Canada
facility: Children's Hospital of Winnipeg (MRD-HCT)
state: Manitoba
city: Toronto
country: Canada
facility: The Hospital for Sick Children (MRD-HCT)
state: Ontario
Study officials:
References: