RECRUITING

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

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

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

Quick Facts

Study Start:2024-06-01
Study Completion:2030-12-01
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT06941389

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.

Ages Eligible for Study:3 Years to 20 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT
Inclusion CriteriaExclusion Criteria
  1. * Pediatric patients aged between 3 and 20.9 years.
  2. * Children diagnosed with sickle cell Anemia (HB SS or HBSB0 Thalassemia)
  3. * For the MRD HCT group, children who are candidates for matched related donor hematopoietic stem cell transplantation (MRD HCT).
  4. * For the NT-DMT group, children who are receiving non-transplant disease-modifying therapies (NT-DMT) for SCD.
  5. * Participants (or their guardians) must provide informed consent to be part of the study.
  6. * Participants must be willing to undergo the necessary assessments and follow-up visits over the 3-year study period.
  1. * Children younger than 3 years or older than 20.9 years.
  2. * Children with significant comorbidities or other health conditions that would interfere with the study's outcomes.
  3. * Children who do not have sickle cell anemia or related conditions.
  4. * For the MRD HCT group, children who are not eligible for the transplant or do not have a matched related donor.
  5. * Children who are currently enrolled in other clinical trials that might interfere with the WeDecide study.
  6. * Children who are unable to adhere to the study protocol or follow-up requirements.

Contacts and Locations

Study Contact

John Horan, MD, MPH
CONTACT
5852742345
John_Horan@URMC.Rochester.edu
Priya Kaushal, MPH
CONTACT
5852750932
Priya_Kaushal@URMC.Rochester.edu

Study Locations (Sites)

University of Alabama (MRD-HCT)
Birmingham, Alabama, 35294
United States
University of Alabama (NT-DMT)
Birmingham, Alabama, 35294
United States
Nemours Children's Hospital (MRD-HCT)
Wilmington, Delaware, 19803
United States
Children's National Hospital (MRD-HCT)
Washington, District of Columbia, 20010
United States
Children's Healthcare of Atlanta (MRD-HCT)
Atlanta, Georgia, 30329
United States
Comer Children's Hospital (MRD-HCT)
Chicago, Illinois, 60637
United States
Riley Children's Hospital (MRD-HCT)
Indianapolis, Indiana, 46202
United States
Riley's Children Hospital (NT-DMT)
Indianapolis, Indiana, 46202
United States
Boston Children's Hospital (MRD-HCT)
Boston, Massachusetts, 02115
United States
Boston Children's Hospital (NT-DMT)
Boston, Massachusetts, 02115
United States
Wahington Univ in St Louis (NT-DMT)
Saint Louis, Missouri, 63130
United States
Hackensack University Hospital (MRD-HCT)
Hackensack, New Jersey, 07601
United States
Children's Hospital at Montefiore (MRD HCT)
Bronx, New York, 10467
United States
Children's Hospital at Montefiore (NT-DMT)
Bronx, New York, 10467
United States
Roswell Park (MRD-HCT)
Buffalo, New York, 14203
United States
Columbia Presbytarian (NT-DMT)
New York, New York, 10032
United States
Cohen's Children Hospital (NT-DMT)
Queens, New York, 11040
United States
University of Rochester (MRD-HCT)
Rochester, New York, 14642
United States
University of Rochester (NT-DMT)
Rochester, New York, 14642
United States
UNC Chapel Hill (NT-DMT)
Chapel Hill, North Carolina, 27599.
United States
Atrium Health (MRD-HCT)
Charlotte, North Carolina, 28203
United States
Nationwide Children's Hospital (NT-DMT)
Columbus, Ohio, 43205
United States
Oklahoma Children's Hospital (MRD-HCT)
Oklahoma City, Oklahoma, 73104
United States
Children's Hospital of Philadelphia (MRD-HCT)
Philadelphia, Pennsylvania, 19104
United States
St Jude Children Hospital (NT-DMT)
Memphis, Tennessee, 38105
United States
St. Jude Children's Research Hospital (MRD-HCT)
Memphis, Tennessee, 38105
United States
Texas Children's Hopsital (NT-DMT)
Houston, Texas, 77030
United States
Texas Children's Hospital (MRD-HCT)
Houston, Texas, 77030
United States
UT San Antonio (NT-DMT)
San Antonio, Texas, 78229
United States

Collaborators and Investigators

Sponsor: University of Rochester

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 Registration Dates

Study Start Date2024-06-01
Study Completion Date2030-12-01

Study Record Updates

Study Start Date2024-06-01
Study Completion Date2030-12-01

Terms related to this study

Keywords Provided by Researchers

  • SCD
  • MRD HCT
  • NT DMT

Additional Relevant MeSH Terms

  • Sickle Cell Disease (SCD)