Major study to decide: transplant or medication for kids with sickle cell?

NCT ID NCT06941389

Recruiting now Knowledge-focused Sponsor: University of Rochester Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This large study will follow 480 children with sickle cell disease to compare two treatment approaches: a stem cell transplant from a matched family donor versus standard disease-modifying therapies. The goal is to see which option leads to better quality of life and thinking skills over three years. By tracking both groups, researchers hope to provide clear information to help families and doctors make informed treatment decisions.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: matched related donor hematopoietic stem cell transplantation (MRD HCT) and non-transplant disease-modifying therapies (NT-DMT)
What this could lead to: If successful, this study could help families and doctors choose the best treatment for children with sickle cell disease by comparing long-term quality of life and thinking abilities.
What could go wrong: This is an observational study, not a controlled trial, so results may be influenced by factors like patient choice. It will take years to complete and may not provide clear answers for every child.

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Conditions

The condition(s) this trial relates to.

sickle cell disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

SICKLE CELL DISEASE (SCD)

Contacts and locations

Locations

Alberta Children's Hospital (MRD-HCT)
RECRUITING

Calgary, Alberta, T3B6A8, Canada
Alberta Children's Hospital (NT-DMT)
NOT_YET_RECRUITING

Calgary, Alberta, T3B 6A8, Canada
Atrium Health (MRD-HCT)
RECRUITING

Charlotte, North Carolina, 28203, United States
Boston Children's Hospital (MRD-HCT)
RECRUITING

Boston, Massachusetts, 02115, United States
Boston Children's Hospital (NT-DMT)
NOT_YET_RECRUITING

Boston, Massachusetts, 02115, United States
British Columbia Children's Hosptial (MRD HCT)
NOT_YET_RECRUITING

Vancouver, British Columbia, Canada
Children's Healthcare of Atlanta (MRD-HCT)
RECRUITING

Atlanta, Georgia, 30329, United States
Children's Hospital at Montefiore (MRD HCT)
NOT_YET_RECRUITING

The Bronx, New York, 10467, United States
Children's Hospital at Montefiore (NT-DMT)
RECRUITING

The Bronx, New York, 10467, United States
Children's Hospital of Philadelphia (MRD-HCT)
RECRUITING

Philadelphia, Pennsylvania, 19104, United States
Children's National Hospital (MRD-HCT)
RECRUITING

Washington D.C., District of Columbia, 20010, United States
Children's of Alabama (MRD-HCT)
RECRUITING

Birmingham, Alabama, 35294, United States
Children's of Alabama (NT-DMT)
NOT_YET_RECRUITING

Birmingham, Alabama, 35294, United States
Cohen's Children Hospital (NT-DMT)
NOT_YET_RECRUITING

Queens, New York, 11040, United States
Columbia Presbytarian (NT-DMT)
NOT_YET_RECRUITING

New York, New York, 10032, United States
Comer Children's Hospital (MRD-HCT)
RECRUITING

Chicago, Illinois, 60637, United States
HSC Winnipeg Children's Hospital/University of Manitoba (MRD-HCT)
RECRUITING

Winnipeg, Manitoba, R3E 0V9, Canada
Hackensack University Hospital (MRD-HCT)
RECRUITING

Hackensack, New Jersey, 07601, United States
Nationwide Children's Hospital (NT-DMT)
NOT_YET_RECRUITING

Columbus, Ohio, 43205, United States
Nemours Children's Hospital, Delaware (MRD-HCT)
RECRUITING

Wilmington, Delaware, 19803, United States
Oklahoma Children's Hospital (MRD-HCT)
NOT_YET_RECRUITING

Oklahoma City, Oklahoma, 73104, United States
Riley Children's Hospital (MRD-HCT)
RECRUITING

Indianapolis, Indiana, 46202, United States
Riley's Children Hospital (NT-DMT)
NOT_YET_RECRUITING

Indianapolis, Indiana, 46202, United States
Roswell Park (MRD-HCT)
RECRUITING

Buffalo, New York, 14203, United States
St Jude Children Hospital (NT-DMT)
NOT_YET_RECRUITING

Memphis, Tennessee, 38105, United States
St. Jude Children's Research Hospital (MRD-HCT)
RECRUITING

Memphis, Tennessee, 38105, United States
Texas Children's Hopsital (NT-DMT)
NOT_YET_RECRUITING

Houston, Texas, 77030, United States
Texas Children's Hospital (MRD-HCT)
RECRUITING

Houston, Texas, 77030, United States
The Hospital for Sick Children (MRD-HCT)
NOT_YET_RECRUITING

Toronto, Ontario, ON M5G 1E8, Canada
UNC Children's Hospital (MRD HCT)
NOT_YET_RECRUITING

Chapel Hill, North Carolina, 27514, United States
UNC Children's Hospital (NT-DMT)
NOT_YET_RECRUITING

Chapel Hill, North Carolina, 27599., United States
UT San Antonio (NT-DMT)
NOT_YET_RECRUITING

San Antonio, Texas, 78229, United States
Universite de Montreal / Ste Justine (MRD HCT)
NOT_YET_RECRUITING

Montreal, Quebec, Canada
University of Rochester (MRD-HCT)
NOT_YET_RECRUITING

Rochester, New York, 14642, United States
University of Rochester (NT-DMT)
NOT_YET_RECRUITING

Rochester, New York, 14642, United States
Wahington Univ in St Louis (NT-DMT)
NOT_YET_RECRUITING

St Louis, Missouri, 63130, United States
Washington University in St Louis (MRD-HCT)
NOT_YET_RECRUITING

St Louis, Missouri, 63130, United States

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