The HOPE Biobank Resource (BMT CTN 2402 HOPE)
Start Date
5/15/2026
Completion Date
3/30/2031
Summary
A prospective, multicenter study that will establish a repository of biospecimens and clinical data from patients undergoing hematopoietic stem cell transplant (HCT) or gene therapy (GT) for treatment of non-malignant blood diseases.
Detailed Description
This is a prospective, multicenter, study that will establish a repository of biospecimens and clinical data from patients undergoing hematopoietic stem cell transplant (HCT) or gene therapy (GT) for treatment of non-malignant blood diseases. The enrollment goal is 375 participants and up to all consenting 100 related donors (approximately 100 are anticipated), accrued over 4 years. Participants will be followed until the end of study, but continued collection of specimens and clinical data may continue to allow up to 15 years of follow-up for all enrolled participants if funds are available. Participants with a diagnosis of Aplastic Anemia or Hemoglobinopathies or bone marrow failure undergoing HCT or GT for management of their underlying disease. There is no treatment intervention in this study. Treatment will be at the provider's discretion. Participants will provide blood and other biospecimens pre and post-intervention at specified intervals. 375 participants and approximately 100 related donors will be enrolled from 35 sites.
Eligibility Criteria
Age Range: No minimum to No maximum
Conditions
Locations
City of Hope
Duarte, California 91010
United States
University of Florida
Gainesville, Florida 32611
United States
Children's Healthcare of Atlanta
Atlanta, Georgia 30329
United States
Lurie Children's Hospital of Chicago
Chicago, Illinois 60611
United States
St. Louis Children's
St Louis, Missouri 63110
United States
Washington University
St Louis, Missouri 63110
United States
Memorial Sloan Kettering Cancer Center
New York, New York 10065
United States
Duke University Medical Center
Durham, North Carolina 27710
United States
Oregon Health and Science University
Portland, Oregon 97239
United States
Vanderbilt University Medical Center
Nashville, Tennessee 37235
United States
Baylor College of Medicine
Houston, Texas 77030
United States
Virginia Commonwealth University
Richmond, Virginia 23298
United States
Fred Hutchinson Cancer Center
Seattle, Washington 98109
United States
Children's Hospital of Wisconsin
Milwaukee, Wisconsin 53226
United States