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Taking people in United StatesNo phase

The HOPE Biobank Resource (BMT CTN 2402 HOPE)

Key facts

Who runs it
Medical College of Wisconsin
Where
Atlanta, Chicago, Duarte, Durham, Gainesville, Houston, Milwaukee, Nashville, New York, Portland, Richmond, Seattle, and St Louis, United StatesSee the 14 sites
Trial code
Status
Recruiting
People taking part
375 people, planned
Started
15 May 2026
Expected to end
30 Mar 2031
Type of study
Observational
Record updated
27 August 2026

Who can take part

Sex
Women and men
Healthy volunteers
Can take part

The registry criteria, word for word

Show all 9 criteriaShow less

Other criteria

  • HCT/GT Inclusion: 1. Patients with a diagnosis of Aplastic Anemia (AA), hemoglobinopathies or bone marrow failure from other causes except for malignant diseases will be eligible for enrollment on this protocol: 1. AA will be defined as having peripheral blood cytopenias with a hypocellular bone marrow for age and a clinical diagnosis of aplastic anemia as determined by their treating physicians. 2. Hemoglobinopathies include sickle cell disease or thalassemia. Patients receiving potentially curative therapy with HCT or GT for hemoglobinopathies will be eligible for this study. 3. Individuals with bone marrow failure due to clinical or molecularly diagnosed inherited bone marrow failure, inborn errors of immunity or other cause will be included
  • Patients must receive an HCT or GT for management of their underlying disease. Allogeneic transplants including all conditioning regimens, donors, and GVHD prophylaxis regimens are eligible. This study does not define how the transplant or transplant-supportive care will be performed
  • Patients or their legal guardian must consent to participate in the CIBMTR "Protocol for a Research Database for Hematopoietic Cell Transplantation and Marrow Toxic Injuries" (NCT 1166009) to allow linkage with the longitudinal clinical data collected by CIBMTR
  • All ages minorities, sexes and genders are eligible for the study, but participants must weigh at least 10 kilograms (kg) at the time of study enrollment given the volume and number of blood draws required
  • All participants or parent/legal guardian must sign an informed consent for this study. If there are questions regarding a patient's eligibility for the study, contact the Protocol Team for review and discussion by emailing. HCT/GT Exclusion 1. Patients with aplastic anemia or hemoglobinopathies who are not pursuing allogeneic HCT or GT
  • Active malignancy
  • Hematologic malignancy or therapy for a prior hematologic malignancy in the previous five 5 years
  • Weight ≤ 10.0 kg at time of study enrollment
  • Prior autologous or allogeneic transplant. Related Donor Inclusion: 1. All related donors for eligible recipients undergoing allogeneic HCT for AA, hemoglobinopathies, or bone marrow failure as defined in the recipient eligibility criteria above are eligible. Note: HCT recipient participants will remain eligible if the related donor declines to participate in the study. Related Donor Exclusion: 1. Donor weight ≤ 10.0 kg at time of study enrollment

Where in United States

  • Children's Healthcare of AtlantaAtlanta, United StatesContact: Judson RussellTaking people
  • Lurie Children's Hospital of ChicagoChicago, United StatesContact: Morgan KearneyTaking people
  • City of HopeDuarte, United StatesContact: Miranda LeeTaking people
  • Duke University Medical CenterDurham, United StatesContact: Quinna LawsonTaking people
  • University of FloridaGainesville, United StatesContact: Giselle Morre-HiggsTaking people
  • Baylor College of MedicineHouston, United StatesContact: Deb DowlinTaking people
  • Children's Hospital of WisconsinMilwaukee, United StatesContact: Kira MielkeTaking people
  • Vanderbilt University Medical CenterNashville, United StatesContact: Melanie Hallowell HallowellTaking people
  • Memorial Sloan-Kettering Cancer CenterNew York, United StatesContact: Alyssa KamrowskiTaking people
  • Oregon Health and Science UniversityPortland, United StatesContact: Denise LackeyTaking people
  • Virginia Commonwealth UniversityRichmond, United StatesContact: Mehreen QureshiTaking people
  • Fred Hutchinson Cancer CenterSeattle, United StatesContact: Sheri BallardTaking people
  • St. Louis Children'sSt Louis, United StatesContact: Lissy KellerTaking people
  • Washington UniversitySt Louis, United StatesContact: Maggie NashTaking people

About the trial

What the trial wants to find out

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.

Questions to ask the study team

  1. How often do I need to come in, and for how long each time?
  2. Are travel costs paid?
  3. Can I keep my own doctor?
  4. What happens if I want to stop?

Contact the study team: see the registry record → Contacts and Locations

Print a page for my doctor

Data and sources

Registry record
Listed on ClinicalTrials.gov.
Official title
Hematopoietic Cell Transplant and Gene Therapy for Non-Malignant Blood Disorders Biobank Resource (BMT CTN 2402)
Record updated
27 August 2026