HOPE Biobank: connecting blood disease patients through stem cell transplant and gene therapy samples

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a multicenter, prospective observational study to establish a repository of biospecimens and associated clinical data from patients receiving hematopoietic stem cell transplant or gene therapy for non-malignant blood disorders. The enrollment target is 375 patients plus up to about 100 related donors, over four years, with potential long-term follow-up of up to 15 years if funds permit. There is no assigned treatment within the protocol; medical decisions are made by the treating clinicians. Participants will provide blood and other samples before and after intervention at specified time points, enabling future analyses of biology and outcomes. Approximately 35 sites are involved, led by the Medical College of Wisconsin with NIH/NCI/NHLBI collaboration.

Why it matters

Who can join: the full rules

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.
2. 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.
3. 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.
4. 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.
5. 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 bmtctn2402@emmes.com.

HCT/GT Exclusion

1. Patients with aplastic anemia or hemoglobinopathies who are not pursuing allogeneic HCT or GT.
2. Active malignancy.
3. Hematologic malignancy or therapy for a prior hematologic malignancy in the previous five 5 years.
4. Weight ≤ 10.0 kg at time of study enrollment.
5. 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 it's running

• City of Hope
  Duarte, California, United States Recruiting
• University of Florida
  Gainesville, Florida, United States Recruiting
• Children's Healthcare of Atlanta
  Atlanta, Georgia, United States Recruiting
• Lurie Children's Hospital of Chicago
  Chicago, Illinois, United States Recruiting
• St. Louis Children's
  St Louis, Missouri, United States Recruiting
• Washington University
  St Louis, Missouri, United States Recruiting

Study contacts

• Study coordinator: Megan Scott
• Email: mscott@emmes.com
• Phone: 301-251-1161

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Dibotatug in adults with bone marrow failure syndromes

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• Donor transplant for high risk blood disorders

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• Using umbilical cord blood to support stem cell transplants for treating aplastic anemia

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• Platform for Rare Blood Disorders in Europe

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  Recruiting Hospital Universitari Vall d'Hebron Research Institute · Barcelona, Catalonia · NCT06250595

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.