The HOPE Biobank Resource (BMT CTN 2402 HOPE)

NCT07227155 | Not Yet Recruiting DMC

• 1 other identifier: BMT CTN 2402
• Study Type: observational
• Target: 375
• Locations: 0 countries
• Sites: N/A

Brief 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.

Conditions

Aplastic Anemias; Hemoglobinopathies; Bone Marrow Failure

Keywords

Biorepository; Biospecimens; Allogenic Transplant; Gene Therapy

Outcome Measures

Primary Outcomes (2)

• Develop a biorepository that will establish mechanistic insights and biologic correlates to key casues of fialure of HCT and GT for Non-malignant Diseases (NMD).

  Retention of biospecimen samples with DNA

  5 Years

• Identify new therapeutic targets for NMD and HCT or GT related complications leading to development of more targeted and effective therapies.

  Retention of biospecimen samples with DNA

  5 Years

Eligibility Criteria

• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients undergoing HCT or GT for treatment of Non-malignant blood disorders: Aplastic Anemia, Hemoglobinopathies or Bone Marrow Failure. Related donors of enrolled participants undergoing HCT are also eligible for the study.

You may qualify if:

• 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:
• 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.
• Hemoglobinopathies include sickle cell disease or thalassemia. Patients receiving potentially curative therapy with HCT or GT for hemoglobinopathies will be eligible for this study.
• 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 bmtctn2402@emmes.com.
• 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.
• \. 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.

You may not qualify if:

• \. Donor weight ≤ 10.0 kg at time of study enrollment

Sponsors & Collaborators

• National Cancer Institute (NCI): collaborator
• National Institutes of Health (NIH): collaborator
• National Heart, Lung, and Blood Institute (NHLBI): collaborator
• Medical College of Wisconsin: lead

Biospecimen

Retention: SAMPLES WITH DNA

Blood, germline (skin, hair, nails), bone marrow, stool

MeSH Terms

Conditions

Anemia, Aplastic; Hemoglobinopathies; Bone Marrow Failure Disorders

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Megan Scott

CONTACT

301-251-1161; mscott@emmes.com

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Target Duration: 5 Years
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 7, 2025
• First Posted: November 12, 2025
• Study Start: March 27, 2026
• Primary Completion (Estimated): March 30, 2031
• Study Completion (Estimated): March 30, 2031
• Last Updated: January 29, 2026

Record last verified: 2026-01