NCT07227155

The HOPE Biobank Resource (BMT CTN 2402 HOPE)

Hematopoietic Cell Transplant and Gene Therapy for Non-Malignant Blood Disorders Biobank Resource (BMT CTN 2402)

• Status: Not Yet Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 375 (estimated)
• Sponsor: Medical College of Wisconsin · Academic / Other
• Sex: All
• Age: —
• Healthy volunteers: Accepted

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.

Conditions

• Aplastic Anemias
• Hemoglobinopathies
• Bone Marrow Failure

Timeline

• Start date: 2026-03-27
• Primary completion: 2031-03-30
• Completion: 2031-03-30
• First posted: 2025-11-12
• Last updated: 2026-01-29