The HOPE Biobank Resource (BMT CTN 2402 HOPE) (HOPE Resource)

January 27, 2026 updated by: Medical College of Wisconsin

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

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.

Study Overview

Status

Not yet recruiting

Conditions

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.

Study Type

Observational

Enrollment (Estimated)

375

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

Yes

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.

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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).
Time Frame: 5 Years
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.
Time Frame: 5 Years
Retention of biospecimen samples with DNA
5 Years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Medical College of Wisconsin

Collaborators

National Cancer Institute (NCI)
National Heart, Lung, and Blood Institute (NHLBI)
National Institutes of Health (NIH)

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

March 27, 2026

Primary Completion (Estimated)

March 30, 2031

Study Completion (Estimated)

March 30, 2031

Study Registration Dates

First Submitted

November 7, 2025

First Submitted That Met QC Criteria

November 7, 2025

First Posted (Actual)

November 12, 2025

Study Record Updates

Last Update Posted (Actual)

January 29, 2026

Last Update Submitted That Met QC Criteria

January 27, 2026

Last Verified

January 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • BMT CTN 2402

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Hemoglobinopathies

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Oman

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe