Not Yet RecruitingNot Applicable

The HOPE Biobank Resource (BMT CTN 2402 HOPE)

375 participantsStarted 2026-03-27

Plain-language 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.

Who can participate

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. 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.

Exclusion criteria

✕. 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.

What they're measuring

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).

Timeframe: 5 Years

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

Timeframe: 5 Years

Trial details

NCT IDNCT07227155

SponsorMedical College of Wisconsin

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2031-03-30

Contact for this trial

Megan Scott

301-251-1161 mscott@emmes.com

View on ClinicalTrials.gov More Aplastic Anemias trials

Who can participate

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. 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.

Exclusion criteria

✕. 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.

What they're measuring

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).

Timeframe: 5 Years

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

Timeframe: 5 Years