The HOPE Biobank Resource (BMT CTN 2402 HOPE)
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorMedical College of Wisconsin
About this trial
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.
Eligibility criteria
This trial accepts healthy volunteersQualifiers
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.
Disqualifiers
None
Trial 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.
Trial design
Design model
Case-only
Time perspective
Prospective
Treatments tested in this trial
Not listed
Trial groups
No trial groups listed
Trial outcomes
Primary outcomes
1
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
Time frame
5 Years2
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
Time frame
5 YearsSponsors and contacts
Click on the lead sponsor to view all of their trials.
Medical College of Wisconsin
Lead sponsor
National Cancer Institute (NCI)
Collaborator
National Institutes of Health (NIH)
Collaborator
National Heart, Lung, and Blood Institute (NHLBI)
Collaborator
Source ClinicalTrials.gov