Stem Cell Transplant for Aplastic Anemia
Trial Summary
What is the purpose of this trial?
Fludarabine-based preparative regimen followed by an allogeneic hematopoietic stem cell transplant using related or unrelated donor in persons 0-70 years of age diagnosed with dyskeratosis congenita or severe aplastic anemia who have bone marrow failure characterized by a requirement for red blood cell and platelet transfusions. Three different preparative regimens are included based on disease and donor type.
Research Team
Jakub Tolar, MD
Principal Investigator
University of Minnesota
Eligibility Criteria
This trial is for people aged 0-70 with dyskeratosis congenita or severe aplastic anemia needing blood transfusions. Participants must have specific blood counts, organ function levels, and genetic mutations related to the conditions. Pregnant individuals, those with uncontrolled infections, certain liver issues, or a diagnosis of Fanconi anemia are excluded.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Preparative Regimen
Participants receive a fludarabine-based preparative regimen including alemtuzumab, cyclophosphamide, and fludarabine, followed by a stem cell transplant
Engraftment Monitoring
Participants are monitored for neutrophil and platelet engraftment
Follow-up
Participants are monitored for regimen-related mortality, acute and chronic graft-versus-host disease, and secondary malignancies
Treatment Details
Interventions
- Alemtuzumab
- Anti-thymocyte globulin
- Cyclophosphamide
- Fludarabine
- Hematopoietic Stem Cell Transplant
- Total Body Irradiation
Alemtuzumab is already approved in United States, European Union for the following indications:
- Chronic lymphocytic leukemia
- Multiple sclerosis
- Multiple sclerosis
- Chronic lymphocytic leukemia
Find a Clinic Near You
Who Is Running the Clinical Trial?
Masonic Cancer Center, University of Minnesota
Lead Sponsor