Stem Cell Transplant for Aplastic Anemia
What You Need to Know Before You Apply
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.
Who Is on the Research Team?
Jakub Tolar, MD
Principal Investigator
University of Minnesota
Are You a Good Fit for This Trial?
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 for a Trial Participant
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
What Are the Treatments Tested in This Trial?
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:
Find a Clinic Near You
Who Is Running the Clinical Trial?
Masonic Cancer Center, University of Minnesota
Lead Sponsor