drugset / Trial / NCT02162420
Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia
Non-randomizedSingle-groupOpen-labelTreatment
Summary
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.
Timeline
- Start
- 2015-01-10
- Primary completion
- 2024-08-08
- Completion
- 2025-03-11
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | ANTILYMPHOCYTE IMMUNOGLOBULIN (HORSE) | Protein / enzyme biologic | 3 mg/kg | Intravenous |
| Background | Alemtuzumab | Monoclonal antibody | 0.2 mg/kg | Intravenous |
| Background | Cyclophosphamide | Other / unclassified | 50 mg/kg | Intravenous |
| Background | Fludarabine | Small molecule | 40 mg/m2 | Intravenous |