drugset / Trial / NCT06412497
MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other Forms of Acquired Bone Marrow Failure.
Non-randomizedParallel-groupOpen-labelTreatment
Summary
A phase II trial of a reduced intensity conditioned (RIC) allogeneic hematopoietic cell transplant (HCT) with post-transplant cyclophosphamide (PTCy) for idiopathic severe aplastic anemia (SAA), paroxysmal nocturnal hemoglobinuria (PNH), acquired pure red cell aplasia (aPRCA), or acquired amegakaryocytic thrombocytopenia (aAT) utilizing population pharmacokinetic (popPK)-guided individual dosing of pre-transplant conditioning and differential dosing of low dose total body irradiation based on age, presence of myelodysplasia and/or clonal hematopoiesis.
Timeline
- Start
- 2024-06-05
- Primary completion
- 2035-05-01
- Completion
- 2036-05-01
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Cyclophosphamide | Other / unclassified | 14.5 mg/kg | — |
| Subject | Cyclophosphamide | Other / unclassified | 50 mg/kg | — |
| Subject | Fludarabine | Small molecule | — | Intravenous |
| Subject | Mycophenolate Mofetil | Small molecule | 15 mg/kg | Oral |
| Subject | Tacrolimus | Small molecule | 0.03 mg/kg | Intravenous |
| Subject | Thymoglobulin | Protein / enzyme biologic | — | — |
| Background | Rituximab | Monoclonal antibody | 375 mg/m2 | Intravenous |
| Background | filgrastim | Protein / enzyme biologic | 5 ug/kg | Subcutaneous |