drugset / Trial / NCT05293509
Pharmacologic Pretransplant Immunosuppression (PTIS) + Reduced Toxicity Conditioning (RTC) Allogeneic Stem Cell Transplantation in Inherited Hematologic Disorders
RandomizedParallel-groupOpen-labelTreatment
Summary
To assess the outcomes of NRM when administering pharmacologic pretransplant immunosuppression (PTIS) followed by pretransplant reduced toxicity conditioning (RTC) and an allogeneic stem cell transplant (allo-SCT) and post-transplant graft-versus-host disease prophylaxis based on post-transplant cyclophosphamide (PT-Cy) in patients with inherited blood disorders.
Timeline
- Start
- 2022-03-02
- Primary completion
- 2023-09-18
- Completion
- 2023-09-18
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Bortezomib | Small molecule | 1.3 mg/m2 | Subcutaneous |
| Subject | Cyclophosphamide | Other / unclassified | 100 mg/m2 | Intravenous |
| Subject | Dexamethasone | Small molecule | 25 mg/m2 | Intravenous |
| Subject | Mycophenolate Mofetil | Small molecule | — | Oral |
| Subject | Rituximab | Monoclonal antibody | 375 mg/m2 | Intravenous |
| Subject | Thymoglobulin | Protein / enzyme biologic | — | Intravenous |
| Background | Busulfan | Small molecule | 110 mg/m2 | Intravenous |
| Background | Cyclosporine | Peptide | — | Intravenous |
| Background | Fludarabine | Small molecule | 40 mg/m2 | Intravenous |
| Background | Tacrolimus | Small molecule | — | Intravenous |