Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06517641 | Clinical Trial of Upfront Haploidentical or Unrelated Donor BMT to Restore Normal Hematopoiesis in Aplastic Anemia | PHASE2 | RECRUITING | 60 | — | — | May 12, 2025 | Feb 1, 2029 | Feb 17, 2026 | 25 | United States |
The primary endpoint is GFFS at 1 year after initiation of conditioning. Events for GFFS include Grade III-IV aGVHD, cGVHD requiring immunosuppression, primary or secondary graft failure requiring second definitive therapy, failure to receive an HSCT infusion, and death. GFFS is defined as the time interval from start of conditioning until the first of these events occurs. For failure to receive an HSCT infusion, the date will be at the time the decision not to proceed to HSCT is made.
| Arm | Type | Description |
|---|---|---|
| Haploidentical transplantation | ACTIVE_COMPARATOR | Patients receiving bone marrow transplanted from a haploidentical related donor will be included in this arm. |
| Unrelated donor transplantation | ACTIVE_COMPARATOR | Patients receiving bone marrow transplanted from an unrelated donor will be included in this arm. |
| Name | Type | Description |
|---|---|---|
| Haploidentical donor bone marrow transplant | DRUG | Drugs: 1. Antithymocyte Globulin (ATG) dose will be 0.5 mg/kg IV on Day -9 over 6 hours and 2 mg/kg IV on Days -8 and -7 over 4 hours. 2. Fludarabine dose will be 30 mg/m\^2 IV daily for 5 days from Day -6 to Day -2. 3. Cyclophosphamide dose will be 14.5 mg/kg IV daily for 2 days (Day -6 to Day -5) prior to transplantation and 50 mg/kg IV daily for 2 days (Day +3 to Day +4) after transplantation. 4. Tacrolimus should be started on Day +5 and administered to maintain a level of 10-15 ng/mL. 5. Mycophenolate mofetil (MMF) dose will be 15 mg/kg PO three times a day (TID) up to 1 gm TID (or IV equivalent) starting on Day +5 through Day +35. 6. G-CSF will be given IV or SQ starting on Day +5 at 5 mcg/kg/day until ANC is \> 1500 for 3 days. Radiation: 1\. Total Body Irradiation (TBI): will be given as a single dose of 400 cGy on Day -1. Procedure: 1\. HSCT: Eligible patients will receive a haploidentical donor bone marrow transplant. |
| Unrelated donor bone marrow transplant | DRUG | Drugs: 1. Antithymocyte Globulin (ATG) dose will be 0.5 mg/kg IV on Day -9 over 6 hours and 2 mg/kg IV on Days -8 and -7 over 4 hours. 2. Fludarabine dose will be 30 mg/m\^2 IV daily for 5 days from Day -6 to Day -2. 3. Cyclophosphamide dose will be 14.5 mg/kg IV daily for 2 days (Day -6 to Day -5) prior to transplantation and 50 mg/kg IV daily for 2 days (Day +3 to Day +4) after transplantation. 4. Tacrolimus should be started on Day +5 and administered to maintain a level of 10-15 ng/mL. 5. Mycophenolate mofetil (MMF) dose will be 15 mg/kg PO three times a day (TID) up to 1 gm TID (or IV equivalent) starting on Day +5 through Day +35. 6. G-CSF will be given IV or SQ starting on Day +5 at 5 mcg/kg/day until ANC is \> 1500 for 3 days. Radiation: 1\. Total Body Irradiation (TBI): will be given as a single dose of 400 cGy on Day -1. Procedure: 1\. HSCT: Eligible patients will receive an unrelated donor bone marrow transplant. |
Inclusion Criteria: 1. Age 3 years to 75 years 2. Confirmed diagnosis of acquired SAA defined as: a. Bone marrow cellularity \< 25% or variable marrow cellularity but with \< 30% residual hematopoietic cells deemed HYPOcellular for age AND b. Two (2) out of 3 of the following (in peripheral blo...