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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT07387926 | Safety and Efficacy of Asciminib in Pediatrics and Young Adults With Relapse/Refractory (r/r) Philadelphia Positive (Ph+) or ABL-class Ph-like Acute Lymphoblastic Leukemia (ALL) | PHASE1 | NOT YET_RECRUITING | 50 | — | — | Jul 30, 2026 | Jun 18, 2036 | May 19, 2026 | - | — |
A dose-limiting toxicity (DLT) is defined as an adverse event which starts between Day 1 and Day 28, is suspected by the investigator to be related to asciminib, and meets one of the several criteria.
An adverse event (AE) is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment.
Participants with Complete Response/Remission (CR) will achieve the following: No circulating blasts or extramedullary disease; No lymphadenopathy, splenomegaly, skin/gum infiltration, testicular mass/CNS involvement; Marrow \<5% blasts (M1) OR \< 1% blasts by flow cytometry. If a discrepancy occurs between disease detection methods, the result of the flow cytometry assay will be used to determine CR status; Peripheral blood count recovery; ANC \> 1000μL and platelets \> 100,000μL. CR evaluable: Participants will be considered CR evaluable for Part 2 if they have relapsed/refractory Ph+ ALL defined as either \>1% bone marrow (BM) blasts by MFC or immunoglobulin/T-cell receptor (IG/TCR) PCR, OR \> 5% BM blasts by morphologic evaluation at enrollment and are treated at RP2D.
| Arm | Type | Description |
|---|---|---|
| Single Arm | EXPERIMENTAL | Asciminib Adult formulation group: escalating doses evaluated Asciminib Pediatric formulation group: dose is based on body weight; dose level being evaluated will be converted into a mg/kg daily dose. For known T315I mutation: Fixed asciminib dose twice daily or the pediatric formulation dose equivalent twice daily. |
| Name | Type | Description |
|---|---|---|
| Asciminib Adult formulation | DRUG | oral, administered daily (twice daily for participants with known T315I mutation); Cycles 1, 2, 3 |
| Asciminib Pediatric formulation | DRUG | oral, administered daily (twice daily for participants with known T315I mutation); Cycles 1, 2, 3 |
| Dexamethasone | DRUG | Fixed doses, oral (preferred) or intravenous (IV) twice daily; Cycle 1, Days 1 - 14; (Cycle 1 = 28 days) |
| Vincristine | DRUG | Fixed doses, IV, weekly; Cycle 1 |
| Blinatumomab | DRUG | Dosing based on bone marrow disease burden and weight. Continuous IV infusion; Cycles 2, 3 |
| Methotrexate (intrathecal) | DRUG | Intrathecal |
| Cytarabine (intrathecal) | DRUG | Intrathecal |
| Hydrocortisone (intrathecal) | DRUG | Intrathecal |
| Prednisolone (intrathecal) | DRUG | Intrathecal |
Inclusion Criteria: * Evidence of Ph+ ALL or ABL1 or ABL2 fusion Ph-like ALL, inclusive of participants with ABL1 T315I mutation * Participants with CNS1, CNS2, CNS3a, or CNS3b at screening * Active B-Cell ALL at screening defined by MFC or IG/TCR PCR of ALL blasts \>0.01% in participants with eith...