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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT04910685 | (HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis | PHASE2 | RECRUITING | 534 | — | — | Nov 30, 2021 | Sep 30, 2032 | May 22, 2026 | 68 | United States, Argentina +17 |
| Arm | Type | Description |
|---|---|---|
| (Part 1) Elenestinib Dose 1 + SDT | EXPERIMENTAL | Participants will receive SDT and elenestinib. SDT will be determined on a per participant basis. Elenestinib will be administered orally, once daily until completion of Part 1. |
| (Part 1) Elenestinib Dose 2 + SDT | EXPERIMENTAL | Participants will receive SDT and elenestinib. SDT will be determined on a per participant basis. Elenestinib will be administered orally, once daily until completion of Part 1. |
| (Part 1) Elenestinib Dose 3 + SDT | EXPERIMENTAL | Participants will receive SDT and elenestinib. SDT will be determined on a per participant basis. Elenestinib will be administered orally, once daily until completion of Part 1. |
| (Part 1) Placebo + SDT | PLACEBO_COMPARATOR | Participants will receive SDT and matching placebo. SDT will be determined on a per participant basis. Placebo will be administered orally, once daily until completion of Part 1. |
| (Part 2) Elenestinib Dose 1 + SDT | EXPERIMENTAL | Participants will receive SDT and elenestinib. SDT will be determined on a per participant basis. Elenestinib will be administered orally, once daily for approximately 48 weeks. |
| (Part 2) Placebo + SDT | PLACEBO_COMPARATOR | Participants will receive SDT and matching placebo. SDT will be determined on a per participant basis. Placebo will be administered orally, once daily for approximately 48 weeks. |
| (Part 3) Elenestinib + SDT | EXPERIMENTAL | Participants will receive SDT and elenestinib. SDT will be determined on a per participant basis. Elenestinib will be administered orally, once daily for up to approximately 5 years. |
| (Part S) Elenestinib Dose 1 + SDT | EXPERIMENTAL | Participants will receive SDT and elenestinib. SDT will be determined on a per participant basis. Elenestinib will be administered orally, once daily for up to approximately 5 years. |
| (Part K) Elenestinib Dose 1 + SDT | EXPERIMENTAL | Participants will receive SDT and elenestinib. SDT will be determined on a per participant basis. Elenestinib will be administered orally, once daily for up to approximately 5 years. |
| (PK groups) Elenestinib + SDT | EXPERIMENTAL | Participants will receive SDT and elenestinib. SDT will be determined on a per participant basis. Elenestinib will be administered orally, once daily for up to approximately 5 years. |
| Name | Type | Description |
|---|---|---|
| Elenestinib | DRUG | Elenestinib oral tablet |
| Placebo | DRUG | Placebo oral tablet |
Key Inclusion Criteria: All Participants: -Participant must have an Eastern Cooperative Oncology Group Performance Status (ECOG PS) of 0 to 2. Part 1 and PK groups: * Participant has confirmed diagnosis of ISM, confirmed by Central Pathology Review * Participant must have failed to achieve adequ...