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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT04924608 | Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas | PHASE3 | ACTIVE NOT_RECRUITING | 145 | — | — | Nov 19, 2021 | Feb 15, 2029 | Mar 23, 2026 | 34 | United States, Australia +11 |
| NCT04590235 | A Study of Selumetinib in Chinese Paediatric and Adult Subjects With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN) | PHASE1 | ACTIVE NOT_RECRUITING | 32 | — | — | Dec 16, 2020 | Aug 31, 2026 | May 14, 2026 | 2 | China |
Objective response rate is defined as the proportion of participants who have a confirmed CR (defined as disappearance of the target PN, confirmed by a consecutive scan within 3 to 6 months after the first response) or confirmed PR (defined as a target PN volume decrease ≥ 20%, compared to baseline, confirmed by a consecutive scan within 3 to 6 months after the first response) by end of Cycle 16 as determined by ICR per REiNS criteria. Increase in the volume of the target PN by 20% or more compared to baseline or the time of best response after documenting a PR is considered as PD.
* Occurrence/frequency. * Relationship to IP as assessed by investigator. * Common Terminology Criteria for Adverse Events (CTCAE) grade. * Seriousness. * Death. * Adverse events leading to discontinuation of IP. * Adverse events of special interest.
AUC0-t after single dose and multiple doses administration
Cmax after single dose and multiple doses administration
t1/2 after single dose and multiple doses administration
| Arm | Type | Description |
|---|---|---|
| Arm A | EXPERIMENTAL | Selumetinib |
| Arm B | PLACEBO_COMPARATOR | Placebo |
| Selumetinib | EXPERIMENTAL | All eligible subjects will first receive a single oral dose of selumetinib 25 mg/m\^2. Then, selumetinib 25 mg/m\^2 oral twice daily will be administered continuously until disease progression or unacceptable drug-related toxicity, whichever occurs first. |
| Name | Type | Description |
|---|---|---|
| Selumetinib | DRUG | Selumetinib oral capsules (10 mg and 25 mg) |
| Placebo | OTHER | Placebo oral capsules for Selumetinib masking (10 mg and 25 mg) |
Key Inclusion Criteria: * Adults ≥ 18 years at enrollment with diagnosis of NF1 with symptomatic, inoperable PN * At least one inoperable target PN measurable by volumetric MRI analysis * Chronic target PN pain score documented for minimum period during screening period * Stable chronic PN pain med...