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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT05309668 | Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With NF1-related Symptomatic, Inoperable PN | PHASE1 | ACTIVE NOT_RECRUITING | 36 | — | — | Jan 21, 2022 | Apr 28, 2028 | Dec 18, 2025 | 16 | United States, Germany +5 |
To determine the pharmacokinetics of selumetinib after administration of the selumetinib granule formulation
To assess the safety and tolerability of the selumetinib granule formulation.
| Arm | Type | Description |
|---|---|---|
| Selumetinib single arm | EXPERIMENTAL | This study consists of a screening period (up to 28 days), a treatment period (25 cycles) and a long term safety follow-up for participants until they are 5 years old or commence an alternative systemic NF1-PN treatment, whichever is the earlier. Participants may continue treatment with selumetinib throughout the long term safety follow-up as long as they are considered to be receiving clinical benefit in the opinion of their Investigator. A safety follow up assessment will be performed 30 days after the last dose of study intervention for all study participants. |
| Name | Type | Description |
|---|---|---|
| Selumetinib granule formulation | DRUG | Selumetinib granule formulation will be administered using BSA-based dosing. The granule formulation dose schema to be used in the study will be established in the dose finding phase. At enrolment participants must have a BSA within the range 0.40 to 1.09 m2; once participants attain a BSA between 1.10 and 1.29 m2 they will be encouraged to transition to the capsule formulation, if feasible, although all participants must remain on the granule formulation until after they have completed their third cycle of treatment. |
| Selumetinib capsule formulation | DRUG | Selumetinib capsule formulation will be administered using BSA-based dosing. Once participants attain a BSA between 1.10 and 1.29 m2 they will be encouraged to transition to the capsule formulation, if feasible, although all participants must remain on the granule formulation until after they have completed their third cycle of treatment. |
Inclusion Criteria: 1. Male and female participants aged ≥ 1 to \< 7 years of age at the time their legally authorised representative (parent or guardian) signs the informed consent. 2. All study participants must be diagnosed with NF1 with symptomatic inoperable PN as defined in protocol. 3. Parti...