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Selumetinib granule formulation · 1 trial · 1 indication
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...
Selumetinib granule formulation is being studied for the treatment of Neurofibromatosis Type 1 (NF1) in children aged 1 to under 7 years with symptomatic, inoperable plexiform neurofibromas. It is an investigational small molecule kinase inhibitor currently in Phase 1 clinical development.
Selumetinib granule formulation is a small molecule kinase inhibitor, belonging to the -tinib class of drugs. It works by inhibiting specific kinase enzymes involved in cell signaling pathways. The drug is being evaluated for its pharmacokinetics, safety, and efficacy in pediatric patients with NF1-related plexiform neurofibromas.
Selumetinib granule formulation is being developed by AstraZeneca PLC, a biopharmaceutical company listed on the stock exchange under the ticker AZN. The company is conducting clinical trials to evaluate the drug's safety and effectiveness in young children with Neurofibromatosis Type 1.
Selumetinib granule formulation is in Phase 1 clinical development. It is an investigational drug and has not been approved by regulatory authorities. The ongoing Phase 1 trial is actively enrolling participants, though recruitment is not yet complete, to assess the drug's pharmacokinetics, safety, and efficacy.
Selumetinib granule formulation is being studied in a Phase 1 clinical trial with the identifier NCT05309668. This open-label, uncontrolled trial is evaluating the drug in children aged 1 to under 7 years with NF1-related symptomatic, inoperable plexiform neurofibromas. The trial is active but not recruiting and has an enrollment target of 36 participants.
Selumetinib granule formulation is a specific formulation of the drug selumetinib, designed as granules for oral administration. It is being developed for use in young children who may have difficulty swallowing capsules. The granule formulation is being studied separately in a Phase 1 trial for pediatric patients with Neurofibromatosis Type 1.