SJ901: Phase 1/2 Evaluation of Single Agent Mirdametinib (PD-0325901), a Brain-Penetrant MEK1/2 Inhibitor, for the Treatment of Children, Adolescents, and Young Adults With Low-Grade Glioma
St. Jude Children's Research Hospital
Summary
This is an open-label, multi-center, Phase 1/2 study of the brain-penetrant MEK inhibitor, mirdametinib (PD-0325901), in patients with pediatric low-grade glioma (pLGG).
Description
The objectives of this study are: Phase 1 Primary Objectives: * To determine the safety and tolerability and estimate the maximum tolerated dose (MTD)/recommended phase 2 dose (RP2D) of mirdametinib dosed twice daily on a continuous schedule in pediatric patients with progressive or recurrent low-grade glioma. * To characterize the plasma pharmacokinetics (PK) of mirdametinib. Phase 2 Cohort 1: Newly diagnosed and/or previously untreated (except surgery) Primary Objectives: * To assess the efficacy, defined as the sustained objective response rate \[a Partial Response (PR), Major Respon…
Eligibility
- Age range
- 2–24 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: Screening Phase * Participants with histologically confirmed or suspected low-grade glioma, including neuronal and mixed neuronal-glial tumors * Participant must have adequate tumor tissue from primary and/or relapsed tumor for central pathology review * For Phase 1: Projected to be ≥ 2 years and \< 25 years at the time of study enrollment * Participant's body surface area (BSA) at time of study enrollment must fall within the range outlined in the protocol for the specific dose level under evaluation: * Phase 1: Dose Finding/Dose-escalation * For Phase 1 participa…
Interventions
- DrugMirdametinib
By mouth, nasogastric (NG) tube or gastrostomy tube (G-tube) BID, days 1-28
Location
- St. Jude Children's Research HospitalMemphis, Tennessee