NCT06735820

Early Phase Study Evaluating MEK and MDM2 Inhibition in Patients With NF1 and MPNST

Early Phase Study to Evaluate the MEK Inhibitor Selumetinib With the MDM2 Inhibitor APG-115 in Patients With Neurofibromatosis Type 1 and Pre-malignant and Malignant Peripheral Nerve Sheath Tumors

• Status: Not Yet Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 45 (estimated)
• Sponsor: AeRang Kim · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This is a phase 0/1/2, multi-site study to evaluate the MEK inhibitor Selumetinib with the MDM2 Inhibitor APG-115 in patients with Neurofibromatosis Type 1 and pre-malignant and malignant peripheral nerve sheath tumors

Detailed description

The first primary objective of the study is to determine the safety, tolerability, pharmacokinetics, and recommended doses of selumetinib and APG-115 in patients with Neurofibromatosis Type 1 (NF1) and refractory/unresectable MPNST (Part A). The second primary objective is to determine the clinical benefit of selumetinib and APG-115 in patients with NF1 and refractory/unresectable MPNST (Part B). The secondary objective is to evaluate the percent apoptosis and tumor proliferation in resectable atypical neurofibromatus neoplasm of uncertain biologic potential (ANNUBP) after exposure to selumetinib and APG-115 (Part C)

Conditions

• Malignant Peripheral Nerve Sheath Tumor (MPNST)
• Neurofibromatosis 1 (NF1)
• Atypical Neurofibroma

Interventions

Type	Name	Description
DRUG	APG-115	Combination therapy of APG-115 and Selumetinib
DRUG	Selumetinib	Combination therapy of APG-115 and Selumetinib

Timeline

• Start date: 2025-10-01
• Primary completion: 2027-10-01
• Completion: 2028-10-01
• First posted: 2024-12-16
• Last updated: 2025-07-03

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study