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
AeRang Kim
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
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 selumeti…
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * AGE: Part A and C: ≥ 18 years of age AGE: Part B: ≥12 years (minimum BSA ≥0.55m2) * Part A and B: Patients with unresectable or metastatic histologically confirmed NF1 associated MPNST. Part C: Patients with NF1 and ANNUBP. Diagnostic criteria based on Miettinen et al, Human Pathol: * MEASURABLE DISEASE: Patients must have measurable disease by RECISTv1.1. Baseline radiologic scans must be performed within 4 weeks of starting treatment. * Therapeutic options: Parts A and B: Patients must have experienced progression after one or more prior regimens of cytotoxic chemother…
Interventions
- DrugAPG-115
Combination therapy of APG-115 and Selumetinib
- DrugSelumetinib
Combination therapy of APG-115 and Selumetinib
Location
- Children's National HospitalWashington D.C., District of Columbia