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

ConditionsMalignant Peripheral Nerve Sheath Tumor (MPNST)Neurofibromatosis 1 (NF1)Atypical Neurofibroma
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorAeRang Kim

About this trial

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

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

AGE: Part A and C: ≥ 18 years of age AGE: Part B: ≥12 years (minimum BSA ≥0.55m2)

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 chemotherapy. Patients who have refused cytotoxic chemotherapy or for whom treatment on this protocol prior to receiving cytotoxic chemotherapy is felt to be in the best interest for the patient by the local investigator will also be eligible. Part C: Patients with ANNUBP that are planned for surgical resection

PRIOR THERAPY

Disqualifiers

A malignancy treated with curative intent and with no known active disease ≥2 years before the first dose of the study intervention and of low potential risk of recurrence.

Adequately treated non-melanoma skin cancer or lentigo maligna without evidence of disease

Adequately treated carcinoma in situ without evidence of disease

Stable optic pathway glioma or low-grade glioma not receiving active therapy

Trial design

Design model

Single group

Treatments tested in this trial

  • APG-115

    Drug

    Combination therapy of APG-115 and Selumetinib

  • Selumetinib

    Drug

    Combination therapy of APG-115 and Selumetinib

Treatment groups

45 Participants
are divided into 1 treatment group
Group A: APG-115 and SelumetinibExperimental treatment 2 interventions

Trial outcomes

Primary outcomes

1

The number of treated patients with adverse events as determined by the common criteria for adverse version 5 (CTCAEv5).

We will be looking at safety and dose recommendations using adverse event evaluation per dose level as determined by CTCAEv5 (Part A)

Time frame
15 months
2

Tumor response by imaging using RECISTv1.1

We will determine the clinical benefit of this combination by evaluating tumor response by imaging using RECISTv1.1 guidelines (Part B)

Time frame
28 months

Secondary outcomes

1

Percent apoptosis and tumor proliferation

To evaluate the percent apoptosis and tumor proliferation in resected tumor tissue ANNUBP after exposure to selumetinib and APG-115 (Part C)

Time frame
12 months

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

AeRang Kim

Lead sponsor

Children's National Research Institute

Sponsor institution

Children's National Research Institute

Collaborator

Source ClinicalTrials.gov
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