SARC031: MEK Inhibitor Selumetinib (AZD6244) in Combination With the mTOR Inhibitor Sirolimus for Patients With Malignant Peripheral Nerve Sheath Tumors

Phase 2

Waitlist Available

Led By Brigitte Widemann, MD

Research Sponsored by Sarcoma Alliance for Research through Collaboration

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Timeline

Screening 3 weeks

Treatment Varies

Follow Up progression-free survival (pfs) is defined as the duration of time from the start of treatment to the time of objective progression or death. [time frame: up to 4 years]

Awards & highlights

No Placebo-Only Group

Summary

This trial tests a combination of two oral medications, selumetinib and sirolimus, for patients with severe NF1-related or sporadic tumors that cannot be removed by surgery or have spread. The drugs work together to block cancer growth signals and stop cell multiplication.

Eligible Conditions

Malignant Peripheral Nerve Sheath Tumor
Neurofibromatosis

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ progression-free survival (pfs) is defined as the duration of time from the start of treatment to the time of objective progression or death. [time frame: up to 4 years]

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~progression-free survival (pfs) is defined as the duration of time from the start of treatment to the time of objective progression or death. [time frame: up to 4 years]

This trial's timeline: 3 weeks for screening, Varies for treatment, and progression-free survival (pfs) is defined as the duration of time from the start of treatment to the time of objective progression or death. [time frame: up to 4 years] for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Clinical benefit rate of selumetinib in combination with sirolimus in patients with unresectable or metastatic NF1 associated or sporadic MPNST.

Secondary study objectives

Assess the impact on pain interference

Pain

Define and describe the toxicities of selumetinib in combination with sirolimus in patients with unresectable or metastatic NF1 associated or sporadic MPNST.

+1 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Selumetinib and SirolimusExperimental Treatment2 Interventions

A Simon's two-stage phase 2 trial of MEK inhibitor selumetinib in combination with the mTOR inhibitor sirolimus to determine the safety and clinical benefit in patients with unresectable or metastatic MPNSTs. Both agents will be given orally on an empty stomach. Selumetinib will be given orally at a dose of 50mg twice daily continuously. Sirolimus will be given orally at a dose of 4mg once daily with a cycle 1 day 1 loading dose of 12mg. Each cycle will be considered 28 days.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Selumetinib

2010

Completed Phase 2

~2080

Sirolimus

2013

Completed Phase 4

~2750