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

Part of paid clinical trials in Washington D.C., District of Columbia.

Sponsor
Sarcoma Alliance for Research through Collaboration
Study ID
NCT03433183
Phase
PHASE2
Status
Completed

Conditions

  • Malignant Peripheral Nerve Sheath Tumors
  • Neurofibromatosis 1

Eligibility Criteria

Sex
ALL
Age
12 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • Selumetinib — DRUG
    Selumetinib (AZD6244) is an oral selective inhibitor of the mitogen-activated protein kinase (MEK) 1/2 currently in development for adult malignancies, pediatric low-grade gliomas and NF1 plexiform neurofibromas. MEK is a critical kinase in the mitogen activated protein (MAP) kinase signal transduction pathway for many growth factor receptors that provide growth signals to cancer cells.
  • Sirolimus — DRUG
    Sirolimus is a macrocyclic lactone produced by Streptomyces hygroscopicus and inhibitor of mammalian Target of Rapamycin (mTOR) serine threonine kinase, which plays a critical role in regulating cellular energy sensing, growth and metabolism.

Study Details

To determine the clinical benefit rate of selumetinib in combination with sirolimus in patients with unresectable or metastatic neurofibromatosis type 1 (NF1) associated or sporadic MPNST.

Key Dates

Start date
Oct 2, 2019
Status verified
Apr 2026
Primary completion
Jun 22, 2022
Completion
Oct 1, 2023

Study Design

Enrollment
21 participants (actual)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Selumetinib and Sirolimus
    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.

Primary Outcome Measure

Clinical Benefit Rate of Selumetinib in Combination With Sirolimus in Patients With Unresectable or Metastatic NF1 Associated or Sporadic MPNST. [ Time Frame: Up to 6 months ]

Locations (5)

FacilityCityStateZIPSite coordinators
Children's National Medical CenterWashington D.C.District of Columbia20010-
Johns Hopkins UniversityBaltimoreMaryland21287-
National Cancer InstituteBethesdaMaryland20892-
Dana Farber Cancer InstituteBostonMassachusetts02215-
Washington UniversitySt LouisMissouri63110-

Find similar trials in Washington D.C., DC

By research site
Children's National Medical Center· Washington D.C., DCJohns Hopkins University· Baltimore, MDNational Cancer Institute· Bethesda, MDDana Farber Cancer Institute· Boston, MAWashington University· St Louis, MO

Related Studies