SARC031: MEK Inhibitor Selumetinib (AZD6244) in Combination With the mTOR Inhibitor Sirolimus for Patients With Malignant Peripheral Nerve Sheath Tumors
Trial Summary
What is the purpose of this trial?
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.
Will I have to stop taking my current medications?
The trial protocol does not specify if you must stop all current medications, but you cannot take other anti-cancer agents or certain medications that affect specific enzymes and transporters. There are also restrictions on using certain foods and supplements like grapefruit and St. John's Wort.
What data supports the effectiveness of the drug Selumetinib?
What safety data exists for Selumetinib (also known as AZD6244, ARRY-142886) in humans?
Selumetinib has been studied in healthy volunteers and patients with various cancers, showing it is generally well-tolerated, but long-term safety data is not fully described. It has been tested in combination with other treatments, and while it shows promise, the full range of potential side effects over time is still being researched.12678
How is the drug Selumetinib unique compared to other treatments?
Selumetinib is unique because it is an oral drug that specifically targets and inhibits MEK1/2, which are proteins involved in cell growth and survival, making it potentially effective for various tumor types. This targeted approach is different from traditional chemotherapy, which affects both cancerous and healthy cells.12369
Research Team
Brigitte Widemann, MD
Principal Investigator
National Cancer Institute (NCI)
AeRang Kim, MD, PhD
Principal Investigator
Children's National Research Institute
Eligibility Criteria
Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive selumetinib and sirolimus orally, with selumetinib given at 50mg twice daily and sirolimus at 4mg once daily, with a cycle 1 day 1 loading dose of 12mg. Each cycle is 28 days.
Follow-up
Participants are monitored for safety, effectiveness, and progression-free survival after treatment
Treatment Details
Interventions
- Selumetinib
- Sirolimus
Selumetinib is already approved in United States, European Union for the following indications:
- Neurofibromatosis type 1 (NF1) in pediatric patients 2 years of age and older with symptomatic, inoperable plexiform neurofibromas (PN)
- Neurofibromatosis type 1 (NF1) in pediatric patients 2 years of age and older with symptomatic, inoperable plexiform neurofibromas (PN)
Find a Clinic Near You
Who Is Running the Clinical Trial?
Sarcoma Alliance for Research through Collaboration
Lead Sponsor
AstraZeneca
Industry Sponsor
Sir Pascal Soriot
AstraZeneca
Chief Executive Officer since 2012
Veterinary Medicine from École nationale vétérinaire d'Alfort, MBA from HEC Paris
Dr. Cristian Massacesi
AstraZeneca
Chief Medical Officer since 2021
MD from Marche Polytechnic University, Oncology training at Royal Marsden Hospital, Kaplan Comprehensive Cancer Center, and European Institute of Oncology
Pascal Soriot
AstraZeneca
Chief Executive Officer since 2012
Veterinary Medicine from École nationale vétérinaire d'Alfort, MBA from HEC Paris
Cristian Massacesi
AstraZeneca
Chief Medical Officer since 2021
MD from Marche Polytechnic University, Medical Oncology training at Royal Marsden Hospital, Kaplan Comprehensive Cancer Center, and European Institute of Oncology
United States Department of Defense
Collaborator