Study on the Safety and Effectiveness of BMS-986504 for Treating Unremovable Malignant Peripheral Nerve Sheath Tumor

Sponsor: Ankit Mangla, MD

Sponsor score: 0

ClinicalTrials ID:NCT07549022

Provider View

This clinical trial is testing a new drug, BMS-986504, to see if it can better treat Malignant Peripheral Nerve Sheath Tumor (MPNST) that cannot be surgically removed. The study focuses on patients whose tumors have a specific gene mutation called MTAP loss, which may make the drug more effective.

Patient Parameters

Program Overview

This study is for people with a type of cancer called Malignant Peripheral Nerve Sheath Tumor (MPNST) that cannot be removed by surgery. Researchers are testing a new drug called BMS-986504 to see if it works better than current treatments. The study is particularly interested in patients whose tumors have a gene mutation known as MTAP loss, which might make the drug more effective at killing cancer cells. This approach is experimental and not yet approved by the FDA for treating MPNST.

Description

Malignant Peripheral Nerve Sheath Tumors (MPNSTs) are aggressive cancers that grow in soft tissues and are a major concern for people with neurofibromatosis type 1 (NF1), a condition that increases cancer risk. MPNSTs are rare, with about 800 to 1,000 new cases in the U.S. each year. The survival rate is about 50% over five years, but drops to 20% if the cancer spreads or can't be surgically removed. Current treatments, like chemotherapy, often don't work well, especially when surgery isn't an option.

MPNSTs often develop from benign nerve tumors called neurofibromas, due to the loss of certain genes on chromosome 9, including MTAP. MTAP is missing in 25-50% of MPNST cases and plays a role in important cell processes. When MTAP is lost, cancer cells become more dependent on another pathway involving an enzyme called PRMT5. Blocking PRMT5 can kill MTAP-deficient cancer cells, a concept known as "synthetic lethality." BMS-986504 is a drug designed to target these MTAP-deficient cells by inhibiting PRMT5.

In a recent Phase 1 trial, BMS-986504 showed promise in shrinking tumors in patients with MTAP-deficient MPNST that couldn't be surgically removed. This study aims to further evaluate the safety and effectiveness of BMS-986504 in such patients, hoping to improve survival and health outcomes.

Eligibility Criteria

Inclusion Criteria

  • Participants must be 12 years or older.
  • Must understand and agree to the study, with consent from a guardian if under 18.
  • Must have a confirmed diagnosis of MPNST.
  • Must have tumors that can be measured by scans and cannot be surgically removed.
  • Must have a specific gene mutation (homozygous MTAP deletion) confirmed by genetic testing.
  • Must have recovered from previous treatments to a certain level of health.
  • Must have normal blood cell counts and organ function.
  • Must be in good overall health, with specific performance status scores.
  • Women and men of childbearing potential must use effective birth control during the study.

Exclusion Criteria

  • Previous treatment with a PRMT5 inhibitor.
  • Inability to swallow tablets.
  • History of certain digestive diseases or surgeries that affect medication absorption.
  • Active drug use.
  • Use of herbal supplements or traditional medicines for the disease within 4 weeks before the study.
  • Need for certain medications that cannot be changed before the study.

Locations

FACILITYZIPCITYSTATE
University Hospitals Seidman Cancer Center, Case Comprehensive Cancer Center44106ClevelandOhio

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