A Phase II Trial to Assess the Safety and Efficacy of BMS-986504 in Unresectable Malignant Peripheral Nerve Sheath Tumor Patients With Homozygous MTAP Deletion
Ankit Mangla, MD
Summary
People who have a cancer called MPNST, or Malignant Peripheral Nerve Sheath Tumor, may be eligible for this study. The purpose of this study is to see if a new medicine called BMS-986504 may work better than other available medicines for people with MPNST. Methylthioadenosine Phosphorylase (MTAP) loss is a gene mutation that some people have. MTAP loss seems to increase the chance that BMS-986504 can kill MPNST cancer cells. People who are missing MTAP from their tumor may be able to enroll in this study. Treating MPNST based on MTAP loss is considered experimental and is not approved by the US Food and Drug Administration (FDA) for determining whether BMS-98650 will be active against cancer. The purpose of this study is to evaluate the safety and effectiveness of BMS-986504 in participants with MPNST.
Description
Malignant Peripheral Nerve Sheath Tumors (MPNSTs) are fast-growing, aggressive cancers that develop in soft tissues. They are the main cause of serious illness and death in people with neurofibromatosis type 1 (NF1), a condition that increases the risk of cancer. MPNSTs are rare, affecting about 1 in 100,000 people. In the United States, about 800 to 1,000 new cases are diagnosed each year. The 5-year survival rate is about 50%. However, this drops to about 20% when the cancer has spread or cannot be removed with surgery. Chemotherapy is often less effective, especially when the tumor cannot b…
Eligibility
- Age range
- 12+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * Male or female participants ≥ 12 years of age at the time of screening. * Ability to understand and willingness to sign documentation of informed consent if ≥ 18 years of age or documentation of assent if 12-17 years of age. * Pathohistological verification of MPNST. * Measurable disease (size of primary tumor and metastatic lesions can be trended by CT or MRI scans). * Unresectable (locally advanced or metastatic) disease. * Confirmation of homozygous MTAP deletion by next generation sequencing * Recovery from the adverse effects of prior therapy at the time of enr…
Interventions
- DrugBMS-986504
Participants will receive 600 milligrams (mg) of BMS-986504 taken orally (by mouth) on Days 1-28 of each 28-day cycle. Treatment will continue until disease progression, unacceptable toxicity, or withdrawal of consent (up to 2 years). Participants who experience dose-limiting toxicity can be transitioned to 400 mg daily, then 200 mg daily of BMS-986504, or taken off the clinical trial. Nine participants will be enrolled in stage 1. If ≥ 1 participant demonstrates a complete or partial response in stage 1, the trial will proceed to stage 2, where an additional eight participants will be enrolled. If there are no responses or significant safety concerns arise, the trial will be halted.
Location
- University Hospitals Seidman Cancer Center, Case Comprehensive Cancer CenterCleveland, Ohio