Safety and Efficacy of BMS-986504 in Unresectable Malignant Peripheral Nerve Sheath Tumor
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age12+
SponsorAnkit Mangla, MD
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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).
Disqualifiers
Participants who have been treated previously with a PRMT5 inhibitor.
Participants who are unable to swallow tablets.
History of gastrointestinal disease, inflammatory bowel disease, major gastric surgery or other gastrointestinal conditions (eg, uncontrolled nausea, vomiting, malabsorption syndrome) likely to alter absorption of study treatment or result in inability to swallow oral medications.
Participants with active drug use.
Trial design
Design model
Single group
Treatments tested in this trial
BMS-986504
DrugParticipants 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.
Treatment groups
17 Participants
are divided into 1 treatment groupGroup A: BMS-986504Experimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Objective response rate (ORR)
ORR is defined as the proportion of participants with a confirmed complete response (CR) or partial response (PR) per Response Evaluation Criteria in Solid Tumors (RECIST) guideline (version 1.1). CR is defined as disappearance of all target lesions. PR is defined as at least a 30% decrease in the sum of diameters of target lesions.
Time frame
Up to 2 yearsSecondary outcomes
1
Progression free survival (PFS)
Progression-Free Survival (PFS) is defined as the duration of time from start of treatment to time of progression or death, whichever occurs first.
Time frame
Up to 2 years2
Overall survival (OS)
OS is defined as the time from treatment initiation to death from any cause.
Time frame
Up to 2 years3
Incidence of adverse events (AEs)
AEs will be defined and graded per the National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0 (CTCAE v5.0).
Time frame
30 days post-treatment discontinuation (up to 2 years)4
Rate of treatment discontinuation
Proportion of participants discontinuing treatment due to toxicity will be calculated.
Time frame
Up to 2 yearsSponsors and contacts
Click on the lead sponsor to view all of their trials.
Ankit Mangla, MD
Lead sponsor
Case Comprehensive Cancer Center
Sponsor institution
Source ClinicalTrials.gov