Fimepinostat for Brain Cancer

(PNOC016 Trial)

Recruiting at 19 trial locations

Overseen By Sabine Mueller, MD, PhD, MAS

Age: < 65

Sex: Any

Trial Phase: Phase < 1

Sponsor: Sabine Mueller, MD, PhD

Must not be taking: HDAC inhibitors, PI3K inhibitors

Disqualifiers: Diabetes, HIV, Cardiac arrhythmia, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 31

30 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial studies how well fimepinostat works in treating patients with newly diagnosed diffuse intrinsic pontine glioma, or medulloblastoma, or high-grade glioma that have come back. Fimepinostat may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications. However, if you are on corticosteroids like dexamethasone, you must be on a stable or decreasing dose for at least 7 days before joining. It's best to discuss your specific medications with the study team.

Research Team

Sabine Mueller, MD, PhD, MAS

Principal Investigator

University of California, San Francisco

Eligibility Criteria

This trial is for children and young adults with specific brain tumors (like diffuse intrinsic pontine glioma or high-grade glioma) that are newly diagnosed or have returned. Participants must be able to swallow medication, have recovered from previous cancer treatments, meet certain health criteria including controlled seizures if present, agree to use contraception, and understand the consent form.

Inclusion Criteria

Your corrected QT interval is less than 480 milliseconds.

My kidney and liver functions meet the required levels for my age and gender.

I can swallow pills without needing to crush or chew them.

Exclusion Criteria

You have had an allergic reaction to drugs similar to fimepinostat.

I do not have any serious illnesses or social situations that would stop me from following the study's requirements.

I have not been treated with HDAC or PI3K inhibitors before.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Initial Treatment

Participants receive fimepinostat orally once daily on days -2 to 0, followed by tumor resection or biopsy on day 0

3 days

1 visit (in-person) for surgery or biopsy

Maintenance

Participants receive fimepinostat by mouth, once daily for days 1-5 each week. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity

12-24 months

Follow-up

Participants are monitored for safety and effectiveness after treatment completion

5 years

Follow-up at 30 days, then every 3 months

Treatment Details

Interventions

• Fimepinostat
• Therapeutic Conventional Surgery

Trial Overview The trial is testing fimepinostat's effectiveness on certain brain tumors by blocking enzymes needed for tumor cell growth. It includes patients who can take the drug orally and have met recovery benchmarks from prior treatments. The study also involves standard care tumor tissue collection.

Participant Groups

Group I: Treatment (fimepinostat, tumor resection) Experimental Treatment 2 Interventions

Patients receive fimepinostat by mouth once daily, on Days -2 to 0. Within 2 hours of receiving fimepinostat on Day 0, patients undergo tumor resection or biopsy as part of their standard of care. MAINTENANCE PHASE: Patients receive fimepinostat by mouth, once daily for days 1-5 each week. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity for up to 12 months from the time treatment begins. Should patients continue to derive clinical benefit, and not experience excess toxicity or progression, patients can continue to receive drug for up to 24 months or longer pending discussion with study chairs and study sponsor.

Who Is Running the Clinical Trial?

Sabine Mueller, MD, PhD

Lead Sponsor

Trials

9

Recruited

440+

Cannonball Kids' Cancer Foundation

Collaborator

Trials

6

Recruited

140+

Pediatric Neuro-Oncology Consortium

Collaborator

Trials

4

Recruited

130+

Pacific Pediatric Neuro-Oncology Consortium

Collaborator

Trials

16

Recruited

840+

Cannonball Kids' Cancer Foundation

Collaborator

Trials

4

Recruited

110+

Curis, Inc.

Industry Sponsor

Trials

17

Recruited

1,100+