PLX3397 + Sirolimus for Sarcoma
(PLX3397 Trial)

Recruiting in Palo Alto (17 mi)

+4 other locations

Overseen byGulam A. Manji, MD

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: Gulam Manji

No Placebo Group

Breakthrough Therapy

Trial Summary

What is the purpose of this trial?

The purpose of this study is to determine if treatment with PLX3397 and Sirolimus will be tolerated and result in shrinking of the cancer or stopping the cancer from growing. In the phase I portion, the maximum tolerate dose of the study drug will be determined. In the Phase II portion, progression free survival will be assessed at the dose level found in Phase I. Participants will continue to take the study drug until they experience an unacceptable side effect or their disease progresses. Funding Source - FDA OOPD

Research Team

Gulam A. Manji, MD

Principal Investigator

Columbia University

Eligibility Criteria

Adults with advanced, unresectable sarcoma or malignant peripheral nerve sheath tumors (MPNSTs) who have had up to three prior treatments. Participants must be able to undergo tumor biopsies, not be pregnant or breastfeeding, and agree to use effective contraception. They should have a performance status allowing daily activity and measurable disease.

Inclusion Criteria

I have had up to 3 treatments for my MPNST, but no radiotherapy.

I am 18 years old or older.

My condition worsened after up to three standard treatments.

See 15 more

Exclusion Criteria

I am not taking strong drugs that affect liver enzyme activity.

Pregnant women

Baseline QTc corrected by Fridericia's formula (QTcF) ≥ 450 ms (males) or ≥ 470 ms (females)

See 14 more

Treatment Details

Interventions

PLX3397 (Tyrosine Kinase Inhibitor)
Sirolimus (mTOR Inhibitor)

Trial OverviewThe trial is testing the combination of PLX3397 and Sirolimus for treating certain types of sarcomas that cannot be surgically removed. The first phase determines the highest dose patients can tolerate without severe side effects; the second phase checks if this dose can stop cancer growth.

Participant Groups

2Treatment groups

Experimental Treatment

Group I: Phase 2: PLX3397 and SirolimusExperimental Treatment2 Interventions

Subjects with unresectable or metastatic Malignant Peripheral Nerve Sheath Tumors (MPNSTs) will take PLX3397 and Sirolimus at the recommended Phase 2 dose (RP2D).

Group II: Phase 1: PLX3397 and SirolimusExperimental Treatment2 Interventions

Subjects with unresectable or metastatic sarcoma will take orally PLX3397 (600 - 1000mg) in combination with Sirolimus (2-6mg) daily.