NCT03761095

A Study of Unesbulin (PTC596) in Combination With Dacarbazine in Participants With Advanced Leiomyosarcoma (LMS)

A Phase 1B Study of Unesbulin (PTC596) in Combination With Dacarbazine in Patients With Locally Recurrent, Unresectable or Metastatic Relapsed/Refractory Leiomyosarcoma

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 41 (actual)
• Sponsor: PTC Therapeutics · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The primary objective of this study is to determine the maximum tolerated dose (MTD) and recommended phase 2 dose (RP2D) of unesbulin in combination with dacarbazine for the treatment of advanced LMS and determine the overall safety profile of unesbulin in combination with dacarbazine. This study will employ the time-to-event continual reassessment method (TITE-CRM) for dose finding. Treatment will be initiated at dose level 2 (DL2) (Dacarbazine 1000 milligrams per square meter \[mg/m\^2\] intravenously \[IV\] every 21 days in combination with unesbulin 200 milligrams \[mg\] orally twice weekly) for the first participant. This dose level represents the investigator's best assessment of the MTD based on available toxicity data for both agents. For subsequent participants, the dose level at which treatment is initiated will be selected based on the TITE-CRM using the most up to date dose-limiting toxicity (DLT) information from all participants previously treated. To enroll additional participants at the RP2D, the study is amended to include an expansion cohort of up to 12 participants (some of whom could be ongoing participants who reconsent). Treatment will continue for each participant until evidence of unacceptable toxicity, disease progression, or treatment discontinuation for another reason.

Conditions

• Leiomyosarcoma

Interventions

Type	Name	Description
DRUG	Unesbulin	Unesbulin will be administered as per the dose and schedule specified in the arm.
DRUG	Dacarbazine	Dacarbazine will be administered as per the dose and schedule specified in the arm.

Timeline

• Start date: 2019-03-13
• Primary completion: 2024-02-05
• Completion: 2024-02-05
• First posted: 2018-12-03
• Last updated: 2024-03-15

Locations

4 sites across 1 country: United States

Regulatory

• FDA-regulated drug study