Phase 2/3 Study to Evaluate Efficacy and Safety of Unesbulin in Leiomyosarcoma
Research type
Research Study
Full title
A PHASE 2/3 STUDY TO EVALUATE THE EFFICACY AND SAFETY OF UNESBULIN IN UNRESECTABLE OR METASTATIC, RELAPSED OR REFRACTORY LEIOMYOSARCOMA
IRAS ID
1005169
Contact name
Mona Wahba
Contact email
Sponsor organisation
PTC Therapeutics, Inc.
Eudract number
2022-000073-12
Clinicaltrials.gov Identifier
Research summary
Leiomyosarcoma is a rare soft tissue cancer that spreads through the bloodstream and grows within the smooth muscle, commonly found in the abdomen or uterus. Current treatments are limited and involve surgery or chemotherapy if it is inoperable or has spread to multiple locations.
PTC596-ONC-008-LMS is a phase 2/3 study to test the effectiveness and safety of unesbulin with dacarbazine for patients with relapsed, unresectable disease or unresponsive to current treatments. The study drug, unesbulin is a novel molecule that disrupts an important binding site involved in the tumour cell’s cytoskeletal structure resulting in cell death, slowing the cancer progression. Dacarbazine is a chemotherapeutic agent (anti-cancer drug) that has shown efficacy in this disease population. The preclinical data of unesbulin in combination with dacarbazine has shown more effective tumour suppression than either drug alone.
Approximately 345 participants will be enrolled into this international, multicentre, randomised, double-blind, placebo-controlled study. Eligible participants will have history of previous drug therapy (300 participants with up to 3 and 45 participants with at least 4 systemic therapies). The participants will be randomly divided into two groups in 2:1 ratio: Unesbulin in combination with Dacarbazine and Placebo in combination with Dacarbazine respectively.
The study is designed as 21 day treatment cycles. Participants will be take either the study drug or placebo orally without food twice weekly. Participants in all treatment groups will be administered dacarbazine using an IV once every 21 days. The study period will be approximately 4 years.
The primary objective is to compare progression free survival (length of time during and after treatment without the cancer getting worse) of unesbulin plus dacarbazine versus placebo plus dacarbazine.
The study is sponsored by PTC Therapeutics Inc.REC name
North East - Newcastle & North Tyneside 2 Research Ethics Committee
REC reference
22/NE/0089
Date of REC Opinion
5 Jul 2022
REC opinion
Further Information Favourable Opinion