Phase 3 Study of Milademetan Versus Trabectedin in Liposarcoma
Research type
Research Study
Full title
A Randomized Multicenter Phase 3 Study of Milademetan Versus Trabectedin in Patients with Dedifferentiated Liposarcoma
IRAS ID
300747
Contact name
Robin Jones
Contact email
Sponsor organisation
Rain Therapeutics, Inc.
Eudract number
2021-001394-23
Duration of Study in the UK
2 years, 4 months, 1 days
Research summary
Research Summary:
This is a phase 3, multicenter, randomized, open-label trial designed to investigate the safety and efficacy of milademetan compared to trabectedin in patients with unresectable or metastatic dedifferentiated liposarcoma. If a patient’s disease got worse on 1 or more prior therapies, then they may be eligible for this study.
Participants will have a 50% chance of being assigned to either milademetan or trabectedin. Milademetan is an experimental drug designed to reduce the activity of a cancer-causing protein known as MDM2 and will be administered orally as capsules on Days 1 to 3 and Days 15 to 17 of each 28 day treatment cycle. Trabectedin has already been approved by regulators as a second line of therapy for patients with liposarcoma and will be administered intravenously over 24 hours on Day 1 of each 21-day treatment cycle.
Total time in the study will be determined by how long the participants can safely be treated without serious side effects, by how long the study doctor determines that they are gaining clinical benefit, and by how long they are willing to take part in this study.
The study will consist of:
• Screening Period (anytime during the 14 days prior to being assigned to a treatment group)
• Treatment Period (until disease worsening, unmanageable side effects of a drug or fulfilment of other discontinuation criteria occur)
• End of Treatment visit (30 days (±7 days) after the last dose of study drug or before starting new anti-cancer treatment)
• Follow-up period of (every 12 weeks (±7 days) to check on the participant’s health status)
About 160 patients are expected to participate in this study at 77 study clinics across 15 countries, including the U.K.
Study assessments include physical examinations, blood and urine samples, questionnaires, electrocardiograms and Computed Tomography or Magnetic Resonance Imaging scans to determine disease status.Lay Summary:
Results from this study showed that although participants receiving milademetan performed better as compared to those receiving trabectedin, the study did not meet its primary objective of demonstrating a clinically significant increase in the time between treatment and signs of cancer regrowth or death for milademetan compared to trabectedin. There were also no statistically significant differences in other key treatment effectiveness measures between the milademetan and trabectedin treatment groups in the study.
Safety data showed that milademetan was well-tolerated and had a side effect profile similar to trabectedin, with the most common side effects being gastrointestinal events or reductions in the number of mature blood cells that were generally manageable through dose modification and/or concomitant treatment.
Analysis of the data from this study does not change the overall benefit-risk of assessment of milademetan.REC name
South Central - Hampshire B Research Ethics Committee
REC reference
22/SC/0304
Date of REC Opinion
11 Oct 2021
REC opinion
Further Information Favourable Opinion