Selpercatinib vs Cabozantinib/Vandetanib in MTC (LIBRETTO-53)
Research type
Research Study
Full title
A Multicenter, Randomized, Open-label, Phase 3 Trial Comparing Selpercatinib to Physicians Choice of Cabozantinib or Vandetanib in Patients with Progressive, Advanced,Kinase Inhibitor Naïve, RET-Mutant Medullary Thyroid Cancer (LIBRETTO-531)
IRAS ID
271871
Contact name
Rosemary Derbyshire
Contact email
Sponsor organisation
Eli Lilly and Company
Eudract number
2019-001978-28
Duration of Study in the UK
5 years, 11 months, 0 days
Research summary
Medullary thyroid cancer (MTC) is a rare type of thyroid cancer that accounts for between 5 to 9% of all thyroid cancers. For most, the cause of MTC is unknown, but roughly 10% are hereditary (genetically transmitted) due to mutation in the RET gene. Majority of non-hereditary MTCs are caused by sporadic RET mutations. The outcome of MTC is highly variable, from tumours that remain unchanged for years to aggressive cancers that can be fatal. Surgery can be curative for most patients. For those with recurrent MTC, after surgery or with cancer spreading outside the thyroid, the current standard treatment is cabozantinib or vandetanib. These drugs have side effects that often lead patients to take lower than recommended dose or to stop taking them.
Selpercatinib is an oral drug currently in clinical development aiming to decrease RET activity in tumours with RET mutations or other genetic changes. Earlier studies of the drug showed manageable side effects and evidence of long-lasting anti-tumour activity in patients with advanced MTC. Selpercatinib may slow or stop the growth of the cancer. It may also help to shrink the cancer.
J2G-MC-JZJB is a randomized trial where both the investigators and participants know which treatment is being administered. There will be approximately 400 patients globally participating in the study, including approximately 8 in the UK. Patients with RET-mutant MTC have a chance to receive either selpercatinib or standard treatment. If the patient is randomized to receive standard treatment, the investigator will decide if cabozantinib or vandetanib is used. Patients randomized to standard treatment whose disease progressed will be allowed to switch treatment to selpercatinib. The main aim of this study is to confirm if selpercatinib is of benefit in patients with advanced/metastatic MTC and to better understand this benefit in the contexts of other available treatments.
REC name
West Midlands - Coventry & Warwickshire Research Ethics Committee
REC reference
20/WM/0056
Date of REC Opinion
6 Apr 2020
REC opinion
Further Information Favourable Opinion