Lori Wirth, MD, affiliate professor, drugs, Harvard Medical College; medical director, Heart for Head and Neck Cancers; Massachusetts Normal Hospital, discusses the section 1/2 LIBRETTO-001 trial (NCT03157128) investigating selpercatinib (Retevmo) in grownup and pediatric sufferers a minimum of 2 years of age with superior or metastatic RET fusion–constructive thyroid most cancers who require systemic remedy and who’re radioactive iodine (RAI) refractory, if RAI is acceptable.
This dialog comes after the June 2024 FDA full approval of selpercatinib for this affected person inhabitants. LIBRETTO-001 demonstrated that sufferers who acquired prior remedy (n = 41) achieved an general response price (ORR) of 85% (95% CI, 71%-94%), and those that had been naive to systemic remedy (n = 24) skilled an ORR of 96% (95% CI, 79%-100%) when handled with selpercatinib.
LIBRETTO-001 was a primary in-human trial that evaluated selpercatinib in sufferers with RET-driven cancers and included a number of totally different cohorts, Wirth begins. One cohort comprised sufferers with non–small cell lung most cancers who had RET fusion–constructive illness and had beforehand been handled with different therapies. Moreover, there have been cohorts of sufferers with RET fusion–constructive thyroid most cancers who had acquired prior multi-kinase inhibitor remedy, she explains.
Furthermore, the trial included a cohort of sufferers with RET fusion–constructive medullary thyroid most cancers who had been remedy naive, Wirth continues. Sufferers with RET fusion–constructive follicular-derived thyroid most cancers had been additionally enrolled, together with these with different RET-driven cancers, she elucidates, including that the preliminary section of the trial targeted on dose discovering, which was essential for figuring out the optimum dosage of selpercatinib.
The first finish level at this stage was to evaluate the protection profile of selpercatinib and set up the beneficial section 2 dose, Wirth stories. Upon transitioning to the enlargement section, the research evaluated further finish factors, similar to ORR, period of response, progression-free survival, and different necessary measures of medical efficacy, she concludes.
