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)
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)
The reason for this study is to see if the study drug selpercatinib is safe and more effective compared to a standard treatment in participants with rearranged during transfection (RET)-mutant medullary thyroid cancer (MTC) that cannot be removed by surgery or has spread to other parts of the body. Participants who are assigned to the standard treatment and discontinue due to progressive disease have the option to potentially crossover to selpercatinib.
Adaptive sample size re-estimation will be performed at interim analysis. The sample size could be increased from approximately 250 to 400 depending on the results of interim analysis.
At least 18 years of age (participants as young as 12 years of age will be allowed if permitted by local regulatory authorities).
Histologically or cytologically confirmed, unresectable, locally advanced and/or metastatic MTC and no prior history of treatment with kinase inhibitors for advanced/metastatic disease.
Radiographic progressive disease per Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 at screening compared with a previous image taken within the prior 14 months as assessed by the BICR. Participants with measurable or non-measurable but evaluable disease are eligible; however, participants with non-measurable disease may not have disease limited to bone sites only.
A defined/acceptable RET gene alteration identified in a tumor, germline deoxyribonucleic acid (DNA) or blood sample.
Eastern Cooperative Oncology Group performance status score of 0 to 2.
Adequate hematologic, hepatic, and renal function and electrolytes.
Men and women of childbearing potential must agree to use a highly effective contraceptive method during treatment with study drug and for 4 months following the last dose of study drug.
Ability to swallow capsules.
Exclusion Criteria:
Sacramento, California 95817, United States
Torrance, California 90502, United States
Columbus, Ohio 43210, United States
Liège, 4000, Belgium
Beijing, Beijing Municipality 100730, China
Nanjing, Jiangsu 210000, China
Bordeaux, Aquitaine 33076, France
Clermont-Ferrand, Puy-de-Dôme 63011, France
Ramat Gan, Central District 5265601, Israel
Turin, Piedmont 10126, Italy
Koto, Tokyo 135-8550, Japan
Amsterdam, North Holland 1066 CX, Netherlands
Kielce, Świętokrzyskie Voivodeship 25-734, Poland
Obninsk, Kalužskaja Oblast' 249036, Russia
Seoul, Seoul-teukbyeolsi [Seoul] 3722, South Korea
L'Hospitalet de Llobregat, Catalunya [Cataluña] 8907, Spain