Table of contents
- Clinical trials overview
- Lung cancer studies
- Thyroid cancer studies
- Pediatric and solid tumor studies
- Access and precision medicine studies
- Trial endpoints and measures
Clinical trials overview
These studies are testing Selpercatinib in people with cancers that have RET changes, which are gene changes that may help drive tumor growth.[1] The trials include adults, children, and teenagers, depending on the study.[2] The phases range from Phase 1 to Phase 3, so the research includes early safety studies and later comparison studies.[2][3]
Lung cancer studies
One Phase 3 study, LIBRETTO-431, compares Selpercatinib with platinum-based therapy, pemetrexed, and with or without pembrolizumab in advanced or metastatic RET fusion-positive non-small cell lung cancer.[1] The main endpoint is progression-free survival, which means how long the cancer stays from getting worse.[1]
Another Phase 3 study, LIBRETTO-432, looks at adjuvant Selpercatinib in stage II-IIIA RET fusion-positive NSCLC after surgery and compares it with placebo.[4] Its main endpoint is event-free survival, which measures how long patients stay free from a cancer-related event such as recurrence or worsening.[4]
The Phase 1/2 study in advanced solid tumors also includes people age 12 years or older with a RET gene alteration in the tumor or blood, and it measures safety first and then tumor response.[3] In Phase 2, the study looks at objective response rate, meaning how many tumors shrink or disappear.[3]
Thyroid cancer studies
A Phase 3 trial is comparing Selpercatinib with cabozantinib or vandetanib in progressive, advanced, kinase inhibitor-naive RET-mutant medullary thyroid cancer.[5] The main endpoint is progression-free survival by blinded independent central review, which means experts who are not part of the treatment team review the scans and results.[5]
Pediatric and solid tumor studies
The pediatric Phase 1/2 study includes children with advanced RET-altered solid tumors or primary central nervous system tumors.[2] Phase 1 checks the frequency, severity, and relatedness of treatment-emergent adverse events and serious adverse events, including dose-limiting toxicities.[2] A dose-limiting toxicity is a side effect that is serious enough to stop dose increases or limit treatment.[2]
In Phase 2 of that pediatric study, the main goal is objective response rate, using RECIST 1.1 or RANO depending on tumor type.[2] RECIST is a method for measuring solid tumors on scans, while RANO is used for brain tumors and other nervous system tumors.[2]
Access and precision medicine studies
The LIBRETTO-201 expanded access program gives access to Selpercatinib for adults with locally advanced or metastatic solid tumors that have activating RET alterations.[6] This study does not have endpoints or data analysis because it is an expanded access program, not a standard efficacy trial.[6]
Two large precision medicine studies also list Selpercatinib among many targeted treatments.[7][8] One is in cancer patients in Norway and looks at whether treatment matches the molecular tumor profile and at serious treatment-related adverse events.[7] The other is the DRUP trial, which studies several tumor types and measures tumor response, disease control, and treatment-related grade 3 or higher and serious adverse events.[8]
Trial endpoints and measures
Across the Selpercatinib trials, the most important endpoints are progression-free survival, event-free survival, objective response rate, and safety measures such as serious adverse events and dose-limiting toxicities.[1][2][3][4][5][7][8]
Some trials compare Selpercatinib with standard treatment, such as chemotherapy or other targeted drugs, while others compare it with placebo or study it in access programs.[1][4][5][6]
