In breve
Questo articolo riassume i trial clinici che studiano Selpercatinib. I trial valutano soprattutto efficacia, sicurezza e benefici in persone con tumori solidi avanzati o metastatici, spesso con alterazioni RET. Le popolazioni includono adulti, bambini e pazienti con tumori specifici come polmone, tiroide e pancreas.
Punti chiave
- Selpercatinib is being studied in clinical trials for several cancer types, especially RET-related tumors. The main areas are advanced or metastatic non-small cell lung cancer, RET-mutant medullary thyroid cancer, pediatric RET-altered tumors, and other solid tumors with RET changes. Most studies are Phase 1, Phase 1/2, or Phase 3, so they are looking at both safety and how well the treatment works. The trials compare Selpercatinib with standard treatments, placebo, or other targeted drugs in different patient groups. Key measures include progression-free survival, overall response rate, event-free survival, and safety outcomes. There is also an expanded access program for patients with advanced solid tumors and activating RET alterations.
Trial overview
The trial data show that Selpercatinib is being studied in several clinical trial settings, mainly for cancers with RET alterations.
The studies include Phase 1, Phase 1/2, Phase 2, and Phase 3 trials, plus an expanded access program that is meant to provide access rather than test a formal endpoint.
Most trials focus on people with advanced, locally advanced, or metastatic cancer, and many require evidence of a RET change in the tumor or blood.
Non-small cell lung cancer studies
One major Phase 3 study, LIBRETTO-431, compares Selpercatinib with platinum-based therapy plus pemetrexed, with or without pembrolizumab, in patients with advanced or metastatic RET fusion-positive non-small cell lung cancer.
The main goal is to compare progression-free survival, which means the time before the cancer gets worse.
Another Phase 3 study, LIBRETTO-432, tests Selpercatinib against placebo after surgery in people with stage II-IIIA RET fusion-positive NSCLC.
This study measures event-free survival, which is the time before the cancer returns, worsens, or another important event happens.
Thyroid cancer studies
Selpercatinib is also being studied in RET-mutant medullary thyroid cancer in a Phase 3 trial that compares it with cabozantinib or vandetanib.
This study looks at progression-free survival by blinded central review, meaning the scans are checked by experts who do not know which treatment the patient received.
The trial includes patients with progressive, advanced disease who have not previously received a kinase inhibitor, which is a type of targeted cancer treatment.
Pediatric and other solid tumor studies
A Phase 1/2 study includes children and teenagers with advanced RET-altered solid tumors or primary central nervous system tumors.
In Phase 1, the study checks safety, including dose-limiting toxicities, which are side effects serious enough to limit treatment.
In Phase 2, the study measures overall response rate, meaning the percentage of patients whose tumors shrink or disappear.
Another large Phase 1/2 study includes patients age 12 years or older with locally advanced or metastatic solid tumors that show a RET gene alteration in the tumor and/or blood.
Its Phase 1 part looks for the maximum tolerated dose and the recommended Phase 2 dose, while Phase 2 measures response by RECIST 1.1 or RANO, depending on the tumor type.
Expanded access and precision medicine programs
The LIBRETTO-201 expanded access program gives Selpercatinib to adults with locally advanced or metastatic solid tumors with activating RET alterations.
This program lists several tumor types, including non-small cell lung cancer, medullary thyroid cancer, colon cancer, breast cancer, pancreatic cancer, papillary thyroid cancer, and other solid tumors with activating RET alterations.
The program does not have endpoints or data analysis, so it is different from a standard efficacy trial.
Selpercatinib also appears in a Phase 2 precision medicine study in advanced pancreatic cancer, where treatment is matched to molecular findings and compared with standard care.
That study measures progression-free survival.
Main endpoints and what they mean
The trial data use several common endpoints, or main results that researchers want to measure.
- Progression-free survival: time without the cancer getting worse.
- Overall response rate: how many patients have a tumor response, such as complete response or partial response.
- Event-free survival: time before the cancer returns or another important event occurs after treatment.
- Dose-limiting toxicities and other safety outcomes: side effects that may limit treatment or show how safe the study treatment is.
- Disease control: in the DRUP trial, this means stable disease at 16 weeks after treatment starts.
Some studies also use blinded independent review or independent review committees to judge scans and responses in a more objective way.
Who can participate
Eligibility depends on the specific trial and cancer type.
Some studies include adults only, such as the expanded access program, while others include children, teenagers, or people age 12 years and older.
Many trials require advanced, metastatic, or locally advanced disease, and several require a RET fusion or another RET alteration in the tumor or blood.