Carcinoid Syndrome Efficacy Study Featuring an Oral Daily Paltusotine Regimen
Research type
Research Study
Full title
A Randomized, Parallel Group, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Paltusotine in Adults with Carcinoid Syndrome due to Well-Differentiated Neuroendocrine Tumors
IRAS ID
1011549
Contact name
Shilpa Kulkarni
Contact email
Sponsor organisation
Crinetics Pharmaceuticals, Inc.
Eudract number
2024-519875-24
Research summary
This is a worldwide study designed to test how well paltusotine works and how safe it is for adults with carcinoid syndrome. Paltusotine is a medication that has been tested in several clinical studies to treat different health conditions, including acromegaly (a hormonal disorder) and carcinoid syndrome (a condition caused by rare tumors). In this Phase 3 study, researchers are comparing 80 mg of paltusotine daily to placebo (a pill with no active drug) to confirm how well it works and how safe it is for long-term use in participants with carcinoid syndrome. The study is comparing how well paltusotine reduces daily flushing and diarrhea compared to the placebo, while also checking its overall safety over a longer period of time. The study has four periods: Screening Period: Lasts up to 11 weeks to check if participants qualify. Double-Blind Period: Lasts 16 weeks, where neither participants nor researchers know who is receiving paltusotine or the placebo. Open-Label Extension Period (OLE): Lasts 104 weeks (about 2 years), where everyone gets paltusotine to study its long-term effects. Follow-Up Period: Lasts 4 weeks to monitor participants after stopping the treatment. Total Study Duration: Lasts up to 135 weeks (about 2.6 years). To participate in the study, participants must be: Adults aged 18 years or older with confirmed diagnosis of carcinoid syndrome requiring medical therapy. Participants must have symptoms of flushing (with or without frequent diarrhea).•Specific type of tumor (neuroendocrine) grade 1 or 2 , not gotten worse in past 6 months. This study is expected to benefit the Sponsor by providing information about how paltusotine works in individuals living with carcinoid syndrome. If the results of this and other studies are favorable, and if regulatory authorities approve paltusotine for the treatment of carcinoid syndrome, there may be benefit for patients in the future.
REC name
Wales REC 5
REC reference
25/WA/0123
Date of REC Opinion
28 Jul 2025
REC opinion
Further Information Favourable Opinion