Carcinoid Syndrome Efficacy Study Featuring an Oral Daily Paltusotine Regimen

To join this study, you must be at least 18 years old and willing to follow all study instructions, including keeping a daily diary of your symptoms. You need to have a confirmed diagnosis of carcinoid syndrome that requires treatment. This means you should be experiencing at least one flushing episode per day over a two-week period. If you are currently taking other medicines for carcinoid syndrome, you might need to stop them for a short time before joining the study to see if your symptoms increase.

Your neuroendocrine tumour (the cause of carcinoid syndrome) must be clearly identified and confirmed by looking at tissue samples. It also needs to be stable, meaning it hasn't significantly grown or spread in the six months before the study starts. You cannot participate if you have severe or uncontrolled diarrhoea that isn't due to carcinoid syndrome, or if you need other specific treatments for your carcinoid syndrome symptoms (like telotristat).

Other reasons you might not be able to join include having undergone major surgery recently (within the last 8 weeks) or having received certain cancer treatments in the past few months. If you've had another type of cancer, you would generally need to have been free of it for at least three years, with some exceptions for common, well-treated skin or cervical cancers.

If you decide to take part, the study will involve several stages. First, there's a 'screening period' that could last up to 11 weeks where we confirm you meet all the requirements. After this, you'll enter the main 'treatment period' which lasts for 16 weeks. During this time, you'll be randomly assigned to receive either paltusotine or a placebo (dummy pill) daily. This part is 'double-blinded,' meaning neither you nor your study doctor will know which treatment you are receiving.

If you complete the main treatment period, you'll then have the option to enter an 'open-label extension period' which can last for up to 104 weeks (about two years). In this phase, everyone receives paltusotine, and both you and your doctor will know what treatment you are on. Throughout the study, you'll have regular visits to the clinic for health checks, blood tests, and to discuss your symptoms and any side effects. You'll also be asked to keep a diary of your symptoms. After your treatment ends, there will be a final follow-up period of 4 weeks to ensure your well-being. The total duration of your involvement could range from several months up to about 2.5 years.