Ongoing Clinical Trials for Carcinoid Syndrome
There are currently 2 clinical trials investigating new treatments for carcinoid syndrome, focusing on an investigational oral medication called paltusotine. These trials are taking place across several European countries including Italy, Poland, Spain, and France, offering patients opportunities to access potential new treatment options for managing symptoms such as flushing and frequent bowel movements.
Clinical trial locations
- France
- Italy
- Poland
- Spain
Efficacy and Safety Study of Paltusotine for Adults with Carcinoid Syndrome due to Well-Differentiated Neuroendocrine Tumors
This trial is investigating whether paltusotine, a daily oral medication, can help reduce the symptoms of carcinoid syndrome in adults with well-differentiated neuroendocrine tumors. The condition occurs when these tumors release certain substances into the bloodstream, causing symptoms like flushing (sudden reddening of the skin, especially on the face) and frequent bowel movements.
Main Focus: The primary goal is to evaluate whether paltusotine can reduce the number of flushing episodes experienced by patients. The study will also assess whether the medication affects bowel movement frequency and examine its overall safety profile. The treatment period lasts 12 weeks, during which participants will be compared to those receiving a placebo (inactive treatment).
Investigational Drug: Paltusotine is taken as a 40 mg tablet once daily. It works by blocking specific hormone receptors involved in the syndrome, similar to other medications in the somatostatin receptor ligand class. Some participants currently taking other similar medications (octreotide or lanreotide) will need to stop those treatments before starting the study.
Who Can Participate: You may be eligible if you are 18 years or older with documented carcinoid syndrome requiring medical therapy. You must have well-differentiated neuroendocrine tumors that are Grade 1 or Grade 2, with evidence of positive somatostatin receptor status on imaging scans. Participants must experience more than one flushing episode per day on average over a 14-day period. You’ll need to keep a daily symptom diary throughout the study and be willing to attend regular study visits for monitoring, which may include blood tests and CT or MRI scans.
Who Cannot Participate: You are not eligible if you have a history of heart valve damage, uncontrolled high blood pressure, significant heart problems, liver or severe kidney disease, or if you are on dialysis. Pregnant or breastfeeding women cannot participate. You also cannot join if you’ve had major surgery within 2 months before the study, have been diagnosed with cancer within the last 5 years (except certain skin cancers), or are currently taking glucocorticoid medications. Recent participation in another clinical trial within 30 days or known allergies to the study medication would also exclude you.
Study on the Safety and Effects of Paltusotine and Octreotide Acetate for Patients with Carcinoid Syndrome
This clinical trial is evaluating paltusotine at different dose levels to understand how the body processes the medication and to assess its safety in people with carcinoid syndrome. The condition causes symptoms like flushing, diarrhea, and wheezing due to hormones released by neuroendocrine tumors.
Main Focus: The study aims to gather information on the safety and tolerability of paltusotine at varying doses (40 mg, 80 mg, or 120 mg per day) and how the body processes the medication. Researchers will also compare it with octreotide acetate, an injectable medication that is a synthetic version of a natural hormone used to control symptoms. The study will monitor participants carefully through regular check-ups, laboratory tests, and heart monitoring to ensure safety.
Investigational Drug: Paltusotine is an oral medication taken as a tablet, also known by its code name CRN00808. It is a non-peptide somatostatin receptor agonist, meaning it mimics the action of somatostatin, a hormone that regulates various bodily functions. The trial will test three different daily doses to find the best balance between effectiveness and minimal side effects.
Who Can Participate: You must be at least 18 years old and have documented carcinoid syndrome requiring medical treatment, along with a well-differentiated neuroendocrine tumor that is Grade 1 or Grade 2. You need to be willing to provide written consent and follow study procedures, including maintaining an electronic symptom diary at least 70% of the time during specific periods. If you’re currently taking medications like lanreotide or octreotide, you must be willing to stop these medications and show worsening symptoms after stopping. You must have positive somatostatin receptor tumor status confirmed by imaging tests. Your disease must not have shown significant progression in the last 6 months.
Who Cannot Participate: You cannot participate if you have any serious health condition that might interfere with the study, are pregnant or breastfeeding, have a history of allergic reactions to similar medications, or have participated in another clinical trial recently. Those with a history of substance abuse, certain mental health conditions that might affect participation, abnormal lab test results not explained by the current condition, certain heart conditions, uncontrolled high blood pressure, or unstable liver or kidney problems are also excluded. You must be able to follow study procedures and instructions to participate.
Summary
Both ongoing clinical trials are focused on investigating paltusotine, an oral medication, as a potential treatment option for managing carcinoid syndrome symptoms. The trials are being conducted across multiple European countries, with Poland participating in both studies and Italy, Spain, and France involved in one of the trials.
The first trial focuses specifically on evaluating whether paltusotine at a 40 mg daily dose can reduce flushing episodes and affect bowel movement frequency over a 12-week period, comparing it directly to a placebo. The second trial takes a broader approach, testing three different dose levels (40 mg, 80 mg, and 120 mg) to understand how the body processes the medication and to establish the safety profile across different doses.
Both studies require participants to have well-differentiated neuroendocrine tumors (Grade 1 or 2) and documented carcinoid syndrome with specific symptom requirements. Importantly, participants may need to stop their current somatostatin receptor ligand treatments before joining these studies. Regular monitoring through symptom diaries, clinical visits, blood tests, and imaging studies will be conducted throughout both trials to ensure participant safety and gather comprehensive data on the medication’s effects.
