DRUG
Paltusotine
Experimental Drug: Randomized
Status
Recruiting
Phase
Phase 3
Enrollment
141
Locations
51
Results
Not posted
Publications
0
Study summary
A Phase 3, randomized, double-blinded, placebo-controlled study to evaluate the efficacy and safety of paltusotine treatment vs placebo as well as the long-term safety of paltusotine in adults with carcinoid syndrome due to well-differentiated neuroendocrine tumors. The purpose of this study is to continue the evaluation of the safety, efficacy, and pharmacokinetics (PK) of paltusotine in participants with carcinoid syndrome.
This is a global, randomized, parallel-group, placebo-controlled study to evaluate the efficacy and safety of paltusotine in adults with carcinoid syndrome. The study includes a screening period of up to 11 weeks, a double-blinded randomized control period of 16 weeks, an open label extension period of 104 weeks, and a follow-up period of 4 weeks.
Interventions
DRUG
Experimental Drug: Randomized
DRUG
Matching Placebo Drug: Randomized
Timeline
First posted
Jul 25, 2025
Study start
Nov 19, 2025
Primary completion
Aug 2027
Study completion
Jan 2030
Results posted
Not reported
Registry updated
Jun 25, 2026
Outcomes
Participants will record the number of flushing per day in a daily diary to assess the efficacy of paltusotine vs placebo in reducing flushing episodes.
Time frame · Measured at Week 12
Treatment group difference of change from baseline to Week 12 in flushing episodes/day averaged over the 14 days prior to Week 12.
Participants will record the number of bowel movements (BMs) per day in a daily diary to assess the efficacy of paltusotine vs placebo in reducing BMs/day.
Time frame · Measured at Week 12
Treatment group difference of change from baseline to Week 12 in BMs/day averaged over the 14 days prior to Week 12.
Eligibility
Inclusion Criteria: * Male or female ≥18 years of age, at the time of Screening. * Willing and able to comply with the study procedures as specified in the protocol, including at least 70% compliance with the study diary for the 2-week period. * Documented carcinoid syndrome requiring medical therapy. Participants must exhibit symptoms of flushing with or without frequent BMs as follows: * For participants who are naïve/not currently treated with somatostatin receptors ligands (SRL), they must exhibit an average of \>1 flushing episode/day over a period of 14 days * For participants who will wash out from SRL treatment, they must be symptomatically controlled and exhibit an increase in daily average flushing episodes and an average of \>1 flushing episode/day over a period of 14 days during the Washout Period. * Evaluable documentation of locally advanced or metastatic histopathologically confirmed well-differentiated neuroendocrine tumor(s) \[NETs\]. * No significant disease progression as assessed by the Investigator within the last 6 months before randomization. Exclusion Criteria: * Diarrhea attributed to any condition(s) other than carcinoid syndrome. * Uncontrolled/severe diarrhea associated with significant volume contraction, dehydration, or hypotension. * Requires second line treatments (eg, telotristat) for control of carcinoid syndrome symptoms in the opinion of the Investigator. * Treatment with specific NET therapy \<4 weeks before Screening (such as everolimus or sunitinib) or hepatic embolization, radiotherapy, peptide receptor radionuclide therapy (PRRT), and/or tumor debulking \<12 weeks before Screening. * Major surgery within 8 weeks before Screening. * History of another primary malignancy \<3 years prior to the date of randomization, except for adequately treated basal or squamous cell carcinoma of the skin, cancer of the breast has been completely locally excised and carcinoma in situ of the cervix has also been resected, previously treated malignancy, if all treatment for that malignancy was completed at least 3 years prior to first dose of study treatment, and no current evidence of disease, concurrent malignancy determined to be clinically stable and not requiring treatment. * Diabetes mellitus treated with insulin for less than 6 weeks prior to the study entry. * Poorly controlled diabetes mellitus defined as having a hemoglobin A1c (HbA1c) ≥8.5% * Unable to administer short-acting (SA) octreotide (octreotide acetate injection), or prior nonresponse documented with somatostatin agonists. * Clinically significant concomitant disease or indicator of disease that is not a result of the primary disease under study, including but not limited to cardiovascular disease, estimated glomerular filtration rate 2×upper limit of normal \[ULN\], and/or total bilirubin (TB) \>1.5×ULN. (Participants with previously diagnosed Gilbert's syndrome not accompanied by other hepatobiliary disorders and associated with TB * Alcohol or drug abuse is not permitted at any time during the study * Diagnosed with symptomatic cholelithiasis
Study locations
Argentina · Brazil · Chile · Colombia · France · Mexico · Spain · United Kingdom · United States. Showing up to 24 locations stored in the fast local snapshot.
Banner MD Anderson Cancer Center
Gilbert, Arizona, United States
Hoag Memorial Hospital Presbyterian
Newport Beach, California, United States
Yale University - New Haven Hospital - Yale Cancer Center
New Haven, Connecticut, United States
University of Miami
Miami, Florida, United States
Moffitt Cancer Center
Tampa, Florida, United States
Winshop Cancer Institute - Emory University
Atlanta, Georgia, United States
University of Iowa Health Care
Iowa City, Iowa, United States
University of Kentucky Medical Center
Lexington, Kentucky, United States
Louisiana State University Health Sciences
Metairie, Louisiana, United States
Henry Ford Cancer - Detroit
Detroit, Michigan, United States
Mayo Clinic
Rochester, Minnesota, United States
Icahn School of Medicine at Mount Sinai
New York, New York, United States
University Hospitals Cleveland Medical Center
Cleveland, Ohio, United States
Hospital of the University of Pennsylvania
Philadelphia, Pennsylvania, United States
Huntsman Cancer Institute, University of Utah
Salt Lake City, Utah, United States
University of Virginia Comprehensive Cancer Center
Charlottesville, Virginia, United States
Medical College of Wisconcin
Milwaukee, Wisconsin, United States
Hospital de Gastroenterologia Dr. Carlos Bonorino Udaondo
Buenos Aires, Argentina
Sanatorio Guemes
Buenos Aires, Argentina
Centro de Endocrinologia y Diabetes Dr. A. Gutman ICM - Investigaciones
Buenos Aires, Argentina
Instituto Médico Especializado Alexander Fleming
Buenos Aires, Argentina
Instituto Médico de la Fundación Estudios Clínicos
Santa Fe, Argentina
AC Camargo Cancer Center
São Paulo, Brazil, Brazil
Fundacao PIO XII - Hospital de Amor Barretos
Barretos, Brazil
Publications
No PMID-linked publications were present in this registry snapshot.
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