Current partner codePEPTIDESDE
NCT07087054·Phase 3·INTERVENTIONAL

Carcinoid Syndrome Efficacy Study Featuring an Oral Daily Paltusotine Regimen

Status

Recruiting

Phase

Phase 3

Enrollment

141

Locations

51

Results

Not posted

Publications

0

Study summary

What the protocol is testing.

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.

Full detailed description

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

Treatment arms and agents.

DRUG

Paltusotine

Experimental Drug: Randomized

DRUG

Placebo

Matching Placebo Drug: Randomized

Timeline

From registration to results.

  1. First posted

    Jul 25, 2025

  2. Study start

    Nov 19, 2025

  3. Primary completion

    Aug 2027

  4. Study completion

    Jan 2030

  5. Results posted

    Not reported

  6. Registry updated

    Jun 25, 2026

Outcomes

What the study measures.

Primary 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.

Secondary outcomes

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

Who can take part.

Minimum age
18 Years
Maximum age
Not reported
Sex
ALL
Healthy volunteers
No

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

51 registered sites.

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

Results and literature.

No PMID-linked publications were present in this registry snapshot.

Primary links

Continue at the source.

Related trials

More studies on Octreotide.

Related PeptideStat pages

Put the record in context.

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