Carcinoid Syndrome Efficacy Study Featuring an Oral Daily Paltusotine Regimen

NCT ID: NCT07087054

Last Updated: 2025-11-28

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

141 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-11-19

Study Completion Date

2030-01-31

Brief Summary

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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.

Detailed Description

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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.

Conditions

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Carcinoid Syndrome Carcinoid Carcinoid Tumor Carcinoid Tumor of Ileum Carcinoid Tumor of Cecum Carcinoid Tumor of Liver Carcinoid Tumor of Pancreas Carcinoid Syndrome Diarrhea Carcinoid Intestine Tumor

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Paltusotine 80 mg daily

Group Type EXPERIMENTAL

Paltusotine

Intervention Type DRUG

Experimental Drug: Randomized

Placebo

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Matching Placebo Drug: Randomized

Interventions

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Paltusotine

Experimental Drug: Randomized

Intervention Type DRUG

Placebo

Matching Placebo Drug: Randomized

Intervention Type DRUG

Eligibility Criteria

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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 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 or cervix in situ, 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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Crinetics Pharmaceuticals Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Hoag Memorial Hospital Presbyterian

Newport Beach, California, United States

Site Status RECRUITING

Yale University - New Haven Hospital - Yale Cancer Center

New Haven, Connecticut, United States

Site Status RECRUITING

Winshop Cancer Institute - Emory University

Atlanta, Georgia, United States

Site Status RECRUITING

University of Kentucky Medical Center

Lexington, Kentucky, United States

Site Status RECRUITING

Louisiana State University Health Sciences

Metairie, Louisiana, United States

Site Status RECRUITING

Henry Ford Cancer - Detroit

Detroit, Michigan, United States

Site Status RECRUITING

Mayo Clinic

Rochester, New York, United States

Site Status RECRUITING

University Hospitals Cleveland Medical Center

Cleveland, Ohio, United States

Site Status RECRUITING

Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, United States

Site Status RECRUITING

University of Virginia Comprehensive Cancer Center

Charlottesville, Virginia, United States

Site Status RECRUITING

Sanatorio Guemes

Buenos Aires, , Argentina

Site Status WITHDRAWN

Centro de Endocrinologia y Diabetes Dr. A. Gutman ICM - Investigaciones

Buenos Aires, , Argentina

Site Status RECRUITING

Instituto Médico Especializado Alexander Fleming

Buenos Aires, , Argentina

Site Status RECRUITING

Instituto Médico de la Fundación Estudios Clínicos

Santa Fe, , Argentina

Site Status RECRUITING

AC Camargo Cancer Center

São Paulo, Brazil, Brazil

Site Status RECRUITING

Sociedade Literaria e Caritativa Santos Agostinho - Hospital Sao José

Criciúma, , Brazil

Site Status RECRUITING

Associacao Hospitalar Moinhos de Vento

Porto Alegre, , Brazil

Site Status RECRUITING

CHRU Tours - Hopital Trousseau

Chambray-lès-Tours, , France

Site Status RECRUITING

Hopital Beaujun - APHP

Clichy, , France

Site Status RECRUITING

APHM- Hopital de la Timone

Marseille, , France

Site Status RECRUITING

Centre Hospitalier Universitaire Nantes

Nantes, , France

Site Status RECRUITING

Centre Antoine Lacassagne

Nice, , France

Site Status RECRUITING

CHU Bordeaux - Hopital Haut-Leveque

Pessac, , France

Site Status RECRUITING

Hospital Universitari Vall d'Hebron

Barcelona, , Spain

Site Status RECRUITING

Hospital Universitario Ramon y Cajal

Madrid, , Spain

Site Status RECRUITING

Hospital Universitario 12 de Octubre

Madrid, , Spain

Site Status RECRUITING

Countries

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United States Argentina Brazil France Spain

Central Contacts

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Crinetics Clinical Trials

Role: CONTACT

833-276-4636

Other Identifiers

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CRN00808-12

Identifier Type: -

Identifier Source: org_study_id

2024-519875-24-00

Identifier Type: CTIS

Identifier Source: secondary_id

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