Carcinoid Syndrome Efficacy Study Featuring an Oral Daily Paltusotine Regimen

NCT07087054 | Recruiting Phase 3 DMC FDA Drug

• 2 other identifiers: CRN00808-122024-519875-24-00
• Study Type: interventional
• Target: 141
• Locations: 9 countries
• Sites: 51

Brief 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.

Conditions

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

Keywords

Neuroendocrine tumor; Paltusotine; CRN00808; Carcinoid syndrome; Lanreotide; Octreotide; Somatostatin agonist

Outcome Measures

Primary Outcomes (1)

• 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.

  Treatment group difference of change from baseline to Week 12 in flushing episodes/day averaged over the 14 days prior to Week 12.

  Measured at Week 12

Secondary Outcomes (1)

• 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.

  Measured at Week 12

Study Arms (2)

Paltusotine 80 mg daily

EXPERIMENTAL

Drug: Paltusotine

Placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

Paltusotine DRUG

Experimental Drug: Randomized

Paltusotine 80 mg daily

Placebo DRUG

Matching Placebo Drug: Randomized

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Crinetics Pharmaceuticals Inc.: lead

Study Sites (51)

Banner MD Anderson Cancer Center

Gilbert, Arizona, 85234, United States

RECRUITING

Hoag Memorial Hospital Presbyterian

Newport Beach, California, 92663, United States

RECRUITING

Yale University - New Haven Hospital - Yale Cancer Center

New Haven, Connecticut, 06510, United States

RECRUITING

University of Miami

Miami, Florida, 33136, United States

RECRUITING

Moffitt Cancer Center

Tampa, Florida, 33612, United States

RECRUITING

Winshop Cancer Institute - Emory University

Atlanta, Georgia, 30322, United States

RECRUITING

University of Iowa Health Care

Iowa City, Iowa, 52242, United States

RECRUITING

University of Kentucky Medical Center

Lexington, Kentucky, 40536, United States

RECRUITING

Louisiana State University Health Sciences

Metairie, Louisiana, 70006, United States

RECRUITING

Henry Ford Cancer - Detroit

Detroit, Michigan, 48202, United States

RECRUITING

Mayo Clinic

Rochester, Minnesota, 55905, United States

RECRUITING

Icahn School of Medicine at Mount Sinai

New York, New York, 10029, United States

RECRUITING

University Hospitals Cleveland Medical Center

Cleveland, Ohio, 44106, United States

RECRUITING

Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

Huntsman Cancer Institute, University of Utah

Salt Lake City, Utah, 84112, United States

RECRUITING

University of Virginia Comprehensive Cancer Center

Charlottesville, Virginia, 22903, United States

RECRUITING

Medical College of Wisconcin

Milwaukee, Wisconsin, 53222, United States

RECRUITING

Hospital de Gastroenterologia Dr. Carlos Bonorino Udaondo

Buenos Aires, 1264, Argentina

RECRUITING

Sanatorio Guemes

Buenos Aires, C1180AAX, Argentina

WITHDRAWN

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

Buenos Aires, C1425BGH, Argentina

RECRUITING

Instituto Médico Especializado Alexander Fleming

Buenos Aires, C1426ANZ, Argentina

RECRUITING

Instituto Médico de la Fundación Estudios Clínicos

Santa Fe, 2000, Argentina

RECRUITING

AC Camargo Cancer Center

São Paulo, Brazil, 01509-010, Brazil

RECRUITING

Fundacao PIO XII - Hospital de Amor Barretos

Barretos, 14784-400, Brazil

RECRUITING

Sociedade Literaria e Caritativa Santos Agostinho - Hospital Sao José

Criciúma, 88811-000, Brazil

RECRUITING

Nucleo de Pesquisa e Desenvolvimento de Medicamentos (MPDM)

Fortaleza, 60430-275, Brazil

RECRUITING

Associacao Hospitalar Moinhos de Vento

Porto Alegre, 90560-032, Brazil

RECRUITING

Instituto Nacional de Cancer (INCA)

Rio de Janeiro, 20230-130, Brazil

RECRUITING

Clinical Universidad Catolica del Maule, Ltda

Maule, Chile, 3460000, Chile

RECRUITING

Centro de Oncologia de Precision

Santiago, Chile, 7560908, Chile

RECRUITING

CECIM-Centro de Estudios Cliicos e Investigaciones Medicas

Santiago, 8320000, Chile

RECRUITING

Hospital Universitario San Ignacio

Bogotá, DC, 110231, Colombia

RECRUITING

Instituto Nacional de Cancerologia

Bogotá, DC, 110411, Colombia

RECRUITING

CHRU Tours - Hopital Trousseau

Chambray-lès-Tours, 37170, France

RECRUITING

Hopital Beaujun - APHP

Clichy, 92110, France

RECRUITING

APHM- Hopital de la Timone

Marseille, 13005, France

RECRUITING

Centre Hospitalier Universitaire Nantes

Nantes, 44093, France

RECRUITING

Centre Antoine Lacassagne

Nice, 06100, France

RECRUITING

CHU Bordeaux - Hopital Haut-Leveque

Pessac, 33600, France

RECRUITING

Centro de Oncolégica VIVA

Mexico City, 06760, Mexico

RECRUITING

Hospital Universitari Vall d'Hebron

Barcelona, 08035, Spain

RECRUITING

Hospital Universitario Ramon y Cajal

Madrid, 28034, Spain

RECRUITING

Hospital Universitario 12 de Octubre

Madrid, 28041, Spain

RECRUITING

King's College Hospital

London, UK, SE59RS, United Kingdom

RECRUITING

Oxford University Hospitals NHS Foundation Trust

Oxford, UK, OX37LE, United Kingdom

RECRUITING

Queen Elizabeth Hospital Brimingham

Birmingham, B15 2TH, United Kingdom

RECRUITING

University Hospital of Wales

Cardiff, CF144XW, United Kingdom

RECRUITING

The Beatson WOS Cancer Centre

Glasgow, G120YN, United Kingdom

RECRUITING

Royal Free Hospital

London, NW3 2QG, United Kingdom

RECRUITING

NIHR Clinical Research Facility, Royal Hallamshire Hospital

Sheffield, S102JF, United Kingdom

RECRUITING

University Hospital Southampton NHS Foundation Trust

Southampton, SO166YD, United Kingdom

RECRUITING

Related Links

• Related Info

MeSH Terms

Conditions

Serotonin Syndrome; Carcinoid Tumor; Carcinoid Tumors, Intestinal; Neuroendocrine Tumors

Condition Hierarchy (Ancestors)

Drug-Related Side Effects and Adverse Reactions; Chemically-Induced Disorders; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Adenocarcinoma; Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue

Central Study Contacts

Crinetics Clinical Trials

CONTACT

833-276-4636; clinicaltrials@crinetics.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 9, 2025
• First Posted: July 25, 2025
• Study Start: November 19, 2025
• Primary Completion (Estimated): August 1, 2027
• Study Completion (Estimated): January 1, 2030
• Last Updated: April 30, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

GB (8); CL (3); CO (2); ME (1); US (17); BR (6); AR (5); FR (6); ES (3)