Study to Evaluate the Safety, PK, and Dose Response of Paltusotine in Subjects With Carcinoid Syndrome

NCT05361668 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: CRN00808-11
• Study Type: interventional
• Target: 36
• Locations: 7 countries
• Sites: 35

Brief Summary

The purpose of this study is to evaluate the safety, pharmacokinetics (PK), and exploratory dose response of paltusotine treatment in subjects with carcinoid syndrome. This study consists of a Randomized Treatment Phase followed by an Open-Label Extension (OLE) Phase.

Conditions

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

Keywords

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

Outcome Measures

Primary Outcomes (1)

• Safety - Incidence of Treatment-emergent Adverse Events (TEAEs)

  Incidence of TEAEs, including serious TEAEs and TEAEs leading to discontinuation; change from baseline to the EOR in safety parameters: clinical laboratory tests, physical exam findings, vital signs, 12-lead ECG, and 24-hour continuous cardiac monitoring (only for subjects on 120 mg dose). In addition to all-cause mortality, deaths were categorized by primary cause (e.g., cardiovascular) based on medical adjudication. Events were coded using MedDRA and summarized by treatment group, system organ class, and preferred term. TEAEs were reported per randomized arm. There were 2 randomized arms (40 mg and 80 mg paltusotine, with up-or down-dose titration permitted for symptom control). Results are analyzed based on the initially assigned randomization arm, regardless of the actual dose received.

  First dose of investigational medicine to End of Randomized Treatment Phase (8 weeks)

Secondary Outcomes (1)

• Pharmacokinetics (PK) of Paltusotine

  Measured at each visit (pre and post dose) up to Week 8 (i.e., End of Randomized Treatment Phase [EOR])

Study Arms (2)

40 mg Paltusotine

EXPERIMENTAL

Participants received paltusotine 40mg, in tablet form, orally, daily, for 8 weeks.

Drug: Randomized: 40 mg Paltusotine

80 mg Paltusotine

EXPERIMENTAL

Participants received paltusotine 80mg, in tablet form, orally, daily for 8 weeks.

Drug: Randomized: 80 mg Paltusotine

Interventions

Randomized: 40 mg Paltusotine DRUG

Two 20 mg tablets QD (Potential post-randomization dose escalation based on efficacy and acceptable tolerability: up to 80 mg)

40 mg Paltusotine

Randomized: 80 mg Paltusotine DRUG

Four 20 mg tablets QD (Potential post-randomization dose escalation based on efficacy and acceptable tolerability: up to 120 mg)

80 mg Paltusotine

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 subjects ≥18 years of age. 2. Documented carcinoid syndrome requiring medical therapy.
• Not currently treated with somatostatin receptor ligands agonists for at least 12 weeks prior to screening and actively symptomatic. This can include treatment-naïve subjects.
• Subjects currently treated with lanreotide, octreotide long acting release, or short acting octreotide (subcutaneous or oral) who are currently symptomatically controlled
• Evaluable documentation of locally advanced or metastatic histopathologically confirmed well-differentiated neuroendocrine tumor (NET). Tumors must be Grade 1 (Ki-67 index \< 3%, or a mitotic count of \< 2 mitoses per 10 high-power fields, if the Ki-67 index is not available) or Grade 2 (Ki-67 index 3-20%, or a mitotic count of 2-20 mitoses per 10 high-power fields, if the Ki-67 index is not available) per the World Health Organization neuroendocrine neoplasm classification (Rindi and Inzani, 2020). Grade 3 tumors are not eligible.
• No significant disease progression as assessed by the Investigator within the last 6 months before initiation of study drug dosing.

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.
• Treatment with specific NET tumor 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.
• History of another primary malignancy \<3 years prior to the date of first dose, except for adequately treated basal or squamous cell carcinoma of the skin, cancer of the breast or cervix in situ, previously treated or 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.

Sponsors & Collaborators

• Crinetics Pharmaceuticals Inc.: lead

Study Sites (35)

Crinetics Study Site

Los Angeles, California, 90048, United States

Location

Crinetics Study Site

Los Angeles, California, 90095, United States

Location

Crinetics Study Site

Newport Beach, California, 92663, United States

Location

Crinetics Study Site

Stanford, California, 94305, United States

Location

Crinetics Study Site

Miami, Florida, 33136, United States

Location

Crinetics Study Site

Iowa City, Iowa, 52242, United States

Location

Crinetics Study Site

Lexington, Kentucky, 40506, United States

Location

Crinetics Study Site

New Orleans, Louisiana, 70112, United States

Location

Crinetics Study Site

Boston, Massachusetts, 02118, United States

Location

Crinetics Study Site

Rochester, Minnesota, 55905, United States

Location

Crinetics Study Site

New York, New York, 10029, United States

Location

Crinetics Study Site

Stony Brook, New York, 11794, United States

Location

Crinetics Study Site

Cleveland, Ohio, 44106, United States

Location

Crinetics Study Site

Columbus, Ohio, 43210, United States

Location

Crinetics Study Site

Houston, Texas, 77030, United States

Location

Crinetics Study Site

CABA, Buenos Aires, C1180 AAX, Argentina

Location

Crinetics Study Site

CABA, Buenos Aires, C1264AAA, Argentina

Location

Crinetics Study Site

CABA, Buenos Aires, C1426ANZ, Argentina

Location

Crinetics Study Site

CABA, C1017AAS, Argentina

Location

Crinetics Study Site

CABA, C1425BGH, Argentina

Location

Crinetics Study Site

Fortaleza, Ceará, 60430-275, Brazil

Location

Crinetics Study Site

Rio de Janeiro, Rio de Janeiro, 20231-092, Brazil

Location

Crinetics Study Site

Rio de Janeiro, Rio de Janeiro, 22281-100, Brazil

Location

Crinetics Study Site

Criciúma, Santa Catarina, 88811508, Brazil

Location

Crinetics Study Site

São Paulo, São Paulo, 01509-010, Brazil

Location

Crinetics Study Site

Rio de Janeiro, 22061-080, Brazil

Location

Crinetics Study Site

Toronto, M4N 3M5, Canada

Location

Crinetics Study Site

Mexico City, Cuauhtemoc, 06100, Mexico

Location

Crinetics Study Site

Querétaro City, Querétaro, 76000, Mexico

Location

Crinetics Study Site

Querétaro City, Querétaro, 76070, Mexico

Location

Crinetics Study Site Peru #1

Lima, 15036, Peru

Location

Crinetics Study Site Peru #2

Lima, 15036, Peru

Location

Crinetics Study Site

Katowice, 40-514, Poland

Location

Crinetics Study Site

Warsaw, 02-351, Poland

Location

Crinetics Study Site

Wroclaw, 53-413, Poland

Location

MeSH Terms

Conditions

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

Interventions

Random Allocation

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

Intervention Hierarchy (Ancestors)

Epidemiologic Research Design; Epidemiologic Methods; Investigative Techniques; Research Design; Methods; Health Care Evaluation Mechanisms; Quality of Health Care; Health Care Quality, Access, and Evaluation; Public Health; Environment and Public Health

Results Point of Contact

• Title: Study Director
• Organization: Crinetics Pharmaceuticals, Inc.

medinfo@crinetics.com

(858) 450-6464

Publication Agreements

• PI is Sponsor Employee: Yes
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 27, 2022
• First Posted: May 5, 2022
• Study Start: April 22, 2022
• Primary Completion: March 7, 2024
• Study Completion: February 24, 2026
• Last Updated: March 19, 2026
• Results First Posted: October 31, 2025

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

ME (3); PE (2); PL (3); BR (6); AR (5); US (15); CA (1)