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

NCT ID: NCT05361668

Last Updated: 2025-10-31

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 36 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-04-22
• Study Completion Date: 2026-02-28

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.

Detailed Description

This is a Phase 2, randomized, open-label, parallel-group, multicenter study designed to evaluate the safety, pharmacokinetics, and efficacy of paltusotine treatment in subjects with carcinoid syndrome. The study was conducted in 2 parts: a Randomized Treatment Phase (RTP) which is completed, and an Open-label Extension (OLE) Phase which is still ongoing. The RTP consisted of paltusotine treatment for 8 weeks. Subjects who completed the RTP were eligible to enter the OLE Phase at the recommendation of the Investigator. In the ongoing OLE Phase, paltusotine is being administered for a further 102 weeks. The total duration of paltusotine treatment for the combined RTP and OLE Phase is up to 110 weeks (28 months).

Conditions

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

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

40 mg Paltusotine

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

Group Type: EXPERIMENTAL

Randomized: 40 mg Paltusotine

Intervention Type: DRUG

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

80 mg Paltusotine

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

Group Type: EXPERIMENTAL

Randomized: 80 mg Paltusotine

Intervention Type: DRUG

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

Interventions

Randomized: 40 mg Paltusotine

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

Intervention Type: DRUG

Randomized: 80 mg Paltusotine

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

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. Male or female subjects ≥18 years of age. 2. Documented carcinoid syndrome requiring medical therapy.

1. 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.

2. Subjects currently treated with lanreotide, octreotide long acting release, or short acting octreotide (subcutaneous or oral) who are currently symptomatically controlled

3. 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.

4. No significant disease progression as assessed by the Investigator within the last 6 months before initiation of study drug dosing.

Exclusion Criteria

1. Diarrhea attributed to any condition(s) other than carcinoid syndrome.

2. Uncontrolled/severe diarrhea associated with significant volume contraction, dehydration, or hypotension.

3. Requires second line treatments (eg, telotristat) for control of carcinoid syndrome symptoms.

4. 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.

5. 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.

6. Diabetes mellitus treated with insulin for less than 6 weeks prior to the study entry.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Crinetics Pharmaceuticals Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Crinetics Study Site

Los Angeles, California, United States

Crinetics Study Site

Los Angeles, California, United States

Crinetics Study Site

Newport Beach, California, United States

Crinetics Study Site

Stanford, California, United States

Crinetics Study Site

Miami, Florida, United States

Crinetics Study Site

Iowa City, Iowa, United States

Crinetics Study Site

Lexington, Kentucky, United States

Crinetics Study Site

New Orleans, Louisiana, United States

Crinetics Study Site

Boston, Massachusetts, United States

Crinetics Study Site

Rochester, Minnesota, United States

Crinetics Study Site

New York, New York, United States

Crinetics Study Site

Stony Brook, New York, United States

Crinetics Study Site

Cleveland, Ohio, United States

Crinetics Study Site

Columbus, Ohio, United States

Crinetics Study Site

Houston, Texas, United States

Crinetics Study Site

CABA, Buenos Aires, Argentina

Crinetics Study Site

CABA, Buenos Aires, Argentina

Crinetics Study Site

CABA, Buenos Aires, Argentina

Crinetics Study Site

CABA, , Argentina

Crinetics Study Site

CABA, , Argentina

Crinetics Study Site

Fortaleza, Ceará, Brazil

Crinetics Study Site

Rio de Janeiro, Rio de Janeiro, Brazil

Crinetics Study Site

Rio de Janeiro, Rio de Janeiro, Brazil

Crinetics Study Site

Criciúma, Santa Catarina, Brazil

Crinetics Study Site

São Paulo, São Paulo, Brazil

Crinetics Study Site

Rio de Janeiro, , Brazil

Crinetics Study Site

Toronto, , Canada

Crinetics Study Site

Mexico City, Cuauhtemoc, Mexico

Crinetics Study Site

Querétaro City, Querétaro, Mexico

Crinetics Study Site

Querétaro City, Querétaro, Mexico

Crinetics Study Site Peru #1

Lima, , Peru

Crinetics Study Site Peru #2

Lima, , Peru

Crinetics Study Site

Katowice, , Poland

Crinetics Study Site

Warsaw, , Poland

Crinetics Study Site

Wroclaw, , Poland

Countries

United States; Argentina; Brazil; Canada; Mexico; Peru; Poland

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

CRN00808-11

Identifier Type: -

Identifier Source: org_study_id