Paltusotine for Carcinoid Syndrome

(CAREFNDR Trial)

Recruiting at 24 trial locations

Overseen By Crinetics Clinical Trials

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Crinetics Pharmaceuticals Inc.

Must be taking: Somatostatin agonists

Must not be taking: Telotristat, Everolimus, Sunitinib

Disqualifiers: Uncontrolled diarrhea, Major surgery, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 16 weeks

141 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called paltusotine to determine its effectiveness for people with carcinoid syndrome, a condition caused by certain tumors that leads to symptoms like flushing and diarrhea. The study compares paltusotine with a placebo (a pill with no active ingredient) to assess its effectiveness and safety. Individuals with carcinoid syndrome, experiencing symptoms like frequent flushing episodes and having tumors confirmed by their doctor, might be suitable for this trial. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants the opportunity to contribute to a potentially groundbreaking treatment.

Do I have to stop taking my current medications for the trial?

The trial requires participants who are currently treated with somatostatin receptor ligands (SRL) to stop taking them during a washout period (time without taking certain medications) before starting the study. Other specific medications like everolimus or sunitinib must not have been taken within 4 weeks before screening.

Is there any evidence suggesting that paltusotine is likely to be safe for humans?

Research has shown that paltusotine is generally safe for individuals with carcinoid syndrome and acromegaly. In previous studies, most participants did not experience serious side effects. Some individuals showed a slight increase in a liver enzyme called ALT, but it did not raise significant concern. Overall, paltusotine's safety profile aligns with the usual symptoms of carcinoid syndrome and the effects of similar treatments. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for carcinoid syndrome?

Unlike the standard treatments for carcinoid syndrome, which typically include somatostatin analogs like octreotide and lanreotide, Paltusotine is a novel oral medication that offers a new approach. Most current therapies require injections, but Paltusotine is taken orally, making it potentially more convenient for patients. Additionally, Paltusotine is designed to maintain stable drug levels in the body, which might improve symptom control and enhance patient quality of life. Researchers are excited about Paltusotine because it could provide a more user-friendly treatment option with effective symptom management.

What evidence suggests that paltusotine might be an effective treatment for carcinoid syndrome?

Research has shown that paltusotine, which participants in this trial may receive, offers promising results for treating carcinoid syndrome, a condition related to certain tumors. In earlier studies, patients experienced a 65% reduction in both the number of bowel movements and flushing episodes, common symptoms of the syndrome. These findings suggest that paltusotine can effectively relieve symptoms. By targeting specific pathways, paltusotine helps control these uncomfortable symptoms, offering hope for better management of the condition. ^{1 2 6 7 8}

Are You a Good Fit for This Trial?

Adults with carcinoid syndrome from well-differentiated neuroendocrine tumors can join this study. Participants should have symptoms despite stable-dose somatostatin analog therapy. Specific details about inclusion and exclusion criteria are not provided, but typically these would cover health status, prior treatments, and other medical conditions.

Inclusion Criteria

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

I have carcinoid syndrome with frequent flushing, needing medication.

My cancer is a well-differentiated neuroendocrine tumor that has spread.

Exclusion Criteria

My diarrhea is not caused by carcinoid syndrome.

I have severe diarrhea causing dehydration or low blood pressure.

I need a second treatment for my carcinoid syndrome symptoms.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

up to 11 weeks

Treatment

Participants receive either paltusotine or placebo in a double-blinded manner

16 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Participants may opt into continuation of paltusotine treatment long-term

104 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Paltusotine

Trial Overview The trial is testing the effectiveness of paltusotine compared to a placebo in managing carcinoid syndrome symptoms. It's a Phase 3 study where participants are randomly assigned to receive either the drug or placebo without knowing which one they're getting (double-blinded).

How Is the Trial Designed?

Group I: Paltusotine 80 mg daily Experimental Treatment 1 Intervention

Group II: Placebo Placebo Group 1 Intervention

Who Is Running the Clinical Trial?

Crinetics Pharmaceuticals Inc.

Lead Sponsor

Trials

13

Recruited

760+

Citations

1. crinetics.com crinetics.com/crinetics-to-highlight-neuroendocrine-tumor-research-progress-at-the-2025-north-american-neuroendocrine-tumor-society-annual-meeting/

Crinetics to Highlight Neuroendocrine Tumor Research ... About Paltusotine​​ Results from a Phase 2 study in carcinoid syndrome demonstrated rapid and sustained reductions in flushing episodes and bowel ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT07087054

NCT07087054 | Carcinoid Syndrome Efficacy Study ... 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 ...

3. netrf.org netrf.org/2023/12/19/crinetics-announces-positive-results-from-phase-ii-study-of-paltusotine/

Crinetics Announces Positive Results From Phase II Study ... Paltusotine resulted in a 65% reduction in bowel movement frequency and 65% reduction in flushing episodes, a finding consistent with prior ...

4. cancertherapyadvisor.com cancertherapyadvisor.com/reports/paltusotine-may-address-unmet-need-in-nets-patients-with-carcinoid-syndrome/

Paltusotine May Address Unmet Need in NETs Patients ... Paltusotine can alleviate symptoms of carcinoid syndrome in patients with neuroendocrine tumors (NETs), according to results of a phase 2 study.

5. crinetics.com crinetics.com/wp-content/uploads/2024/03/Pal-CS-Ph2-Investor-Deck_FINAL_3-12-24_Upload.pdf

Results from Paltusotine Carcinoid Syndrome Open Label ... * The fatal outcome of one SAE (cardiac failure, most likely secondary to carcinoid heart disease) occurred 26 days after treatment ...

6. crinetics.com crinetics.com/pipeline/paltusotine/

Paltusotine, Oral SST2 Agonist The safety and efficacy of paltusotine have not been established in this patient population. In clinical studies, paltusotine has been well-tolerated and the ...

7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12199097/

Oral paltusotine, a nonpeptide selective somatostatin ... ... safety information. Paltusotine was well tolerated in clinical trials involving patients with acromegaly or carcinoid syndrome and had a safety ...

8. s206.q4cdn.com s206.q4cdn.com/492797646/files/doc_news/NEW-LONG-TERM-SAFETY-AND-EFFICACY-DATA-PRESENTED-WITH-PALTUSOTINE-IN-PARTICIPANTS-LIVING-WITH-ACROMEGALY-09-06-2022-2022.pdf

NEW LONG-TERM SAFETY AND EFFICACY DATA ... Paltusotine, a somatostatin receptor type 2 (SST2) agonist, is in Phase 3 clinical development for acromegaly and Phase 2 clinical development ...