Paltusotine for Carcinoid Syndrome

Not currently recruiting at 32 trial locations

Overseen By Crinetics Clinical Trials

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Crinetics Pharmaceuticals Inc.

Must be taking: Somatostatin receptor ligands

Must not be taking: Telotristat, Everolimus, Sunitinib

Disqualifiers: Uncontrolled diarrhea, Recent malignancy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

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

Compensation: Varies

Number of Visits: Unknown

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 safety and effectiveness for people with carcinoid syndrome, a condition often causing symptoms like flushing and diarrhea. The study involves two groups receiving different doses of paltusotine to identify the optimal dose. It is open to individuals with carcinoid syndrome who have not recently used certain treatments or whose symptoms are controlled with current medication. Those managing carcinoid syndrome without specific treatment may find this trial suitable. As a Phase 2 trial, this research focuses on evaluating the treatment's effectiveness in an initial, smaller group of participants.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications. However, if you are currently treated with certain medications like lanreotide or octreotide and are symptomatically controlled, you may continue with them. If you are on other specific NET tumor therapies, you may need to stop them at least 4 to 12 weeks before screening.

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

Research has shown that paltusotine is generally well-tolerated by people with carcinoid syndrome. Clinical studies have demonstrated a safety profile for paltusotine similar to the usual symptoms of carcinoid syndrome and other similar treatments. For example, only a few cases showed increased liver enzyme levels, and these were not serious.

Other studies have also found that paltusotine is well-tolerated by patients with both carcinoid syndrome and acromegaly, a condition where the body produces too much growth hormone. This suggests that paltusotine is safe for different conditions. While researchers continue to study the safety and effectiveness of paltusotine, the results so far are promising for its use in humans. ^{1 2 3 4 5}

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

Paltusotine is unique because it offers a new oral option for treating carcinoid syndrome, which is typically managed with injectable somatostatin analogs like octreotide and lanreotide. Researchers are excited about paltusotine because it works by reducing the production of hormones that cause the symptoms of carcinoid syndrome, potentially offering a less invasive alternative with the convenience of a pill. This oral administration could improve patient compliance and quality of life, setting it apart from the current standard treatments.

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

Research has shown that paltusotine may help treat carcinoid syndrome. Studies found that patients taking paltusotine experienced 65% fewer bowel movements and flushing episodes, common symptoms of this condition. This drug reduces these symptoms, potentially improving life for people with neuroendocrine tumors. The consistent symptom relief observed in studies suggests that paltusotine could be an effective treatment. Although more research is needed, the early results are promising. ^{1 2 4 6 7}

Are You a Good Fit for This Trial?

This trial is for adults with carcinoid syndrome not currently treated with certain somatostatin receptor ligands or those controlled on lanreotide/octreotide. Participants should have stable disease, no recent other NET treatments, and can't have insulin-treated diabetes less than 6 weeks old, severe diarrhea from other causes, or require second-line treatments.

Inclusion Criteria

I am taking lanreotide or octreotide and my symptoms are under control.

I haven't taken hormone therapy for my symptoms in the last 12 weeks.

My condition hasn't worsened significantly in the last 6 months.

Exclusion Criteria

I haven't had specific cancer treatments like everolimus or surgery to remove part of the tumor in the last 4 weeks.

I have no cancer except possibly treated skin cancer or cervical cancer.

I have been on insulin for my diabetes for less than 6 weeks.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Randomized Treatment

Participants receive paltusotine treatment to evaluate safety, pharmacokinetics, and dose response

8 weeks

Open-Label Extension

Participants may opt into continuation of treatment long-term

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Paltusotine

Trial Overview The study tests the safety and dose response of Paltusotine (80 mg or 40 mg) in patients with carcinoid syndrome. It includes a randomized phase where doses are compared by chance and an open-label phase where all receive the drug.

How Is the Trial Designed?

Group I: 80 mg Paltusotine Experimental Treatment 1 Intervention

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

Group II: 40 mg Paltusotine Experimental Treatment 1 Intervention

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

Who Is Running the Clinical Trial?

Crinetics Pharmaceuticals Inc.

Lead Sponsor

Trials

13

Recruited

760+

Citations

1. 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 ... Paltusotine is an investigational drug in clinical studies for the treatment of acromegaly and carcinoid syndrome. Flushing: 63% reduction in ...

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

NCT07087054 | Carcinoid Syndrome Efficacy Study ... This is a global, randomized, parallel-group, placebo-controlled study to evaluate the efficacy and safety of paltusotine in adults with carcinoid syndrome. The ...

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. ir.crinetics.com ir.crinetics.com/news/news-details/2025/Crinetics-to-Highlight-Neuroendocrine-Tumor-Research-Progress-at-the-2025-North-American-Neuroendocrine-Tumor-Society-Annual-Meeting/default.aspx

News Details “For the first time, we will present progression-free survival data from our Phase 2 study of paltusotine for the treatment of carcinoid ...

5. 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.

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 ...