Paltusotine for Acromegaly

(PATHFNDR-1 Trial)

Not currently recruiting at 133 trial locations

Overseen By Crinetics Clinical Trials

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Crinetics Pharmaceuticals Inc.

Must be taking: Somatostatin receptor ligands

Must not be taking: Pasireotide, Pegvisomant, Dopamine agonists

Disqualifiers: Malignancy, HIV, Hepatitis, 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: 12 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests paltusotine, a pill designed to help people with acromegaly, a condition where excessive growth hormone causes enlarged body parts and other health issues. The study aims to determine if paltusotine is safe and effective compared to a placebo (a non-active pill). Individuals with acromegaly who are on stable treatment with specific medications may be suitable candidates for this trial. As a Phase 3 trial, it represents the final step before FDA approval, offering participants the opportunity to contribute to a potentially groundbreaking treatment.

Will I have to stop taking my current medications?

The trial requires that participants have been on a stable dose of somatostatin receptor ligand therapy, but you must stop taking pasireotide LAR, pegvisomant, dopamine agonists, or short-acting somatostatin analogs before joining. If you're on these, you'll need to stop them 12 to 24 weeks before the trial.

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

Research has shown that paltusotine is generally well tolerated. In earlier studies, patients with acromegaly who switched from injections to paltusotine maintained stable IGF-I levels, which is crucial for managing the condition. Specifically, findings from the Advance OLE study indicated that paltusotine's safety profile is similar to current injection treatments, with no unexpected side effects. Additionally, paltusotine reduced the frequency of symptom flare-ups, suggesting it is a promising and safe option for managing acromegaly. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Paltusotine is unique because it offers a new oral treatment option for acromegaly, a condition typically managed with injectable therapies like somatostatin analogs, dopamine agonists, or growth hormone receptor antagonists. Unlike these standard treatments, Paltusotine is designed to be taken as a pill, which could significantly improve patient convenience and adherence. Additionally, Paltusotine targets the same receptors as injectable somatostatin analogs but in a potentially more patient-friendly form. Researchers are excited about Paltusotine because it might provide effective symptom control without the need for frequent injections, making life easier for people managing acromegaly.

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

Research has shown that paltusotine, which participants in this trial may receive, effectively treats acromegaly, a condition where the body produces too much growth hormone. Studies indicate that paltusotine lowers levels of IGF-1, a substance often elevated in people with acromegaly. Specifically, 92.6% of patients experienced a drop in IGF-1 levels by the end of the treatment. The treatment also maintained stable hormone levels. These findings suggest that paltusotine can effectively manage acromegaly in patients who have switched from other treatments. ^{1 3 6 7 8}

Are You a Good Fit for This Trial?

Adults diagnosed with acromegaly, previously treated with somatostatin receptor ligand therapy and have their condition under control. Participants must not be pregnant or breastfeeding, should use birth control if necessary, and provide consent. Excluded are those who've used investigational drugs recently, have significant health risks, a history of certain infections or cancer (except some skin cancers), substance abuse within the last year, or specific heart conditions.

Inclusion Criteria

My acromegaly is under control with stable medication.

Willing to provide signed informed consent

I am not pregnant or breastfeeding, and I cannot become pregnant or am using birth control.

Exclusion Criteria

I haven't taken any experimental drugs recently.

I had pituitary surgery within the last 6 months or have had pituitary radiation therapy.

I have not had cancer, except for certain skin cancers, in the last 5 years.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive paltusotine or placebo to evaluate safety and efficacy

12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Paltusotine
• Placebo

Trial Overview The trial is testing Paltusotine's safety and effectiveness compared to a placebo in adults with acromegaly who were on stable doses of somatostatin receptor ligands. It's randomized and controlled; participants won't know if they're getting the actual drug or a dummy pill.

How Is the Trial Designed?

Group I: Paltusotine 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/two-year-data-from-advance-ole-study-presented-at-cbem/

New Long-Term Safety and Efficacy Data Presented with ... These results show that paltusotine, an investigational compound, lowered and maintained insulin-like growth factor-1 (IGF-1) at levels comparable to prior ...

2. academic.oup.com academic.oup.com/jes/article/8/Supplement_1/bvae163.1201/7812139

12535 Efficacy And Safety Of Once-daily Oral Paltusotine In ... In the paltusotine group, IGF-I was reduced in 92.6% of patients at end of treatment, with the majority of the effect seen by Wks 2 to 4. The ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10099171/

ACROBAT Edge: Safety and Efficacy of Switching Injected ... These results suggest once-daily oral paltusotine was effective in maintaining IGF-I values in patients with acromegaly who switched from injected SRLs.

4. crinetics.com crinetics.com/crinetics-paltusotine-achieved-primary-and-secondary-endpoints-in-phase-3-pathfndr-1-acromegaly-study/

Crinetics' Once-Daily Oral Paltusotine Achieved the ... Paltusotine, an oral, once-daily investigational compound, achieved positive results by meeting the primary endpoint and all secondary endpoints of the Phase 3 ...

5. checkrare.com checkrare.com/data-on-paltusotine-for-the-treatment-of-acromegaly/

Data on Paltusotine for the Treatment of Acromegaly Data from a long-term extension study of two phase 3 studies of paltusotine showed the ability of the treatment to stabilize biochemical control ...

6. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/36353760/

Safety and Efficacy of Switching Injected SRLs to Oral ... These results suggest once-daily oral paltusotine was effective in maintaining IGF-I values in patients with acromegaly who switched from injected SRLs.

7. crinetics.com crinetics.com/crinetics-presents-new-data-at-endo-2024-that-increases-that-increases-body-of-evidence-positioning-once-daily-oral-paltusotine-as-potential-first-choice-treatment-option-for-acromegaly/

Crinetics Presents New Data at ENDO 2024 that Increases ... Paltusotine was associated with statistically significant reductions in the frequency of breakthrough acromegaly symptom exacerbations for total ...

8. clinicaltrials.gov clinicaltrials.gov/study/NCT03792555

A Study to Evaluate the Safety and Efficacy of Paltusotine ... A Phase 2 double-blind, placebo-controlled, randomized withdrawal study is designed to evaluate the safety, efficacy, and pharmacokinetics of paltusotine.