Paltusotine for Acromegaly

(PATHFNDR-2 Trial)

Not currently recruiting at 85 trial locations

Overseen By Crinetics Clinical Trials

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Crinetics Pharmaceuticals Inc.

Must not be taking: Pegvisomant, Cabergoline, Pasireotide

Disqualifiers: Malignancy, HIV, Hepatitis, Alcohol abuse, 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: 24 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial evaluates the safety and effectiveness of the oral drug paltusotine for people with acromegaly, a condition where the body produces excess growth hormone, leading to enlarged features and health issues. Participants will receive either paltusotine or a placebo (a pill with no active medicine) to compare results. It is open to adults diagnosed with acromegaly who have not yet received medication or are willing to discontinue their current treatment. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants the opportunity to contribute to potentially groundbreaking treatment advancements.

Will I have to stop taking my current medications?

The trial requires participants to either be medically naïve, not currently treated, or willing to stop their current medications during the study screening period. If you are on certain medications, you may need to stop taking them to participate.

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 acromegaly, a condition where the body produces excess growth hormone. In one study that tracked patients for two years, paltusotine demonstrated a safety profile similar to other drugs in its group, indicating no unexpected side effects. Another study found no new safety issues, suggesting the drug remains safe over time. These findings suggest that paltusotine is safe for most people, with side effects similar to other treatments in this category. ^{1 2 3 4 5}

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

Unlike the standard treatments for acromegaly, which often involve injectable medications like somatostatin analogs (e.g., octreotide, lanreotide) or surgery, Paltusotine is unique because it is an oral medication. Researchers are excited about Paltusotine because it offers a potentially more convenient option for patients, eliminating the need for frequent injections. Additionally, Paltusotine works by targeting the same receptors as somatostatin analogs but does so in a pill form, which could improve patient compliance and quality of life. This could be a game-changer for those looking for effective but less invasive treatment options.

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 excess growth hormone. Studies found that paltusotine lowered a key growth factor, IGF-1, in 92.6% of patients. Most patients noticed this improvement within two to four weeks. Additionally, patients who switched from injections to paltusotine maintained steady IGF-1 levels. Long-term evidence demonstrated that paltusotine helped sustain these levels over time. ^{1 2 3 4 6}

Are You a Good Fit for This Trial?

This trial is for adults over 18 with acromegaly who haven't been treated pharmacologically or are willing to stop current treatments during the study. Participants must not be pregnant, should use birth control if necessary, and cannot have had certain cancer treatments or suffer from specific heart conditions.

Inclusion Criteria

I have acromegaly and am either not on treatment or willing to stop my current treatment for the study.

I have acromegaly and am either not on treatment or willing to stop my current treatment for the study.

I am not pregnant or breastfeeding, and I am either unable to have children, past menopause, or using birth control.

Exclusion Criteria

I have not had pituitary radiation therapy in the last 3 years.

Use of any investigational drug within the past 30 days or 5 half-lives, whichever is longer

I have heart conditions or take medications that affect my heart rhythm.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either paltusotine or placebo for the treatment of acromegaly

24 weeks

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 trial is testing Paltusotine's safety and effectiveness compared to a placebo in treating acromegaly. It's a randomized study where participants will either receive the actual drug or a placebo without knowing which one they're getting.

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. 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.

3. 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 ...

4. 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 ...

5. 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.

6. clinicaltrials.gov clinicaltrials.gov/study/NCT05192382

NCT05192382 | A Study to Evaluate the Safety and ... A randomized, placebo-controlled study designed to evaluate the safety and efficacy of paltusotine (formerly CRN00808; an oral selective nonpeptide somatostatin ...