Active not recruitingPHASE3INTERVENTIONAL

A Study to Evaluate the Safety and Efficacy of Paltusotine for the Treatment of Acromegaly (PATHFNDR-2)

This research study is looking at a new oral medication called paltusotine for individuals diagnosed with acromegaly. Acromegaly is a condition where the body produces too much growth hormone, leading to various health problems. The study aims to find out if paltusotine is safe and effective in treating acromegaly for people who haven't had previous medicine for it or have stopped their current treatment. Participants will be randomly assigned to receive either paltusotine or a 'dummy pill' (placebo). This helps researchers understand the true effects of the new medicine. It's important to remember that participating in a study like this is a personal decision, and you should discuss it thoroughly with your doctor.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Crinetics Pharmaceuticals Inc.

Enrolment target

111

Start

17 Dec 2021

Estimated completion

01 Jan 2028

Acromegaly

Results

Results from this study

Posted January 2026

Results have been published for this study.

Primary outcome

Percentage of Participants With a Biochemical Response in Insulin-like Growth Factor-1 (IGF-1) at the End of the Randomized Control Phase (EOR)

A value \>1.0 indicates IGF-1 levels above the age- and sex-adjusted ULN. Response is defined as an IGF-1 level ≤1.0×ULN based on the average of last 2 measurements.

Full results on the registry

What is this study about?

This study, called PATHFNDR-2, is investigating a new oral medication named paltusotine for a condition known as acromegaly. Our bodies normally produce growth hormone, but in acromegaly, too much growth hormone is made. This can lead to various symptoms like enlarged hands and feet, changes in facial features, joint pain, and other health issues. Managing acromegaly is important to improve quality of life and prevent long-term complications.

Paltusotine is a type of medicine that works by targeting specific areas in the body that control growth hormone production. The aim of this study is to see how well paltusotine works compared to a placebo (a tablet with no active medicine) in people who haven't taken medication for their acromegaly before, or who have stopped their previous treatment. By comparing the new drug to a placebo, researchers can get a clear picture of its benefits and any side effects.

Taking part in a clinical trial like this helps researchers learn more about new treatments. The information gathered from studies like PATHFNDR-2 is crucial for developing better ways to help people living with acromegaly. It's a way for you to contribute to medical science while potentially benefiting from a new treatment option.

Key takeaways

Exploring a new pill (paltusotine) for acromegaly treatment.
Comparing the new pill to a dummy pill (placebo) to see if it works.
Suitable for adults with acromegaly who haven't had previous drug treatment or can stop it.
Requires regular clinic visits for monitoring and tests.
You can stop participating at any time.
Helps advance understanding of acromegaly treatments.

Who may be eligible?

This study is looking for adults aged 18 and over who have been diagnosed with acromegaly. You might be suitable if you haven't taken medication for acromegaly before, or if you're able to stop your current treatment before the study starts.

There are certain reasons why you might not be able to join. For example, if you've had certain types of radiation treatment to your pituitary gland in the last three years, or if you've previously taken paltusotine. Also, if you have a history of serious heart problems, active cancer (apart from certain treated skin cancers), or some serious infections like HIV or hepatitis, you might not be eligible. If you're pregnant, breastfeeding, or could become pregnant and aren't using effective contraception, you won't be able to participate.

The research team will carefully review your medical history and current health to make sure the study is a safe and appropriate option for you. They will also check if you are taking any other medications that might interfere with the study drug.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 18 years old or older?
Do you have a confirmed diagnosis of acromegaly?
Have you not taken medication for acromegaly before, or can you stop your current treatment?
Are you able to attend regular clinic appointments?
Are you not pregnant or breastfeeding, and if female, using effective contraception if needed?
Do you have no serious heart conditions or active cancers (other than certain treated skin cancers)?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you'll be assigned to either receive the new medicine, paltusotine, or a dummy pill (placebo). Neither you nor your study doctor will know which one you're receiving. You'll need to visit the study clinic regularly for appointments. These visits will involve various health checks, such as blood tests, physical examinations, and possibly other tests to monitor your acromegaly and how your body is responding to the treatment.

During your time in the study, you'll be asked to take the study medication as instructed. The total duration of your participation in the study, including all visits and follow-up, will be explained in full by the study team. You'll have ongoing support and monitoring from the medical team throughout the trial.

Potential risks and benefits

Participating in this study might offer potential benefits, such as access to a new treatment for acromegaly that isn't yet widely available, and close medical monitoring of your condition. However, like all medications, paltusotine may have side effects, which could range from mild to serious. The study also involves regular clinic visits and tests, which may be inconvenient. You will be fully informed about all known risks and discomforts before you decide to join. It's important to remember that you are free to withdraw from the study at any time, for any reason, without affecting your future medical care.

Locations (57)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Crinetics Study Site
Verified postcode
Aurora, United States
Crinetics Study Site
Verified postcode
Boston, United States
Crinetics Study Site
Verified postcode
Columbus, United States
Crinetics Study Site
Verified postcode
Portland, United States
Crinetics Study Site
Verified postcode
Philadelphia, United States
Crinetics Study Site
Verified postcode
Pittsburgh, United States
Crinetics Study Site
Unverified
CABA, Argentina
Crinetics Study Site
Verified postcode
Buenos Aires, Argentina
Crinetics Study Site
Unverified
CABA, Argentina
Crinetics Study Site
Unverified
CABA, Argentina
Crinetics Study Site
Verified postcode
Córdoba, Argentina
Crinetics Study Site
Verified postcode
Fortaleza, Brazil

Common questions

What is acromegaly?

Acromegaly is a rare condition where the body makes too much growth hormone, leading to enlarged hands, feet, and face, as well as other health issues.

What is a 'placebo'?

A placebo is a 'dummy pill' that looks like the study drug but contains no active medicine. It helps researchers compare the real effects of the new drug.

Will I know if I'm getting the new medicine or the placebo?

No, this is a 'blinded' study, meaning neither you nor your study doctor will know whether you are receiving paltusotine or the placebo.

What if I change my mind after joining?

You are free to leave the study at any time, for any reason, without it affecting your medical care.

What does 'non-pharmacologically treated' mean?

It means you either haven't taken medicine for your acromegaly before, or you've stopped taking any previous medications before the study starts.