Active not recruitingPHASE3INTERVENTIONAL

A Study to Evaluate the Safety and Efficacy of Paltusotine for the Treatment of Acromegaly

This study is testing a new oral medicine called paltusotine for adults with acromegaly. Acromegaly is a condition where your body produces too much growth hormone. The study aims to find out if paltusotine is safe and effective in controlling the symptoms of acromegaly in people who are already managing their condition with other somatostatin-based treatments. Some participants will receive paltusotine, while others will receive a dummy pill (placebo) to fairly compare the new medicine. This is a Phase 3 study, meaning it's one of the final stages of testing before a medicine might become widely available.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Crinetics Pharmaceuticals Inc.

Enrolment target

Start

12 May 2021

Estimated completion

01 Jun 2027

Acromegaly

Results

Results from this study

Posted January 2026

Results have been published for this study.

Primary outcome

Percentage of Participants Who Maintain Biochemical Response in IGF-1 (≤1.0× the Upper Limit of Normal [ULN]) 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 (weeks 34 and 36).

Full results on the registry

What is this study about?

This research study is focused on a health condition called acromegaly. If you have acromegaly, your body makes too much growth hormone, which can lead to various health problems. While there are existing treatments, this study is exploring a new oral medication called paltusotine. This medicine works in a similar way to some current treatments but offers the convenience of a pill instead of an injection.

The main goals of this study are twofold: first, to ensure that paltusotine is safe for people to take, and second, to see how well it works in controlling acromegaly. Specifically, the researchers want to know if it can help keep your hormone levels, like IGF-1, within a healthy range. To get clear results, the study will compare paltusotine with a placebo, which is a pill that looks like the study drug but contains no active medicine. This allows researchers to see the true effects of paltusotine.

This study is for adults who have already been diagnosed with acromegaly and are currently managing their condition with a type of medication called somatostatin receptor ligands (SRLs). It's a 'Phase 3' study, which means it’s one of the final and most important steps in testing a new medicine before it can be considered for wider use. Participating could help scientists learn more about acromegaly treatment and potentially lead to new options for patients.

Key takeaways

New oral medicine (paltusotine) for acromegaly is being tested.
Aims to check safety and effectiveness in controlling acromegaly.
For adults whose acromegaly is already managed by other treatments.
Involves health checks, blood tests, and taking either paltusotine or a dummy pill.
This is a Phase 3 trial, an important step before wider availability.

Who may be eligible?

To join this study, you must be an adult aged 18 or older with a confirmed diagnosis of acromegaly. Your acromegaly should currently be under control with existing somatostatin-based treatments, and your IGF-1 hormone levels should be within an acceptable range. Women who might get pregnant must be using reliable birth control or past menopause, and not be pregnant or breastfeeding. You also need to be willing to understand and sign a consent form to take part.

There are also reasons why you might not be able to join. For example, if you haven't been treated for acromegaly before, or if you've recently had pituitary surgery or radiation. You also can't have certain other serious health conditions like specific heart problems, a recent history of cancer (excluding some skin cancers), or some infectious diseases. If you've recently taken other experimental drugs, or certain specific acromegaly medications, you also won't be able to participate. The study team will review all your health information carefully to make sure it's safe for you to take part.

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 acromegaly that is currently well-controlled with existing medication?
Are your IGF-1 hormone levels within a stable, acceptable range?
For women, are you not pregnant or breastfeeding, and using reliable birth control if needed?
Have you not had pituitary surgery in the last 6 months or pituitary radiation therapy?
Do you have good overall health without certain serious conditions like specific heart problems or recent cancers?

Answer every question to see your result.

What does participation involve?

If you decide to join this study, you would be randomly assigned to either receive the new medicine, paltusotine, or a dummy pill (placebo). You wouldn't know which one you are taking. You would need to take the study medicine as instructed. This study involves several visits to the clinic where you would have health checks, blood tests to measure your hormone levels, and other assessments. The doctors will monitor your health closely throughout the study. You will also have follow-up appointments after you finish taking the study medicine to ensure your well-being. The exact length and number of visits would be explained in detail by the study team, but typically, Phase 3 studies can last for several months to over a year.

Potential risks and benefits

Taking part in a clinical trial may offer potential benefits, such as access to a new treatment before it's widely available and closer monitoring of your health. However, there are also potential risks; the new medicine might cause side effects, some of which could be serious, or it might not work as well as expected. We don't yet know all the effects of paltusotine. You will be fully informed about all known risks before you make a decision, and you have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (39)

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

Crinetics Study Site
Verified postcode
Los Angeles, United States
Crinetics Study Site
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Torrance, United States
Crinetics Study Site
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Baltimore, United States
Crinetics Study Site
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Boston, United States
Crinetics Study Site
Verified postcode
Philadelphia, United States
Crinetics Study Site
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Pittsburgh, United States
Crinetics Study Site
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Nashville, United States
Crinetics Study Site
Unverified
CABA, Argentina
Crinetics Study Site
Unverified
Ciudad Autonoma de Buenos Aire, Argentina
Crinetics Study Site
Verified postcode
Córdoba, Argentina
Crinetics Study Site
Verified postcode
Ghent, Belgium
Crinetics Study Site
Verified postcode
Fortaleza, Brazil

Common questions

What is acromegaly?

Acromegaly is a condition where your body makes too much growth hormone, causing parts of your body to grow unusually large over time.

What is paltusotine?

Paltusotine is a new oral medicine being tested for acromegaly. It works similarly to some existing injection treatments to help control the condition.

What is a placebo?

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

Can I still take my usual acromegaly medicine?

The study focuses on people who are already on stable somatostatin-based treatments, but specific instructions about your current medications will be given by the study team.

What phase is this study?

This is a Phase 3 study, which means it's one of the final stages of testing to check if the new medicine is safe and works well enough to be considered for wider use.