Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

A Randomized, Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Paltusotine in Subjects with Non-pharmacologically Treated Acromegaly (PATHFNDR-2)

The PATHFNDR-2 study is a phase 3 clinical trial investigating a new medication called Paltusotine for people with acromegaly who are not currently taking other drugs for their condition. Acromegaly is a condition where the body produces too much growth hormone. In this study, some participants will receive Paltusotine tablets, while others will receive a placebo (an inactive dummy pill that looks the same). The main goal is to see how many people taking Paltusotine can get their hormone levels (specifically IGF-1) back to a healthier range compared to those taking the placebo. Researchers also want to check if the drug helps with acromegaly symptoms and if it's safe to use. This is a crucial step in potentially making a new treatment available.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Crinetics Pharmaceuticals Inc.

Enrolment target

Start

19 Sep 2024

Acromegaly

What is this study about?

This study, called PATHFNDR-2, is looking for a new way to treat acromegaly, a condition caused by your body making too much growth hormone. This excess hormone can lead to various health problems over time. Currently, treatments often involve surgery or other medications, but this study is testing a new drug called Paltusotine.

The main aim of the study is to see if Paltusotine can help bring a specific hormone level, called IGF-1, back into a normal range. IGF-1 is often high in people with acromegaly. Researchers will compare people taking Paltusotine with a group taking a placebo, which is an inactive tablet that looks just like the real drug. This comparison helps scientists understand if any changes are truly due to the new medication or simply from other factors.

Beyond checking hormone levels, the study will also look at how Paltusotine affects symptoms of acromegaly and its overall safety. Finding a safe and effective new treatment could offer more options for people living with this condition, potentially improving their health and quality of life.

Key takeaways

This study is testing a new drug called Paltusotine for acromegaly.
It's for adults whose acromegaly isn't currently being treated with medication.
The study compares Paltusotine to a dummy pill (placebo) to check effectiveness and safety.
The main goal is to see if Paltusotine helps lower high hormone levels (IGF-1).
Participation involves regular clinic visits, blood tests, and symptom tracking.
You can leave the study at any time without affecting your normal medical care.

Who may be eligible?

To join this study, you need to be an adult, at least 18 years old, and have been diagnosed with acromegaly.

A key requirement is that you should not currently be taking any medication specifically for your acromegaly. This helps the researchers clearly see the effects of the new drug being tested.

Both men and women can participate in the study. Your medical team will review all your health information to make sure this study is the right fit for you.

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?
Have you been diagnosed with acromegaly?
Are you currently NOT taking any medication specifically for your acromegaly?
Are you willing and able to attend regular clinic appointments?

Answer every question to see your result.

What does participation involve?

If you join the study, you'll be given either Paltusotine tablets or a dummy pill (placebo) to take regularly. You won't know which one you're taking, nor will your study doctor, which helps ensure the results are fair. Throughout the study, you'll have regular visits to the clinic for check-ups where doctors will monitor your hormone levels (like IGF-1) with blood tests. They'll also ask you about any symptoms you're experiencing and how you're feeling, possibly using a diary to track symptoms. The total duration of your participation in the study, including initial assessments and follow-up, will be explained in detail by the study team, but typically phase 3 studies involve several months of monitoring.

Potential risks and benefits

Taking part in any clinical trial involves potential benefits and risks. A potential benefit of joining this study is that you might receive access to a new medication, Paltusotine, which could help manage your acromegaly symptoms and hormone levels more effectively than current options or without needing other types of treatment. However, there's also a chance you might receive the placebo and not experience direct benefits from the active drug during the main study phase. Potential risks could include side effects from the medication, which would be carefully monitored by the study team. You have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (7)

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

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Unverified
Germany
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Unverified
France
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Unverified
Hungary
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Unverified
Poland
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Unverified
Italy
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Unverified
Bulgaria
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Unverified
Greece

Common questions

What is acromegaly?

Acromegaly is a condition where your body produces too much growth hormone, often due to a non-cancerous tumour in your pituitary gland. This leads to bigger hands and feet, changes in facial features, and other health issues.

What is Paltusotine?

Paltusotine is a new medication being tested in this study to see if it can help control the high levels of hormones, like IGF-1, found in people with acromegaly.

What does 'placebo' mean?

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

Will I know if I'm taking the real drug or the placebo?

No, neither you nor your study doctor will know whether you are taking Paltusotine or the placebo. This is called 'blinding' and helps ensure the study results are unbiased.

How long will the study last for me?

The exact length of time for your participation will be explained by the study team, but these types of studies usually involve several months of monitoring and clinic visits.