What side effects are associated with this type of intervention?

Common treatments for acromegaly include somatostatin receptor ligands (SRLs) such as octreotide, lanreotide, and pasireotide, as well as nonpeptide somatostatin agonists like paltusotine. Known side effects of these treatments can include gastrointestinal issues (nausea, diarrhea, abdominal pain), gallstones, and potential alterations in glucose metabolism leading to hyperglycemia or diabetes. Pasireotide, in particular, has a higher incidence of hyperglycemia and diabetes. Additionally, there can be injection site reactions for injectable formulations.

What prior FDA approvals exist?

The FDA has approved several treatments for acromegaly, primarily focusing on somatostatin receptor ligands (SRLs). These include octreotide and lanreotide, which are long-acting somatostatin analogs. Pasireotide, another somatostatin analog, is also approved for patients who do not respond adequately to other treatments. These drugs work by mimicking the action of somatostatin, a hormone that inhibits growth hormone release. Paltusotine, currently under study, is an orally administered nonpeptide somatostatin agonist, representing a novel approach compared to the injectable peptide-based treatments.

What other types of research exist?

Promising alternatives to Paltusotine for Acromegaly patients include other somatostatin analogs such as octreotide and lanreotide, which have been well-studied and are effective. Additionally, growth hormone receptor antagonists like pegvisomant are also viable options. Emerging therapies and novel drug formulations currently in clinical trials may also provide new options in the near future.

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Somatostatin Agonist

Paltusotine for Acromegaly (PATHFNDR-1 Trial)

Phase 3

Waitlist Available

Research Sponsored by Crinetics Pharmaceuticals Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Confirmed diagnosis of acromegaly and controlled (as measured by IGF-1 ≤1.0×ULN) via stable dose of protocol defined somatostatin receptor ligand therapy

Females must be non-pregnant and non-lactating, and either surgically sterile, post-menopausal, or using effective method(s) of birth control

Must not have

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

Pituitary surgery within 24 weeks prior to Screening or history of pituitary radiation therapy

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

Pivotal Trial

Summary

This trial is testing paltusotine, a pill that mimics a natural hormone, in patients with acromegaly. The goal is to see if it can help control their high growth hormone levels. Paltusotine is being developed for the treatment of acromegaly and neuroendocrine tumors.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Specific side effects for Paltusotine aren't listed here but generally could include digestive issues, changes in blood sugar levels, gallstones symptoms if present before treatment starts, fatigue or allergic reactions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My acromegaly is under control with stable medication.

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

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My acromegaly is under control with stable medication.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I haven't taken any experimental drugs recently.

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

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I have acromegaly and have not started or have stopped treatment.

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I have been treated with paltusotine before.

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I have painful gallstones.

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I have a history of HIV, hepatitis B, or active hepatitis C.

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

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: PaltusotineExperimental Treatment1 Intervention

Group II: PlaceboPlacebo Group1 Intervention

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Paltusotine

2018

Completed Phase 2

~60

0 / 10Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Acromegaly include somatostatin analogs such as octreotide, lanreotide, and pasireotide, which work by mimicking the action of somatostatin, a hormone that inhibits the release of growth hormone (GH). By binding to somatostatin receptors on pituitary tumors, these analogs reduce GH secretion and subsequently lower insulin-like growth factor 1 (IGF-1) levels, which are responsible for the symptoms and complications of Acromegaly. Paltusotine, a nonpeptide somatostatin agonist, operates through a similar mechanism but is orally administered, potentially improving patient compliance and quality of life. Effective management of GH and IGF-1 levels is crucial for preventing the severe complications associated with Acromegaly, such as cardiovascular disease, diabetes, and joint problems.