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

Study Overview
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Brief Summary
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A randomized, placebo-controlled study designed to evaluate the safety and efficacy of paltusotine (also known as CRN00808; an orally administered nonpeptide somatostatin agonist) in subjects with acromegaly previously treated with somatostatin receptor ligand (SRL) based treatment regimens.

Official Title
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A Randomized, Controlled, Multi-Center Study to Evaluate the Safety and Efficacy of Paltusotine in Subjects With Acromegaly Treated With Long-acting Somatostatin Receptor Ligands (PATHFNDR-1)

Conditions
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Acromegaly

Intervention / Treatment
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* Drug: Paltusotine
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Male and female subjects ≥18 years of age 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method(s) of birth control Willing to provide signed informed consent Exclusion Criteria: Treatment naïve or treatment-withdrawn acromegaly subjects Prior treatment with paltusotine Pituitary surgery within 24 weeks prior to Screening or history of pituitary radiation therapy History or presence of malignancy except adequately treated basal cell and squamous cell carcinomas of the skin within the past 5 years Use of any investigational drug within the past 30 days or 5 half-lives, whichever is longer Known history of HIV, hepatitis B, or active hepatitis C History of alcohol or substance abuse in the past 12 months Any condition that in the opinion of the investigator would jeopardize the subject's appropriate participation in this study Cardiovascular conditions or medications associated with prolonged QT or those which predispose subjects to heart rhythm abnormalities Subjects with symptomatic cholelithiasis Subjects with clinically significant abnormal findings during the Screening Period, or any other medical condition(s) or laboratory findings that, in the opinion of the Investigator, might jeopardize the subject's safety or ability to complete the study Subjects currently taking pasireotide LAR (within 24 weeks prior to Screening) or pegvisomant, dopamine agonists (within 12 weeks prior to Screening), or short acting somatostatin analogs (within 12 weeks prior to first dose of study drug)

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Paltusotine<br> | Drug: Paltusotine<br>* Paltusotine, tablets, once daily by mouth<br>* Other names: CRN00808;|
| Placebo Comparator: Placebo<br> | Drug: Placebo<br>* Placebo, tablets, once daily by mouth<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Proportion of subjects who maintain biochemical response in IGF-1 (≤1.0× the upper limit of normal [ULN]) at the End of the Randomized Control Phase (EOR) |  | 36 Weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from baseline in IGF-1, in units of ULN, to EOR |  | 36 Weeks | 
| Proportion of subjects with GH <1.0 ng/mL at Week 34, out of those who had GH <1.0 ng/mL at baseline |  | 34 Weeks | 
| Change from baseline in Total Acromegaly Symptoms Diary (ASD) score to EOR |  | 36 Weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Acromegaly, PATHFNDR, Paltusotine, CRN00808