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

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 (formerly CRN00808; an oral selective nonpeptide somatostatin receptor type 2 biased agonist) in subjects with non-pharmacologically treated acromegaly.

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

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 either medically naïve, not currently treated, or willing to washout during the study screening period. 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: Pituitary radiation therapy within 3 years of Screening Prior treatment with paltusotine History of ineffective or intolerance to octreotide or lanreotide 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 or previously using pegvisomant or cabergoline (within 16 weeks prior to Screening) or pasireotide LAR (within 24 weeks prior to Screening)

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 achieve biochemical response in IGF-1 (≤1.0× the upper limit of normal [ULN]) at the End of the Randomized Control Phase (EOR) |  | 24 Weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from baseline in IGF-1, in units of ULN, to EOR |  | 24 Weeks | 
| Proportion of subjects with IGF-1 <1.3×ULN at EOR |  | 24 Weeks | 
| Change from baseline in Total Acromegaly Symptoms Diary (ASD) score to EOR | The total ASD score ranges from 0 and 70; lower scores indicate lower symptom burden. | 24 Weeks | 
| Proportion of subjects with GH <1.0 ng/mL at Week 22 |  | 24 Weeks | 

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