Clinical trial NCT04837040: A Study to Evaluate the Safety and Efficacy of Paltusotine for the Treatment of Acromegaly
Official title: 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)
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Acromegaly
Keywords: Acromegaly
PATHFNDR
Paltusotine
CRN00808
Lead sponsor: Crinetics Pharmaceuticals Inc.
Sponsor class: INDUSTRY
First posted: 2021-04-08
Enrollment: 58
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: Inclusion Criteria:

1. Male and female subjects ≥18 years of age
2. Confirmed diagnosis of acromegaly and controlled (as measured by IGF-1 ≤1.0×ULN) via stable dose of protocol defined somatostatin receptor ligand therapy
3. Females must be non-pregnant and non-lactating, and either surgically sterile, post-menopausal, or using effective method(s) of birth control
4. Willing to provide signed informed consent

Exclusion Criteria:

1. Treatment naïve or treatment-withdrawn acromegaly subjects
2. Prior treatment with paltusotine
3. Pituitary surgery within 24 weeks prior to Screening or history of pituitary radiation therapy
4. History or presence of malignancy except adequately treated basal cell and squamous cell carcinomas of the skin within the past 5 years
5. Use of any investigational drug within the past 30 days or 5 half-lives, whichever is longer
6. Known history of HIV, hepatitis B, or active hepatitis C
7. History of alcohol or substance abuse in the past 12 months
8. Any condition that in the opinion of the investigator would jeopardize the subject's appropriate participation in this study
9. Cardiovascular conditions or medications associated with prolonged QT or those which predispose subjects to heart rhythm abnormalities
10. Subjects with symptomatic cholelithiasis
11. 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
12. 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)

Primary outcomes:
measure: 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)
timeFrame: 36 Weeks
Secondary outcomes:
measure: Change from baseline in IGF-1, in units of ULN, to EOR
timeFrame: 36 Weeks
measure: Proportion of subjects with GH <1.0 ng/mL at Week 34, out of those who had GH <1.0 ng/mL at baseline
timeFrame: 34 Weeks
measure: Change from baseline in Total Acromegaly Symptoms Diary (ASD) score to EOR
timeFrame: 36 Weeks
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Crinetics Study Site
state: California
city: Torrance
country: United States
facility: Crinetics Study Site
state: California
city: Baltimore
country: United States
facility: Crinetics Study Site
state: Maryland
city: Boston
country: United States
facility: Crinetics Study Site
state: Massachusetts
city: Philadelphia
country: United States
facility: Crinetics Study Site
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Crinetics Study Site
state: Pennsylvania
city: Nashville
country: United States
facility: Crinetics Study Site
state: Tennessee
city: Caba
country: Argentina
facility: Crinetics Study Site
state: Buenos Aires
city: Ciudad Autonoma de Buenos Aire
country: Argentina
facility: Crinetics Study Site
state: Buenos Aires
city: Córdoba
country: Argentina
facility: Crinetics Study Site
city: Gent
country: Belgium
facility: Crinetics Study Site
city: Fortaleza
country: Brazil
facility: Crinetics Study Site
state: Ceará
city: Curitiba
country: Brazil
facility: Crinetics Study Site
state: Paraná
city: Porto Alegre
country: Brazil
facility: Crinetics Study Site
state: Rio Grande Do Sul
city: Rio De Janeiro
country: Brazil
facility: Crinetics Study Site
city: Rio De Janeiro
country: Brazil
facility: Crinetics Study Site
city: Sofia
country: Bulgaria
facility: Crinetics Study Site
city: Bron
country: France
facility: Crinetics Study Site
city: Pessac
country: France
facility: Crinetics Study Site
city: Budapest
country: Hungary
facility: Crinetics Study Site
city: Pécs
country: Hungary
facility: Crinetics Study Site
city: Be'er Sheva
country: Israel
facility: Crinetics Study Site
city: Petach Tikva
country: Israel
facility: Crinetics Study Site
city: Roma
country: Italy
facility: Crinetics Study Site
city: San Isidro
country: Peru
facility: Crinetics Study Site
state: Lima
city: Bydgoszcz
country: Poland
facility: Crinetics Study Site
city: Poznań
country: Poland
facility: Crinetics Study Site
city: Kemerovo
country: Russian Federation
facility: Crinetics Study Site
state: Kemerovo Oblast
city: Novosibirsk
country: Russian Federation
facility: Crinetics Study Site
state: Novosibirsk Oblast
city: Novosibirsk
country: Russian Federation
facility: Crinetics Study Site
state: Novosibirsk Oblast
city: Kazan
country: Russian Federation
facility: Crinetics Study Site
state: Republic Of Tatarstan
city: Samara
country: Russian Federation
facility: Crinetics Study Site
state: Samara Oblast
city: Moscow
country: Russian Federation
facility: Crinetics Study Site
city: Moscow
country: Russian Federation
facility: Crinetics Study Site
city: Moscow
country: Russian Federation
facility: Crinetics Study Site
city: Belgrade
country: Serbia
facility: Crinetics Study Site
city: Sheffield
country: United Kingdom
facility: Crinetics Study Site
state: South Yorkshire
city: Coventry
country: United Kingdom
facility: Crinetics Study Site
state: West Midlands
city: London
country: United Kingdom
facility: Crinetics Study Site
Study officials:
References: