Clinical trial NCT06420297: OLE Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome
Official title: A Long-term, Open-label Extension Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome
Study type: INTERVENTIONAL
Overall status: ENROLLING_BY_INVITATION
Phase: PHASE3
Conditions: Hyperphagia in Prader-Willi Syndrome
Keywords: Opel-label extension
safety
tolerability
Lead sponsor: ACADIA Pharmaceuticals Inc.
Sponsor class: INDUSTRY
First posted: 2024-05-20
Enrollment: 160
Sex: ALL
Minimum age (years): 5
Maximum age (years): 30
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: To evaluate long-term safety and tolerability of carbetocin nasal spray (3.2 mg TID) in subjects with PWS

Detailed description: This is a long-term, OLE study to evaluate long-term safety and tolerability of carbetocin nasal spray (3.2 mg TID) in subjects with PWS. Subjects who complete the antecedent double-blind study (ACP-101-302) will be invited to participate in the present study.

Eligibility criteria: Inclusion Criteria:

* Has completed the Week12/EOT visit of the antecedent, Study ACP-101-302
* Met all entry criteria for the antecedent study
* May benefit from long-term treatment with open-label carbetocin in the judgment of the Investigator.
* Lives with a caregiver who understands and is willing and able to adhere to study-related procedures and is willing to participate in all study visits.

Exclusion Criteria:

* History of, or current, cerebrovascular disease, brain trauma, epilepsy, or frequent migraines. A history of febrile seizures is not exclusionary.
* Active psychotic symptoms, a history of psychotic symptoms, or a psychotic disorder
* History of suicide attempt or inpatient psychiatric hospitalization
* Has a clinically significant abnormality in vital signs at Baseline
* Has an average QTcF interval of \>450 ms on the Baseline ECG performed before the first dose of carbetocin is given in the present study (i.e., the ECG performed at the EOT visit of the antecedent study)
* Has developed a clinically significant ECG finding during the antecedent study
* Subject is judged by the Investigator or the Medical Monitor to be inappropriate for the study due to AEs, medical condition, or noncompliance with investigational product or study procedures in the antecedent study

Additional inclusion/exclusion criteria apply. Subjects will be evaluated at screening to ensure that all criteria for study participation are met.

Primary outcomes:
measure: Treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), withdrawals due to adverse events (AEs), potentially clinically important changes in other safety assessments, device incidents or device malfunctions
timeFrame: Baseline to 36 months
Secondary outcomes:
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Children's of Alabama
state: Alabama
city: Phoenix
country: United States
facility: Phoenix Children's Hospital
state: Arizona
city: Orange
country: United States
facility: University of California Irvine
state: California
city: Palo Alto
country: United States
facility: Stanford University School of Medicine
state: California
city: San Diego
country: United States
facility: Rady Children's Hospital San Diego
state: California
city: Aurora
country: United States
facility: Children's Hospital Colorado
state: Colorado
city: Chicago
country: United States
facility: Ann &amp; Robert H. Lurie Children's Hospital of Chicago
state: Illinois
city: Iowa City
country: United States
facility: University of Iowa
state: Iowa
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Kansas City
country: United States
facility: Children's Mercy Kansas City
state: Missouri
city: Saint Louis
country: United States
facility: SSM Health/Saint Louis University
state: Missouri
city: Brooklyn
country: United States
facility: Maimonides Medical Center
state: New York
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
city: Pittsburgh
country: United States
facility: UPMC-Children's Hospital Pittsburgh
state: Pennsylvania
city: Nashville
country: United States
facility: Vanderbilt Clinical Research Center
state: Tennessee
city: Fort Worth
country: United States
facility: Cook Children's Health Care System
state: Texas
city: San Antonio
country: United States
facility: Christus Children's
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah
state: Utah
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
city: Edmonton
country: Canada
facility: Alberta Diabetes Institute
state: Alberta
city: Montreal
country: Canada
facility: CHU Sainte-Justine
state: Quebec
city: Augsburg
country: Germany
facility: KJF Klinik Josefinum gGmbH
city: Essen
country: Germany
facility: Universitätsklinikum Essen
city: Barcelona
country: Spain
facility: Parc Taulí Hospital Universitari
city: Madrid
country: Spain
facility: Hospital General Universitario Gregorio Marañón
city: Málaga
country: Spain
facility: Hospital Regional Universitario de Málaga
city: Birmingham
country: United Kingdom
facility: University Hospitals Birmingham NHS Foundation Trust - Heartlands Hospital
city: Glasgow
country: United Kingdom
facility: Royal Hospital for Children Glasgow Clinical
city: London
country: United Kingdom
facility: Barts Health NHS Trust - The Royal London Hospital
Study officials:
References: