Clinical trial NCT06173531: Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome
Official title: A Phase 3, Randomized, Double-blind, Placebo-controlled, Parallel-group Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Hyperphagia in Prader-Willi Syndrome
Keywords: Randomized
Placebo-controlled
Lead sponsor: ACADIA Pharmaceuticals Inc.
Sponsor class: INDUSTRY
First posted: 2023-12-15
Enrollment: 170
Sex: ALL
Minimum age (years): 5
Maximum age (years): 30
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 12-week, randomized, double-blind, placebo-controlled, parallel-group study of carbetocin nasal spray for the treatment of hyperphagia in Prader-Willi syndrome (PWS)

Detailed description: This is a 12-week, multicenter, randomized, double-blind, placebo-controlled, parallel-group study comparing carbetocin nasal spray 3.2 mg TID with placebo (matched placebo nasal spray TID) in subjects with PWS.

Eligibility criteria: Inclusion Criteria:

* Male or female and 5 through 30 years of age
* Prader-Willi syndrome with a documented disease-causing mutation
* Increased appetite with decreased satiety accompanied by food seeking (consistent with PWS Nutritional Phase 3)
* HQ-CT total score of ≥13 at Screening and Baseline
* CGI-S score for hyperphagia in PWS of ≥4 at Screening and Baseline
* 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:

* Genetically diagnosed with Schaaf-Yang syndrome or another genetic, hormonal, or chromosomal cognitive impairment besides PWS
* An active upper respiratory infection at the Screening visit or the Baseline visit
* Any clinically significant cardiovascular disorder, renal, hepatic, gastrointestinal, or respiratory disease, including severe asthma
* History of, or current, cerebrovascular disease, brain trauma, epilepsy, or frequent migraines. A history of febrile seizures is not exclusionary
* Nasal surgery within 1 month of Screening visit or planning to have nasal surgery during the study.
* Unwilling to abstain from nasal saline, other nasal irrigation, and other intranasal medications during the Screening period and through the treatment period of the study
* Clinically significant irritability or agitation, requiring initiation of or increase in the dose of antipsychotic medication, within the 6 months prior to the Screening visit
* Used prostaglandins, prostaglandin analogues, or prostaglandin agonists in the 3 months prior to the Baseline visit. Inhibitors of prostaglandin synthesis, such as nonsteroidal anti-inflammatory drugs, are not exclusionary.
* Started a glucagon-like peptide 1 (GLP-1) agonist within the 6 months prior to the Screening visit. Treatment with GLP-1 agonist is allowed if the subject has been taking it for more than 6 months prior to Screening.
* Used oxytocin, desmopressin (DDAVP), or tesofensine within 6 months prior to the Baseline visit
* Active psychotic symptoms, a history of psychotic symptoms, or a psychotic disorder
* History of suicide attempt or inpatient psychiatric hospitalization
* New food-related interventions, including environment or dietary restrictions, within 1 month of the Screening visit

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

Primary outcomes:
measure: Change from Baseline at Week 12 in caregiver-rated Hyperphagia Questionnaire for Clinical Trials (HQ-CT) score
timeFrame: Baseline to Week 12
Secondary outcomes:
measure: Change from Baseline at Week 12 in caregiver-rated Clinical Global Impression-Severity (CGI-S) score for PWS
timeFrame: Baseline to Week 12
measure: Clinical Global Impression-Change (CGI-C) for PWS score at Week 12
timeFrame: Score at Week 12
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 & 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 Hospital
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: Toulouse
country: France
facility: Centre Hospitalier Universitaire (CHU) de Toulouse - Hôpital des Enfants
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 Research Facility
city: London
country: United Kingdom
facility: Barts Health NHS Trust - The Royal London Hospital
Study officials:
References: