Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome

Hyperphagia in Prader-Willi Syndrome Clinical Trial

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06173531
• Other study ID #: ACP-101-302
• Secondary ID: 2023-506200-24
• Status: Recruiting
• Phase: Phase 3
• Start date: November 27, 2023
• Est. completion date: June 2026

Study information

• Verified date: June 2024
• Source: ACADIA Pharmaceuticals Inc.
• Contact: Andrew McGinn
• Phone: 610-299-6639
• Email: andrew.mcginn[@]acadia-pharm.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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)

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 170
• Est. completion date: June 2026
• Est. primary completion date: May 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 5 Years to 30 Years

Eligibility

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.

Related Conditions & MeSH terms

• Hyperphagia
• Hyperphagia in Prader-Willi Syndrome
• Prader-Willi Syndrome
• Syndrome

Intervention

Drug:
• Carbetocin
Carbetocin nasal spray 3.2 mg three times daily (TID)
• Placebo
Placebo given TID, identical in appearance respective to carbetocin treatment

Locations

Country	Name	City	State
Canada	Alberta Diabetes Institute	Edmonton	Alberta
Canada	CHU Sainte Justine	Montreal	Quebec
United Kingdom	University Hospitals Birmingham NHS Foundation Trust - Heartlands Hospital	Birmingham
United Kingdom	Royal Hospital for Children Glasgow Clinical Research Facility	Glasgow
United States	Children's Hospital Colorado	Aurora	Colorado
United States	Children's of Alabama	Birmingham	Alabama
United States	Maimonides Medical Center	Brooklyn	New York
United States	Ann & Robert H. Lurie Children's Hospital of Chicago	Chicago	Illinois
United States	Cook Children's Health Care System	Fort Worth	Texas
United States	University of Iowa	Iowa City	Iowa
United States	Children's Mercy Hospital	Kansas City	Missouri
United States	Vanderbilt Clinical Research Center	Nashville	Tennessee
United States	Stanford University School of Medicine	Palo Alto	California
United States	Christus Children's	San Antonio	Texas
United States	Rady Children's Hospital San Diego	San Diego	California

Sponsors (1)

Lead Sponsor	Collaborator
ACADIA Pharmaceuticals Inc.

Countries where clinical trial is conducted

United States,  Canada,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change from Baseline at Week 12 in caregiver-rated Hyperphagia Questionnaire for Clinical Trials (HQ-CT) score	The HQ-CT is a nine-item questionnaire designed to be completed by caregivers of subjects with PWS. It is a revision of the 11-item HPWSQ-R and has been further validated. The Foundation for Prader-Willi Research has made the HQ-CT available for clinical studies in PWS, and it is the consensus instrument within the PWS research community for measuring observable behaviors that stem from subjects' excessive drive to eat.; The HQ-CT should be completed by the same caregiver throughout the study. The HQ-CT will be administered to the caregiver by a rater using standardized prompts. The Food Safe Zone should be administered immediately before administration of the HQ-CT.; A higher score on the HQ-CT indicates greater severity of hyperphagia.	Baseline to Week 12
Secondary	Change from Baseline at Week 12 in caregiver-rated Clinical Global Impression-Severity (CGI-S) score for PWS	The CGI-S is a rating scale that records a clinician's global impression of the current severity of illness on a seven-point scale, using a range of responses from 1 (normal) to 7 (among the most severely ill subjects).	Baseline to Week 12
Secondary	Clinical Global Impression-Change (CGI-C) for PWS score at Week 12	The CGI-C is a rating scale that records a clinician's global impression of change in severity of illness, using a range of responses from 1 (very much improved) to 7 (very much worse).	Score at Week 12