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

A Phase 3, Randomized, Double-blind, Placebo-controlled, Parallel-group Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome

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

Study Overview

• Status: Recruiting
• Conditions: Hyperphagia in Prader-Willi Syndrome
• Intervention / Treatment: Drug: Carbetocin; Drug: Placebo

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.

• Study Type: Interventional
• Enrollment (Estimated): 170
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Andrew McGinn; Phone Number: 610-299-6639; Email: andrew.mcginn@acadia-pharm.com
• Study Contact Backup: Name: Kelly O'Rourke-Kosko; Phone Number: 609-250-6909; Email: kkosko@acadia-pharm.com

Study Locations

• Canada
  • Alberta
    • Edmonton, Alberta, Canada, T3B 6A8
      • Recruiting
      • Alberta Diabetes Institute
      • Contact: Andrew McGinn
• United States
  • Alabama
    • Birmingham, Alabama, United States, 35233
      • Recruiting
      • Children's of Alabama
      • Contact: Andrew McGinn
  • California
    • Palo Alto, California, United States, 94304
      • Recruiting
      • Stanford University School of Medicine
      • Contact: Andrew McGinn
    • San Diego, California, United States, 92123
      • Recruiting
      • Rady Children's Hospital San Diego
      • Contact: Andrew McGinn
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Recruiting
      • Ann & Robert H. Lurie Children's Hospital of Chicago
      • Contact: Andrew McGinn
  • Iowa
    • Iowa City, Iowa, United States, 52242
      • Recruiting
      • University of Iowa
      • Contact: Andrew McGinn
  • Missouri
    • Kansas City, Missouri, United States, 64108
      • Recruiting
      • Children's Mercy Hospital
      • Contact: Andrew McGinn
  • New York
    • Brooklyn, New York, United States, 11219
      • Recruiting
      • Maimonides Medical Center
      • Contact: Andrew McGinn
  • Tennessee
    • Nashville, Tennessee, United States, 37232
      • Recruiting
      • Vanderbilt Clinical Research Center
      • Contact: Andrew McGinn
  • Texas
    • Fort Worth, Texas, United States, 79104
      • Recruiting
      • Cook Children's Health Care System
      • Contact: Andrew McGinn

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo	Drug: Placebo; Placebo given TID, identical in appearance respective to carbetocin treatment
Experimental: Carbetocin; Carbetocin nasal spray 3.2 mg three times daily (TID)	Drug: Carbetocin; Carbetocin nasal spray 3.2 mg three times daily (TID); Other Names: ACP-101

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
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
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

Collaborators and Investigators

• Sponsor: ACADIA Pharmaceuticals Inc.

Study record dates

Study Major Dates

• Study Start (Actual): November 27, 2023
• Primary Completion (Estimated): May 1, 2026
• Study Completion (Estimated): June 1, 2026

Study Registration Dates

• First Submitted: December 8, 2023
• First Submitted That Met QC Criteria: December 8, 2023
• First Posted (Actual): December 15, 2023

Study Record Updates

• Last Update Posted (Actual): April 2, 2024
• Last Update Submitted That Met QC Criteria: April 1, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Keywords: Placebo-controlled; Randomized
• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Neurologic Manifestations; Neurobehavioral Manifestations; Disease; Congenital Abnormalities; Signs and Symptoms, Digestive; Overnutrition; Nutrition Disorders; Overweight; Genetic Diseases, Inborn; Intellectual Disability; Abnormalities, Multiple; Chromosome Disorders; Obesity; Imprinting Disorders; Syndrome; Prader-Willi Syndrome; Hyperphagia; Physiological Effects of Drugs; Reproductive Control Agents; Oxytocics; Carbetocin
• Other Study ID Numbers: ACP-101-302; 2023-506200-24 (Other Identifier: EU CT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No