Summary

Eligibility
for people ages 5-30 (full criteria)
Location
at UC Irvine
Dates
study started
completion around

Description

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)

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

Details

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.

Keywords

Hyperphagia in Prader-Willi Syndrome, Randomized, Placebo-controlled, Prader-Willi Syndrome, Syndrome, Hyperphagia, Carbetocin

Eligibility

You can join if…

Open to people ages 5-30

  • 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

You CAN'T join if...

  • 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.

Locations

  • University of California Irvine accepting new patients
    Orange California 92697 United States
  • Rady Children's Hospital San Diego accepting new patients
    San Diego California 92123 United States
  • Stanford University School of Medicine accepting new patients
    Palo Alto California 94304 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
ACADIA Pharmaceuticals Inc.
ID
NCT06173531
Phase
Phase 3 Prader-Willi Syndrome Research Study
Study Type
Interventional
Participants
Expecting 170 study participants
Last Updated