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Carbetocin nasal spray for treating hunger 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

Phase 3 Interventional ACADIA Pharmaceuticals Inc. · NCT06173531

This study is testing if a nasal spray called carbetocin can help reduce excessive hunger in young people with Prader-Willi syndrome.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment170 (estimated)
Ages5 Years to 30 Years
SexAll
SponsorACADIA Pharmaceuticals Inc. Industry-sponsored
Locations33 sites (Birmingham, Alabama and 32 other locations)
Trial IDNCT06173531 on ClinicalTrials.gov

What this trial studies

This is a 12-week, multicenter, randomized, double-blind, placebo-controlled study evaluating the efficacy of carbetocin nasal spray in reducing hyperphagia in individuals with Prader-Willi syndrome (PWS). Participants aged 5 to 30 years with a documented mutation causing PWS will be enrolled and randomly assigned to receive either carbetocin or a matched placebo. The study aims to assess changes in appetite and satiety through various clinical measures over the treatment period.

Who should consider this trial

Good fit: Ideal candidates are males and females aged 5 to 30 years diagnosed with Prader-Willi syndrome and experiencing increased appetite and decreased satiety.

Not a fit: Patients with other genetic syndromes or significant comorbid conditions may not benefit from this treatment.

Why it matters

Potential benefit: If successful, this treatment could significantly reduce excessive hunger and improve quality of life for patients with Prader-Willi syndrome.

How similar studies have performed: While this approach is novel for treating hyperphagia in PWS, similar interventions targeting appetite regulation have shown promise in other conditions.

Eligibility criteria

Show full inclusion / exclusion 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.

Where this trial is running

Birmingham, Alabama and 32 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Hyperphagia in Prader-Willi SyndromeRandomizedPlacebo-controlled
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.