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

Acadia Pharmaceuticals

Status and phase: Enrolling; Phase 3

Conditions: Hyperphagia in Prader-Willi Syndrome

Treatments: Drug: Placebo; Drug: Carbetocin

Study type: Interventional

Funder types: Industry

Identifiers

NCT06173531

2023-506200-24 (EudraCT Number)

ACP-101-302

Details and patient eligibility

About

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

Full 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.

Enrollment: 170 estimated patients

Sex: All

Ages: 5 to 30 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

---

170 participants in 2 patient groups, including a placebo group

Carbetocin

Experimental group

Description:

Carbetocin nasal spray 3.2 mg three times daily (TID)

Treatment:

Drug: Carbetocin

Placebo

Placebo Comparator group

Description:

Placebo

Treatment:

Drug: Placebo