Overview
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.
Principal investigator
Eligibility 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.
* 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.
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