A Phase 3, Randomized, Double-blind, Placebo-controlled, Parallel-group Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome
Samir Gupta, MD
Primary Investigator
Brief description of study
What is the purpose of this study?
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 Prader-Willi Syndrome (PWS).
Detailed description of study
What will happen during the study?
Experimental: Carbetocin
Carbetocin nasal spray 3.2 mg three times daily (TID) Drug: Carbetocin
Placebo Comparator: Placebo
Placebo given TID, identical in appearance respective to carbetocin treatment
Eligibility of study
You may be eligible for this study if you meet the following criteria:
- Conditions: Hyperphagia, Prader-Willi Syndrome, PWS, Riley
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Age: 5 years - 30 years
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Gender: All
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.
