Hyperphagia in Prader-Willi Syndrome Clinical Trial
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
12-week, randomized, double-blind, placebo-controlled, parallel-group study of carbetocin nasal spray for the treatment of hyperphagia in Prader-Willi syndrome (PWS)
| Status | Recruiting |
| Enrollment | 170 |
| Est. completion date | June 2026 |
| Est. primary completion date | May 2026 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 5 Years to 30 Years |
| Eligibility | 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. |
| Country | Name | City | State |
|---|---|---|---|
| Canada | Alberta Diabetes Institute | Edmonton | Alberta |
| United Kingdom | Royal Hospital for Children Glasgow Clinical Research Facility | Glasgow | |
| United States | Children's of Alabama | Birmingham | Alabama |
| United States | Maimonides Medical Center | Brooklyn | New York |
| United States | Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois |
| United States | Cook Children's Health Care System | Fort Worth | Texas |
| United States | University of Iowa | Iowa City | Iowa |
| United States | Children's Mercy Hospital | Kansas City | Missouri |
| United States | Vanderbilt Clinical Research Center | Nashville | Tennessee |
| United States | Stanford University School of Medicine | Palo Alto | California |
| United States | Rady Children's Hospital San Diego | San Diego | California |
| Lead Sponsor | Collaborator |
|---|---|
| ACADIA Pharmaceuticals Inc. |
United States, Canada, United Kingdom,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Change from Baseline at Week 12 in caregiver-rated Hyperphagia Questionnaire for Clinical Trials (HQ-CT) score | The HQ-CT is a nine-item questionnaire designed to be completed by caregivers of subjects with PWS. It is a revision of the 11-item HPWSQ-R and has been further validated. The Foundation for Prader-Willi Research has made the HQ-CT available for clinical studies in PWS, and it is the consensus instrument within the PWS research community for measuring observable behaviors that stem from subjects' excessive drive to eat. The HQ-CT should be completed by the same caregiver throughout the study. The HQ-CT will be administered to the caregiver by a rater using standardized prompts. The Food Safe Zone should be administered immediately before administration of the HQ-CT. A higher score on the HQ-CT indicates greater severity of hyperphagia. |
Baseline to Week 12 | |
| Secondary | Change from Baseline at Week 12 in caregiver-rated Clinical Global Impression-Severity (CGI-S) score for PWS | The CGI-S is a rating scale that records a clinician's global impression of the current severity of illness on a seven-point scale, using a range of responses from 1 (normal) to 7 (among the most severely ill subjects). | Baseline to Week 12 | |
| Secondary | Clinical Global Impression-Change (CGI-C) for PWS score at Week 12 | The CGI-C is a rating scale that records a clinician's global impression of change in severity of illness, using a range of responses from 1 (very much improved) to 7 (very much worse). | Score at Week 12 |
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Completed |
NCT01968187 -
Treatment of Hyperphagia Behavioral Symptoms in Children and Adults Diagnosed With Prader-Willi Syndrome
|
Phase 2 |