Clinical trial NCT01968187: Treatment of Hyperphagia Behavioral Symptoms in Children and Adults Diagnosed With Prader-Willi Syndrome
Official title: A Prospective, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Effectiveness of Intranasal Carbetocin in Subjects With Prader-Willi Syndrome (PWS)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Hyperphagia in Prader-Willi Syndrome
Lead sponsor: Ferring Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2013-10-23
Enrollment: 38
Sex: ALL
Minimum age (years): 10
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to evaluate the safety and effectiveness of intranasal FE 992097 in children and adults with Prader-Willi Syndrome.


Eligibility criteria: Inclusion Criteria:

* Male or female 10-18 years of age (both inclusive)
* Genetically confirmed diagnosis of Prader-Willi Syndrome
* Determined to be in nutritional phase 3 Prader-Willi Syndrome based on Miller et al, 2011

Exclusion Criteria:

* Known genetic, hormonal, or chromosomal cause of cognitive impairment other than Prader-Willi Syndrome
* Presence of currently active psychotic symptoms
* Presence of any cardiovascular disorders, epilepsy, frequent migraines or severe asthma
* Previous diagnosis of autism spectrum disorder by a qualified healthcare provider
* Prior or concomitant use of a selective serotonin reuptake inhibitor (SSRI) or selective norepinephrine reuptake inhibitor (SNRI), antipsychotic medication, wakefulness-promoting drug, or thyroid hormone unless dosage has been stable ≥6 months at time of screening

Primary outcomes:
measure: Change From Baseline in Hyperphagia in Prader-Willi Syndrome (PWS) Questionnaires- Responsiveness (HPWSQ-R) Total Score at End-of-treatment (Day 15)
timeFrame: From Day 1 (baseline) to Day 15
Secondary outcomes:
measure: Clinical Global Impression- Improvement After Treatment (CGI-I) Score at End-of-treatment (Day 15)
timeFrame: At Day 15
measure: Change From Baseline in HPWSQ-R Domain Scores (Behavior, Drive and Severity) at End-of-treatment (Day 15)
timeFrame: From Day 1 (baseline) to Day 15
measure: Change From Baseline in Children's Yale-Brown Obsessive Compulsive Scale Score (CY-BOCS) at End-of-treatment (Day 15)
timeFrame: From Day 1 (baseline) to Day 15
measure: Change From Baseline in the Food Domain Score of the Reiss Profile at End-of-treatment (Day 15)
timeFrame: From Day 1 (baseline) to Day 15
Other outcomes:
Locations:
city: Gainesville
country: United States
facility: Florida University
state: Florida
city: Mineola
country: United States
facility: Winthrop University
state: New York
city: Nashville
country: United States
facility: Vanderbilt University
state: Tennessee
Study officials:
affiliation: Ferring Pharmaceuticals
name: Clinical Development Support
role: STUDY_DIRECTOR
References:
citation: Dykens EM, Miller J, Angulo M, Roof E, Reidy M, Hatoum HT, Willey R, Bolton G, Korner P. Intranasal carbetocin reduces hyperphagia in individuals with Prader-Willi syndrome. JCI Insight. 2018 Jun 21;3(12):e98333. doi: 10.1172/jci.insight.98333. eCollection 2018 Jun 21.