Treatment of Hyperphagia Behavioral Symptoms in Children and Adults Diagnosed With Prader-Willi Syndrome

NCT01968187 | Completed Phase 2 Results FDA Drug

• 1 other identifier: 000114
• Study Type: interventional
• Target: 38
• Locations: 1 country
• Sites: 3

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.

Conditions

Hyperphagia in Prader-Willi Syndrome

Outcome Measures

Primary Outcomes (1)

• Change From Baseline in Hyperphagia in Prader-Willi Syndrome (PWS) Questionnaires- Responsiveness (HPWSQ-R) Total Score at End-of-treatment (Day 15)

  The HPWSQ-R is an 11-item questionnaire examining the psychological, developmental, and neurobiological correlates of hyperphagia in PWS. The items are classified into 3 domains; behavior, drive, and severity with each item rated on a five-point scale (1: not at all/none of the time/extremely easy to 5: extremely/all of the time/extremely hard). The questionnaire was completed by the parent/caregiver using a 1-week recall period. Total score was the sum of all the items in the three domains and ranged from 11 (no hyperphagia behaviors) to 55 (most severe hyperphagia behaviors). Change from baseline in HPWSQ-R Total Score at Day 15 is presented for this outcome measure.

  From Day 1 (baseline) to Day 15

Secondary Outcomes (4)

• Clinical Global Impression- Improvement After Treatment (CGI-I) Score at End-of-treatment (Day 15)

  At Day 15

• Change From Baseline in HPWSQ-R Domain Scores (Behavior, Drive and Severity) at End-of-treatment (Day 15)

  From Day 1 (baseline) to Day 15

• Change From Baseline in Children's Yale-Brown Obsessive Compulsive Scale Score (CY-BOCS) at End-of-treatment (Day 15)

  From Day 1 (baseline) to Day 15

• Change From Baseline in the Food Domain Score of the Reiss Profile at End-of-treatment (Day 15)

  From Day 1 (baseline) to Day 15

Study Arms (2)

Carbetocin (FE 992097)

EXPERIMENTAL

Drug: FE 992097

Placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

FE 992097 DRUG

Each spray pump actuation delivered a 50 μL volume of solution that contained 1.6 mg carbetocin (FE 992097); each dose consisted of 3 spray pump actuations in each nostril that delivered a total of 9.6 mg carbetocin. Parents were instructed to administer 3 intranasal spray actuations in each nostril 3 times daily before meals for 14 days.

Carbetocin (FE 992097)

Placebo DRUG

Each spray pump actuation delivered a 50 μL volume of sterile sodium chloride solution 0.9%; each dose consisted of 3 spray pump actuations in each nostril. Parents were instructed to administer 3 intranasal spray actuations in each nostril 3 times daily before meals for 14 days.

Placebo

Eligibility Criteria

• Age: 10 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Ferring Pharmaceuticals: lead

Study Sites (3)

Florida University

Gainesville, Florida, United States

Location

Winthrop University

Mineola, New York, United States

Location

Vanderbilt University

Nashville, Tennessee, United States

Location

Related Publications (1)

• 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.

  PMID: 29925684 DERIVED

Results Point of Contact

• Title: Global Clinical Compliance
• Organization: Ferring Pharmaceuticals

Disclosure@ferring.com

+1 862-286-5200

Study Officials

• Clinical Development Support

  Ferring Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 9, 2013
• First Posted: October 23, 2013
• Study Start: January 20, 2014
• Primary Completion: July 16, 2014
• Study Completion: July 16, 2014
• Last Updated: March 27, 2025
• Results First Posted: March 27, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)