Phase 2 N=38 Randomized Double-blind Treatment

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

Hyperphagia in Prader-Willi Syndrome

Bottom Line

View on ClinicalTrials.gov: NCT01968187 ↗

Enrolled (actual)

Serious AEs

0.0%

Results posted

Mar 2025

Primary outcome: Primary: Change From Baseline in Hyperphagia in Prader-Willi Syndrome (PWS) Questionnaires- Responsiveness (HPWSQ-R) Total Score at End-of-treatment (Day 15) — -15.6; -8.9 score on a scale — p=0.0290

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: FE 992097 (Drug); Placebo (Drug)
Age: Pediatric, Adult · 10+ yrs
Sex: All
Sponsor: Ferring Pharmaceuticals
Primary completion: Jul 2014

Outcome Measures

Outcome	Result	p-value
PRIMARY Change From Baseline in Hyperphagia in Prader-Willi Syndrome (PWS) Questionnaires- Responsiveness (HPWSQ-R) Total Score at End-of-treatment (Day 15)	-15.6; -8.9	0.0290 sig
SECONDARY Clinical Global Impression- Improvement After Treatment (CGI-I) Score at End-of-treatment (Day 15)	2.7; 3.6	0.0233 sig
SECONDARY Change From Baseline in HPWSQ-R Domain Scores (Behavior, Drive and Severity) at End-of-treatment (Day 15)	-5.9; -3.9; -4.5; -2.9; -3.3; -1.8	0.1172
SECONDARY Change From Baseline in Children's Yale-Brown Obsessive Compulsive Scale Score (CY-BOCS) at End-of-treatment (Day 15)	-8.8; -2.6	0.0047 sig
SECONDARY Change From Baseline in the Food Domain Score of the Reiss Profile at End-of-treatment (Day 15)	-6.9; -2.5	0.0132 sig

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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01968187). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.