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

Phase 2

Completed

Conditions: Hyperphagia in Prader-Willi Syndrome

Interventions: Drug: FE 992097
Drug: Placebo

Registration Number: NCT01968187

Lead Sponsor: Ferring Pharmaceuticals

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.

Detailed Description: Not available

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 38

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

Study & Design

Study Type: INTERVENTIONAL

Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Carbetocin (FE 992097)	FE 992097	-
Placebo	Placebo	-

Primary Outcome Measures

Name	Time	Method
Change From Baseline in Hyperphagia in Prader-Willi Syndrome (PWS) Questionnaires- Responsiveness (HPWSQ-R) Total Score at End-of-treatment (Day 15)	From Day 1 (baseline) to 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.

Secondary Outcome Measures

Name	Time	Method
Clinical Global Impression- Improvement After Treatment (CGI-I) Score at End-of-treatment (Day 15)	At Day 15	The Clinical Global Impression (CGI) scale consists of a 7-point clinician rating of illness severity (1 = normal, not at all ill, 7 = among the most extremely ill patients), at the beginning of the trial (baseline) - Clinical Global Impression-Severity Rating (CGI-S) and a 7-point clinician rating of improvement of patient condition (1=very much improved since baseline/initiation of treatment, 7=very much worse from baseline), during and at the end of the trial (Day 15) - CGI-I. The CGI-I score at Day 15 is presented for this outcome measure.
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	The HPWSQ-R is an 11-item questionnaire examining the psychological, developmental, and neurobiological correlates of hyperphagia in PWS. The items were classified into 3 domains; behavior, drive, and severity with each item rated on a five-point score range from 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. Changes in the HPWSQ-R Total Score and in the Domain Scores (behavior, drive, and severity) at Day 15 is presented for this outcome measure. HPWSQ-R Behavior, Drive and Severity scores range from 5-25, 4-20, and 2-10, respectively, with higher scores indicating a worse outcome. Change from baseline is presented = (Day 15 score minus Baseline score).
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	The CY-BOCS is a clinician rated, semi-structured inventory of specific symptoms and symptom severity in pediatric obsessive-compulsive disorder (OCD). Total scores on the CY-BOCS are calculated using a symptom checklist and severity scale. The 10 severity items are summed to produce an Obsessions Severity Score (5 items), Compulsions Severity Score (5 items), and Total score (sum of all 10 severity items). The total score is calculated by summing the 10 individual scores and ranges from 0 (no obsessions or compulsions) to 40 (most severe OC).
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	The Food Domain Score of the Reiss Profile consisted of 7 questions that pertain to food seeking behavior. The questions were rated on a five point scale ranging from -2 (strongly disagree; this phrase is not at all characteristic of the person) to 2 (strongly agree; this phrase is definitely characteristic of the person). Total score was defined as the sum of all individual item scores. The total score ranged from -14 to 14 with higher scores indicating higher severity. Change from baseline in Food Domain of the Reiss Profile at Day 15 is presented for this outcome measure.

Trial Locations

Locations (3): Florida University
🇺🇸
Gainesville, Florida, United States
Winthrop University
🇺🇸
Mineola, New York, United States
Vanderbilt University
🇺🇸
Nashville, Tennessee, United States