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Completed

NCT Number: NCT01968187

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

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

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Key information

Conditions

Hyperphagia in Prader-Willi Syndrome

Age range

10 year–18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Florida University, Gainesville, Florida, United States

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

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.

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.

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)

    Time frame: 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 outcomes

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

    Time frame: 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.

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

    Time frame: 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).

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

    Time frame: 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).

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

    Time frame: 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.

Sponsors and collaborators

Lead sponsor

Ferring Pharmaceuticals

Industry

Registry information

Official study 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)

Important dates

Study start
2014
Primary completion
2014
Study completion
2014
First posted
Oct 23, 2013
Registry last updated
Mar 27, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

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This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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