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Trial · Interventional

CompletedPhase 2Results postedRegistry data

Oxytocin vs. Placebo for the Treatment Hyperphagia in Children and Adolescents With Prader-Willi Syndrome

Short name OXT-PWS

NCTNCT02629991 (opens ClinicalTrials.gov in a new tab)Other id14-10-427-01
Open at ClinicalTrials.govAsk about this trial

Overview

Status
Completed
Phase
Phase 2
Enrollment
23 participants (actual)
Sponsor
Montefiore Medical Center · Other
Design
Randomized · Quadruple masking
Countries
1 country

United States

Start
Oct 2015
Primary completion
Dec 2016
Completion
May 2018
First posted
15 Dec 2015 (estimated)
Last update posted
7 Oct 2021
Conditions
Prader-Willi Syndrome · Hyperphagia

Timeline

1 trial · 1 completed · 1 with posted results

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
20152016201720182019
  1. Ph 2

    OXT-PWS

    NCT02629991

    Oct 2015 to May 2018

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

The investigators propose a randomized double-blind 8 week treatment trial of intranasal oxytocin (IN-OXT) vs. placebo in 24 subjects aged 5 to 18 years with PWS in order to assess IN-OXT's affect on (1) Eating behaviors (2) Repetitive and disruptive behaviors and (3) Salivary OXT levels.

Interventions

  • Intranasal Oxytocin (IN-OXT)Drug
  • Matched PlaceboOther

Arms2

  • Experimental

    Intranasal Oxytocin

    Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).

  • Placebo comparator

    Matched Placebo

    Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).

Primary outcomes4

Outcomes the sponsor listed as primary.

  1. Hyperphagia Questionnaire (HQ)- Total Score

    Time frame · Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported.

    Registry description

    The Hyperphagia Questionnaire (HQ)- Total Score. The HQ is a 11-item clinician-rated instrument that was designed to measure food related behaviors in PWS. Items map onto 3 subscales; Hyperphagic Behavior, Hyperphagic Drive, and Hyperphagia Severity. The subscales are summed together to compute the Total Score. The Total Score ranges from 11-55, with higher scores indicating more hyperphagia and a worse outcome.

  2. Hyperphagia Questionnaire (HQ)- Behavior Factor Score

    Time frame · Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported.

    Registry description

    The Hyperphagia Questionnaire (HQ)- Behavior Factor Score assesses food seeking behaviors (e.g., stealing food). The Behavior Factor Score ranges from 5-25, with higher scores indicating more hyperphagic behaviors and a worse outcome.

  3. Hyperphagia Questionnaire (HQ)- Drive Factor Score

    Time frame · Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported.

    Registry description

    The Hyperphagia Questionnaire (HQ)- Drive Factor Score assesses the persistence in asking for food. The Drive Factor Score ranges from 4-20, with higher scores indicating higher hyperphagic drive and a worse outcome

  4. Hyperphagia Questionnaire (HQ) -Severity Factor Score

    Time frame · Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported.

    Registry description

    The Hyperphagia Questionnaire (HQ)- Severity Factor Score assesses the severity of hyperphagia. The Severity Factor Score ranges from 2-10, with higher scores indicating higher severity.

Secondary outcomes(6)

  1. Repetitive Behavior Scale-Revised (RBS-R) Ritualistic Behavior Factor Score

    Time frame · Trends over multiple time points (Weeks 0, 4, and 8).

    Registry description

    The Repetitive Behavior Scale-Revised (RBS-R) Ritualistic Behavior Factor Score assesses ritualistic behaviors. Scores range from 0-18, with higher scores indicating more ritualistic behaviors and a worse outcome.

  2. Repetitive Behavior Scale-Revised (RBS-R) Sameness Factor Score

    Time frame · Trends over multiple time points (Weeks 0, 4, and 8).

    Registry description

    The Repetitive Behavior Scale-Revised (RBS-R) Sameness Factor Score assesses the need for sameness in the environment. Scores range from 0-33, with higher scores indicating more need for sameness and a worse outcome.

  3. Repetitive Behavior Scale-Revised (RBS-R) Stereotyped Behavior Factor Score

    Time frame · Trends over multiple time points (Weeks 0, 4, and 8).

    Registry description

    The Repetitive Behavior Scale-Revised (RBS-R) Stereotyped Behavior Factor Score assesses stereotyped behaviors. Scores range from 0-18, with higher scores indicating more stereotyped behaviors and a worse outcome.

  4. Repetitive Behavior Scale-Revised (RBS-R) Compulsive Behavior Factor Score

    Time frame · Trends over multiple time points (Weeks 0, 4, and 8).

    Registry description

    The Repetitive Behavior Scale-Revised (RBS-R) Compulsive Behavior Factor Score assesses compulsive behaviors. Scores range from 0-24, with higher scores indicating more compulsive behaviors and a worse outcome.

  5. Repetitive Behavior Scale-Revised (RBS-R) Self-Injurious Factor Score

    Time frame · Trends over multiple time points (Weeks 0, 4, and 8).

    Registry description

    The Repetitive Behavior Scale-Revised (RBS-R) Self-Injurious Factor Score assesses self-injurious behaviors. Scores range from 0-24, with higher scores indicating more self-injurious behaviors and a worse outcome.

  6. Repetitive Behavior Scale-Revised (RBS-R) Restricted Interest Factor Score

    Time frame · Trends over multiple time points (Weeks 0, 4, and 8).

    Registry description

    The Repetitive Behavior Scale-Revised (RBS-R) Restricted Interest Factor Score assesses restricted interests. Scores range from 0-12, with higher scores indicating more restricted interests and a worse outcome.

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

Primary outcome results

Primary outcomeMean· Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported.

Hyperphagia Questionnaire (HQ)- Total Score

GroupValue (score on a scale)Spread
Intranasal Oxytocin27.9110.77
Matched Placebo30.588.08
Primary outcomeMean· Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported.

Hyperphagia Questionnaire (HQ)- Behavior Factor Score

GroupValue (score on a scale)Spread
Intranasal Oxytocin9.555.01
Matched Placebo9.753.47
Primary outcomeMean· Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported.

Hyperphagia Questionnaire (HQ)- Drive Factor Score

GroupValue (score on a scale)Spread
Intranasal Oxytocin10.824.81
Matched Placebo13.584.44
Primary outcomeMean· Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported.

Hyperphagia Questionnaire (HQ) -Severity Factor Score

GroupValue (score on a scale)Spread
Intranasal Oxytocin (IN-OXT)7.552.62
Matched Placebo7.252.09
Secondary and other outcome results(4)
Secondary outcomeMean· Trends over multiple time points (Weeks 0, 4, and 8).

Repetitive Behavior Scale-Revised (RBS-R) Ritualistic Behavior Factor Score

GroupValue (score on a scale)Spread
Intranasal Oxytocin6.273.74
Matched Placebo9.425.00
Secondary outcomeMean· Trends over multiple time points (Weeks 0, 4, and 8).

Repetitive Behavior Scale-Revised (RBS-R) Sameness Factor Score

GroupValue (score on a scale)Spread
Intranasal Oxytocin9.364.72
Matched Placebo14.178.07
Secondary outcomeMean· Trends over multiple time points (Weeks 0, 4, and 8).

Repetitive Behavior Scale-Revised (RBS-R) Stereotyped Behavior Factor Score

GroupValue (score on a scale)Spread
Intranasal Oxytocin2.182.52
Matched Placebo3.332.84
Secondary outcomeMean· Trends over multiple time points (Weeks 0, 4, and 8).

Repetitive Behavior Scale-Revised (RBS-R) Compulsive Behavior Factor Score

GroupValue (score on a scale)Spread
Intranasal Oxytocin5.186.9
Matched Placebo7.54.89

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
GroupDeathsSerious eventsOther events
Intranasal Oxytocin (IN-OXT)Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).0 / 110 / 117 / 11
Matched PlaceboParticipants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).0 / 120 / 127 / 12

Each cell reads participants affected / participants at risk, as the registry posted them.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies0

Studies that cite this trial.

No study cites this trial.

Eligibility

Who could join, as posted.

Sex
All
Age
from 5 Years up to 18 Years
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
Inclusion Criteria: 1. Male or Female child outpatients aged 5 to 18 years 2. Diagnosis of PWS confirmed by genetic testing and patient medical records and history 3. Stable pharmacologic, educational, behavioral and/or dietary interventions for 4 weeks prior to the study start, and for the duration of the study. 4. Have a physical exam and laboratory results that are within the norms for PWS 5. Have a parent/caregiver/guardian that is able to consent for their participation and complete assessments regarding the child's development and behavior improvement throughout the study. Exclusion Criteria: 1. Exposure to any investigational agent in the 30 days prior to randomization 2. Prior chronic treatment with oxytocin. 3. A primary psychiatric diagnosis other than ASD, including bipolar disorder, psychosis, schizophrenia, PTSD or major depressive disorder. These patients will be excluded due to potential confounding results. 4. Pregnant or lactating patients. IN-OXT has not been studied in pregnant or lactating women. 5. A medical condition that might interfere with the conduct of the study, confound interpretation of study results or endanger their own well-being. 6. Plan to initiate or change nonpharmacologic or pharmacologic interventions during the course of the study. 7. Females using an estrogen-based contraceptive. As an alternative to an estrogen based contraceptive, subjects will be counseled to use progesterone-based contraceptives; cervical caps; cervical sponges; or spermicidal foam in combination with a condom. Subjects will need to use a double barrier method to be in the study.

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