Phase 2 N=23 Randomized Quadruple-blind Treatment

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

Prader-Willi Syndrome · Hyperphagia

Bottom Line

View on ClinicalTrials.gov: NCT02629991 ↗

Enrolled (actual)

Serious AEs

0.0%

Results posted

Oct 2021

Primary outcome: Primary: Hyperphagia Questionnaire (HQ)- Total Score — 27.91; 30.58; 27.4; 23.33 score on a scale — p=0.059

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Intranasal Oxytocin (IN-OXT) (Drug); Matched Placebo (Other)
Age: Pediatric, Adult · 5+ yrs
Sex: All
Sponsor: Montefiore Medical Center
Primary completion: Dec 2016

Outcome Measures

Outcome	Result	p-value
PRIMARY Hyperphagia Questionnaire (HQ)- Total Score	27.91; 30.58; 27.4; 23.33; 26.91; 25.09	0.059
PRIMARY Hyperphagia Questionnaire (HQ)- Behavior Factor Score	9.55; 9.75; 9.4; 7.22; 9.64; 8.09	0.088
PRIMARY Hyperphagia Questionnaire (HQ)- Drive Factor Score	10.82; 13.58; 11; 10.44; 10.36; 10.55	0.027 sig
PRIMARY Hyperphagia Questionnaire (HQ) -Severity Factor Score	7.55; 7.25; 7.00; 5.67; 6.91; 6.45	0.16
SECONDARY Repetitive Behavior Scale-Revised (RBS-R) Ritualistic Behavior Factor Score	6.27; 9.42; 4.50; 6.27; 4.90; 6.64	0.69
SECONDARY Repetitive Behavior Scale-Revised (RBS-R) Sameness Factor Score	9.36; 14.17; 6.6; 8; 7.3; 7.55	0.034 sig
SECONDARY Repetitive Behavior Scale-Revised (RBS-R) Stereotyped Behavior Factor Score	2.18; 3.33; 2.5; 2; 2.2; 1.09	0.009 sig
SECONDARY Repetitive Behavior Scale-Revised (RBS-R) Compulsive Behavior Factor Score	5.18; 7.5; 4.9; 5.27; 4.5; 5.09	0.033 sig
SECONDARY Repetitive Behavior Scale-Revised (RBS-R) Self-Injurious Factor Score	5.00; 2.82; 4.40; 3.45; 4.40; 3.36	0.78
SECONDARY Repetitive Behavior Scale-Revised (RBS-R) Restricted Interest Factor Score	3.27; 5.17; 2.20; 4.00; 2.10; 3.73	0.53

Summary

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.

Eligibility Criteria

Inclusion Criteria

Male or Female child outpatients aged 5 to 18 years
Diagnosis of PWS confirmed by genetic testing and patient medical records and history
Stable pharmacologic, educational, behavioral and/or dietary interventions for 4 weeks prior to the study start, and for the duration of the study.
Have a physical exam and laboratory results that are within the norms for PWS
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

Exposure to any investigational agent in the 30 days prior to randomization
Prior chronic treatment with oxytocin.
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.
Pregnant or lactating patients. IN-OXT has not been studied in pregnant or lactating women.
A medical condition that might interfere with the conduct of the study, confound interpretation of study results or endanger their own well-being.
Plan to initiate or change nonpharmacologic or pharmacologic interventions during the course of the study.
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.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02629991). 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.