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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02629991
• Date of registration: 18/10/2015
• Prospective Registration: No
• Primary sponsor: Montefiore Medical Center
• Public title: Oxytocin vs. Placebo for the Treatment Hyperphagia in Children and Adolescents With Prader-Willi Syndrome OXT-PWS
• Scientific title: Oxytocin vs. Placebo for the Treatment Hyperphagia in Children and Adolescents With Prader-Willi Syndrome
• Date of first enrolment: October 2015
• Target sample size: 24
• Recruitment status: Unknown status
• URL: https://clinicaltrials.gov/show/NCT02629991
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

United States

Key inclusion & exclusion criteria

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.



Age minimum: 5 Years
Age maximum: 18 Years
Gender: All

Health Condition(s) or Problem(s) studied

Hyperphagia

Prader-Willi Syndrome

Intervention(s)

Drug: Intranasal Oxytocin (IN-OXT)

Drug: Matched Placebo

Primary Outcome(s)

Change in Revised Dykens Hyperphagia Scale from Baseline to Endpoint. [Time Frame: From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks)]

Secondary Outcome(s)

Change in Aberrant Behavior Checklist (ABC) from Baseline to Endpoint. [Time Frame: From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks)]

Change in Repetitive Behavior Scale (RBS-R) from Baseline to Endpoint. [Time Frame: From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks)]

Change in Social Responsiveness Scale (SRS) from Baseline to Endpoint. [Time Frame: From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks)]

Change in Children's Yale-Brown Obsessive Compulsive Scale (CYBOCS) from Baseline to Endpoint. [Time Frame: From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks)]

Secondary ID(s)

14-10-427-01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Foundation for Prader-Willi Research