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

NCT ID: NCT02629991

Last Updated: 2021-10-07

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 23 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2015-10-31
• Study Completion Date: 2018-05-31

Brief 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.

Detailed Description

The investigators propose to conduct a treatment study of intranasal oxytocin (IN-OXT) vs. placebo in children and adolescents with Prader-Willi Syndrome (PWS). OXT has already been proven safe and effective in a treatment study of socialization and disruptive behavior in adults with PWS and is being used in infants with PWS in an ongoing clinical trial. The investigators hypothesize that OXT will be superior to placebo and have a positive effect on child and adolescent PWS eating and repetitive behaviors. Additional knowledge of OXT's ability to reduce overeating could lead to improvement of patient's quality of life and physical health and reduction in familial stress.

The investigators propose a randomized double-blind 8 week treatment trial of intranasal 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. If superior to placebo, this data will add to the current knowledge that OXT is an effective treatment for hyperphagia as well as other problematic symptomatology of PWS.

Conditions

Prader-Willi Syndrome; Hyperphagia

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Intranasal Oxytocin

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

Group Type: EXPERIMENTAL

Intranasal Oxytocin (IN-OXT)

Intervention Type: DRUG

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

Matched Placebo

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

Group Type: PLACEBO_COMPARATOR

Matched Placebo

Intervention Type: OTHER

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

Interventions

Intranasal Oxytocin (IN-OXT)

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

Intervention Type: DRUG

Matched Placebo

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

Intervention Type: OTHER

Other Intervention Names

Syntocinon

Eligibility 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.

• Minimum Eligible Age: 5 Years
• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Foundation for Prader-Willi Research

OTHER

Sponsor Role: collaborator

Montefiore Medical Center

OTHER

Sponsor Role: lead

Responsible Party

Eric Hollander

Professor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Eric Hollander, MD

Role: PRINCIPAL_INVESTIGATOR

Montefiore Medical Center

Locations

Montefiore Medical Center, Albert Einstein College of Medicine

The Bronx, New York, United States

Countries

United States

Related Links

http://www.fpwr.org/therapeutics-in-development-for-pws/

Foundation for Prader Willi Research

Other Identifiers

14-10-427-01

Identifier Type: -

Identifier Source: org_study_id