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

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ClinicalTrials.gov Identifier: NCT02629991
Recruitment Status : Active, not recruiting
First Posted : December 15, 2015
Last Update Posted : April 2, 2018
Sponsor:
Collaborator:
Foundation for Prader-Willi Research
Information provided by (Responsible Party):
Eric Hollander, Montefiore Medical Center

Brief Summary:
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 (1) Eating behaviors (2) Repetitive and disruptive behaviors (3) social cognition measures and (4) Salivary OXT levels and plasma levels of ghrelin, pancreatic polypeptide and leptin. The investigators will determine if IN-OXT reduces eating behaviors in addition to improving social cognition and reducing repetitive/disruptive behaviors in children with PWS. 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.

Condition or disease Intervention/treatment Phase
Prader-Willi Syndrome Hyperphagia Drug: Intranasal Oxytocin (IN-OXT) Drug: Matched Placebo Phase 2

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, and social cognition. If proven superior, this data will add to the current knowledge that OXT is a beneficial treatment with minimal side effects for PWS symptoms. 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 (1) Eating behaviors (2) Repetitive and disruptive behaviors (3) social cognition measures and (4) Salivary OXT levels and plasma levels of ghrelin, pancreatic polypeptide and leptin. The investigators will determine if IN-OXT reduces eating behaviors in addition to improving social cognition and reducing repetitive/disruptive behaviors in children with PWS. 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.


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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 24 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Oxytocin vs. Placebo for the Treatment Hyperphagia in Children and Adolescents With Prader-Willi Syndrome
Study Start Date : October 2015
Actual Primary Completion Date : December 2016
Estimated Study Completion Date : May 2018

Resource links provided by the National Library of Medicine

Drug Information available for: Oxytocin

Arm Intervention/treatment
Experimental: Intranasal Oxytocin (IN-OXT)
Syntocinon (synthetic oxytocin) will be used in this protocol. Each subject will receive a dose of 16 IU BID (32 IU a day), and will be instructed to inhale 2 puffs per nostril (4 IU each) twice a day.
Drug: Intranasal Oxytocin (IN-OXT)
Each subject will receive a dose of 16 IU BID (32 IU a day), and will be instructed to inhale 2 puffs per nostril (4 IU each) twice a day.
Other Name: Syntocinon

Placebo Comparator: Matched Placebo
Each subject will receive a dose of 16 IU BID (32 IU a day), and will be instructed to inhale 2 puffs per nostril (4 IU each) twice a day.
Drug: Matched Placebo
Each subject will receive a dose of 16 IU BID (32 IU a day), and will be instructed to inhale 2 puffs per nostril (4 IU each) twice a day.




Primary Outcome Measures :
  1. 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) ]
    Assesses Eating Behaviors and Hyperphagia in PWS. Repeated Measures Analysis.


Secondary Outcome Measures :
  1. 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) ]
    Repeated Measures Analysis.

  2. 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) ]
    Repeated Measures Analysis.

  3. 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) ]
    Repeated Measures Analysis.

  4. 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) ]
    Repeated Measures Analysis.


Other Outcome Measures:
  1. Change in Salivary Oxytocin Levels from Baseline to Endpoint. [ Time Frame: From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks) ]
    Repeated Measures Analysis.



Information from the National Library of Medicine

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Ages Eligible for Study:   5 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02629991


Locations
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United States, New York
Montefiore Medical Center, Albert Einstein College of Medicine
Bronx, New York, United States, 10467
Sponsors and Collaborators
Montefiore Medical Center
Foundation for Prader-Willi Research

Additional Information:
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Responsible Party: Eric Hollander, Professor, Montefiore Medical Center
ClinicalTrials.gov Identifier: NCT02629991     History of Changes
Other Study ID Numbers: 14-10-427-01
First Posted: December 15, 2015    Key Record Dates
Last Update Posted: April 2, 2018
Last Verified: March 2018
Keywords provided by Eric Hollander, Montefiore Medical Center:
Prader-Willi Syndrome (PWS)
Hyperphagia
Additional relevant MeSH terms:
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Prader-Willi Syndrome
Syndrome
Hyperphagia
Disease
Pathologic Processes
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn
Obesity
Overnutrition
Nutrition Disorders
Signs and Symptoms, Digestive
Signs and Symptoms
Oxytocin
Oxytocics
Reproductive Control Agents
Physiological Effects of Drugs