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Oxytocin Trial in Prader-Willi Syndrome

Primary Purpose

Prader Willi Syndrome

Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Intranasal oxytocin
Placebo
Sponsored by
University of Florida
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Prader Willi Syndrome focused on measuring Prader Willi syndrome, Oxytocin

Eligibility Criteria

5 Years - 11 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Children with genetically confirmed PWS
  • Ages at ≥ 5 years and ≤ 11 years (must start treatment prior to 11th birthday)
  • Child must be in nutritional phase 2b or 3, as determined by the PI at each site.
  • Must currently be on growth hormone treatment, and have been receiving growth hormone treatment for at least one year prior to screening date.
  • Treatment cannot have been interrupted for more than 1 week within 3 months prior to screening date.
  • Priority will be given to children currently enrolled in the RDCRN Natural History study

Exclusion Criteria:

  • Inability to tolerate intranasal administration of medication
  • Hepatic insufficiency (AST/ALT greater than 3 times the normal levels for age)
  • Renal insufficiency (BUN/Creatinine greater than 3 times the normal levels for age)
  • History of an abnormal ECG (as determined by a cardiologist). If there is any question about cardiac function, ECG reports will be reviewed with a cardiologist prior to enrollment in the study.
  • Child not receiving growth hormone treatment
  • Child with hypertension or hypotension for age and sex (blood pressure >97% for age and sex or blood pressure <3% for age and sex)
  • Diabetes mellitus
  • Pregnant or lactating.
  • Schizophrenia or psychosis
  • Taking any psychotropic medications

Sites / Locations

  • University of California, Irvine
  • University of Florida
  • University of Kansas Medical Center

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Placebo Comparator

Arm Label

Intranasal oxytocin

Placebo

Arm Description

Intranasal oxytocin. 16 IU intranasal oxytocin x 5 days. One month interval between arms of treatment.

Placebo will be administered via nasal spray - 1 spray in each nostril x5 days.

Outcomes

Primary Outcome Measures

Safety of intranasal oxytocin in children with Prader-Willi syndrome
Occurrence of adverse event, description and quantification of clinical and behavior severity, pre- and post- intranasal oxytocin and placebo administration.

Secondary Outcome Measures

Evaluation of food intake in Prader-Willi syndrome
Quantitative evaluation of hyperphagia via the Hyperphagia Questionnaire obtained on days 1, 4 and 6 during the 7 day study protocol and conducted in the evening on these days. Score will range from 0 (no hyperphagia behaviors) to 96 (most severe hyperphagia behaviors). Additionally quantity of food consumed will be recorded on Days 1,4, and 6.

Full Information

First Posted
December 11, 2013
Last Updated
April 4, 2018
Sponsor
University of Florida
Collaborators
National Institutes of Health (NIH)
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1. Study Identification

Unique Protocol Identification Number
NCT02013258
Brief Title
Oxytocin Trial in Prader-Willi Syndrome
Official Title
Oxytocin Trial in Prader-Willi Syndrome
Study Type
Interventional

2. Study Status

Record Verification Date
April 2018
Overall Recruitment Status
Completed
Study Start Date
March 2015 (undefined)
Primary Completion Date
August 2015 (Actual)
Study Completion Date
August 2015 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
University of Florida
Collaborators
National Institutes of Health (NIH)

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
Individuals with Prader-Willi syndrome (PWS) have been found to have a deficit of oxytocin-producing neurons and decreased oxytocin receptor gene function, so the purpose of this study is to determine if oxytocin (OT) administration will improve some of the aspects of Prader-Willi syndrome that are particularly troublesome for children and their families (the insatiable appetite and social behaviors). The research questions are: Does intranasal oxytocin cause any side effects in children with PWS? Does intranasal oxytocin administration alter appetite or behaviors in PWS?
Detailed Description
This study is to investigate if intranasal oxytocin will improve hyperphagia, social skills, and behaviors in subjects with Prader-Willi syndrome. This will be a randomized placebo controlled pilot study. The primary outcome measure is to determine if intranasal administration of oxytocin will cause any adverse events in subjects with Prader-Willi syndrome. Secondarily, the investigators will also perform evaluations to determine if intranasal oxytocin has any effect on social skills, behaviors, or appetite in children with Prader-Willi syndrome.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Prader Willi Syndrome
Keywords
Prader Willi syndrome, Oxytocin

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Crossover Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
24 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Intranasal oxytocin
Arm Type
Active Comparator
Arm Description
Intranasal oxytocin. 16 IU intranasal oxytocin x 5 days. One month interval between arms of treatment.
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo will be administered via nasal spray - 1 spray in each nostril x5 days.
Intervention Type
Drug
Intervention Name(s)
Intranasal oxytocin
Other Intervention Name(s)
Pitocin, Syntocinon
Intervention Description
This is a double-blind placebo controlled 2x2 study. Subjects will receive OT for 5 consecutive days during their 7 day stay. This will be followed by a wash out period of 4-6 weeks.
Intervention Type
Other
Intervention Name(s)
Placebo
Intervention Description
This is a double-blind placebo controlled 2x2 study. Placebo will be given via intranasal spray, one spray in each nostril daily x 5 days. One month interval between arms of treatment.
Primary Outcome Measure Information:
Title
Safety of intranasal oxytocin in children with Prader-Willi syndrome
Description
Occurrence of adverse event, description and quantification of clinical and behavior severity, pre- and post- intranasal oxytocin and placebo administration.
Time Frame
3 months
Secondary Outcome Measure Information:
Title
Evaluation of food intake in Prader-Willi syndrome
Description
Quantitative evaluation of hyperphagia via the Hyperphagia Questionnaire obtained on days 1, 4 and 6 during the 7 day study protocol and conducted in the evening on these days. Score will range from 0 (no hyperphagia behaviors) to 96 (most severe hyperphagia behaviors). Additionally quantity of food consumed will be recorded on Days 1,4, and 6.
Time Frame
3 months
Other Pre-specified Outcome Measures:
Title
Evaluation of social effects of intranasal oxytocin in children with Prader-Willi syndrome
Description
Evaluation of anxiety, food issues, irritability, social communication and behavioral issues will be measured on days 1, 4 and 6 using: Aberrant Behavior Checklist: Behavior rating scale used to measure behavior problems of children and adults with intellectual disabilities ages 6 - 54 years. Scores will range from 0 (no problem behaviors) to 174 (severe aberrant behaviors). Social Responsiveness Scale:designed to measure the breadth of repetitive behaviors. Scores will range from 64 (no repetitive behaviors) to 260 (severe repetitive behaviors). Repetitive Behavior Scales-Revised: Designed to measure the breadth of repetitive behaviors in autism spectrum disorder including: Ritualistic/Sameness Behavior; Stereotypic Behavior; Self-injurious Behavior; Compulsive Behavior; and Restricted Interests. Scores will range from 0 (no autistic behaviors) to 100 (severe autistic behaviors).
Time Frame
3 months
Title
Effects of intranasal oxytocin on appetite-regulating hormones
Description
Evaluation of plasma OT, ghrelin and other neuroendocrine hormones involved in appetite regulation (cortisol, orexin A, ghrelin, leptin, oxytocin, insulin).
Time Frame
3 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
5 Years
Maximum Age & Unit of Time
11 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Children with genetically confirmed PWS Ages at ≥ 5 years and ≤ 11 years (must start treatment prior to 11th birthday) Child must be in nutritional phase 2b or 3, as determined by the PI at each site. Must currently be on growth hormone treatment, and have been receiving growth hormone treatment for at least one year prior to screening date. Treatment cannot have been interrupted for more than 1 week within 3 months prior to screening date. Priority will be given to children currently enrolled in the RDCRN Natural History study Exclusion Criteria: Inability to tolerate intranasal administration of medication Hepatic insufficiency (AST/ALT greater than 3 times the normal levels for age) Renal insufficiency (BUN/Creatinine greater than 3 times the normal levels for age) History of an abnormal ECG (as determined by a cardiologist). If there is any question about cardiac function, ECG reports will be reviewed with a cardiologist prior to enrollment in the study. Child not receiving growth hormone treatment Child with hypertension or hypotension for age and sex (blood pressure >97% for age and sex or blood pressure <3% for age and sex) Diabetes mellitus Pregnant or lactating. Schizophrenia or psychosis Taking any psychotropic medications
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Jennifer L Miller, MD
Organizational Affiliation
University of Florida
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of California, Irvine
City
Orange
State/Province
California
ZIP/Postal Code
92686
Country
United States
Facility Name
University of Florida
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32610
Country
United States
Facility Name
University of Kansas Medical Center
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States

12. IPD Sharing Statement

Citations:
PubMed Identifier
21702900
Citation
Tauber M, Mantoulan C, Copet P, Jauregui J, Demeer G, Diene G, Roge B, Laurier V, Ehlinger V, Arnaud C, Molinas C, Thuilleaux D. Oxytocin may be useful to increase trust in others and decrease disruptive behaviours in patients with Prader-Willi syndrome: a randomised placebo-controlled trial in 24 patients. Orphanet J Rare Dis. 2011 Jun 24;6:47. doi: 10.1186/1750-1172-6-47.
Results Reference
background
Links:
URL
https://www.ncbi.nlm.nih.gov/pubmed/28371242
Description
Oxytocin treatment in children with Prader-Willi syndrome: A double-blind, placebo-controlled, crossover study. Miller JL, Tamura R, Butler MG, Kimonis V, Sulsona C, Gold JA, Driscoll DJ. Am J Med Genet A. 2017 May;173(5):1243-1250.

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Oxytocin Trial in Prader-Willi Syndrome

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