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Follow-up of Prader Willi Syndrome Infants Treated by Oxytocin and Comparison With Not-treated Infants. (OT2SUITE)

Primary Purpose

Prader-Willi Syndrome

Status
Completed
Phase
Not Applicable
Locations
France
Study Type
Interventional
Intervention
Oxytocin
Control
Sponsored by
University Hospital, Toulouse
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, Oxytocin, Communication skills, Oral skills, Brain activity, Hormonal disease

Eligibility Criteria

3 Years - 4 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Infants with Prader Willi Syndrome (genetic diagnosis confirmed)
  • For treated group : infant included in the ancient study
  • For not treated group: infant never treated with oxytocin

Exclusion Criteria:

  • Subject involved in another search including an exclusion period still in progress at the time of inclusion.
  • Impossibility to give parents or legal guardian informed information
  • No coverage by a Social Security scheme
  • Refusal of parents or legal representative to sign consent.

If a patient has a contraindication to Magnetic resonance imaging, it may be included in the study but Magnetic resonance imaging will not be performed.

Sites / Locations

  • Centre de référence du syndrome de Prader-Willi Hôpital des Enfants

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

Oxytocin

Control

Arm Description

Groups of children with Prader Willi Syndrome treated by oxytocin for 7 days during their first 6 months of life.

Groups of children with Prader Willi Syndrome not treated by oxytocin for 7 days during their first 6 months of life.

Outcomes

Primary Outcome Measures

Evaluation of communication skills.
Assessed by Vineland-II scale.

Secondary Outcome Measures

Evaluation of adaptative behavior composite and 3 domains : "Daily living skills", "Socialization", "Motor skills".
Assessed by Vineland-II scale.
Evaluation of behavioral troubles.
Assessed by Child Behaviour Check List questionnaire.
Evaluation of global development.
Assessed by Bayley Scales of Infant and Toddler Development.
Evaluation of orality and eating behaviour.
Assessed by: A questionnaire on eating behavior. An oral evaluation, which combines a clinical examination carried out by the reference center physician, and the assessment of eating behavior during the meal. The fluoroscopy of swallowing.
Evaluation of brain activity.
Assessed by a morphological Magnetic resonance imaging, a resting functional Magnetic resonance imaging.
Evaluation of plasma levels of ghrelin and other peptides involved in feeding behaviour or energy metabolism.
Circulating levels of acylated and non-acylated ghrelin and some peptides and neuropeptides involved in appetite regulation (leptin, cortisol, insulin, Glucagon like peptide-1, pancreatic polypeptide, orexin A, alpha-melanocyte stimulating hormone...).

Full Information

First Posted
March 10, 2017
Last Updated
January 29, 2019
Sponsor
University Hospital, Toulouse
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1. Study Identification

Unique Protocol Identification Number
NCT03081832
Brief Title
Follow-up of Prader Willi Syndrome Infants Treated by Oxytocin and Comparison With Not-treated Infants.
Acronym
OT2SUITE
Official Title
Long Term Evaluation of Infants Aged From 3 to 4 Years Old Included in the Ancient Study (Repeated Administrations of Oxytocin in Infants With Prader Willi Syndrome Aged From 0 to 6 Months) and Comparison With Not Treated and Age-matched Prader Willi Syndrome Infants (OT2SUITE)
Study Type
Interventional

2. Study Status

Record Verification Date
January 2019
Overall Recruitment Status
Completed
Study Start Date
January 2017 (Actual)
Primary Completion Date
May 2018 (Actual)
Study Completion Date
December 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
University Hospital, Toulouse

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

Brief Summary
The objective of this study is to collect data on tolerance and effects of early treatment with oxytocin in children with Prader Willi Syndrome aged from 3 to 4 years and to compare these infants with not treated age-matched infants with Prader Willi Syndrome.
Detailed Description
In accordance with recommendations of regulatory authorities, we want to collect long term data of patients treated with oxytocin before the age of 6 months. Moreover clinical observations of these infants support long term effects on communication skills, global development and behaviour.

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, Oxytocin, Communication skills, Oral skills, Brain activity, Hormonal disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
34 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Oxytocin
Arm Type
Experimental
Arm Description
Groups of children with Prader Willi Syndrome treated by oxytocin for 7 days during their first 6 months of life.
Arm Title
Control
Arm Type
Experimental
Arm Description
Groups of children with Prader Willi Syndrome not treated by oxytocin for 7 days during their first 6 months of life.
Intervention Type
Drug
Intervention Name(s)
Oxytocin
Intervention Description
Infant included in the ancient study (repeated administrations of oxytocin in infants with Prader Willi Syndrome aged from 0 to 6 months)
Intervention Type
Other
Intervention Name(s)
Control
Intervention Description
Not treated.
Primary Outcome Measure Information:
Title
Evaluation of communication skills.
Description
Assessed by Vineland-II scale.
Time Frame
Day 1
Secondary Outcome Measure Information:
Title
Evaluation of adaptative behavior composite and 3 domains : "Daily living skills", "Socialization", "Motor skills".
Description
Assessed by Vineland-II scale.
Time Frame
Day 1
Title
Evaluation of behavioral troubles.
Description
Assessed by Child Behaviour Check List questionnaire.
Time Frame
Day 1
Title
Evaluation of global development.
Description
Assessed by Bayley Scales of Infant and Toddler Development.
Time Frame
Day 2 and 3
Title
Evaluation of orality and eating behaviour.
Description
Assessed by: A questionnaire on eating behavior. An oral evaluation, which combines a clinical examination carried out by the reference center physician, and the assessment of eating behavior during the meal. The fluoroscopy of swallowing.
Time Frame
Day 2
Title
Evaluation of brain activity.
Description
Assessed by a morphological Magnetic resonance imaging, a resting functional Magnetic resonance imaging.
Time Frame
Day 3
Title
Evaluation of plasma levels of ghrelin and other peptides involved in feeding behaviour or energy metabolism.
Description
Circulating levels of acylated and non-acylated ghrelin and some peptides and neuropeptides involved in appetite regulation (leptin, cortisol, insulin, Glucagon like peptide-1, pancreatic polypeptide, orexin A, alpha-melanocyte stimulating hormone...).
Time Frame
Day 1

10. Eligibility

Sex
All
Minimum Age & Unit of Time
3 Years
Maximum Age & Unit of Time
4 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Infants with Prader Willi Syndrome (genetic diagnosis confirmed) For treated group : infant included in the ancient study For not treated group: infant never treated with oxytocin Exclusion Criteria: Subject involved in another search including an exclusion period still in progress at the time of inclusion. Impossibility to give parents or legal guardian informed information No coverage by a Social Security scheme Refusal of parents or legal representative to sign consent. If a patient has a contraindication to Magnetic resonance imaging, it may be included in the study but Magnetic resonance imaging will not be performed.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Maïthé Tauber, Pr
Organizational Affiliation
Centre de référence du syndrome de Prader-Willi- CHU Toulouse
Official's Role
Principal Investigator
Facility Information:
Facility Name
Centre de référence du syndrome de Prader-Willi Hôpital des Enfants
City
Toulouse
ZIP/Postal Code
31059
Country
France

12. IPD Sharing Statement

Plan to Share IPD
No
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Follow-up of Prader Willi Syndrome Infants Treated by Oxytocin and Comparison With Not-treated Infants.

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