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Oxytocin Treatment in Neonates and Infants With Prader-Willi Syndrome (OTBB3)

Primary Purpose

Prader-Willi Syndrome

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
OT
Placebo comparator
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

Eligibility Criteria

1 Day - 92 Days (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Male or female neonate or infant, with PWS genetically confirmed.
  2. Age <92 days (plus a tolerance of up to 8 days maximum) (for preterm infants, born before 37 weeks amenorrhea, corrected age will be applied).
  3. Signed informed consent obtained from the parents/holders of parental authority.
  4. Parents willing and able to comply with all study procedures.

Exclusion Criteria:

  • 1. Neonate or infant currently admitted to the emergency care unit for ongoing life-threatening comorbidities like severe respiratory, cardiovascular or neurological abnormalities.

    2. Neonate or infant with prolongation of the QT interval. 3. Neonate or infant without medical insurance. 4. Neonate or infant with hypersensitivity to oxytocin or excipients of the product.

    5. Neonate or infant with concomitant treatment prolonging QT interval 6. Neonate or infant with family history of genetic pathology causing QT interval prolongation.

    7. Neonate or infant with hypokalemia (clinically relevant at the discretion of the doctor).

    8. Neonate or infant participating simultaneously in another interventional study.

    9. Neonates or infants whose parents' situations may jeopardize the interpretation of the results.

    10. Neonates or infants whose parents' refuse video recording, required to respond to the primary objective of the study.

Sites / Locations

  • Cliniques Saint Luc
  • Hôpital Femme Mère Enfant
  • Hôpital Jeanne de Flandre
  • Hôpital de la Timone Enfant
  • Groupe Hospitalier Necker - Enfants Malades
  • Centre de réfrence Prader-Willi, Hospital of infants
  • Klinik für Kinderheilkunde II

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Oxytocin

Placebo

Arm Description

intranasal administration of OT

intranasal administration of placebo

Outcomes

Primary Outcome Measures

Neonatal Oral-Motor Assessment Scale (NOMAS) scale
the score goes from 8 to 28, the higher the score meaning a worse outcome

Secondary Outcome Measures

Ghrelin dosage
Concentration of ghrelin (unacylated/UAG and acylated/AG)
Oxytocin dosage
Plasma Oxytocin concentration
Proficiency score
The volume of milk taken in the first five minutes of feeding

Full Information

First Posted
February 19, 2020
Last Updated
May 31, 2023
Sponsor
University Hospital, Toulouse
Collaborators
International Clinical Trials Association, Epidemiological and Clinical Research Information Network
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1. Study Identification

Unique Protocol Identification Number
NCT04283578
Brief Title
Oxytocin Treatment in Neonates and Infants With Prader-Willi Syndrome
Acronym
OTBB3
Official Title
Oxytocin Treatment in Neonates and Infants Aged From 0 to 3 Months With Prader-Willi Syndrome : a Study of Safety and Efficacy on Oral and Social Skills and Feeding Behavior of Intranasal Administration of Oxytocin vs Placebo
Study Type
Interventional

2. Study Status

Record Verification Date
May 2023
Overall Recruitment Status
Completed
Study Start Date
March 10, 2020 (Actual)
Primary Completion Date
October 16, 2021 (Actual)
Study Completion Date
March 14, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
University Hospital, Toulouse
Collaborators
International Clinical Trials Association, Epidemiological and Clinical Research Information Network

4. Oversight

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

5. Study Description

Brief Summary
Prospective, randomized, placebo-controlled, double-blind part of the phase III trial to assess the safety and efficacy of 4 weeks oxytocin (OT) administration on oral and social skills in neonates/infants with Prader-Willi Syndrome (PWS) aged less than 3 months at inclusion. Phase III clinical trial.

6. Conditions and Keywords

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

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
52 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Oxytocin
Arm Type
Experimental
Arm Description
intranasal administration of OT
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
intranasal administration of placebo
Intervention Type
Drug
Intervention Name(s)
OT
Other Intervention Name(s)
oxytocin
Intervention Description
One group will receive OT for 4 weeks, then placebo or OT for 8 weeks
Intervention Type
Drug
Intervention Name(s)
Placebo comparator
Intervention Description
One group will receive Placebo for 4 weeks, then Placebo or OT for 8 weeks.
Primary Outcome Measure Information:
Title
Neonatal Oral-Motor Assessment Scale (NOMAS) scale
Description
the score goes from 8 to 28, the higher the score meaning a worse outcome
Time Frame
4 weeks
Secondary Outcome Measure Information:
Title
Ghrelin dosage
Description
Concentration of ghrelin (unacylated/UAG and acylated/AG)
Time Frame
Day 0, Week 1 and week 4
Title
Oxytocin dosage
Description
Plasma Oxytocin concentration
Time Frame
Day 0, week 4
Title
Proficiency score
Description
The volume of milk taken in the first five minutes of feeding
Time Frame
Day 0, Week and week 4

10. Eligibility

Sex
All
Minimum Age & Unit of Time
1 Day
Maximum Age & Unit of Time
92 Days
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Male or female neonate or infant, with PWS genetically confirmed. Age <92 days (plus a tolerance of up to 8 days maximum) (for preterm infants, born before 37 weeks amenorrhea, corrected age will be applied). Signed informed consent obtained from the parents/holders of parental authority. Parents willing and able to comply with all study procedures. Exclusion Criteria: 1. Neonate or infant currently admitted to the emergency care unit for ongoing life-threatening comorbidities like severe respiratory, cardiovascular or neurological abnormalities. 2. Neonate or infant with prolongation of the QT interval. 3. Neonate or infant without medical insurance. 4. Neonate or infant with hypersensitivity to oxytocin or excipients of the product. 5. Neonate or infant with concomitant treatment prolonging QT interval 6. Neonate or infant with family history of genetic pathology causing QT interval prolongation. 7. Neonate or infant with hypokalemia (clinically relevant at the discretion of the doctor). 8. Neonate or infant participating simultaneously in another interventional study. 9. Neonates or infants whose parents' situations may jeopardize the interpretation of the results. 10. Neonates or infants whose parents' refuse video recording, required to respond to the primary objective of the study.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Maithé TAUBER, MD
Organizational Affiliation
University Hospital, Toulouse
Official's Role
Principal Investigator
Facility Information:
Facility Name
Cliniques Saint Luc
City
Bruxelles
Country
Belgium
Facility Name
Hôpital Femme Mère Enfant
City
Bron
Country
France
Facility Name
Hôpital Jeanne de Flandre
City
Lille
Country
France
Facility Name
Hôpital de la Timone Enfant
City
Marseille
Country
France
Facility Name
Groupe Hospitalier Necker - Enfants Malades
City
Paris
Country
France
Facility Name
Centre de réfrence Prader-Willi, Hospital of infants
City
Toulouse
ZIP/Postal Code
31059
Country
France
Facility Name
Klinik für Kinderheilkunde II
City
Essen
Country
Germany

12. IPD Sharing Statement

Plan to Share IPD
No
Citations:
PubMed Identifier
33232306
Citation
Borie AM, Dromard Y, Guillon G, Olma A, Manning M, Muscatelli F, Desarmenien MG, Jeanneteau F. Correction of vasopressin deficit in the lateral septum ameliorates social deficits of mouse autism model. J Clin Invest. 2021 Jan 19;131(2):e144450. doi: 10.1172/JCI144450.
Results Reference
derived

Learn more about this trial

Oxytocin Treatment in Neonates and Infants With Prader-Willi Syndrome

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