NCT04283578

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.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
52

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Mar 2020

Geographic Reach
3 countries

7 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

February 19, 2020

Completed
6 days until next milestone

First Posted

Study publicly available on registry

February 25, 2020

Completed
14 days until next milestone

Study Start

First participant enrolled

March 10, 2020

Completed
1.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 16, 2021

Completed
5 months until next milestone

Study Completion

Last participant's last visit for all outcomes

March 14, 2022

Completed
Last Updated

June 1, 2023

Status Verified

May 1, 2023

Enrollment Period

1.6 years

First QC Date

February 19, 2020

Last Update Submit

May 31, 2023

Conditions

Prader-Willi Syndrome

Outcome Measures

Primary Outcomes (1)

  • Neonatal Oral-Motor Assessment Scale (NOMAS) scale

    the score goes from 8 to 28, the higher the score meaning a worse outcome

    4 weeks

Secondary Outcomes (3)

  • Ghrelin dosage

    Day 0, Week 1 and week 4

  • Oxytocin dosage

    Day 0, week 4

  • Proficiency score

    Day 0, Week and week 4

Study Arms (2)

Oxytocin

EXPERIMENTAL

intranasal administration of OT

Drug: OTDrug: Placebo comparator

Placebo

PLACEBO COMPARATOR

intranasal administration of placebo

Drug: OTDrug: Placebo comparator

Interventions

OTDRUG

One group will receive OT for 4 weeks, then placebo or OT for 8 weeks

Also known as: oxytocin
OxytocinPlacebo
Placebo comparatorDRUG

One group will receive Placebo for 4 weeks, then Placebo or OT for 8 weeks.

OxytocinPlacebo

Eligibility Criteria

Age1 Day - 92 Days
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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.

You may not qualify if:

  • \. Neonate or infant currently admitted to the emergency care unit for ongoing life-threatening comorbidities like severe respiratory, cardiovascular or neurological abnormalities.
  • \. 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.
  • \. Neonate or infant with concomitant treatment prolonging QT interval 6. Neonate or infant with family history of genetic pathology causing QT interval prolongation.
  • \. Neonate or infant with hypokalemia (clinically relevant at the discretion of the doctor).
  • \. Neonate or infant participating simultaneously in another interventional study.
  • \. Neonates or infants whose parents' situations may jeopardize the interpretation of the results.
  • \. Neonates or infants whose parents' refuse video recording, required to respond to the primary objective of the study.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (7)

Cliniques Saint Luc

Brussels, Belgium

Location

Hôpital Femme Mère Enfant

Bron, France

Location

Hôpital Jeanne de Flandre

Lille, France

Location

Hôpital de la Timone Enfant

Marseille, France

Location

Groupe Hospitalier Necker - Enfants Malades

Paris, France

Location

Centre de réfrence Prader-Willi, Hospital of infants

Toulouse, 31059, France

Location

Klinik für Kinderheilkunde II

Essen, Germany

Location

Related Publications (1)

  • 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.

    PMID: 33232306DERIVED

MeSH Terms

Conditions

Prader-Willi Syndrome

Interventions

Oxytocin

Condition Hierarchy (Ancestors)

Intellectual DisabilityNeurobehavioral ManifestationsNeurologic ManifestationsNervous System DiseasesAbnormalities, MultipleCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesChromosome DisordersGenetic Diseases, InbornImprinting DisordersObesityOverweightOvernutritionNutrition DisordersNutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Pituitary Hormones, PosteriorPituitary HormonesPeptide HormonesHormonesHormones, Hormone Substitutes, and Hormone AntagonistsPeptidesAmino Acids, Peptides, and Proteins

Study Officials

  • Maithé TAUBER, MD

    University Hospital, Toulouse

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 19, 2020

First Posted

February 25, 2020

Study Start

March 10, 2020

Primary Completion

October 16, 2021

Study Completion

March 14, 2022

Last Updated

June 1, 2023

Record last verified: 2023-05

Data Sharing

IPD Sharing
Will not share

Locations

DE(1)FR(5)BE(1)