Evaluation of Tolerance, Suckling and Food Intake After Repeated Nasals Administrations of Oxytocin in PWS Infants

NCT02205034 | Completed Phase 1 Results DMC

• 2 other identifiers: 12 391 021239102
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 1

Brief Summary

The Prader-Willi syndrome (PWS) includes severe neonatal hypotonia with impaired suckling leading to failure to thrive in the most severe cases, subsequently followed by an early onset of morbid obesity with hyperphagia and deficit of satiety, combined with other endocrine dysfunction probably due to hypothalamic dysfunction. The pathophysiological mechanism of the occurrence of the 2 main nutritional phases of PWS is unknown. Swaab reported a deficit in the oxytocin (OT)-producing neurons of the paraventricular nucleus in the brain of these patients. In addition of its well-known anorexigenic effect, OT is involved in establishing and maintaining social codes. Moreover in a PWS mouse model generated from a MAGEL2 KO gene a single OT injection at 5 hr of life prevent the early death observed in 50 % of the new born mice by recovering normal suckling. Interestingly this effect is no longer observed if OT injection takes place later. Our hypothesis is that early administration of OT in babies with PWS may improve suckling and possibly infant-mother interactions. In our recent study (manuscript in preparation), we have shown that a single intranasal administration of OT is well tolerated. This escalating dose study is designed to evaluate the tolerance of repeated intranasal administration of OT in 3 steps (4IU every other day, 4 IU daily, 4IU twice daily) in babies younger than 5 months with PWS.

Conditions

Prader Willi Syndrome

Keywords

Prader Willi

Outcome Measures

Primary Outcomes (1)

• Occurence of Adverse Event

  Occurrence of adverse event, description and quantification of their severity, imputability to repeated intranasal administration of OT (4IU every other day, 4 IU daily, 4IU twice daily) during the 7 days following the first administration.

  up to day 8 (Visit 8)

Secondary Outcomes (2)

• NOMAS Score

  Before and after 7 days of treatment

• Videofluoroscopy of Swallowing Score

  before and after 7 days of treatment

Study Arms (3)

first arm

ACTIVE COMPARATOR

4IU of oxytocin every other day

Drug: oxytocin

second arm

ACTIVE COMPARATOR

4 IU of oxytocin daily

Drug: oxytocin

third arm

ACTIVE COMPARATOR

4 IU of oxytocin twice daily

Drug: oxytocin

Interventions

oxytocin DRUG

Intranasal administration

Also known as: Oxytocin (Syntocinon)

first arm; second arm; third arm

Eligibility Criteria

• Age: 1 Month - 5 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Infants with PWS genetically confirmed
• Aged less than 5 months

You may not qualify if:

• Infants presenting hepatic insufficiency
• Infants presenting renal insufficiency
• Infants with abnormal ECG

Sponsors & Collaborators

• University Hospital, Toulouse: lead

Study Sites (1)

Centre de réfrence Prader-Willi, Hospital of infants

Toulouse, 31059, France

Location

Related Publications (3)

• Tauber M, Boulanouar K, Diene G, Cabal-Berthoumieu S, Ehlinger V, Fichaux-Bourin P, Molinas C, Faye S, Valette M, Pourrinet J, Cessans C, Viaux-Sauvelon S, Bascoul C, Guedeney A, Delhanty P, Geenen V, Martens H, Muscatelli F, Cohen D, Consoli A, Payoux P, Arnaud C, Salles JP. The Use of Oxytocin to Improve Feeding and Social Skills in Infants With Prader-Willi Syndrome. Pediatrics. 2017 Feb;139(2):e20162976. doi: 10.1542/peds.2016-2976.

  PMID: 28100688 RESULT

• Viaux-Savelon S, Rosenblum O, Guedeney A, Diene G, Cabal-Berthoumieu S, Fichaux-Bourin P, Molinas C, Faye S, Valette M, Bascoul C, Cohen D, Tauber M. Dyssynchrony and perinatal psychopathology impact of child disease on parents-child interactions, the paradigm of Prader Willi syndrom. J Physiol Paris. 2016 Nov;110(4 Pt B):427-433. doi: 10.1016/j.jphysparis.2017.08.001. Epub 2017 Sep 1.

  PMID: 28823614 RESULT

• 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: 33232306 DERIVED

MeSH Terms

Conditions

Prader-Willi Syndrome

Interventions

Oxytocin

Condition Hierarchy (Ancestors)

Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn; Imprinting Disorders; Obesity; Overweight; Overnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Pituitary Hormones, Posterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins

Results Point of Contact

• Title: Pr Maithe Tauber
• Organization: CHU Toulouse

tauber.mt@chu-toulouse.fr

+33534558551

Study Officials

• Maïthé TAUBER

  Endocrinologie pédiatrique, Hospital of Toulouse

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 22, 2014
• First Posted: July 31, 2014
• Study Start: May 1, 2013
• Primary Completion: July 1, 2014
• Study Completion: July 1, 2014
• Last Updated: February 21, 2024
• Results First Posted: December 4, 2023

Record last verified: 2024-01

Locations

FR (1)