Primary outcomeNumber· up to day 8 (Visit 8)
Occurence of Adverse Event
| Group | Value (number of adverse events) |
|---|---|
| First Arm | 10 |
| Second Arm | 9 |
| Third Arm | 6 |
Trial · Interventional
CompletedPhase 1/2Results postedRegistry dataEvaluation of Tolerance, Suckling and Food Intake After Repeated Nasals Administrations of Oxytocin in PWS Infants
Short name OTBB2
Overview
- Status
- Completed
- Phase
- Phase 1/2
- Enrollment
- 18 participants (actual)
- Sponsor
- University Hospital, Toulouse · Other
- Design
- Not randomized · Open label (no masking)
- Countries
1 country
France
- Start
- May 2013
- Primary completion
- Jul 2014
- Completion
- Jul 2014
- First posted
- 31 Jul 2014 (estimated)
- Last update posted
- 21 Feb 2024
- Conditions
- Prader Willi Syndrome
Timeline
1 trial · 1 completed · 1 with posted results
- Completed
- Ongoing
- Not yet recruiting
- Stopped
- Estimated dates (est.)
- Results posted
201320142015
- Ph 1/2
Registry summary
Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.
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.
Interventions
- oxytocinDrug
Arms3
Active comparator
first arm
4IU of oxytocin every other day
Active comparator
second arm
4 IU of oxytocin daily
Active comparator
third arm
4 IU of oxytocin twice daily
Primary outcomes1
Outcomes the sponsor listed as primary.
Occurence of Adverse Event
Time frame · up to day 8 (Visit 8)
Registry description
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.
Secondary outcomes(2)
NOMAS Score
Time frame · Before and after 7 days of treatment
Registry description
NOMAS score evaluate sucking/swallowing abilitites of infants during feeding; endpoint is the % of infants who reached a NOMAS score \<= 10 (normal score)
Videofluoroscopy of Swallowing Score
Time frame · before and after 7 days of treatment
Registry description
Videofluoroscopy of swallowing score (VFSS score)
Posted results
Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.
Primary outcome results
Secondary and other outcome results(2)
Secondary outcome· Before and after 7 days of treatment
NOMAS Score
| Group | Value |
|---|
Secondary outcome· before and after 7 days of treatment
Videofluoroscopy of Swallowing Score
| Group | Value |
|---|
Adverse events, as posted
| Group | Deaths | Serious events | Other events |
|---|---|---|---|
| First Arm4IU of oxytocin every other day oxytocin: Intranasal administration | 0 / 6 | 1 / 6 | 4 / 6 |
| Second Arm4 IU of oxytocin daily oxytocin: Intranasal administration | 0 / 6 | 0 / 6 | 5 / 6 |
| Third Arm4 IU of oxytocin twice daily oxytocin: Intranasal administration | 0 / 6 | 0 / 6 | 4 / 6 |
Each cell reads participants affected / participants at risk, as the registry posted them.
Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works
Studies1
Studies that cite this trial.
Studies with results
1 study on this page
Indexing hint: Clinical trial · 2017
10 results
The Use of Oxytocin to Improve Feeding and Social Skills in Infants With Prader-Willi Syndrome.
Tauber et al. 2017Pediatricscited by 126
PMID 28100688DOI 10.1542/peds.2016-2976PUB-28100688
Full text at the publisher10 results
Other registry references
References listed at the registry that are not in PepBank. They link to PubMed.
- ResultViaux-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
- DerivedBorie 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
Eligibility
Who could join, as posted.
- Sex
- All
- Age
- from 1 Month up to 5 Months
- Healthy volunteers
- Not accepted
Inclusion and exclusion criteria, as posted
Inclusion Criteria:
* Infants with PWS genetically confirmed
* Aged less than 5 months
Exclusion Criteria:
* Infants presenting hepatic insufficiency
* Infants presenting renal insufficiency
* Infants with abnormal ECG
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