- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01548521
Tolerance of Intranasal Administration of OT in Prader-Willi Newborn Babies (OTBB)
February 21, 2017 updated by: University Hospital, Toulouse
Tolerance of Intranasal Administration of OT in Prader-Willi Newborn Babies and Effect on Suck and Food Intake.
Background: Prader-Willi syndrome (PWS) is a rare, complex multisystem genetic disorder arising from the lack of expression of paternally inherited imprinted genes on chromosome 15q11-q13.
The syndrome 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 insatiable hunger, 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.
A deficit in the oxytocin (OT)-producing neurons of the paraventricular nucleus in the brain of these patients has been reported.
In addition of its well-known anorexigenic effect, OT is involved in establishing and maintaining social codes.
Indeed, we have recently shown in a double blind placebo study, that OT administration to adult patients with PWS significantly decreased depressive mood tendencies and tantrums while increasing trust in others with some data on a trend to decrease appetite with higher satiety.
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.
These data, OT deficit in PWS, good tolerance of OT and its effect after intranasal administration in adult patients with PWS and the recent striking data obtained in the MAGEL2 mouse model, prompted us to evaluate the tolerance of a single administration of intranasal OT in PWS newborns and its possible effect on suckling and food intake.
Nowadays the diagnosis of PWS is done during the first months of life in our country.
At this age, children still present with poor suckling suggesting that OT may be still efficient.
Moreover in adult patients with PWS we have shown that OT improves some typical behavioral troubles.
Therefore we first want to evaluate the tolerance of the intranasal administration of OT in 6 infants with PWS genetically confirmed and its effect on suckling, milk intake and weight gain.
Study Overview
Detailed Description
We want to evaluate the tolerance of the intranasal administration of OT in 6 infants with PWS genetically confirmed and its effect on suckling, milk intake and weight gain.
The three first patients will have single nasal administration of 2 IU of Oxytocin and, if no adverse event has been observed, the 3 following patients will have single nasal administration of 4 IU of Oxytocin.
We will monitor cardiac pulse, blood pressure, urine emission and measure biological safety parameters (glycemia, natremia and kaliemia).
Video will be performed during 1 day before the drug administration and the 3 days after in order to qualify the suckling.
Quantitative evaluation of the milk intake during each feeding and per day will be also evaluated.
Biological parameters will be measured OT, ghrelin, others neuroendocrine hormones involved in appetite regulation (leptin, cortisol, insulin, GLP-1, PYY, pancreatic polypeptide, orexin A, aMSH) taking advantage of blood samples for safety biological measurements.
These infants will stay 8 days (which is less than the mean duration of hospitalization of these infants) with data records by phone at 1 month and then have a final visit after 3 months.
Study Type
Interventional
Enrollment (Actual)
5
Phase
- Phase 2
- Phase 1
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Toulouse, France, 31059
- Children Hospital of Toulouse Purpan
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
2 weeks to 5 months (Child)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
Inclusion Criteria:
- neonates with genetic diagnosis of Prader-Willi syndrome
- aged from 15 days to 5 months
Exclusion Criteria:
- exclusive tube feeding
- arrhythmia
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Oxytocin
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2 ui intranasal administration for the 3 first patients, 4UI for the 3 following patients.
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Occurrence of adverse event, description and quantification of their severity, imputability to oxytocin administration.
Time Frame: up to day 8
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up to day 8
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Quantitative evaluation of food intake
Time Frame: from day 1 to month 3
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food intake is measured in ml
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from day 1 to month 3
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Evaluation of plasmatic OT, ghrelin and others neuroendocrine hormones involved in appetite regulation (leptin, cortisol, insulin, GLP-1, PYY, pancratic polypeptide, orexin A, aMSH)
Time Frame: from day 1 to month 3
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from day 1 to month 3
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Principal Investigator: Maithe TAUBER, MD, Hospital of Toulouse
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
July 1, 2011
Primary Completion (Actual)
February 1, 2012
Study Completion (Actual)
April 1, 2012
Study Registration Dates
First Submitted
December 30, 2011
First Submitted That Met QC Criteria
March 7, 2012
First Posted (Estimate)
March 8, 2012
Study Record Updates
Last Update Posted (Actual)
February 23, 2017
Last Update Submitted That Met QC Criteria
February 21, 2017
Last Verified
February 1, 2017
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Nervous System Diseases
- Neurologic Manifestations
- Neurobehavioral Manifestations
- Congenital Abnormalities
- Overnutrition
- Nutrition Disorders
- Genetic Diseases, Inborn
- Intellectual Disability
- Abnormalities, Multiple
- Chromosome Disorders
- Obesity
- Prader-Willi Syndrome
- Physiological Effects of Drugs
- Reproductive Control Agents
- Oxytocics
- Oxytocin
Other Study ID Numbers
- 10 152 02
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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