Early Treatment Outcomes in Pierre-Robin-Like Phenotype

Pierre Robin Syndrome Clinical Trial

Official title:

Cohort Study on Prevalence and Early Treatment Outcomes in Pierre-Robin-Like Phenotype

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02266043
• Other study ID #: FACE-Subproject SP5-Tuebingen
• Status: Completed
• Start date: January 1, 2013
• Est. completion date: December 31, 2016

Study information

• Verified date: May 2018
• Source: University Hospital Tuebingen
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

In a longitudinal cohort study, we want to assess the effect of the pre-epiglottic baton plate, the main treatment approach currently used in Germany for Pierre-Robin-Like phenotype, on upper airway obstruction and failure to thrive using objective criteria (mixed-obstructive apnea index assessed by polysomnography, standard deviation score for body weight) upon admission and 3 months after hospital discharge.

Description:

Pierre-Robin sequence consists of a small lower jaw, a retropositioned tongue (glossoptosis), and optionally cleft palate, leading to severe upper airway obstruction and failure to thrive; occasionally even to sudden infant death. Treatment approaches are heterogeneous, some are considerably invasive; few have been evaluated by objective tests.

In a longitudinal cohort study, we want to assess the effect of the pre-epiglottic baton plate on upper airway obstruction and failure to thrive using objective criteria (mixed-obstructive apnea index assessed by polysomnography, standard deviation score for body weight) upon admission and 3 months after hospital discharge. The pre-epiglottic baton plate is the main treatment approach currently used in Germany for this condition and practised standard care in the three participating study centers.

These data will help to compare the effect of the main treatment currently used in Germany with international data on more invasive treatments used in this rare condition.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 50
• Est. completion date: December 31, 2016
• Est. primary completion date: August 31, 2016
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 12 Months

Eligibility

Inclusion Criteria:

• Infants < 1 year at admission, Pierre-Robin-like phenotype

Exclusion Criteria:

• no parental consent; need for immediate tracheotomy

Related Conditions & MeSH terms

• Pierre Robin Syndrome

Intervention

Device:
• Pre-epiglottic baton plate (PEBP)
modified acrylic palatal plate in which a velar extension resembling a spur or baton shifts the base of the tongue forward. This treatment is the standard care for infants with Pierre-Robin-Like Phenotype in the participating study centers and not assigned by protocol.

Locations

Country	Name	City	State
Germany	Centre of Competence for Oro- and Craniofacial Malformations and Department of Neonatology, Univ. Hospital of Cologne	Cologne
Germany	Dept. of Neonatology and Interdisciplinary Centre for Craniofacial Malformations, University Hospital Tuebingen	Tuebingen
Germany	Dept. of Orthodontics and University Children's Hospital, University Hospital of Wuerzburg	Würzburg

Sponsors (2)

Lead Sponsor	Collaborator
University Hospital Tuebingen	German Federal Ministry of Education and Research

Country where clinical trial is conducted

Germany, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in mixed-obstructive apnea index (MOAI)	Change in mixed-obstructive apnea index (MOAI) assessed by polygraphy from pre- to post-intervention as a marker of Pierre-Robin-sequence-related breathing disorder	upon admission within the first year of life (before start of treatment) and 3 months after hospital discharge
Secondary	Change in standard deviation score for body weight	Body weight will be obtained by electronic scales and the standard deviation score for weight calculated	upon admission within the first year of life (before start of treatment) and 3 months after hospital discharge
Secondary	Duration of hospital stay		admission within the first year of life for initiation of study intervention
Secondary	Treatment failure		3 months after admission for initiation of study intervention