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Early Treatment Outcomes in Pierre-Robin-Like Phenotype

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ClinicalTrials.gov Identifier: NCT02266043
Recruitment Status : Completed
First Posted : October 16, 2014
Last Update Posted : May 10, 2018
Sponsor:
Collaborator:
German Federal Ministry of Education and Research
Information provided by (Responsible Party):
University Hospital Tuebingen

Tracking Information
First Submitted Date September 30, 2014
First Posted Date October 16, 2014
Last Update Posted Date May 10, 2018
Actual Study Start Date January 1, 2013
Actual Primary Completion Date August 31, 2016   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: October 10, 2014)
Change in mixed-obstructive apnea index (MOAI) [ Time Frame: upon admission within the first year of life (before start of treatment) and 3 months after hospital discharge ]
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
Original Primary Outcome Measures Same as current
Change History Complete list of historical versions of study NCT02266043 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures
 (submitted: October 10, 2014)
  • Change in standard deviation score for body weight [ Time Frame: upon admission within the first year of life (before start of treatment) and 3 months after hospital discharge ]
    Body weight will be obtained by electronic scales and the standard deviation score for weight calculated
  • Duration of hospital stay [ Time Frame: admission within the first year of life for initiation of study intervention ]
  • Treatment failure [ Time Frame: 3 months after admission for initiation of study intervention ]
Original Secondary Outcome Measures Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Early Treatment Outcomes in Pierre-Robin-Like Phenotype
Official Title Cohort Study on Prevalence and Early Treatment Outcomes in Pierre-Robin-Like Phenotype
Brief 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.
Detailed 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.

Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population Infants <1 year admitted with Pierre-Robin-like phenotype to participating centres
Condition 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.

Study Groups/Cohorts Not Provided
Publications * Poets CF, Maas C, Buchenau W, Arand J, Vierzig A, Braumann B, Müller-Hagedorn S. Multicenter study on the effectiveness of the pre-epiglottic baton plate for airway obstruction and feeding problems in Robin sequence. Orphanet J Rare Dis. 2017 Mar 9;12(1):46. doi: 10.1186/s13023-017-0602-8.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Completed
Estimated Enrollment
 (submitted: May 11, 2016)
50
Original Estimated Enrollment
 (submitted: October 10, 2014)
40
Actual Study Completion Date December 31, 2016
Actual Primary Completion Date August 31, 2016   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion Criteria:

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

Exclusion Criteria:

  • no parental consent; need for immediate tracheotomy
Sex/Gender
Sexes Eligible for Study: All
Ages up to 12 Months   (Child)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries Germany
Removed Location Countries  
 
Administrative Information
NCT Number NCT02266043
Other Study ID Numbers FACE-Subproject SP5-Tuebingen
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party University Hospital Tuebingen
Study Sponsor University Hospital Tuebingen
Collaborators German Federal Ministry of Education and Research
Investigators
Principal Investigator: Christian F Poets, MD Dept. of Neonatology and Interdisciplinary Centre forCraniofacial Malformations, University Hospital Tuebingen
Principal Investigator: Siegmar Reinert, MD, PhD Dept. of Craniofacial Surgery, University Hospital Tuebingen
PRS Account University Hospital Tuebingen
Verification Date May 2018