Early Treatment Outcomes in Pierre-Robin-Like Phenotype

NCT02266043 | Completed

• 1 other identifier: FACE-Subproject SP5-Tuebingen
• Study Type: observational
• Target: 50
• Locations: 1 country
• Sites: 3

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.

Conditions

Pierre Robin Syndrome

Keywords

Upper airway obstruction

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (3)

• Change in standard deviation score for body weight

  upon admission within the first year of life (before start of treatment) and 3 months after hospital discharge

• Duration of hospital stay

  admission within the first year of life for initiation of study intervention

• Treatment failure

  3 months after admission for initiation of study intervention

Interventions

Pre-epiglottic baton plate (PEBP) DEVICE

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.

Eligibility Criteria

• Age: Up to 12 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• Sampling Method: Non-Probability Sample

Study Population

Infants \<1 year admitted with Pierre-Robin-like phenotype to participating centres

You may qualify if:

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

You may not qualify if:

• no parental consent; need for immediate tracheotomy

Sponsors & Collaborators

• University Hospital Tuebingen: lead
• German Federal Ministry of Education and Research: collaborator

Study Sites (3)

Centre of Competence for Oro- and Craniofacial Malformations and Department of Neonatology, Univ. Hospital of Cologne

Cologne, 50924, Germany

Location

Dept. of Neonatology and Interdisciplinary Centre for Craniofacial Malformations, University Hospital Tuebingen

Tübingen, 72076, Germany

Location

Dept. of Orthodontics and University Children's Hospital, University Hospital of Wuerzburg

Würzburg, 97080, Germany

Location

Related Publications (1)

• Poets CF, Maas C, Buchenau W, Arand J, Vierzig A, Braumann B, Muller-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.

  PMID: 28274235 DERIVED

MeSH Terms

Conditions

Pierre Robin Syndrome; Airway Obstruction

Condition Hierarchy (Ancestors)

Jaw Abnormalities; Jaw Diseases; Musculoskeletal Diseases; Maxillofacial Abnormalities; Craniofacial Abnormalities; Musculoskeletal Abnormalities; Stomatognathic Diseases; Stomatognathic System Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Respiratory Insufficiency; Respiration Disorders; Respiratory Tract Diseases

Study Officials

• 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

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 30, 2014
• First Posted: October 16, 2014
• Study Start: January 1, 2013
• Primary Completion: August 31, 2016
• Study Completion: December 31, 2016
• Last Updated: May 11, 2018

Record last verified: 2018-05

Locations

DE (3)