Pierre Robin SyndromeNCT02266043
Early Treatment Outcomes in Pierre-Robin-Like Phenotype
An observational study of Pierre Robin Syndrome, run by University Hospital Tuebingen at 3 sites in Germany.
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… Read more about this study
- Who is it for?
- Ages Up to 12 months
- Conditions the study is for
- Pierre Robin Syndrome
- What is being tested?
- Pre-epiglottic baton plate (PEBP)
- Type of treatment
- Medical device
- How many people
- 50 participants
- Where
- 3 sitesGermany
- Study dates
- 1 Jan 2013to 31 Dec 2016
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 12 months
- All sexes
- No healthy volunteers
- Child
Study population: Infants <1 year admitted with Pierre-Robin-like phenotype to participating centres
Who may be able to join
- Infants < 1 year at admission, Pierre-Robin-like phenotype
Who cannot join
- no parental consent; need for immediate tracheotomy
Trial locations and contacts
3 sites in 1 country.
Germany3 sites
Centre of Competence for Oro- and Craniofacial Malformations and Department of Neonatology, Univ. Hospital of Cologne
Cologne, 50924
Dept. of Neonatology and Interdisciplinary Centre for Craniofacial Malformations, University Hospital Tuebingen
Tübingen, 72076
Dept. of Orthodontics and University Children's Hospital, University Hospital of Wuerzburg
Würzburg, 97080
About this study
Official title: Cohort Study on Prevalence and Early Treatment Outcomes in Pierre-Robin-Like Phenotype
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.
Read the 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.
- Conditions
- Pierre Robin Syndrome
- Being tested
- Pre-epiglottic baton plate (PEBP)
- Keywords
- Upper airway obstruction
- Medical subject headings
- Pierre Robin Syndrome, Airway Obstruction
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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.
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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
Body weight will be obtained by electronic scales and the standard deviation score for weight calculated
Measured: upon admission within the first year of life (before start of treatment) and 3 months after hospital discharge
Duration of hospital stay
Measured: admission within the first year of life for initiation of study intervention
Treatment failure
Measured: 3 months after admission for initiation of study intervention
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Oct 2014 (estimated)
- Study start1 Jan 2013
- Primary completion31 Aug 2016
- Study completion31 Dec 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Pierre Robin SyndromeStudies of the same condition, open and under way.
- More trials run by University Hospital TuebingenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University Hospital Tuebingen
- Sponsor type
- University, hospital or other
- Collaborators
- German Federal Ministry of Education and Research
- Responsible party
- Sponsor
- Investigators
- Christian F Poets, MD, Principal investigator, Dept. of Neonatology and Interdisciplinary Centre forCraniofacial Malformations, University Hospital TuebingenSiegmar Reinert, MD, PhD, Principal investigator, Dept. of Craniofacial Surgery, University Hospital Tuebingen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02266043
- Sponsor's protocol ID
- FACE-Subproject SP5-Tuebingen
- Status last verified
- May 2018
- First submitted
- 30 Sep 2014
- Last updated
- 11 May 2018
- Results posted
- No
- Data monitoring committee
- No
Publications
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.
PubMed 28274235
Study at a glance
From the registry record, last updated by the sponsor on 11 May 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Cohort Study on Prevalence and Early Treatment Outcomes in Pierre-Robin-Like Phenotype
- ClinicalTrials.gov ID
- NCT02266043
- Acronym / sponsor ID
- FACE-Subproject SP5-Tuebingen
- Conditions
- Pierre Robin Syndrome
- Keywords
- Upper airway obstruction
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- University Hospital TuebingenUniversity, hospital or other
- Collaborators
- German Federal Ministry of Education and Research
- Study start
- 1 Jan 2013 (actual)
- Primary completion
- 31 Aug 2016 (actual)
- Study completion
- 31 Dec 2016 (actual)
- First posted
- 16 Oct 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- Up to 12 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Infants <1 year admitted with Pierre-Robin-like phenotype to participating centres
Key inclusion criteria (1 listed)
- Infants < 1 year at admission, Pierre-Robin-like phenotype
Key exclusion criteria (1 listed)
- no parental consent; need for immediate tracheotomy
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 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. | Device | – |
Primary outcome
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
3 secondary outcomes
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
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 May 2018.
- Derived from this study: PubMed 28274235Poets 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.
Related trials for Pierre Robin Syndrome
Active, not recruitingObservational study
3D-CT-Based Prediction of Difficult Laryngoscopy in Infants With Pierre Robin Sequence
Pierre Robin Sequence (PRS), Difficult Laryngoscopy
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Christian F Poets, MD principal investigator
Dept. of Neonatology and Interdisciplinary Centre forCraniofacial Malformations, University Hospital Tuebingen
Siegmar Reinert, MD, PhD principal investigator
Dept. of Craniofacial Surgery, University Hospital Tuebingen
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Pierre Robin Syndrome, run by University Hospital Tuebingen at 3 sites in Germany.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 11 May 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02266043, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 May 2018.