Pierre Robin SequenceDYSROBINNCT03423017
Brainstem Dysfunction Involvement in the Pathogenesis of Pierre Robin Sequence
A study testing Recording of data in people with Pierre Robin Sequence and Brainstem Dysfunction, run by Hospices Civils de Lyon at 2 sites in France.
Trial summary
Introduction Pierre Robin Sequence, PRS, incidence is about one hundred births per year in France. The main neonatal clinical manifestations are secondary to airway obstruction and food difficulties related to swallowing disorders. Despite recent progress, the pathogenesis of PRS is not fully understood. Read more about this study
- Who is it for?
- Ages 1 day to 2 months
- Conditions the study is for
- Pierre Robin Sequence, Brainstem Dysfunction
- What is being tested?
- Recording of data
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 113 participants
- Where
- 2 sitesFrance
- Study dates
- 24 May 2018to 23 Mar 2023
- Phase
- Interventional
- 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 1 day to 2 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- infants with PRS
- Inclusion Criteria for the group 2 - infant with isolated airway obstruction
- healthy infant : siblings of sudden unexpected death of the infant or with any known pathology
- During one hospitalization or program for one hospitalization
- Parental consent
- Social safety affiliation
Who cannot join
- Birth before 37 SA
- Neonatal complication
- Group 2 only: AWO with neurological disease including brainstem dysfunction
- Group 3 only : AWO, ENT disease or syndromic disease, neurological disease including brainstem dysfunction, Intra uterine growth retardation
Trial locations and contacts
2 sites in 1 country.
France2 sites
Hopital Femme Mère Enfant
Bron, 69500
APHP-Necker
Paris, 75015 Paris
About this study
Introduction Pierre Robin Sequence, PRS, incidence is about one hundred births per year in France. The main neonatal clinical manifestations are secondary to airway obstruction and food difficulties related to swallowing disorders. Despite recent progress, the pathogenesis of PRS is not fully understood.
The hypothesis is that brainstem dysfunction, BSD, plays a central role in the pathogenesis of PRS.
Read more
The purpose of the study is to achieve a complete evaluation of BSD to specify its role in the pathogenesis of PRS.
The primary objective is to compare central apnea index (CAI) of infants with PRS with those of infants with isolated airway obstruction (AWO) and those of healthy infants in order to clarify the direct role of BSD.
Material and Methods This prospective interventional study will be carried out in Lyon at the Hôpital Femme-Mère-Enfant and in Paris at the Hôpital Necker-Enfants Malades for 2 years. 3 groups of patients will be studied: PRS, 50 patients, AWO, 50 patients and healthy, 30 patients, included before 2 months of life. Infants will be followed for a maximum of 10 months. The evaluations will be carried out for 48 hours between birth and 2 months of life and then for 24 hours between 6 and 10 months of life for PRS and AWO group. Concerning the healthy group, the evaluation will be carried out during 48h during a single hospitalization before 2 months. Polysomnography, holter-ECG, 24h gas exchange, impedance-pH monitoring and mental region EEG will be performed. The central apnea index (mean number per hour), obstructive apnea index, non-nutritive swallowing index (NNS), gastroesophageal reflux and NNS-respiration coordination will be assessed for each stage of sleep and compared between the three groups of patients.
- Conditions
- Pierre Robin Sequence, Brainstem Dysfunction
- Being tested
- Recording of data
- Keywords
- Pierre Robin sequence, Brainstem, pathogenesis, swallowing, apnea
- Medical subject headings
- Pierre Robin Syndrome, Apnea
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Recording of dataOther
Recording during one hospitalization of 48 hours between birth and 2 months of life and one hospitalization of 24 hours between 6 and 10 months of life
- General data: age, term of birth, birth weight, gender, diseases, weight and size at recording, cranial perimeter, food, drugs : first and second visit
- Respiratory rate, respiratory signs, stridor : first and second visit
- 24h holter-ECG : first and second visit
- 24h impedance pH-metry : first visit
- Nocturne polysomnography : 50% prone position, 50% supine : sleep analyze, obstructive apnea hypopnea index (OAHI), central apnea index (CAI), periodical breathing, micro arousals index, non-nutritive swallowing analyze,(mental region electromyogram) : first and second visit
- 24 h gaz exchanges : mean, maximal PtcCO2 and % of time spent above 50 mmHg , SpO2 under 90%, 85% et 80% and desaturations index : first and second visit
- succimetry procedure : at first visit only between birth and 2 months
Recording of dataOther
Recording during one hospitalization of 48 hours between birth and 2 months of life
- General data: age, term of birth, birth weight, gender, diseases, weight and size at recording, cranial perimeter, food, drugs
- Respiratory rate, respiratory signs, stridor
- 24h holter-ECG
- 24h impedance pH-metry
- Nocturne polysomnography : 100% supine position : sleep analyze, obstructive apnea hypopnea index (OAHI), central apnea index (CAI), periodical breathing, micro arousals index, non-nutritive swallowing analyze,(mental region electromyogram)
- 24 h gas exchanges : mean, maximal PtcCO2 and % of time spent above 50 mmHg , SpO2 under 90%, 85% et 80% and desaturations index
- Succimetry procedure : at first visit only between birth and 2 months
| Study group | Role | What they receive |
|---|---|---|
| Pierre Robin sequence | Other | Infants with PRS : retrognathism, glossoptosis, cleft palate
Group 1a : isolated PRS Group 1b : PRS with bone disease or collagen disease (Stickler) Group 1c : syndromic PRS or associated PRS without bone disease or collagen disease Receives: Other: Recording of data |
| Superior airway obstruction, AWO | Other | Infants with AWO : laryngomalacia, tracheal stenosis, laryngeal stenosis, others etiology Receives: Other: Recording of data |
| Healthy infants | Other | Healthy infants : siblings of sudden unexpected death of the infant Receives: Other: Recording of data |
What is being measured
The primary outcome is the main question the study is designed to answer.
Central apnea index (CAI) (mean number per hour)
To compare central apnea index (CAI) (mean number per hour), according to the ASSM guidelines 2007, recording by one nocturne polysomnography, between 3 groups of infants by sleep step, between birth and 2 months of life, in order to clarify the direct role of BSD.
According to the ASSM guidelines 2007, the central apnea index is measured : mean number of central apnea per hour.
Measured: 2 months of life during one nocturne polysomnography
Secondary outcomes (7)
Obstructive apnea hypopnea index (OAHI)(mean number per hour)
To compare obstructive apnea hypopnea index (OAHI)(mean number per hour) according to the guidelines ASSM 2007, recording by one nocturne polysomnography, between 3 groups of infants by sleep step, between birth and 2 months of life According to the ASSM guidelines 2007, the obstructive apnea hypopnea index is measured: mean number of obstructive apnea and hypopnea per hour.
Measured: 2 months
Comparison of OAHI(mean number per hour) and CAI
To compare obstructive apnea hypopnea index (OAHI)(mean number per hour) and central apnea index (CAI) (mean number per hour), between PRS group and AWO group by sleep step between 6 and 10 months of life
According to the ASSM guidelines 2007, the obstructive apnea hypopnea index, mean number of obstructive apnea and hypopnea per hour, and the central apnea index are measured, mean number of central apnea per hour.
Measured: between 6 and 10 months of life
Micro arousals index (mean number per hour)
To compare micro arousals index (mean number per hour) between the 3 groups by sleep step between birth and 2 months of life and between PRS group and AWO group between 6 and 10 months of life to clarify the role of BSD in sleep microstructure
According to the ASSM guidelines 2007, micro arousals index (mean number per hour) are measured.
Measured: up to 2 months of life
Micro arousals index (mean number per hour)
To compare micro arousals index (mean number per hour) between the 3 groups by sleep step between birth and 2 months of life and between PRS group and AWO group between 6 and 10 months of life to clarify the role of BSD in sleep microstructure
According to the ASSM guidelines 2007, micro arousals index (mean number per hour) are measured.
Measured: up to 10 months of life
Non-nutritive swallowing index (NNS) and apnea secondary to non-nutritive swallowing
To compare non-nutritive swallowing index (NNS) and apnea secondary to non-nutritive swallowing between the 3 groups by sleep step between birth and 2 months of life and between PRS group and AWO group between 6 and 10 months of life to clarify the role of BSD in swallowing disorders and NNS-respiration coordination
non-nutritive swallowing index (NNS), mean number per hour, and apnea secondary to non-nutritive swallowing, mean number per hour, monitoring by the mental region EEG and the one nocturne polysomnography
Measured: up to 2 months of life
Non-nutritive swallowing index (NNS) and apnea secondary to non-nutritive swallowing
To compare non-nutritive swallowing index (NNS) and apnea secondary to non-nutritive swallowing between the 3 groups by sleep step between birth and 2 months of life and between PRS group and AWO group between 6 and 10 months of life to clarify the role of BSD in swallowing disorders and NNS-respiration coordination
non-nutritive swallowing index (NNS), mean number per hour, and apnea secondary to non-nutritive swallowing, mean number per hour, monitoring by the mental region EEG and the one nocturne polysomnography
Measured: up to 10 months of life
Positive and negative pressures, their temporal organizations evaluated using succimetry procedure between the 3 groups of patients at 0 and 2 months of life
Compare the positive and negative pressures, their temporal organizations evaluated using succimetry procedure between the 3 groups of patients at 0 and 2 months of life.
Measured: up to 2 months of life
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Feb 2018
- Study start24 May 2018
- Primary completion23 Mar 2023
- Study completion23 Mar 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pierre Robin SequenceStudies of the same condition, open and under way.
- More trials run by Hospices Civils de LyonThe 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 113 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hospices Civils de Lyon
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03423017
- Sponsor's protocol ID
- 69HCL17_0816
- Acronym
- DYSROBIN
- Status last verified
- Jan 2024
- First submitted
- 12 Jan 2018
- Last updated
- 10 Jan 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 10 Jan 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Brainstem Dysfunction Involvement in the Pathogenesis of Pierre Robin Sequence
- ClinicalTrials.gov ID
- NCT03423017
- Acronym / sponsor ID
- DYSROBIN / 69HCL17_0816
- Conditions
- Pierre Robin Sequence, Brainstem Dysfunction
- Keywords
- Pierre Robin sequence, Brainstem, pathogenesis, swallowing, apnea
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 113 participants (actual)
- Lead sponsor
- Hospices Civils de LyonUniversity, hospital or other
- Study start
- 24 May 2018 (actual)
- Primary completion
- 23 Mar 2023 (actual)
- Study completion
- 23 Mar 2023 (actual)
- First posted
- 6 Feb 2018 (actual)
- 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.
Introduction Pierre Robin Sequence, PRS, incidence is about one hundred births per year in France. The main neonatal clinical manifestations are secondary to airway obstruction and food difficulties related to swallowing disorders. Despite recent progress, the pathogenesis of PRS is not fully understood.
The hypothesis is that brainstem dysfunction, BSD, plays a central role in the pathogenesis of PRS.
The purpose of the study is to achieve a complete evaluation of BSD to specify its role in the pathogenesis of PRS.
The primary objective is to compare central apnea index (CAI) of infants with PRS with those of infants with isolated airway obstruction (AWO) and those of healthy infants in order to clarify the direct role of BSD.
Material and Methods This prospective interventional study will be carried out in Lyon at the Hôpital Femme-Mère-Enfant and in Paris at the Hôpital Necker-Enfants Malades for 2 years. 3 groups of patients will be studied: PRS, 50 patients, AWO, 50 patients and healthy, 30 patients, included before 2 months of life. Infants will be followed for a maximum of 10 months. The evaluations will be carried out for 48 hours between birth and 2 months of life and then for 24 hours between 6 and 10 months of life for PRS and AWO group. Concerning the healthy group, the evaluation will be carried out during 48h during a single hospitalization before 2 months. Polysomnography, holter-ECG, 24h gas exchange, impedance-pH monitoring and mental region EEG will be performed. The central apnea index (mean number per hour), obstructive apnea index, non-nutritive swallowing index (NNS), gastroesophageal reflux and NNS-respiration coordination will be assessed for each stage of sleep and compared between the three groups of patients.
Study design
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Pierre Robin sequence | Other | Other: Recording of dataInfants with PRS : retrognathism, glossoptosis, cleft palate Group 1a : isolated PRS Group 1b : PRS with bone disease or collagen disease (Stickler) Group 1c : syndromic PRS or associated PRS without bone disease or collagen disease |
| Superior airway obstruction, AWO | Other | Other: Recording of dataInfants with AWO : laryngomalacia, tracheal stenosis, laryngeal stenosis, others etiology |
| Healthy infants | Other | Other: Recording of dataHealthy infants : siblings of sudden unexpected death of the infant |
Who may qualify
- Ages
- 1 day to 2 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- infants with PRS
- Inclusion Criteria for the group 2 - infant with isolated airway obstruction
- healthy infant : siblings of sudden unexpected death of the infant or with any known pathology
- During one hospitalization or program for one hospitalization
- Parental consent
Key exclusion criteria (4 listed)
- Birth before 37 SA
- Neonatal complication
- Group 2 only: AWO with neurological disease including brainstem dysfunction
- Group 3 only : AWO, ENT disease or syndromic disease, neurological disease including brainstem dysfunction, Intra uterine growth retardation
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 |
|---|---|---|
| Recording of dataRecording during one hospitalization of 48 hours between birth and 2 months of life and one hospitalization of 24 hours between 6 and 10 months of life * General data: age, term of birth, birth weight, gender, diseases, weight and size at recording, cranial perimeter, food, drugs : first and second visit * Respiratory rate, respiratory signs, stridor : first and second visit * 24h holter-ECG : first and second visit * 24h impedance pH-metry : first visit * Nocturne polysomnography : 50% prone position, 50% supine : sleep analyze, obstructive apnea hypopnea index (OAHI), central apnea index (CAI), periodical breathing, micro arousals index, non-nutritive swallowing analyze,(mental region electromyogram) : first and second visit * 24 h gaz exchanges : mean, maximal PtcCO2 and % of time spent above 50 mmHg , SpO2 under 90%, 85% et 80% and desaturations index : first and second visit * succimetry procedure : at first visit only between birth and 2 months | Other | Pierre Robin sequence, Superior airway obstruction, AWO |
| Recording of dataRecording during one hospitalization of 48 hours between birth and 2 months of life * General data: age, term of birth, birth weight, gender, diseases, weight and size at recording, cranial perimeter, food, drugs * Respiratory rate, respiratory signs, stridor * 24h holter-ECG * 24h impedance pH-metry * Nocturne polysomnography : 100% supine position : sleep analyze, obstructive apnea hypopnea index (OAHI), central apnea index (CAI), periodical breathing, micro arousals index, non-nutritive swallowing analyze,(mental region electromyogram) * 24 h gas exchanges : mean, maximal PtcCO2 and % of time spent above 50 mmHg , SpO2 under 90%, 85% et 80% and desaturations index * Succimetry procedure : at first visit only between birth and 2 months | Other | Healthy infants |
Primary outcome
Central apnea index (CAI) (mean number per hour)
Time frame: 2 months of life during one nocturne polysomnography
To compare central apnea index (CAI) (mean number per hour), according to the ASSM guidelines 2007, recording by one nocturne polysomnography, between 3 groups of infants by sleep step, between birth and 2 months of life, in order to clarify the direct role of BSD. According to the ASSM guidelines 2007, the central apnea index is measured : mean number of central apnea per hour.
7 secondary outcomes
Obstructive apnea hypopnea index (OAHI)(mean number per hour)
Time frame: 2 months
Comparison of OAHI(mean number per hour) and CAI
Time frame: between 6 and 10 months of life
Micro arousals index (mean number per hour)
Time frame: up to 2 months of life
Micro arousals index (mean number per hour)
Time frame: up to 10 months of life
Non-nutritive swallowing index (NNS) and apnea secondary to non-nutritive swallowing
Time frame: up to 2 months of life
Non-nutritive swallowing index (NNS) and apnea secondary to non-nutritive swallowing
Time frame: up to 10 months of life
Positive and negative pressures, their temporal organizations evaluated using succimetry procedure between the 3 groups of patients at 0 and 2 months of life
Time frame: up to 2 months of life
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 10 Jan 2024.
- Individual participant dataSharing planned: Undecided
Related trials for Pierre Robin Sequence
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.
The record lists no contact. Use the official record to reach the sponsor.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Recording of data in people with Pierre Robin Sequence and Brainstem Dysfunction, run by Hospices Civils de Lyon at 2 sites in France.
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 6 inclusion and 4 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 10 Jan 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in France. 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?
Central apnea index (CAI) (mean number per hour) (time frame: 2 months of life during one nocturne polysomnography). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 10 Jan 2024.