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Trial registered on ANZCTR


Registration number
ACTRN12624000007538
Ethics application status
Approved
Date submitted
1/12/2023
Date registered
9/01/2024
Date last updated
23/06/2024
Date data sharing statement initially provided
9/01/2024
Type of registration
Prospectively registered

Titles & IDs
Public title
A longitudinal pilot study assessing the medical and surgical treatment of babies with congenital upper airway conditions.
Scientific title
Prospective Longitudinal Assessment of Treatment options for Young Patients with Upper airway Structural abnormalities (PLATYPUS): a pilot study
Secondary ID [1] 310988 0
NIL
Universal Trial Number (UTN)
Trial acronym
PLATYPUS
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Young Children undergoing general anaesthesia
332166 0
Young Children with laryngomalacia 332167 0
Young Children having supraglottoplasty surgery 332168 0
Condition category
Condition code
Anaesthesiology 328884 328884 0 0
Anaesthetics
Respiratory 328885 328885 0 0
Normal development and function of the respiratory system
Surgery 328886 328886 0 0
Other surgery

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Infants with symptoms of laryngomalacia will be recruited through the Ear, Nose and Throat (ENT) service. Participants will be divided into two groups according to their clinical presentation and the judgement of the treating ENT consultant in line with the current clinical guidelines and routine clinical practice of the ENT service. There will be no changes in the management or any aspect of clinical care due to trial participation.

Planned surgical management group: Patients who are planned for diagnostic laryngotracheobronchoscopy (LTB) +/- supraglottoplasty for the treatment of suspected laryngomalacia. Supraglottoplasty will be defined as any surgical procedure done to stabilise the tissue of the supraglottic larynx.

Medical management group: Patients with suspected laryngomalacia who are for conservative non-surgical treatment after ENT appointment.

Patients born premature (before 36 weeks gestation) will have their ages corrected to 40 weeks. Patients born at term will be referred to by their birth age.

Enrolled patients will undergo oximetry monitoring using a Nonin oximeter device (WristOx 3150, Nonin Medical, Plymouth, MN, United States). The Nonin WristOx 3150 is a small, lightweight pulse oximeter ideal for use in the patient’s home and has probes that are designed for use with paediatric patients. It is easy to use as it is activated when the probe is placed on the patients foot or toe. The monitor can be attached easily to the patient's crib. Patients will wear the oximeter continuously for 48 hours following their initial consultation with the Perth Children’s Hospital ENT team. The medical management group will do 48 hours of Nonin WristOx monitoring following their regular scheduled ENT appointment at 3 months age/3 months corrected age, as well as each subsequent ENT clinic appointment until they reach 6 months of age/6 months corrected age if premature, or they are discharged from the ENT surgical clinic, whichever occurs first. After each round of oximetry recordings, the data will be downloaded using Nonin nVision software to assess the length of time for which the device was worn and the quality of the recording (e.g., artefact burden).

Patients in the surgical management group will undergo oximetry monitoring using the Nonin WristOx 3150 and transcutaneous carbon dioxide monitoring using the SenTec digital monitoring system (SenTec, Lincoln, RI, United States) at the time of their surgery. Patients who are inpatients prior to their surgery will also have Sentec monitoring for 1 night pre operatively. SenTec monitoring involves a probe on the patient’s skin, usually on the forehead or shoulder. Following SenTec monitoring, the data will be downloaded and assessed by a trained researcher to establish the length of time for which the device was worn and the quality of the recording. Patients will undergo oximetry again 8 weeks following their supraglottoplasty procedure. For most patients, this will occur at approximately 3 months of age/3 months corrected age, in line with the oximetry done for the medical management group. Patients in the surgical management group will repeat oximetry at each subsequent appointment until they reach 6 months of age/6 months corrected age, or until they are discharged from the ENT surgical clinic, whichever occurs first.

As per routine care, these appointments can be in person, via telehealth or over the phone. If the appointment is not in person, the oximeter will be posted to the families.
Intervention code [1] 327495 0
Diagnosis / Prognosis
Comparator / control treatment
Patients who are allocated to the medical management group following their initial ENT clinic appointment i.e., patients with suspected laryngomalacia who are for conserative non-surgical treatment after ENT appointment.

Participants' medical and surgical management will not be affected in any way by participation in this research study. Patients will be divided into the two groups depending on their treatment plan as per the ENT team based on their routine clinical care and current clinical guidelines. However, for the purpose of making comparisons between patients in the medical management and surgical management groups, we will identify the medical management group as our comparator.
Control group
Active

Outcomes
Primary outcome [1] 336695 0
To assess the rate of successful partipant recruitment as determined by a review of screening logs and enrollment logs at completion of recruitment.
Timepoint [1] 336695 0
From beginning of study to the completion of recruitment.
Primary outcome [2] 336696 0
To assess the viability of measurements of oxygenation in infants with severe laryngomalacia.
Timepoint [2] 336696 0
The quality and duration of measurements will be asssessed following each continuous monitoring episode for each patient.
Primary outcome [3] 336697 0
Assessment of the accetability of study procedures to parents/legal guardians.
Timepoint [3] 336697 0
Parents/legal guardians will be asked to rate the acceptability of study procedures following every at-home measurement period completed by their child.
Secondary outcome [1] 429346 0
Assessment of the acceptability of study procedures to clinical staff.
Timepoint [1] 429346 0
Nurses directly involved in the participant's inpatient hospital care whilst they are on WristOx and/or SenTec monitoring will be asked to rate acceptability of study procedures at the end of their involvment in the care of the participant.
Secondary outcome [2] 429829 0
Primary outcome [4]: To assess the viability of measurements of ventilation in infants with severe laryngomalacia.
Timepoint [2] 429829 0
Primary outcome [4]: The quality and duration of measurements will be asssessed following each continuous monitoring episode for each patient.
Secondary outcome [3] 429840 0
Symptomology of patients being treated for laryngomalacia
Timepoint [3] 429840 0
Patients will be assessed at all appointments between their first appointment with ENT at approximately 2 weeks of age, and their final appointment at approximately 6 months of age (or 6 months corrected age if born premature).
Secondary outcome [4] 429841 0
Oxygenation of patients being treated for laryngomalacia
Timepoint [4] 429841 0
Patients will complete 48 hours of oximetry following each appointment with the ENT surgical team between their first appointment with ENT at approximately 2 weeks of age, and their final appointment at approximately 6 months of age (or 6 months corrected age if born premature). Patients in the surgical management group will do an additional 48 hours of oximetry following their supraglottoplasty procedure.
Secondary outcome [5] 429842 0
Perioperative ventilation of patients being treated for laryngomalacia
Timepoint [5] 429842 0
Participants in the surgical group who are inpatients prior to their surgery will complete 12 hours of continuous tcCO2 monitoring immediately pre-operatively. All patients in the surgical group will complete 12 hours of tcCO2 monitoring on the first night post-operatively.

Eligibility
Key inclusion criteria
Patients up to 6 months of age/6 months corrected age (if born premature) who have been referred to the Ear, Nose and Throat (ENT) team at PCH for management of suspected laryngomalacia will be recruited.
Minimum age
No limit
Maximum age
6 Months
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Children with any syndromic features or neuromuscular conditions
Children with known significant cardiac disease
Children unable to complete at-home oximetry
Families without a sufficient level of English to be consented.

Study design
Purpose of the study
Diagnosis
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
A study of two groups of patients undergoing different treatments as determined by the ENT team based on routine clinical care and current institutional guidelines. Both groups will undergo measurements of their oximetry using the Nonin WristOx - the time points at which these measurements occur will depend on their treatment plan. Patients having supraglottoplasty surgery will have additional ventilation measurements with the SenTec digital monitoring system.
Phase
Not Applicable
Type of endpoint/s
Statistical methods / analysis
Analysis will be conducted in the R statistical environment, and statistical significance will be taken at 5% (p<0.05). Data will be explored using graphical and numeric summaries. To assess the feasibility objectives, the proportion of successful enrolments out of the total number of eligible patients will be reported, overall and specific to the surgical and non-surgical groups. The proportion of protocol violations out of the number of enrolled participants will also be reported. The proportion of recruited patients with successful WristOx measurements at every timepoint will also be reported, where a successful measurement is defined as greater than or equal to 12 hours of data for each time they were asked to wear the device.

The values of WristOx variables for each participant will be graphed over the timepoints, with separate graphs shown for both the surgical and non-surgical groups, in order to assess any trends from immediately after the outpatient appointment to 12-16 weeks after the appointment. To assess associations between pre-operative oximetry and the success of supraglottoplasty, surgical group participants will be classified as having had “successful” surgery (defined as resolution of all symptoms and the resumption of normal feeding and weight gain) or not, a binary outcome. Relationships between pre-surgical WristOx variables (including mean oxygen saturation, minimum oxygen saturation, ODI3, among others) and surgery outcome will be explored using graphical and numerical summaries. Logistic regression models will be fitted to the binary outcome of surgery success, with covariates including WristOx variables, patient characteristics and surgical details. Longitudinal models will be used to quantify the relationship between continuous outcomes that are measured over time against covariates.

For the surgical group, the values of SenTec variables following surgery will be summarised.

Recruitment
Recruitment status
Recruiting
Date of first participant enrolment
Anticipated
11/03/2024
Actual
19/04/2024
Date of last participant enrolment
Anticipated
1/02/2026
Actual
Date of last data collection
Anticipated
1/08/2026
Actual
Sample size
Target
120
Accrual to date
16
Final
Recruitment in Australia
Recruitment state(s)
WA
Recruitment hospital [1] 25893 0
Perth Children's Hospital - Nedlands
Recruitment postcode(s) [1] 41727 0
6009 - Nedlands

Funding & Sponsors
Funding source category [1] 315252 0
Hospital
Name [1] 315252 0
Perth Children's Hospital
Country [1] 315252 0
Australia
Funding source category [2] 315307 0
Government body
Name [2] 315307 0
NHMRC
Country [2] 315307 0
Australia
Primary sponsor type
Hospital
Name
Perth Children's Hospital
Address
15 Hospital Avenue Nedlands WA 6009
Country
Australia
Secondary sponsor category [1] 317280 0
None
Name [1] 317280 0
Address [1] 317280 0
Country [1] 317280 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 314170 0
Child and Adolescent Health Service Human Research Ethics Committee
Ethics committee address [1] 314170 0
15 Hospital Avenue Nedlands Western Australia 6009
Ethics committee country [1] 314170 0
Australia
Date submitted for ethics approval [1] 314170 0
27/11/2023
Approval date [1] 314170 0
20/12/2023
Ethics approval number [1] 314170 0
RGS0000006281

Summary
Brief summary
Laryngomalacia is the most common congenital airway abnormality in young children. In the most severe cases, surgical intervention is necessary to prevent serious complications from arising and to allow for the normal development of breathing and feeding. Supraglottoplasty is the most common surgical procedure employed to address severe laryngomalacia. The procedure reduces or stabilises the problematic laryngeal tissue preventing further obstruction from occurring. Existing literature on the efficacy of this surgery has demonstrated that this procedure is not always effective at resolving symptoms. The factors that contribute to the success or failure of supraglottoplasty have been difficult to elucidate but are likely closely related to the patient’s pre-operative clinical condition. We propose a prospective study investigating the use of continuous pre-operative pulse oximetry as a tool to better understand the nature of the laryngomalacia present and identify patients most likely to benefit from supraglottoplasty surgery.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 130662 0
Prof Britta Regli-von Ungern-Sternberg
Address 130662 0
Perth Children's Hospital Department of Anaesthesia and Pain Medicine 15 Hospital Avenue Nedlands WA 6009
Country 130662 0
Australia
Phone 130662 0
+61 8 64564805
Fax 130662 0
Email 130662 0
Britta.Regli-VonUngern@health.wa.gov.au
Contact person for public queries
Name 130663 0
Prof Britta Regli-von Ungern-Sternberg
Address 130663 0
Perth Children's Hospital Department of Anaesthesia and Pain Medicine 15 Hospital Avenue Nedlands WA 6009
Country 130663 0
Australia
Phone 130663 0
+61 8 64564805
Fax 130663 0
Email 130663 0
Britta.Regli-VonUngern@health.wa.gov.au
Contact person for scientific queries
Name 130664 0
Prof Britta Regli-von Ungern-Sternberg
Address 130664 0
Perth Children's Hospital Department of Anaesthesia and Pain Medicine 15 Hospital Avenue Nedlands WA 6009
Country 130664 0
Australia
Phone 130664 0
+61 8 64564805
Fax 130664 0
Email 130664 0
Britta.Regli-VonUngern@health.wa.gov.au

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment
Investigators for the study have not yet decided on data sharing.


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
No additional documents have been identified.