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Trial registered on ANZCTR


Registration number
ACTRN12622001328763
Ethics application status
Approved
Date submitted
15/09/2022
Date registered
14/10/2022
Date last updated
3/05/2023
Date data sharing statement initially provided
14/10/2022
Type of registration
Prospectively registered

Titles & IDs
Public title
Treating auditory problems in children with neurofibromatosis type 1
Scientific title
A randomised controlled trial to determine the effect of remote microphone listening devices on speech perception and functional hearing in children with neurofibromatosis type 1 and auditory deficits
Secondary ID [1] 307462 0
81244
Universal Trial Number (UTN)
U1111-1279-8971
Trial acronym
TAP-iN
Linked study record
This randomised controlled trial is the parent study for ACTRN12622001329752, an extension trial.

Health condition
Health condition(s) or problem(s) studied:
Neurofibromatosis type 1 326854 0
Condition category
Condition code
Human Genetics and Inherited Disorders 324067 324067 0 0
Other human genetics and inherited disorders
Ear 324068 324068 0 0
Other ear disorders
Neurological 324069 324069 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
This is a multisite phase II/III clinical trial evaluating the use of a remote microphone listening (RML) device in treating speech perception in noise difficulties in children with neurofibromatosis type 1 (NF1).

The investigational device is the Phonak Roger Touchscreen Microphone paired with Phonak Roger Focus Receivers with SlimTubes and open domes. The receivers are designed for children with normal hearing thresholds, so they provide a safe, comfortable, and adjustable volume for wearers. The receivers are small devices that sit behind each pinna and are held in place by a soft, vented rubber earpiece inserted into the ear canal. They are minimally visible, do not block the ear and allow the wearer access to environmental sound. The Touchscreen Microphone is a compact device, worn by the teacher on a lanyard.

The registration form describes the parent randomised controlled trial (RCT) component of the study. The RCT is a 4-week trial with a 2-period crossover design. Participants will be randomised to one of two treatment sequences:
- 2 weeks of device use followed by 2 weeks of treatment as usual (no device use)
- 2 weeks of treatment as usual (no device use) followed by 2 weeks of device use

Consistent with other clinical trials of RML devices, no washout period is included and carryover effects are not anticipated.

Children and teachers will be asked to wear the RML device Monday - Friday for the time the child is in the classroom. To determine the amount of time the device is used, we will ask the teacher to complete a simple daily compliance check.

Children and their family will be instructed on how to use the RML device including checking device function, ensuring communication between the microphone and the receiver, and how to charge the batteries. Teachers will also be provided the option of a training session conducted by a member of the research team via videoconferencing or a telephone call. Teachers will then assist children in the use of the device in the classroom. Training will occur within 2 weeks prior to the intervention period.
Intervention code [1] 323919 0
Treatment: Devices
Comparator / control treatment
Treatment as usual. For this condition, children will be able to continue to receive interventions for behaviour and learning such as stimulant medication or speech therapy, but will not wear the RML device.
Control group
Active

Outcomes
Primary outcome [1] 331868 0
Performance on the Consonant-Nucleus-Consonant test, measured by % correct phonemes
Timepoint [1] 331868 0
Assessments will be undertaken with the device (aided) and without the device (unaided), conducted during the pre-intervention baseline assessment in a randomised order by the study audiologist.
Primary outcome [2] 331869 0
Total score on the Listening Inventory for Education-Revised questionnaire (child version)
Timepoint [2] 331869 0
Baseline, end of treatment period 1 (2 weeks post-randomisation), end of treatment period 2 (4-weeks post-randomisation)
Primary outcome [3] 332706 0
Total score on the Listening Inventory for Education-Revised questionnaire (teacher version)
Timepoint [3] 332706 0
Baseline, end of treatment period 1 (2 weeks post-randomisation), end of treatment period 2 (4-weeks post-randomisation)
Secondary outcome [1] 411427 0
Integrated Visual and Auditory-Quick Screen test, in background noise
Timepoint [1] 411427 0
Assessments will be undertaken with the device (aided) and without the device (unaided), conducted during the pre-intervention baseline assessment in a randomised order by the study audiologist.
Secondary outcome [2] 414434 0
Adverse events after short term use, assessed using a short Tolerability and Adverse Events survey written by the investigator team. While uncommon, possible adverse events may include:
- discomfort related to the positioning of the device on the child's ear
- loud volume of signal transmitted to the child’s ear
Timepoint [2] 414434 0
Baseline, end of treatment period 1 (2 weeks post-randomisation), end of treatment period 2 (4-weeks post-randomisation)

Eligibility
Key inclusion criteria
• Children aged 6-12 years
• Satisfy the revised diagnostic criteria for NF1
• Participant and at least one caregiver have sufficient English to complete study outcomes, understand and comply with study requirements and to communicate any adverse effects
• Has a legally acceptable parent/guardian capable of understanding the informed consent document and providing consent on the participant’s behalf
• Demonstrate a functional hearing difficulty on the Listening in Spatialized Noise - Sentences (LiSN-S) test [Note: defined as 2 standard deviations below the normative age range for the Different Voice 90°, Same Voice 90° and/or spatial advantage conditions]
• School/teachers willing to participate in the study (of any age)
Minimum age
6 Years
Maximum age
12 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
• School/teachers unwilling to participate
• Active ear infection at the time of screening
• Evidence of sensory hearing loss (defined by a 4-frequency average hearing loss (average of 0.5-, 1-, 2-, and 4 kHz) of >20dbHL in both ears, or use of corrective hearing device such as a hearing aid or cochlear implant.
• Full Scale IQ (FSIQ) <70 on standardised test of intellectual functioning.
• Plan to commence or change medication (or dosage) for ADHD symptoms during the 4-week RCT. In these cases, children will be considered eligible if they have been on a stable dose of ADHD medication for 4 weeks prior to screening (and they meet all other eligibility requirements).
• Symptomatic or progressive intracranial pathology that may affect scores on audiological, cognitive, or behavioural outcome measures (e.g., acquired brain injury, or hydrocephalus). Asymptomatic or stable low-grade gliomas that are not thought to impact on outcome measures will not result in exclusion.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
The first randomisation schedule will be to counterbalance the order in which the participant completes the in-clinic Consonant-Nucleus-Consonant test and Integrated Visual and Auditory Quick Screen assessment at the baseline assessment. This will only be provided to the site audiologist who will conduct the testing.

The second randomisation schedule will be to determine the order of conditions for the in-school trial: device followed by treatment as usual, or treatment as usual followed by device. This randomisation schedule will be provided to the site audiologist and study coordinator.
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
There will be two randomisations in this trial which will be completely independent of each other.

The first is required to counterbalance the order of in-clinic testing for the two conditions (device aided vs unaided) for the (1) Consonant-Nucleus-Consonant test and the (2) Integrated Visual-Auditory Quick Screen Test.

The second randomisation is required to enrol eligible participants into the crossover randomised controlled trial. For this, children will be randomly assigned to one of the following two sequences:

1. Device followed by treatment as usual, or
2. Treatment as usual followed by device.

Randomisation will be performed through web-based randomisation by a member of the research team (study investigators or trained staff) while the child is in the clinic. A randomisation schedule will be prepared by a statistician independent from the study in the Clinical Epidemiology and Biostatistic Unit at the Murdoch Children’s Research Institute. The randomisation schedule will have an allocated ratio of 1:1 for each sequence arm and will be assigned in randomly permuted blocks of different sizes.
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?


The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Crossover
Other design features
Phase
Not Applicable
Type of endpoint/s
Efficacy
Statistical methods / analysis
Data analysis for the study will be performed by the trial statistician who is part of the Clinical Epidemiology and Biostatistic Unit at the Murdoch Children’s Research Institute. Statistical analysis will follow standard methods for randomised trials and the primary analysis will be by intention to treat, including all enrolled participants. Categorical variables will be presented as the number and proportion in each category. Continuous variables will be presented as means and standard deviations, or medians and interquartile ranges for skewed data, and the range.

Between-group comparisons of the primary and secondary outcomes will be presented as the mean difference with 95% CI, calculated from linear mixed effects models. This allows us to retain participants with missing data due to drop-out. The model will include a fixed effect for the intervention (device vs control) and period, and a random effect for individual.

For non-continuous secondary outcomes (binary or categorical), the appropriate generalised linear model (GLM) will be used to estimate the effect of the device on the outcome of interest compared to the control group

Recruitment
Recruitment status
Recruiting
Date of first participant enrolment
Anticipated
1/05/2023
Actual
Date of last participant enrolment
Anticipated
24/11/2025
Actual
Date of last data collection
Anticipated
19/12/2025
Actual
Sample size
Target
124
Accrual to date
0
Final
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 22666 0
The Royal Childrens Hospital - Parkville
Recruitment hospital [2] 23148 0
The Children's Hospital at Westmead - Westmead
Recruitment postcode(s) [1] 37943 0
3052 - Parkville
Recruitment postcode(s) [2] 37944 0
2145 - Westmead

Funding & Sponsors
Funding source category [1] 311735 0
Government body
Name [1] 311735 0
Medical Research Future Fund, National Health and Medical Research Council
Country [1] 311735 0
Australia
Primary sponsor type
Other Collaborative groups
Name
Murdoch Children's Research Institute
Address
MCRI
Royal Children's Hospital
50 Flemington Road
Parkville VIC 3052
Country
Australia
Secondary sponsor category [1] 313196 0
None
Name [1] 313196 0
Address [1] 313196 0
Country [1] 313196 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 311182 0
The Royal Children’s Hospital Human Research Ethics Committee
Ethics committee address [1] 311182 0
Ethics committee country [1] 311182 0
Australia
Date submitted for ethics approval [1] 311182 0
25/11/2021
Approval date [1] 311182 0
07/03/2022
Ethics approval number [1] 311182 0
HREC/81244/RCHM-2022

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 120242 0
A/Prof Jonathan Payne
Address 120242 0
MCRI
Royal Children's Hospital
50 Flemington Road
Parkville VIC 3052
Country 120242 0
Australia
Phone 120242 0
+61 3 99366761
Fax 120242 0
Email 120242 0
jonathan.payne@mcri.edu.au
Contact person for public queries
Name 120243 0
Alice Maier
Address 120243 0
MCRI
Royal Children's Hospital
50 Flemington Road
Parkville VIC 3052
Country 120243 0
Australia
Phone 120243 0
+61 3 99366150
Fax 120243 0
Email 120243 0
alice.maier@mcri.edu.au
Contact person for scientific queries
Name 120244 0
Alice Maier
Address 120244 0
MCRI
Royal Children's Hospital
50 Flemington Road
Parkville VIC 3052
Country 120244 0
Australia
Phone 120244 0
+61 3 99366150
Fax 120244 0
Email 120244 0
alice.maier@mcri.edu.au

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
Individual participant data underlying published results.
When will data be available (start and end dates)?
Beginning no less than 6 months following main results publications. No end date determined.
Available to whom?
Case-by-case basis at the discretion of the study PI. Researchers must be from a recognised research institute whose proposed use of the data has been ethically reviewed an approved by an independent committee and who accept the Sponsors conditions of access.
Available for what types of analyses?
To achieve aims approved by the PI.
How or where can data be obtained?
Access subject to approvals by the Sponsor and PI.
Principal Investigator contact: jonathan.payne@mcri.edu.au


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.