Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12622001329752
Ethics application status
Approved
Date submitted
7/10/2022
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: extension trial
Scientific title
An extension trial to determine the effect of remote microphone listening devices on speech perception, functional hearing, literacy skills and auditory neural conduction in children with neurofibromatosis type 1 and auditory deficits
Secondary ID [1] 308132 0
81244 (extension)
Universal Trial Number (UTN)
U1111-1283-6942
Trial acronym
TAP-iN
Linked study record
This trial is the extension of the parent randomised controlled trial ACTRN12622001328763

Health condition
Health condition(s) or problem(s) studied:
Neurofibromatosis type 1 327848 0
Condition category
Condition code
Human Genetics and Inherited Disorders 324925 324925 0 0
Other human genetics and inherited disorders
Ear 324926 324926 0 0
Other ear disorders
Neurological 324927 324927 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
This is a multisite 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.

Children enrolled in this trial will have consented for participation in the parent study, which is a shorter randomised controlled trial (RCT) of RML devices. After completing participation in the parent study, participants will be invited into this extension trial where they will wear the RML device for a longer period of time (approximately 3 school terms). This will enable us to determine whether longer-term device use can improve learning outcomes for children with NF1.

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 weekly compliance check.
Intervention code [1] 324587 0
Treatment: Devices
Comparator / control treatment
Treatment as usual. Children in the treatment as usual control 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] 332737 0
This study does not have any primary outcomes. Please refer to the primary outcomes listed in record ACTRN12622001328763, the related parent study.
Timepoint [1] 332737 0
N/A
Secondary outcome [1] 414563 0
Phonological Awareness Composite from the Comprehensive Test of Phonological Processing
Timepoint [1] 414563 0
Baseline, end of post treatment (30 weeks post baseline)
Secondary outcome [2] 414564 0
Sight Word condition from the Test of Word Reading Efficiency-2
Timepoint [2] 414564 0
Baseline, end of post treatment (30 weeks post baseline)
Secondary outcome [3] 414565 0
Phonemic Decoding condition from the Test of Word Reading Efficiency-2
Timepoint [3] 414565 0
Baseline, end of post treatment (30 weeks post baseline)
Secondary outcome [4] 414566 0
Word Reading subtest from the Wechsler Individual Achievement Test-3
Timepoint [4] 414566 0
Baseline, end of post treatment (30 weeks post baseline)
Secondary outcome [5] 414567 0
Pseudoword Decoding subtest from the Wechsler Individual Achievement Test-3
Timepoint [5] 414567 0
Baseline, end of post treatment (30 weeks post baseline)
Secondary outcome [6] 414568 0
Spelling subtest from the Wechsler Individual Achievement Test-3
Timepoint [6] 414568 0
Baseline, end of post treatment (30 weeks post baseline)
Secondary outcome [7] 414569 0
Listening in Spatialized Noise-Sentences test
Timepoint [7] 414569 0
Baseline, end of post treatment (30 weeks post baseline)
Secondary outcome [8] 414570 0
Total score on the Listening Inventory for Education-Revised (child version)
Timepoint [8] 414570 0
Baseline, end of post treatment (30 weeks post baseline)
Secondary outcome [9] 414571 0
Total score on the Listening Inventory for Education-Revised (teacher version)
Timepoint [9] 414571 0
Baseline, end of post treatment (30 weeks post baseline)
Secondary outcome [10] 414572 0
Conners-3 (short form) DSM-5 Inattentive Content Scale (parent version)
Timepoint [10] 414572 0
Baseline, end of post treatment (30 weeks post baseline)
Secondary outcome [11] 414573 0
Conners-3 (short form) DSM-5 Inattentive Content Scale (teacher version)
Timepoint [11] 414573 0
Baseline, end of post treatment (30 weeks post baseline)
Secondary outcome [12] 414574 0
Social Skills Scale from the Behavior Assessment System for Children-3 (parent version)
Timepoint [12] 414574 0
Baseline, end of post treatment (30 weeks post baseline)
Secondary outcome [13] 414575 0
Social Skills Scale from the Behavior Assessment System for Children-3 (teacher version)
Timepoint [13] 414575 0
Baseline, end of post treatment (30 weeks post baseline)
Secondary outcome [14] 414576 0
Interpersonal Relations Scale from the Behavior Assessment System for Children-3 (child version)
Timepoint [14] 414576 0
Baseline, end of post treatment (30 weeks post baseline)
Secondary outcome [15] 414577 0
Anxiety Scale from the Behavior Assessment System for Children-3 (parent version)
Timepoint [15] 414577 0
Baseline, end of post treatment (30 weeks post baseline)
Secondary outcome [16] 414578 0
Anxiety Scale from the Behavior Assessment System for Children-3 (child version)
Timepoint [16] 414578 0
Baseline, end of post treatment (30 weeks post baseline)
Secondary outcome [17] 414579 0
Anxiety Scale from the Behavior Assessment System for Children-3 (teacher version)
Timepoint [17] 414579 0
Baseline, end of post treatment (30 weeks post baseline)
Secondary outcome [18] 414580 0
Psychosocial score from the PedsQL Generic Module (parent version)
Timepoint [18] 414580 0
Baseline, end of post treatment (30 weeks post baseline)
Secondary outcome [19] 414581 0
Psychosocial score from the PedsQL Generic Module (child version)
Timepoint [19] 414581 0
Baseline, end of post treatment (30 weeks post baseline)
Secondary outcome [20] 414582 0
Cognitive Fatigue Score from the PedsQL Multidimensional Fatigue Module (parent version)
Timepoint [20] 414582 0
Baseline, end of post treatment (30 weeks post baseline)
Secondary outcome [21] 414583 0
Cognitive Fatigue Score from the PedsQL Multidimensional Fatigue Module (child version)
Timepoint [21] 414583 0
Baseline, end of post treatment (30 weeks post baseline)
Secondary outcome [22] 414584 0
Auditory Brain Response (ABR).

Acoustic click stimuli will be presented to each ear individually at 90 dBnHL (decibels hearing level) at varying rates from 8-100 Hertz. Each test run will comprise 2000 sweeps, with a minimum of two runs completed at each stimulus rate. Runs runs will be compared to determine waveform repeatability. Absolute latencies and interlatencies of ABR waves I, III and V will be determined for each stimulus rate.
Timepoint [22] 414584 0
Baseline, end of post treatment (30 weeks post baseline)
Secondary outcome [23] 414585 0
Adverse events after long 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 [23] 414585 0
Baseline, end of post treatment (30 weeks post baseline)

Eligibility
Key inclusion criteria
• Children aged 6-12 years.
• Satisfy the revised diagnostic criteria for NF1.
• Consented to and screened for the parent RCT trial.
• 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.
• School/teacher 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.
• Consent and screening not undertaken for the parent RCT.
• 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.
• Starting secondary school/high school within 9 months of commencing this extension phase of the study
• 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
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
Parallel
Other design features
There are several pathways a child can take to participate in this extension trial. All of them will require informed consent of the parent RCT. Children with NF1 can participate in this extension trial via the following pathways:

1. Children with NF1 screen eligible to complete the parent RCT and following completion of that trial, they opt to continue wearing the RML device in the classroom for 3 school terms. In this case, they will be enrolled in the treated condition of the extension trial.

2. Children with NF1 screen eligible to complete the parent RCT and following completion of that trial, they opt to not continue wearing the RML device in the classroom. In this case, they will be invited into the treatment as usual control condition of the extension trial.

3. Children with NF1 pass all screening criteria to be eligible for the parent RCT except that they do not demonstrate a difficulty perceiving speech in noise as part of their screening assessment. These children will not enter the RCT trial, but be directly invited into the treatment as usual control condition of the extension trial.

For children in the treated condition, they will be asked to wear the RML device Monday to 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 weekly compliance check so we can determine the amount of time the device was used.
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.

For this extension trial, between-group comparisons of secondary continuous outcomes will be presented as mean difference and 95% CI, calculated from generalised linear regression models, adjusted for baseline scores and characteristics (age, sex). We will also perform additional analyses which take into account interventions received and compliance to device across the 30-week follow-up.

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
19/12/2025
Actual
Date of last data collection
Anticipated
30/09/2026
Actual
Sample size
Target
100
Accrual to date
0
Final
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 23328 0
The Royal Childrens Hospital - Parkville
Recruitment hospital [2] 23329 0
The Children's Hospital at Westmead - Westmead
Recruitment postcode(s) [1] 38702 0
3052 - Parkville
Recruitment postcode(s) [2] 38703 0
2145 - Westmead

Funding & Sponsors
Funding source category [1] 312388 0
Government body
Name [1] 312388 0
Medical Research Future Fund, National Health and Medical Research Council
Country [1] 312388 0
Australia
Primary sponsor type
Other Collaborative groups
Name
Murdoch Children's Research Institute
Address
Murdoch Children's Research Institute
Royal Children's Hospital
50 Flemington Road
Parkville VIC 3052
Country
Australia
Secondary sponsor category [1] 313960 0
None
Name [1] 313960 0
Address [1] 313960 0
Country [1] 313960 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 311745 0
The Royal Children’s Hospital Human Research Ethics Committee
Ethics committee address [1] 311745 0
Research Ethics & Governance Office
The Royal Children's Hospital
Level 4, South Building
50 Flemington Road
Parkville Vic 3052
Ethics committee country [1] 311745 0
Australia
Date submitted for ethics approval [1] 311745 0
25/11/2021
Approval date [1] 311745 0
07/03/2022
Ethics approval number [1] 311745 0

Summary
Brief summary
Learning disorders are one of the greatest causes of morbidity in children with the genetic syndrome, neurofibromatosis type 1 (NF1) and result in academic underachievement, reduced quality of life, and are of significant concern to families and their teachers. There are minimal evidence-based interventions for these problems in NF1, and there is an urgent need for trials targeting this area of clinical need.

This registration form details the extension study from the Treating Auditory Problems in NF1 "TAP-iN" trial. The extension study described on this registry form will enable us to establish the efficacy of an RML device in treating central auditory deficits in children with NF1 and will allow us to determine whether treatment benefits extend to broader areas of learning and behaviour. Outcomes are clinically meaningful and include measures of speech perception, literacy skills, attention, fatigue, social function and quality of life. If realized, this study will provide powerful evidence for a novel, non-invasive intervention targeting a common and impairing problem in NF1.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 122222 0
A/Prof Jonathan Payne
Address 122222 0
MCRI
Royal Children's Hospital
50 Flemington Road
Parkville VIC 3052
Country 122222 0
Australia
Phone 122222 0
+61399366761
Fax 122222 0
Email 122222 0
[email protected]
Contact person for public queries
Name 122223 0
Ms Alice Maier
Address 122223 0
MCRI
Royal Children's Hospital
50 Flemington Road
Parkville VIC 3052
Country 122223 0
Australia
Phone 122223 0
+61 3 99366150
Fax 122223 0
Email 122223 0
[email protected]
Contact person for scientific queries
Name 122224 0
Ms Alice Maier
Address 122224 0
MCRI
Royal Children's Hospital
50 Flemington Road
Parkville VIC 3052
Country 122224 0
Australia
Phone 122224 0
+61 3 99366150
Fax 122224 0
Email 122224 0
[email protected]

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 and 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: [email protected]


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.