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Trial registered on ANZCTR


Registration number
ACTRN12619000525189
Ethics application status
Approved
Date submitted
27/03/2019
Date registered
2/04/2019
Date last updated
26/05/2020
Date data sharing statement initially provided
2/04/2019
Date results information initially provided
26/05/2020
Type of registration
Prospectively registered

Titles & IDs
Public title
Pilot Study of Assistive Listening Devices in Treating Auditory Processing Deficits in Neurofibromatosis Type 1
Scientific title
Feasibility of using Assistive Listening Devices in Treatment of Auditory Processing Deficits in Neurofibromatosis Type 1: A pilot study
Secondary ID [1] 297817 0
Nil
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
auditory processing deficits in children with Neurofibromatosis Type 1 312184 0
Condition category
Condition code
Human Genetics and Inherited Disorders 310734 310734 0 0
Other human genetics and inherited disorders
Ear 310747 310747 0 0
Other ear disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
This is a single site, randomised, blinded, two period crossover pilot study of 10 participants investigating the use of remote microphone assistive listening devices (ALD) in treating auditory processing deficits in children with Neurofibromatosis Type (NF1).

Eligible participants will have their hearing assessed under two conditions (with and without ALD) and testing order will be randomised. This is to confirm that the ALD confers a functional hearing benefit. Children will then be randomised to one of two treatment sequences: 1) being fitted with an inactive ALD for two weeks, followed by active ALD use for two weeks or 2) being fitted with an activated ALD for two weeks, following a period of two weeks of use with a deactivated ALD. This is to assess whether the device confers a hearing benefit in everyday listening environments. All hearing tests will be conducted, and all ALD fitted, by an appropriately trained study audiologist. No wash out period is included, consistent with other clinical trials investigating classroom benefits of ALD’s in children. Carryover effects are not expected.

This pilot study will assess the feasibility of conducting a large scale, randomised, study of the use of assistive listening devices in the treatment of auditory processing deficits in children with NF1. This study further aims to assess whether assistive listening devices ameliorate auditory processing deficits in children with NF1 and will also explore if assistive listening devices impact ADHD symptomatology in children with NF1. Throughout the trial, parents, children and their teachers will complete questionnaire measures regarding their hearing/listening and behaviour, the acceptability of the trial and the tolerability of the device. Compliance will be assessed using teacher and parent responses on a daily adherence checklist.
Intervention code [1] 314061 0
Treatment: Devices
Comparator / control treatment
The ALD will be deactivated by the study audiologist by unpairing the microphone from the receiver. This means that no auditory signal will be transmitted from the microphone to the earpiece receiver. Even when unpaired, the earpiece receiver and microphone devices will still outwardly appear functional (i.e., power lights will still be on; screens functioning etc). The use of a placebo phase will allow us to identify any potential practice effects and distinguish whether any changes in the treatment group are larger than those accounted for by practice effects (i.e., a treatment effect).
Control group
Placebo

Outcomes
Primary outcome [1] 319582 0
Recruitment strategy, including:
- Recruitment rate (% of eligible participants enrolled)
- Suitability of selection criteria (% screening failures and % successfully randomised).
Timepoint [1] 319582 0
Baseline (prior to the commencement of period 1 and 2)
Primary outcome [2] 319583 0
Feasibility of trial design for patients with NF1: ability and acceptability of parent/carer to comply with the demands of the protocol, including:
- Study visit attendance (assessed at the end of period 2)
- Number of participants who complete only period 1 of the study.
- Number of participants who withdraw during the study (assessed at the end of period 2).
- Number of participants who commit major protocol violations (assessed at end of period 2 via parent/child and teacher questionnaire completion)
- Number of protocol violations (assessed at the end of period 2 via parent/child and teacher questionnaire completion).
Timepoint [2] 319583 0
End of period 1 (first two week intervention period)
End of period 2 (second two week intervention period)
Primary outcome [3] 319584 0
Feasibility of trial design for patients with NF1: tolerability and adherence to ALD, including:
- Tolerability questionnaires developed for the purposes of this study, administered to children and their parents.
- Device compliance checklist completed by participant's parents and teachers.
Timepoint [3] 319584 0
- Tolerability questionnaire: end of period 2 (i.e., at the end of the intervention period).

- Device compliance checklist: completed daily throughout intervention and collected at the end of periods 1 and 2 (i.e., at the end of the first and second two week intervention period).
Secondary outcome [1] 368784 0
Auditory processing deficits as assessed via the Consonant-Nucleus- Consonant speech perception test.
Timepoint [1] 368784 0
Baseline (device aided versus unaided; assessed in clinic by study audiologist prior to commencing period 1)
Secondary outcome [2] 368785 0
Auditory processing deficits as assessed via teacher, parent and child questionnaire (Listening Inventory for Education Questionnaire Revised; Abbreviated Profile of Hearing Aid Benefit)
Timepoint [2] 368785 0
Baseline
End of period 1 (first two week intervention period)
End of period 2 (second two week intervention period)
Secondary outcome [3] 368786 0
Inattentive ADHD behaviours assessed via parent and teacher questionnaire (Connors 3)
Timepoint [3] 368786 0
Baseline
End of period 1 (first two week intervention period)
End of period 2 (second two week intervention period)

Eligibility
Key inclusion criteria
1) Satisfy the NIH diagnostic criteria for NF1
2) Objective evidence of auditory processing deficits, as identified by a qualified audiologist on 2 or more objective measures of auditory processing (including the Listening in specialised noise test; Gap detection test, Consonant-Nucleus-Consonant test, and auditory brainstem response)
3) Written informed consent from parent/legal guardian and verbal consent from child
Minimum age
6 Years
Maximum age
17 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Non-English speaking parents.
2. Abnormal sound detection ability as indicated through behavioural audiogram and oto-accoustic emissions.
3. Use of cochlear implants, hearing aids or other type of corrective hearing device.
4. Active ear infection.
5. Full scale IQ < 70 on standardised cognitive assessment on verified records of testing performed within 2 years of enrolment.

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)
Treatment order (active vs non active ALD use in classroom) will be allocated by a statistician not directly involved in the study at the central site. They will provide the treatment schedule to the study audiologists via a password protected document in a confidential email.

Children and their families and teachers will not be advised of which treatment order they have been randomised to until they complete, or withdraw from, the trial. Study clinicians who are not directly involved in the fitting of the devices will also remain blinded to treatment order.
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Treatment order will be set up by a statistician not directly involved in the analysis of the trial results, who will prepare the randomisation schedule using a using a computer generated sequence.
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s

The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Crossover
Other design features
Phase
Phase 1
Type of endpoint(s)
Efficacy
Statistical methods / analysis
Screening and baseline characteristics of the participants will be presented using means and standard deviations for continuous data (or medians and inter-quartile ranges for non-normal data) and proportions for categorical data.

When a participant withdraws from the study, the reasons for withdrawal shall be recorded by the investigator. Because this is a pilot protocol and the main outcome of the study is the feasibility of the protocol, participant withdrawal numbers and reasons represent data, and will be reported as such.

The following information will be presented: number of children approached regarding the study, number of children who agree to be in the study, number of children who refuse to be in the study, number of children who start the study, number of children who complete period 1 of the study, number of children who complete period 1 and period 2 of the study, number of children who withdraw from the study categorized by reasons and period, number of children who discontinue from treatment categorized by reasons and period, number of children with adverse reactions, number of children fully compliant with the treatments, number of children with protocol violations.

All the secondary outcome measures will be presented using means and standard deviations for continuous data (or medians and inter-quartile ranges for non-normal data) and proportions for categorical data, by treatment (active ADL vs non-active ADL) and by period (1 vs 2).

The existence of carryover effects will be ruled out performing unpaired t-tests, and the existence of period effects using paired t-test. If the carryover effect is not significant between the two sequences, then the data from two periods will be combined and analysed using paired t-tests. Otherwise, the data only from period 1 will be used to estimate the treatment effect. The differences between treatment effects on the secondary outcomes will then be assessed by means of a standard t-tests for independent samples.


Recruitment
Recruitment status
Completed
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 13509 0
The Royal Childrens Hospital - Parkville
Recruitment postcode(s) [1] 26128 0
3052 - Parkville

Funding & Sponsors
Funding source category [1] 302341 0
Charities/Societies/Foundations
Name [1] 302341 0
Murdoch Children's Research Institute
Address [1] 302341 0
Royal Children's Hospital
50 Flemington Road
Parkville VIC 3052
Country [1] 302341 0
Australia
Primary sponsor type
Charities/Societies/Foundations
Name
Murdoch Children's Research Institute
Address
Royal Children's Hospital
50 Flemington Road
Parkville VIC 3052
Country
Australia
Secondary sponsor category [1] 302227 0
None
Name [1] 302227 0
Address [1] 302227 0
Country [1] 302227 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 303016 0
Royal Children's Hospital Human Research Ethics Committee.
Ethics committee address [1] 303016 0
Royal Children's Hospital
50 Flemington Road
Parkville VIC 3052
Ethics committee country [1] 303016 0
Australia
Date submitted for ethics approval [1] 303016 0
09/01/2019
Approval date [1] 303016 0
22/03/2019
Ethics approval number [1] 303016 0

Summary
Brief summary
NF1 is a common genetic condition with a prevalence rate of at least 1 in 2,700 live births. While not diagnostic of the condition, cognitive and behavioural impairments are the most commonly cited childhood complications of NF1. NF1 is also highly comorbid with ADHD (40%) and autism spectrum disorders (ASD; 25%). Auditory Processing Disorder (APD) is characterised by abnormal auditory nerve function in the presence of normal functioning cochlear sensory hair cells. Common impairments include a reduced ability to perceive rapid changes in auditory signals (e.g., brief gaps or amplitude fluctuations) and speech perception difficulties in the presence of background noise, even at signal to noise ratios that would be expected in everyday listening environments (e.g., classrooms; playgrounds). Our recent phenotyping study of 36 NF1 individuals indicated that a significant proportion presented with convincing evidence of APD, compared to age matched healthy control participants. The children in our study who demonstrated clinically impaired performances on two or more tests of auditory processing, on average, also scored within the clinical range on parental questionnaires of inattentive ADHD symptoms. The clinical question that remains is whether auditory processing deficits in NF1 children can be treated.

Intervention research suggests that assistive listening devices (ALD) attenuate speech processing deficits associated with APD in the general population. While there is evidence that ALD can improve listening and educational outcomes in other hearing-impaired populations, these systems have not yet been explored as an intervention option for children with NF1.

The current clinical trial is a pilot study assessing all elements of the study design (recruitment strategy, tolerability of the study product, study duration, study procedures and outcome measures) to assess whether ALDs are acceptable and feasible for the conduct of a full-scale randomized clinical trial to treat auditory processing deficits in NF1 children.

Eligible participants will have their hearing assessed under two conditions (with and without ALD) and testing order will be randomised. They will then be randomised to one of two treatment sequences: 1) being fitted with an inactive ALD for two weeks, followed by active ALD use for two weeks or 2) being fitted with an activated ALD for two weeks, followed by a period of two weeks of use with an inactivated ALD.

We hypothesise that children will exhibit improved hearing outcomes while wearing the active ALD, as measured by an objective audiology assessment, and by questionnaire ratings from children, their parents and teachers.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 92174 0
Dr Jonathan Payne
Address 92174 0
Murdoch Children's Research Institute
Royal Children's Hospital
50 Flemington Road
Parkville VIC 3052
Country 92174 0
Australia
Phone 92174 0
+61 3 9936 6761
Fax 92174 0
Email 92174 0
jonathan.payne@mcri.edu.au
Contact person for public queries
Name 92175 0
Dr Jonathan Payne
Address 92175 0
Murdoch Children's Research Institute
Royal Children's Hospital
50 Flemington Road
Parkville VIC 3052
Country 92175 0
Australia
Phone 92175 0
+61 3 9936 6761
Fax 92175 0
Email 92175 0
jonathan.payne@mcri.edu.au
Contact person for scientific queries
Name 92176 0
Prof Gary Rance
Address 92176 0
The University of Melbourne
Audiology & Speech Pathology
550 Swanston Street
Carlton VIC 3010
Country 92176 0
Australia
Phone 92176 0
+61 3 9035 5342
Fax 92176 0
Email 92176 0
grance@unimelb.edu.au

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment
What supporting documents are/will be available?
No other documents available
Summary results
Have study results been published in a peer-reviewed journal?
No
Other publications
Have study results been made publicly available in another format?
Results – basic reporting
Results – plain English summary