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Trial registered on ANZCTR


Registration number
ACTRN12611000779976
Ethics application status
Approved
Date submitted
26/07/2011
Date registered
26/07/2011
Date last updated
10/12/2015
Type of registration
Prospectively registered

Titles & IDs
Public title
The neural basis and treatment of reading disability in children with neurofibromatosis type 1
Scientific title
The neural basis and treatment of reading disability in children with neurofibromatosis type 1
Secondary ID [1] 262687 0
Nil
Universal Trial Number (UTN)
U1111-1123-1136
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Neurofibromatosis type 1 268391 0
Reading difficulties 270405 0
Condition category
Condition code
Human Genetics and Inherited Disorders 268532 268532 0 0
Other human genetics and inherited disorders
Neurological 270549 270549 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
There are 2 parts to the study. Participants can choose to complete Part A or Part A and B.
Part A involves participants using a computerised phonics treatment which trains letter recognition, parsing, letter-sound rules, and blending. Participants train at home for 30 minutes per day, 5 days per week, for 8 weeks.
Part B involves completing a magnetic resonance imaging (MRI) of the brain which lasts approximately 20 mins. Participants willl only complete the MRI once at baseline.
Intervention code [1] 267039 0
Treatment: Other
Comparator / control treatment
This study uses a double-baseline control. At the start of the study, each child completes the test battery (Time 1). After 8 weeks of *no* treatment, they redo the same tests (Time 2). We will then subtract their scores at Time 1 from Time 2 to reveal their test retest effects on each test. These test retest effects will then be subtracted from each child’s subsequent post treatment scores (i.e. Time 3 - after 8 weeks of training) and maintenance scores (Time 4 - 8 weeks after completing training). This allows us to determine how much each child improved due to training alone (i.e. excluding test retest effects) and to determine whether any effects were maintained after completion of training.
Control group
Active

Outcomes
Primary outcome [1] 269282 0
Castles and Coltheart 2 Nonword Reading Test
Timepoint [1] 269282 0
At Time 1, Time 2 (after 8 weeks of no treatment; baseline control), Time 3 (after 8 weeks of training) , and at Time 4 (8 weeks after completing training).
Primary outcome [2] 269283 0
Castles and Coltheart 2 Irregular Word Reading Test
Timepoint [2] 269283 0
At Time 1, Time 2 (after 8 weeks of no treatment; baseline control), Time 3 (after 8 weeks of training) , and at Time 4 (8 weeks after completing training).
Secondary outcome [1] 279292 0
Test of Word Reading Efficiency (TOWRE) Sight Word Efficiency and Phonetic Decoding Efficiency
Timepoint [1] 279292 0
At Time 1, Time 2 (after 8 weeks of no treatment; baseline control), Time 3 (after 8 weeks of training) , and at Time 4 (8 weeks after completing training).
Secondary outcome [2] 279293 0
Phonological recoding subskills (letter identification, parsing, letter-sound conversion, blending)
Timepoint [2] 279293 0
At Time 1, Time 2 (after 8 weeks of no treatment; baseline control), Time 3 (after 8 weeks of training) , and at Time 4 (8 weeks after completing training).
Secondary outcome [3] 279294 0
The Test of Everyday Reading Comprehension
Timepoint [3] 279294 0
At Time 1, Time 2 (after 8 weeks of no treatment; baseline control), Time 3 (after 8 weeks of training) , and at Time 4 (8 weeks after completing training).
Secondary outcome [4] 279295 0
The Kohnen Spelling Test for Nonwords and the Kohnen Spelling Test for Irregular Words
Timepoint [4] 279295 0
At Time 1, Time 2 (after 8 weeks of no treatment; baseline control), Time 3 (after 8 weeks of training) , and at Time 4 (8 weeks after completing training).

Eligibility
Key inclusion criteria
1. All participants must fulfil the diagnosis of NF1 based on NIH criteria
2. Participants must have a full scale IQ greater than or equal to 80 (WISC-IV) or if a significant discrepancy is present between PRI and VCI the higher will be used. This index used must be 80 or above
3. Participants must have access to a computer and the internet
Minimum age
7 Years
Maximum age
12 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Intracranial pathology such as epilepsy, hydrocephalus, diagnosed traumatic brain injury, or progressive intracranial tumours (children with asymptomatic or static lesions will be eligible);
2. Significant visual and/or hearing problems;
3. Currently taking psychotropic medication
4. Evidence/history of severe psychological disturbance
5. Insufficient English to complete the assessments

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)
After informed consent is obtained children are screened to determine whether they have an IQ greater than or equal to 80.
Part 1: All children who met this criteria will then complete a number of tests to provide information regarding their reading and cognitive profile.
Part 2: Those children who fall below the average range on nonword reading but within or above the average range on irregular word reading (phonological dyslexia) will be invited to participate in the computer treatement program.
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Children will not be randomised. All children participating in the treatment phase of the study will recieve the intervention and act as their own control (double-baseline).
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Not Applicable
Type of endpoint(s)
Efficacy
Statistical methods / analysis

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)

Funding & Sponsors
Funding source category [1] 267511 0
Hospital
Name [1] 267511 0
The Children's Hospital at Westmead
Address [1] 267511 0
Corner of Hawkesbury Road and Hainsworth Street
Locked Bag 4001
Westmead NSW 2145
Country [1] 267511 0
Australia
Primary sponsor type
Hospital
Name
The Children's Hospital at Westmead
Address
Corner of Hawkesbury Road and Hainsworth Street
Locked Bag 4001
Westmead NSW 2145
Country
Australia
Secondary sponsor category [1] 266553 0
None
Name [1] 266553 0
Address [1] 266553 0
Country [1] 266553 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 269477 0
Children's Hospital at Westmead Human Research Ethics Commitee
Ethics committee address [1] 269477 0
Corner of Hawkesbury Road and Hainsworth Street
Locked Bag 4001
Westmead NSW 2145
Ethics committee country [1] 269477 0
Australia
Date submitted for ethics approval [1] 269477 0
Approval date [1] 269477 0
01/06/2011
Ethics approval number [1] 269477 0
11/CHW/28

Summary
Brief summary
The study has two parts - Part A and Part B. Participants can choose to participate in Part A alone or both parts of the study.
The aim of Part A is to see if a computer-based training program can improve the reading of children with neurofibromatosis type 1 (NF1) and reading difficulties. Many children with NF1 experience difficulty reading, particularly when sounding out unfamiliar words (phonological dyslexia). It is hypothesised that by teaching these children letter-sound relationships (phonics) their reading and reading related skills (e.g. spelling) will improve.
The aim of Part B is to identify parts of brain tissue and structure that may be related to children's reading abilities.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 32913 0
Dr Belinda Barton
Address 32913 0
Children’s Hospital Education Research Institute
Suite 11, Level 2, Children's Hospital Medical Centre, Hainsworth St, Westmead NSW, 2145.
Country 32913 0
Australia
Phone 32913 0
+61 02 9845 0415
Fax 32913 0
Email 32913 0
belinda.barton@health.nsw.gov.au
Contact person for public queries
Name 16160 0
Mrs Shelley Arnold (Robertson)
Address 16160 0
NF1 Learning Disorders Clinic
c/- Clinical Research Rooms, Level 1
The Children's Hospital at Westmead
Locked Bag 4001
Westmead NSW 2145
Country 16160 0
Australia
Phone 16160 0
+61 02 9845 3057
Fax 16160 0
+61 02 9845 3945
Email 16160 0
shelley.arnold@health.nsw.gov.au
Contact person for scientific queries
Name 7088 0
Dr Jonathan Payne
Address 7088 0
The Children's Hospital at Westmead
Locked Bag 4001
Westmead NSW 2145
Country 7088 0
Australia
Phone 7088 0
+61 02 9845 3698
Fax 7088 0
+61 02 9845 3389
Email 7088 0
jonathan.payne@health.nsw.gov.au

No information has been provided regarding IPD availability
Summary results
Have study results been published in a peer-reviewed journal?
Other publications
Have study results been made publicly available in another format?
Results – basic reporting
Results – plain English summary