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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05185583




Registration number
NCT05185583
Ethics application status
Date submitted
29/11/2021
Date registered
11/01/2022

Titles & IDs
Public title
Methylphenidate in Childhood Apraxia of Speech
Scientific title
A Phase II Proof-of-concept Trial of Methylphenidate in Children With Apraxia of Speech: a Double-blind, Randomised, Placebo-controlled, Cross-over Trial
Secondary ID [1] 0 0
77169
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Childhood Apraxia of Speech 0 0
Condition category
Condition code
Neurological 0 0 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Methylphenidate Hydrochloride
Treatment: Drugs - Placebo

Experimental: Sequence A: Methylphenidate, Placebo - Participants will first receive methylphenidate capsules twice daily for four weeks. Doses will be administered four hours apart. The maximum dose is determined based on the participant's weight. After a 2-day washout, participants then receive Placebo (matching methylphenidate capsules) twice daily for four weeks.

Experimental: Sequence B: Placebo, Methylphenidate - Participants will first receive Placebo capsules twice daily for four weeks. Doses will be administered four hours apart. After a 2-day washout, participants then receive methylphenidate capsules (matching Placebo capsules) twice daily for four weeks. The maximum dose is determined based on the participant's weight.


Treatment: Drugs: Methylphenidate Hydrochloride
Participants will receive twice daily doses of Methylphenidate Hydrochloride four hours apart. There will be three dosage schedules, determined based on three weight ranges (20-30kg; 30-40kg; =40kg). For children weighing 20-30kg, the maximum daily dose will be 20mg. For children weighing 30-40kg, the maximum daily dose will be 30mg. For children weighing =40kg, the maximum daily dose will be 40mg.

Treatment: Drugs: Placebo
Participants will receive twice daily doses of placebo capsules. Gelatine placebo capsules will contain hypromellose, an inert substance.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change From Baseline in Preschool Speech Intelligibility Measure Score at 4 weeks
Timepoint [1] 0 0
Baseline and 4 weeks
Secondary outcome [1] 0 0
Change From Baseline in Assessment of the Intelligibility of Dysarthric Speech-II (ASSIDS-II) Score at 4 weeks
Timepoint [1] 0 0
Baseline and 4 weeks
Secondary outcome [2] 0 0
Number of children at screening who refuse, are eligible, or are ineligible (and reason).
Timepoint [2] 0 0
At study recruitment, up to 4 weeks before starting treatment.
Secondary outcome [3] 0 0
Number of children who withdraw, discontinue, and/or experience 1 or more protocol violations.
Timepoint [3] 0 0
During the 4 week treatment phase.
Secondary outcome [4] 0 0
Adherence to dose regimen during each 4 week treatment period
Timepoint [4] 0 0
4 weeks
Secondary outcome [5] 0 0
Parent/caregiver experience of tolerability and utility
Timepoint [5] 0 0
Baseline and 4 weeks
Secondary outcome [6] 0 0
Paediatric patient experience of tolerability and utility
Timepoint [6] 0 0
Baseline and 4 weeks
Secondary outcome [7] 0 0
Change From Baseline in total number of grammatical features (Language Assessment, Remediation and Screening Procedure; LARSP) at 4 weeks
Timepoint [7] 0 0
Baseline and 4 weeks
Secondary outcome [8] 0 0
Change From Baseline in total number of sentences (Language Assessment, Remediation and Screening Procedure; LARSP) at 4 weeks
Timepoint [8] 0 0
Baseline and 4 weeks
Secondary outcome [9] 0 0
Change From Baseline in mean number of sentences per turn (Language Assessment, Remediation and Screening Procedure; LARSP) at 4 weeks
Timepoint [9] 0 0
Baseline and 4 weeks
Secondary outcome [10] 0 0
Change From Baseline in mean sentence length (Language Assessment, Remediation and Screening Procedure; LARSP) at 4 weeks
Timepoint [10] 0 0
Baseline and 4 weeks
Secondary outcome [11] 0 0
Change From Baseline in stage of grammatical development (Language Assessment, Remediation and Screening Procedure; LARSP) at 4 weeks
Timepoint [11] 0 0
Baseline and 4 weeks
Secondary outcome [12] 0 0
Change From Baseline in types of clausal structures used (Language Assessment, Remediation and Screening Procedure; LARSP) at 4 weeks
Timepoint [12] 0 0
Baseline and 4 weeks
Secondary outcome [13] 0 0
Change From Baseline in syllable repetition at 4 weeks
Timepoint [13] 0 0
Baseline and 4 weeks
Secondary outcome [14] 0 0
Change From Baseline in Intelligibility in Context Scale Score at 4 weeks
Timepoint [14] 0 0
Baseline and 4 weeks
Secondary outcome [15] 0 0
Change From Baseline in Children's Non-word Repetition (CNRep) Test Score at 4 weeks
Timepoint [15] 0 0
Baseline and 4 weeks
Secondary outcome [16] 0 0
Change From Baseline in Swanson, Nolan, and Pelham (SNAP-IV) Parent 18-Item Rating Scale (SNAP-IV) Score at 4 weeks
Timepoint [16] 0 0
Baseline and 4 weeks

Eligibility
Key inclusion criteria
* Has childhood apraxia of speech
* Aged 6-12 years
* Can perform the speech tasks for the trial (able to speak single words and short sentences)
* English as a first language
* Has adequate hearing
* Has a legally acceptable representative capable of understanding the informed consent document and providing consent on their behalf
* Passes the health and medical examination including examination of heart rate and blood pressure for age and weight norms
* Can commit to the time requirements of the trial
* Lives within 250 kilometres of the study site (MCRI)
* Able to swallow a capsule
* Scores 13 or more out of 27 on either the inattention and/or hyperactivity subscales of the SNAP-IV Parent 18-Item Rating Scale, suggesting clinically significant symptoms of inattention and/or hyperactivity
Minimum age
6 Years
Maximum age
12 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Is unable to commit to the time requirements of the trial (8 weeks + 2 days)
* Has a diagnosis of severe intellectual disability, or other significant neurodevelopmental conditions (e.g., Fragile X, Down Syndrome, etc.)
* Has epilepsy or other seizure disorders
* Is taking medication(s) for another health condition(s) that is known to interfere with MPH
* Has any contraindication to the stimulant (methylphenidate) medication, including severe anxiety, depression, severe Tourette syndrome, glaucoma, psychotic symptoms, hypertension, congenital heart disease, known past or present diagnosed substance abuse or dependence
* Has a score of moderate or high risk of suicidality, assessed with the Columbia Suicidality Severity Rating Scale (C-SSRS)
* Has used psychostimulants within the past 3 months (e.g., Ritalin, Concerta, Focalin)
* Lives more than 250 kilometres from the study site
* Unable to swallow a capsule

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)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Crossover
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
14/03/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/07/2025
Actual
Sample size
Target
24
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Murdoch Children's Research Institute - Parkville
Recruitment postcode(s) [1] 0 0
3052 - Parkville

Funding & Sponsors
Primary sponsor type
Other
Name
Murdoch Childrens Research Institute
Address
Country

Ethics approval
Ethics application status

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

Contacts
Principal investigator
Name 0 0
Angela Morgan, PhD
Address 0 0
Murdoch Childrens Research Institute
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Angela Morgan, PhD
Address 0 0
Country 0 0
Phone 0 0
+613 8341 6458
Fax 0 0
Email 0 0
angela.morgan@mcri.edu.au
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
The de-identified data set collected for this analysis of the trial will be available six months after publication of the primary outcome. The study protocol may be obtained from the Murdoch Children's Research Institute. Prior to releasing any data the following are required: a data access agreement must be signed between relevant parties, the trial investigators must see and approve the analysis plan describing how the data will be analyzed, there must be an agreement around appropriate acknowledgment and any additional costs involved must be covered. Should the study investigators be unavailable, this role is delegated to the Murdoch Children's Research Institute. Data will only be shared with a recognized research institute which has approved the proposed analysis plan.

Supporting document/s available: Study protocol, Statistical analysis plan (SAP), Informed consent form (ICF)
When will data be available (start and end dates)?
6 months after publication of primary outcome
Available to whom?
Prior to releasing any data the following are required: a data access agreement must be signed between relevant parties, the trial investigators must see and approve the analysis plan describing how the data will be analyzed, there must be an agreement around appropriate acknowledgment and any additional costs involved must be covered.
Available for what types of analyses?
How or where can data be obtained?


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05185583