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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
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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
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Secondary ID [1]
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77169
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Childhood Apraxia of Speech
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Condition category
Condition code
Neurological
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Other neurological disorders
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Intervention/exposure
Study type
Interventional
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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.
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Change From Baseline in Preschool Speech Intelligibility Measure Score at 4 weeks
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Assessment method [1]
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Single word speech intelligibility will be measured using the Preschool Speech Intelligibility Measure (PSIM). Twenty (20) items from the PSIM will be administered at each testing time point. Participants are required to repeat each test word after the researcher. Higher scores are indicative of greater unintelligibility (range 0-80). Mean change from baseline will be reported at the group level.
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Timepoint [1]
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Baseline and 4 weeks
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Secondary outcome [1]
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Change From Baseline in Assessment of the Intelligibility of Dysarthric Speech-II (ASSIDS-II) Score at 4 weeks
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Assessment method [1]
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The Assessment of the Intelligibility of Dysarthric Speech-II (ASSIDS-II) will be used to measure sentence level speech intelligibility. Five (5) 5-word sentences will be administered at each assessment time point. A phonemic accuracy score will be calculated by determining the percentage of correct phonemes across the 5 sentences at each timepoint. Mean change from baseline will be reported at the group level.
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Timepoint [1]
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Baseline and 4 weeks
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Secondary outcome [2]
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Number of children at screening who refuse, are eligible, or are ineligible (and reason).
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Assessment method [2]
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The frequency of children at screening who refuse, are eligible, or are ineligible (and reason).
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Timepoint [2]
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At study recruitment, up to 4 weeks before starting treatment.
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Secondary outcome [3]
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Number of children who withdraw, discontinue, and/or experience 1 or more protocol violations.
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Assessment method [3]
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Number of children in either sequence A or B who:
1. withdraw
2. discontinue the treatment prior to 4 weeks
3. experience 1 or more protocol violations.
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Timepoint [3]
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During the 4 week treatment phase.
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Secondary outcome [4]
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Adherence to dose regimen during each 4 week treatment period
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Assessment method [4]
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Adherence will be determined by calculating the percentage of prescribed doses achieved based on the medication diary entries and reconciling against returned dispensed medication. A participant will be considered adherent during a 4-week period if 80% of the prescribed doses are achieved. Data will be aggregated such that the proportion of children who adhere to the dosing regimen in each 4-week period will be reported.
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Timepoint [4]
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4 weeks
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Secondary outcome [5]
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Parent/caregiver experience of tolerability and utility
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Assessment method [5]
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Measured using the investigators' parent/caregiver tolerability and utility survey which is a dedicated electronic Case Report Form. This provides data on caregiver(s)'s views of usability (e.g., "It was convenient to give my child the capsules"), tolerability (e.g., "It was not a burden to participate in this trial") and experience of the trial (Vogel, 2016) (e.g., "I am glad my child participated in this trial"), and whether speech was improved (e.g., "I think the capsule improved my child's speech"). Items will be rated 1-5 where a score of 1 indicates the parent/caregiver strongly disagrees and a score of 5 indicates the parent/caregiver strongly agrees. One item related to school can be rated as 0 if the child is on holidays or in a COVID-19 lockdown for the trial duration. Total scores range from 20-105, where a higher score indicates a greater tolerability/utility of the trial as rated by the parent. Mean change from baseline will be reported at the group level.
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Timepoint [5]
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Baseline and 4 weeks
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Secondary outcome [6]
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Paediatric patient experience of tolerability and utility
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Assessment method [6]
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Measured using the investigators' patient tolerability and utility survey which is a dedicated electronic Case Report Form (eCRF). This provides data on participants' views of usability (e.g., "It was easy to take the capsules"), tolerability/burden (e.g., "I had enough energy to play when taking the capsules") and experience of the trial (Vogel, 2016) (e.g., "I would do this trial again"), and whether speech was improved (e.g.,"I think my speech is better after taking the capsules"). Items will be rated 1-5 where a score of 1 indicates the participant strongly disagrees with the statement and a score of 5 indicates the participant strongly agrees. One item related to school can be rated as 0 if the child is on holidays or in a COVID-19 lockdown for the duration of the trial. Total scores range from 20-105, where, a higher score indicates a greater tolerability/utility of the trial as rated by the child participant. Mean change from baseline will be reported at the group level.
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Timepoint [6]
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Baseline and 4 weeks
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Secondary outcome [7]
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Change From Baseline in total number of grammatical features (Language Assessment, Remediation and Screening Procedure; LARSP) at 4 weeks
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Assessment method [7]
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Children's conversational speech will be analysed to determine the total number of grammatical features present using the Language Assessment, Remediation and Screening Procedure (LARSP). Mean change from baseline will be reported at the group level.
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Timepoint [7]
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Baseline and 4 weeks
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Secondary outcome [8]
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Change From Baseline in total number of sentences (Language Assessment, Remediation and Screening Procedure; LARSP) at 4 weeks
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Assessment method [8]
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Children's conversational speech will be analysed to determine the total number of sentences present using the Language Assessment, Remediation and Screening Procedure (LARSP). Mean change from baseline will be reported at the group level.
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Timepoint [8]
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Baseline and 4 weeks
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Secondary outcome [9]
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Change From Baseline in mean number of sentences per turn (Language Assessment, Remediation and Screening Procedure; LARSP) at 4 weeks
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Assessment method [9]
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Children's conversational speech will be analysed to determine the total number of sentences per turn using the Language Assessment, Remediation and Screening Procedure (LARSP). Mean change from baseline will be reported at the group level.
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Timepoint [9]
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Baseline and 4 weeks
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Secondary outcome [10]
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Change From Baseline in mean sentence length (Language Assessment, Remediation and Screening Procedure; LARSP) at 4 weeks
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Assessment method [10]
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Children's conversational speech will be analysed to determine the mean sentence length produced using the Language Assessment, Remediation and Screening Procedure (LARSP). Mean change from baseline will be reported at the group level.
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Timepoint [10]
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Baseline and 4 weeks
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Secondary outcome [11]
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Change From Baseline in stage of grammatical development (Language Assessment, Remediation and Screening Procedure; LARSP) at 4 weeks
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Assessment method [11]
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Children's conversational speech will be analysed to determine the child's stage of grammatical development, identified using the Language Assessment, Remediation and Screening Procedure (LARSP). Mean change from baseline will be reported at the group level.
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Timepoint [11]
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Baseline and 4 weeks
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Secondary outcome [12]
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Change From Baseline in types of clausal structures used (Language Assessment, Remediation and Screening Procedure; LARSP) at 4 weeks
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Assessment method [12]
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Children's conversational speech will be analysed to determine types of clausal structures used by the child, identified using the Language Assessment, Remediation and Screening Procedure (LARSP). Aggregated data will be reported.
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Timepoint [12]
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Baseline and 4 weeks
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Secondary outcome [13]
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Change From Baseline in syllable repetition at 4 weeks
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Assessment method [13]
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Syllable repetition will be measured by analysing the average number of syllables produced, average period or duration between consonant-vowel syllable voicing offsets (milliseconds) and variance of the diadochokinetic period (percentage reported). Mean change from baseline will be reported at the group level, and will be compared between Sequence A and Sequence B.
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Timepoint [13]
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Baseline and 4 weeks
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Secondary outcome [14]
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Change From Baseline in Intelligibility in Context Scale Score at 4 weeks
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Assessment method [14]
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The Intelligibility in Context Scale (ICS) will measure functional speech intelligibility using parent/caregiver ratings across 7 items, where high scores are indicative of poor intelligibility of speech (range 7-35). Mean change from baseline will be reported at the group level.
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Timepoint [14]
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Baseline and 4 weeks
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Secondary outcome [15]
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Change From Baseline in Children's Non-word Repetition (CNRep) Test Score at 4 weeks
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Assessment method [15]
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The Children's Non-word Repetition (CNRep) Test will measure phonological working memory. Ten CNRep items will be administered at each of the four assessment timepoints to control for learning effects. The percentage of correct responses will be calculated by syllable (i.e., 2, 3, 4 and 5 syllable words) and for the total list of words. Mean change from baseline will be reported at the group level.
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Timepoint [15]
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Baseline and 4 weeks
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Secondary outcome [16]
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Change From Baseline in Swanson, Nolan, and Pelham (SNAP-IV) Parent 18-Item Rating Scale (SNAP-IV) Score at 4 weeks
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Assessment method [16]
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The Swanson, Nolan, and Pelham (SNAP-IV) Questionnaire is a measure of Attention Deficit Hyperactivity Disorder (ADHD) symptoms using ADHD diagnostic criteria specified in the Diagnostic and Statistical Manual of Mental Disorders, Fourth Edition (DSM-IV). Parent ratings on the Attention and Hyperactivity subscales will be used to measure attention deficit and hyperactivity symptoms. Higher scores indicate greater inattentive and/or hyperactive behaviours. Scores on each subscale range from 0-27. A score below 13 is indicative of symptoms not being clinically significant. A score of 13-17 indicates mild symptoms; a score of 18-22 indicates moderate symptoms and a score of 22-27 indicates severe symptoms. Mean change from baseline will be reported at the group level.
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Timepoint [16]
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Baseline and 4 weeks
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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
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Minimum age
6
Years
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Maximum age
12
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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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
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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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
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Intervention assignment
Crossover
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
14/03/2022
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
1/07/2025
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Actual
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Sample size
Target
24
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
VIC
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Recruitment hospital [1]
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Murdoch Children's Research Institute - Parkville
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Recruitment postcode(s) [1]
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3052 - Parkville
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Funding & Sponsors
Primary sponsor type
Other
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Name
Murdoch Childrens Research Institute
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
The purpose of this study is to describe the possible effects of methylphenidate (MPH) on speech intelligibility in children with childhood apraxia of speech (CAS) aged 6-12 years. This outcome will be compared between MPH intake and placebo intake.
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Trial website
https://clinicaltrials.gov/study/NCT05185583
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Trial related presentations / publications
Fraile R, Saenz-Lechon N, Godino-Llorente JI, Osma-Ruiz V, Fredouille C. Automatic detection of laryngeal pathologies in records of sustained vowels by means of mel-frequency cepstral coefficient parameters and differentiation of patients by sex. Folia Phoniatr Logop. 2009;61(3):146-52. doi: 10.1159/000219950. Epub 2009 Jul 1. Sapir S, Ramig LO, Spielman JL, Fox C. Formant centralization ratio: a proposal for a new acoustic measure of dysarthric speech. J Speech Lang Hear Res. 2010 Feb;53(1):114-25. doi: 10.1044/1092-4388(2009/08-0184). Epub 2009 Nov 30. Vergis, Ballard, K. J., Duffy, J. R., McNeil, M. R., Scholl, D., & Layfield, C. (2014). An acoustic measure of lexical stress differentiates aphasia and aphasia plus apraxia of speech after stroke. Aphasiology, 28(5), 554-575. https://doi.org/10.1080/02687038.2014.889275 Vogel, A., Skarrat, J., Castles, J., Synofzik, M. . (2016). Video game-based speech rehabilitation for reducing dysarthria severity in adults with degenerative ataxia. European Journal of Neurology, 23(227).
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Public notes
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Contacts
Principal investigator
Name
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Angela Morgan, PhD
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Address
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Murdoch Childrens Research Institute
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Angela Morgan, PhD
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Address
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Country
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Phone
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+613 8341 6458
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Fax
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Email
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[email protected]
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Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
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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)
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When will data be available (start and end dates)?
6 months after publication of primary outcome
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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.
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Available for what types of analyses?
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How or where can data be obtained?
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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