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Methylphenidate in Childhood Apraxia of Speech
PHASE2RECRUITING
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.
Study details:
This is a randomised, double-blind, placebo-controlled, two-period crossover proof-of-concept trial of methylphenidate (MPH) for children with childhood apraxia of speech (CAS). 24 children aged 6 to 12 years with CAS will be recruited. If children pass the screening procedure, which includes a physical exam conducted by a medical officer at the Melbourne Children's Campus, children will be enrolled into the 8 weeks + 2-day trial (includes 2-day washout).
Participants will be randomly assigned to sequence A (4 weeks of MPH, followed by 4 weeks of placebo) or sequence B (4 weeks of placebo, followed by 4 weeks of MPH). After 4 weeks in period 1, a two-day washout period will occur before participants crossover to period 2 for 4 weeks. Pre- and post-treatment speech outcomes will be measured.
The investigators' primary objective is to provide proof-of-concept that speech intelligibility could demonstrate greater improvements from baseline to 4 weeks following a 4-week period of MPH use compared with placebo in children with CAS. The secondary objectives are to describe feasibility, tolerability and change from baseline in: connected speech intelligibility, quality of language production, speech quality, functional speech intelligibility, phonological working memory, attentional and hyperactive behaviour.
Eligibility criteria
Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.
Inclusion criteria
Exclusion criteria
Eligibility
Age eligible for study : 6 and older
Healthy volunteers accepted : No
Gender eligible for study: All
Things to know
Study dates
Study start: 2022-03-14
Primary completion: 2025-07-01
Study completion finish: 2025-07-01
Study type
TREATMENT
Phase
PHASE2
Trial ID
NCT05185583
Intervention or treatment
DRUG: Methylphenidate Hydrochloride
DRUG: Placebo
Conditions
- • Childhood Apraxia of Speech
Find a site
Closest Location:
Murdoch Children's Research Institute
Research sites nearby
Select from list below to view details:
Murdoch Children's Research Institute
Parkville, Victoria, Australia
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Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
| Participant Group/Arm | Intervention/Treatment |
|---|---|
EXPERIMENTAL: Sequence A: Methylphenidate, Placebo
| DRUG: Methylphenidate Hydrochloride
|
EXPERIMENTAL: Sequence B: Placebo, Methylphenidate
| DRUG: Methylphenidate Hydrochloride
|
What is the study measuring?
Primary outcome
| Primary Outcome Measure | Primary Outcome Description | Primary Outcome Time Frame |
|---|---|---|
| Change From Baseline in Preschool Speech Intelligibility Measure Score at 4 weeks | 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. | Baseline and 4 weeks |
Secondary outcome
| Secondary Outcome Measure | Secondary Outcome Description | Secondary Outcome Time Frame |
|---|---|---|
| Change From Baseline in Assessment of the Intelligibility of Dysarthric Speech-II (ASSIDS-II) Score at 4 weeks | 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. | Baseline and 4 weeks |
| Number of children at screening who refuse, are eligible, or are ineligible (and reason). | The frequency of children at screening who refuse, are eligible, or are ineligible (and reason). | At study recruitment, up to 4 weeks before starting treatment. |
| Number of children who withdraw, discontinue, and/or experience 1 or more protocol violations. | 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. | During the 4 week treatment phase. |
| Adherence to dose regimen during each 4 week treatment period | 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. | 4 weeks |
| Parent/caregiver experience of tolerability and utility | 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. | Baseline and 4 weeks |
| Paediatric patient experience of tolerability and utility | 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. | Baseline and 4 weeks |
| Change From Baseline in total number of grammatical features (Language Assessment, Remediation and Screening Procedure; LARSP) at 4 weeks | 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. | Baseline and 4 weeks |
| Change From Baseline in total number of sentences (Language Assessment, Remediation and Screening Procedure; LARSP) at 4 weeks | 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. | Baseline and 4 weeks |
| Change From Baseline in mean number of sentences per turn (Language Assessment, Remediation and Screening Procedure; LARSP) at 4 weeks | 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. | Baseline and 4 weeks |
| Change From Baseline in mean sentence length (Language Assessment, Remediation and Screening Procedure; LARSP) at 4 weeks | 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. | Baseline and 4 weeks |
| Change From Baseline in stage of grammatical development (Language Assessment, Remediation and Screening Procedure; LARSP) at 4 weeks | 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. | Baseline and 4 weeks |
| Change From Baseline in types of clausal structures used (Language Assessment, Remediation and Screening Procedure; LARSP) at 4 weeks | 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. | Baseline and 4 weeks |
| Change From Baseline in syllable repetition at 4 weeks | 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. | Baseline and 4 weeks |
| Change From Baseline in Intelligibility in Context Scale Score at 4 weeks | 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. | Baseline and 4 weeks |
| Change From Baseline in Children's Non-word Repetition (CNRep) Test Score at 4 weeks | 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. | Baseline and 4 weeks |
| Change From Baseline in Swanson, Nolan, and Pelham (SNAP-IV) Parent 18-Item Rating Scale (SNAP-IV) Score at 4 weeks | 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. | Baseline and 4 weeks |
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