A Phase II Proof-of-concept Trial of Methylphenidate in Children With Apraxia of Speech: a Double-blind, Randomised, Placebo-controlled, Cross-over Trial
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 18
- 试验地点
- 1
- 主要终点
- Change From Baseline in Preschool Speech Intelligibility Measure Score at 4 weeks
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 6 Years 至 12 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
研究组 & 干预措施
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.
干预措施: Methylphenidate Hydrochloride (Drug)
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.
干预措施: Placebo (Drug)
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.
干预措施: Methylphenidate Hydrochloride (Drug)
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.
干预措施: Placebo (Drug)
结局指标
主要结局
Change From Baseline in Preschool Speech Intelligibility Measure Score at 4 weeks
时间窗: Baseline and 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.
次要结局
- Number of children at screening who refuse, are eligible, or are ineligible (and reason).(At study recruitment, up to 4 weeks before starting treatment.)
- Adherence to dose regimen during each 4 week treatment period(4 weeks)
- Parent/caregiver experience of tolerability and utility(Baseline and 4 weeks)
- Number of children who withdraw, discontinue, and/or experience 1 or more protocol violations.(During the 4 week treatment phase.)
- Change From Baseline in stage of grammatical development (Language Assessment, Remediation and Screening Procedure; LARSP) at 4 weeks(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(Baseline and 4 weeks)
- Change From Baseline in Assessment of the Intelligibility of Dysarthric Speech-II (ASSIDS-II) Score at 4 weeks(Baseline and 4 weeks)
- Paediatric patient experience of tolerability and utility(Baseline and 4 weeks)
- Change From Baseline in total number of grammatical features (Language Assessment, Remediation and Screening Procedure; LARSP) at 4 weeks(Baseline and 4 weeks)
- Change From Baseline in total number of sentences (Language Assessment, Remediation and Screening Procedure; LARSP) at 4 weeks(Baseline and 4 weeks)
- Change From Baseline in Intelligibility in Context Scale Score at 4 weeks(Baseline and 4 weeks)
- Change From Baseline in Children's Non-word Repetition (CNRep) Test Score at 4 weeks(Baseline and 4 weeks)
- Change From Baseline in mean number of sentences per turn (Language Assessment, Remediation and Screening Procedure; LARSP) at 4 weeks(Baseline and 4 weeks)
- Change From Baseline in mean sentence length (Language Assessment, Remediation and Screening Procedure; LARSP) at 4 weeks(Baseline and 4 weeks)
- Change From Baseline in syllable repetition at 4 weeks(Baseline and 4 weeks)
- Change From Baseline in types of clausal structures used (Language Assessment, Remediation and Screening Procedure; LARSP) at 4 weeks(Baseline and 4 weeks)