Autism Spectrum Disorder
Conditions
Brief summary
Paediatric Autism Communication Therapy (PACT) is a naturalistic developmental behavioural interventions to reduce autism symptoms. The aim of this trial is to assess the beneficial and harmful effects of PACT in 2-6 year-old children with a recent diagnosis of autism spectrum disorder.
Detailed description
Autism spectrum disorder (ASD) is a neurodevelopmental disorder affecting approximately 2% of children and young people worldwide. ASD is considered a lifelong disorder and interventions significantly reducing the core autistic symptoms have been sparse. Paediatric Autism Communication Therapy (PACT) is among the first naturalistic developmental behavioural interventions to show promising results for reduction in autism symptoms. The aim of this trial is to assess the beneficial and harmful effects of PACT in 2-6 year-old children with a recent diagnosis of ASD. This trial is an investigator-initiated, independently funded, pragmatic, national, parallel group, superiority, randomised clinical trial comparing PACT combined with management as usual to management as usual alone.
Interventions
BEHAVIORALPACT
Paediatric Autism Communication Therapy
BEHAVIORALMAU
Management as usual
Sponsors
Copenhagen Trial Unit, Center for Clinical Intervention Research
Region Capital Denmark
Region Zealand
Region of Southern Denmark
Central Denmark Region
Regionshospital Nordjylland
University of Southern Denmark
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Investigator, Outcomes Assessor)
Masking description
Due to the nature of the intervention, blinding of parents and therapists is impossible. The primary outcome measure, Autism Diagnostic Observation Schedule, Calibrated Severity Score (ADOS-CSS), and further outcomes (Brief Observation of Social Communication Change (BOSCC), Dyadic Communication Measure for Autism (DCMA) and Measure of Naturalistic Developmental Behavioral Intervention Strategy Implementation - Caregiver Change (MONSI-CC)) are videotaped, leaving raters blinded to treatment allocation. At the clinical follow-up assessment, families are asked not to reveal group allocation. Data from the DAN-PACT trial will be kept separate from research data and will not be accessible to the research team. We will follow the rule that statistical analyses are conducted with the intervention groups coded. The steering committee will write two abstracts while the blinding is intact assuming either group as intervention group and control, respectively. After this, the code will be broken
Eligibility
Sex/Gender
ALL
Age
2 Years to 6 Years
Healthy volunteers
No
Inclusion criteria
* Children aged 2 through 6 years (both inclusive) * A diagnosis of ASD meeting the criteria for International Classification of Diseases; Tenth Edition (ICD-10): Diagnose codes: DF84.0, DF84.1, DF84.5, DF84.8 * An Autism Diagnostic Observation Schedule, 2nd Edition Calibrated Severity Score (ADOS-2 CSS) ≥ 4 * The ASD diagnosis must be verified by the Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-5), Autism Spectrum Disorder Checklist * The ASD diagnosis must be the primary developmental disorder (comorbid conditions allowed) and conferred at a clinical conference * Parents must have sufficient Danish (or English) language skills to communicate with the therapist * Signed informed consent by parents or holders of legal guardianship
Exclusion criteria
* Children having a sibling already included in the trial * Hearing and visual impairment in child or parent * Parent not available for regular sessions with the therapist, evaluated at the consent meeting
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Autism Symptoms | End of intervention (14 months) | Autism symptoms are measured by Diagnostic Observation schedule, 2nd Edition, Calibrated Severity Score (ADOS-2 CSS). Range 0-10, with 10 indicating the highest symptom severity. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Quality of Life (child) | End of intervention (14 months) | Parents' assessment of their child's quality of life with Pediatric Quality of Life Inventory (PEDSQL), a questionnaire including 23 items covering four dimensions of health related quality of life. Each item is rated between 0 and 100. A higher score indicates a higher level of health-related quality of life. The mean item score is used as outcome. |
| Child adaptive functioning | End of intervention (14 months) | Child adaptive functioning, or personal and social skills needed for everyday living will be measured by the Vineland Adaptive Behavior Scales, 3rd Edition (VABS-3), an online questionnaire filled in by parents. Each of four domains, Communication, Daily Living Skills, Socialization and Motor Skills are divided into 3 sub-domains. Each item is scored on a Likert-type format with scores 0 (never), 1(sometimes), and 2 (usually or often). Individual items scores are summed to a raw composite score and converted into a standard scale scores. Higher scores indicate higher functioning. |
| Social interaction of the child | At 6 month and end of intervention (14 months) | Brief Observation of Social Communication Change (BOSCC) is rated on a 12-minute videotaped parent-child play-session. Each of the 15 behavioural items is coded using an empirically based decision tree that captures information regarding the behaviour including frequency and quality. Each item is rated on a 6- point scale from 0 (abnormality is not present) to 5 (abnormality is present and significantly impairs functioning). For measuring change only item 1-12 (range 0-60) are used. |
| Quality of life (parents) | End of intervention (14 months) | Parent quality of life is measured by the questionnaire World Health Organization Quality of Life assessment- BREF (WHOQOL-BREF), including 28 items of quality of life for parents in 4 dimensions: Physiological, emotional, social and context. The item scores range from 1 to 5 with higher scores denoting higher quality of life. As the number of items differs for each subscale, the mean item score is used as outcome. |
Other
| Measure | Time frame | Description |
|---|---|---|
| Parental reflective functioning | End of intervention (12 months) | Parental reflective functioning will be measured by the Parental Reflective Functioning Questionnaire (PRFQ-1) with 18 items all rated on a 7-point scale with (7 if you strongly agree; and 1 if you strongly disagree. The midpoint, if you are neutral or undecided, is 4). High score indicate highly or hyper- reflective |
| Child attachment | End of intervention (14 months) | Parents' perception of their child's attachment is rated on the Maternal Perception of Child Attachment (MPCA). This parent-report measure consists of 23 items rated on a 5-point rating scale, ranging from frequently (1) to never (5). High scores indicate maternal perceptions of strong child attachment. |
| Adverse events | End of intervention (14 months) | Adverse events that occur during the study will be measured by the Negative Effects Questionnaire (NEQ). The NEQ questionnaire measures six factors; symptoms, quality, dependency, stigma, hopelessness, and failure. The self-report measure consists of three parts. First, respondents endorse specific items in case they have occurred or not during treatment, yes/no (1/0). Second, the respondents rate how negatively the negative effect was on four-point Likert-scale, ranging from Not at all to Extremely (0-4). Third, the respondents attribute the negative effect to The treatment I received yes/no (1/0). |
| Serious Adverse Events | End of intervention (14 months) | Serious Adverse Events (SAE) defined as any adverse event (AE) that results in death, is life-threatening, requires hospitalisation or prolongation of existing hospitalisation, or results in persistent or significant disability or incapacity is registered continuously through patient files |
| Parent-child synchrony and initiative in communication | At 6 month and end of intervention (14 months) | Dyadic Communication Measure for Autism (DCMA) is used to rate parent synchrony and responsiveness to the child, child communicative initiations, responses and communicative functions, and amount of mutual shared attention between parent and child will be measured by the DCMA. DCMA is rated on a 12-minute videotaped parent-child play-session. Raters will be blinded to treatment allocation. |
| Parents' perceptions of the intervention | End of intervention (14 months) | Parents' perceptions of the intervention will be explored in a subsample of parents in each intervention group by qualitative interviews |
| Receptive language | End of intervention (14 months) | Child receptive language as measured by the Mullens Scales of Early Learning (MSEL). The MSEL consists of four cognitive scales: Visual Reception, Fine Motor, Receptive Language, Expressive Language, as well as a Gross Motor Scale, and can be administered to infants and children up to 5 years, 6 months of age. The MSEL receptive language raw scores will be used for the total study population in order to use the same instrument for all children, even when some of them may be older than the validated target group. |
| Language | End of intervention (14 months) | Child language as measured by the New Reynell Developmental Language Scale (NRDLS). The NRDLS covers a number of important aspects of language acquisition including vocabulary, sentence structure, verb morphology, inference, and grammaticality judgement. NRDLS is developed for children 2-7 years of age. The NRDLS raw scores will be used for the total study population in order to use the same instrument for all children, even when some of them may older than the validated target group at follow-up. |
| Cost-effectiveness | End of intervention (14 months) | The costs of delivering PACT combined with MAU compared to MAU alone will be estimated through cost-effectiveness analyses of intervention deliveries and parents' productivity loss |
| Parent implementation of strategy | End of intervention (14 months) | Caregivers' implementation of PACT strategies will be measured by the Measure of Naturalistic Developmental Behavioral Intervention Strategy Implementation - Caregiver Change (MONSI-CC) rated on a 12-minute videotaped parent-child play-session. Each of 21 items are rated between 1 (strategy was rarely implemented) and 5 (strategies were well-timed) and summed on different subscales. Raters will be blinded to treatment allocation. |
| Behavioural and emotional problems | End of intervention (14 months) | Child symptoms and problems are measured by the Child Behavior Checklist for ages 1½-5 (CBCL/1½-5). A 99 item questionnaire generating a Total problem Score (range 0-198). Higher score indicating more problems |
| Family life functioning | End of intervention (14 months) | Participant family life functioning will be measured by the Autism Family Experience Questionnaire (AFEQ). A parent-reported questionnaire with 48 items (range 48-240). High score indication low function. |
| Social impairment | End of intervention (14 months) | The presence and severity of social impairment will be measured by the Social Responsiveness Scale, 2nd Edition (SRS-2). A parent reported questionnaire with 65 items identifying the presence and severity of social impairment. Each item is scored on a 4 point Likert-scale: 1 (not true); 2 (sometimes true); 3 (often true); and 4 (almost always true). Scores are obtained for five Treatment Subscales: Social Awareness; Social Cognition; Social Communication; Social Motivation; and Restricted Interests and Repetitive Behavior. Higher scores indicating more severe impairment. |
Countries
Denmark
Contacts
Primary ContactNiels Bilenberg, Professor
Outcome results
None listed