A Study to Evaluate Scales for Repetitive and Restricted Behaviors in Children, Adolescents, and Adults With Autism Spectrum Disorder (ASD)

NCT03611075 | Completed Phase 1 Results

• 2 other identifiers: BP403312019-003524-20
• Study Type: interventional
• Target: 144
• Locations: 3 countries
• Sites: 18

Brief Summary

This is a non-drug study seeking to characterize different scales to measure repetitive and restrictive behaviors in different ASD sub-populations over time. This study will also explore the use of digital biomarkers.

Conditions

Autism Spectrum Disorder

Outcome Measures

Primary Outcomes (7)

• Children's Yale-Brown Obsessive Compulsive Scale Modified for Autism Spectrum Disorder (CY-BOCS-ASD) - Total Score

  The CY-BOCS-ASD is where the interviewer asks the parent/caregiver about past and present repetitive behaviors, guided by the revised repetitive behavior checklist with a list of 25 behaviors, classified into the following categories: Hoarding/ritualistic behavior, Sensorymotor and arranging, Insistence on routines /Self-injurious behaviors, Stereotypy and Restricted interests. This checklist was expanded to include repetitive behaviors commonly seen in children with ASD. A target symptom list comprising the four most troublesome behaviors is established by the interviewer. The severity (0 to 4) of each target behavior is rated for the following five items: time spent, interference, distress, resistance and degree of control to generate a total score (0-20). The ratings evaluate the symptom severity over the past week and are based on the information collected from the child and parent/caregiver during the interview. Values at visits are reported.

  Baseline, Weeks 2 and 12

• Montefiore Einstein Rigidity Scale (MERS-R)

  The MERS-R was to assess 3 domains of rigid behavior in children and adults. It's a clinician-administered scale in which the clinician uses all available information to rate. Each domains is assessed separately, during examples are discussed and related behaviors are probed for more individualized exemplars. A list of relevant behaviors is compiled for each domain, and ratings for the items are based on the average occurrence of the behaviors over the past one week. Total values at visits are reported and domains are assessed: ASD: 1. Behavioral Rigidity (e.g., insistence on sameness, things must be done in his/her way) 2. Cognitive Rigidity (e.g., inflexible adherence to rules) 3. Protest (in response to deviation from rigidity; e.g., verbal objection, tantrum, physical aggression). These domain scores can range from 0 (no problems) to 4 (severe problems). The total score represents the sum of the domain scores ranging from 0-48, where higher scores indicate more severe problems.

  Baseline, Weeks 2 and 12

• Repetitive Behavior Scale-Revised (RBS-R)

  The Repetitive Behavior Scale-Revised (RBS-R) is a 43-item caregiver-report questionnaire to measure breadth of repetitive behaviors in children, adolescents, and adults with ASD. It provides a quantitative measure of the full spectrum of repetitive behaviors of 6 subscales: Stereotype, Self-injurious, Compulsive, Ritualistic, Sameness and Restricted Behaviors. Caregivers are asked to read a list of behaviors and choose for each item a score that best describes how much of a problem the behavior has been over the last month. Behaviors are rated on a 4-point scale: 0-Behavior does not occur, 1-Behavior occurs and is a mild problem, 2-Behavior occurs and is a moderate problem, 3-Behavior occurs and is a severe problem. The total score is the sum of all 43 items and can range from 0 to 129. Higher scores indicate more severe problems with repetitive behaviors. The sums of score values at visits are reported.

  Baseline, Weeks 2 and 12

• Repetitive Behavior Questionnaire for Children (RBQ-2)

  The RBQ-2 is a 20 items questionnaire to assess the spectrum of restricted and repetitive behaviors observed in individuals with ASD such as repetitive motor movements, rigidity/adherence to routine, preoccupation with restricted patterns of interest and unusual sensory Interest (Leekman et al 2007; Honey et al 2012). The RBQ-2 exists in two different versions, a caregiver reported version for pediatric subjects RBQ-2 and a participant-reported version for adults RBQ-2A (Barrett et al 2015). The RBQ-2, the caregiver version is used for this reported data set. To ensure for this study a comparable the mean total score is calculated as the sum of the items, total scores at visits are reported. For the RBQ-2, the possible total score can range between 20-60, with higher scores indicating more frequent RRB.

  Baseline, Weeks 2 and 12

• Restricted Behavior Questionnaire for Adults (RBQ-2A)

  The RBQ-2 is a 20 items questionnaire to assess the spectrum of restricted and repetitive behaviorsobserved in individuals with ASD such as repetitive motor movements, rigidity/adherence to routine, preoccupation with restricted patterns of interest and unusual sensory Interest (Leekman et al 2007; Honey et al 2012). The RBQ-2 exists in two different versions, a caregiver reported version for pediatric subjects RBQ-2 and a participant-reported version for adults RBQ-2A (Barrett et al 2015). The RBQ-2, the caregiver version is used for this reported data set. Total score is calculated as the sum of the items, total scores at visits are reported. For the RBQ-2A, the possible total score can range between 20 to 60, with higher scores indicating more frequent and severe RRB.

  Baseline, Weeks 2 and 12

• Childhood Routines Inventory-Revised (CRI-R)

  The CRI-R (for children and adolescents) and ARI (for adults) were developed together and are closely related scales (Evans et al 2017).The CRI-R and ARI questionnaires capture a wide range of restricted and repetitive behaviors, including stereotypies, tics, compulsions, habits, sensory sensitivities, and focused interests, in the context of typical and atypical development in children, adolescents and adults across the entire lifespan. All items will be answered on a five-point Likert scale from not at all/ never, a little/rarely, somewhat/ sometimes, quite a lot/ often, and very much / always. The caregiver-completed CRI-R scale for pediatric subjects includes 62 items. Total score is calculated as the sum of the items, total scores at visits are reported. For the CRI-R, the possible total score can range between 62 - 310, with higher scores indicating more frequent RRB.

  Baseline, Weeks 2 and 12

• Adult Routines Inventory (ARI)

  The CRI-R (for children and Adolescents) and ARI (for adults) were developed together and are closely related scales (Evans et al 2017).The CRI-R and ARI questionnaires capture a wide range of RRBs, including stereotypies, tics, compulsions, habits, sensory sensitivities, and focused interests, in the context of typical and atypical development in children, adolescents and adults across the entire lifespan. All items will be answered on a five-point Likert scale from not at all/ never, a little/rarely, somewhat/ sometimes, quite a lot/ often, and very much / always. The caregiver-completed CRI-R scale for pediatric subjects includes 62 items. The participant-reported ARI scale for adult subjects includes 55 items and will be completed by the participant. Total score is calculated as the sum of the items, mean total scores at visits are reported. For the ARI, the possible total score can range between 55-275, with higher scores indicating more frequent RRB.

  Baseline, Weeks 2 and 12

Secondary Outcomes (16)

• Clinical Global Impression Scales (CGI)

  Baseline, Weeks 2 and 12

• Child's Sleep Habits Questionnaire (CSHQ)

  Baseline, Weeks 2 and 12

• Pittsburg Sleep Quality Index (PSQI)

  Baseline, Weeks 2 and 12

• Reading the Mind in the Eyes Test -R (RMET-R)

  Baseline, Week 12

• Reading the Mind in the Eyes Test Child -C (RMET-C)

  Baseline, Week 12

Study Arms (9)

Low-Functioning Autism Spectrum Disorder (ASD) Children

EXPERIMENTAL

Participants will be 5-12 years old, with Intelligence Quotient (IQ) scores between 50-70

Other: No Intervention

High-Functioning ASD Children

EXPERIMENTAL

Participants will be 5-12 years old, with IQ scores of 70 or above

Other: No Intervention

Low-Functioning ASD Adolescents

EXPERIMENTAL

Participants will be 13-17 years old, with IQ scores between 50-70

Other: No Intervention

High-Functioning ASD Adolescents

EXPERIMENTAL

Participants will be 13-17 years old, with IQ scores of 70 or above

Other: No Intervention

Low-Functioning ASD Adults

EXPERIMENTAL

Participants will be 18-45 years old, with IQ scores between 50-70

Other: No Intervention

High-Functioning ASD Adults

EXPERIMENTAL

Participants will be 18-45 years old, with IQ scores of 70 or above

Other: No Intervention

Typically Developing (TD) Healthy Participant Children

NO INTERVENTION

Participants will be 5-12 years old

TD Healthy Participant Adolescents

NO INTERVENTION

Participants will be 13-17 years old

TD Healthy Participant Adults

NO INTERVENTION

Participants will be 18-45 years old

Interventions

No Intervention OTHER

No interventions are administered in this study

High-Functioning ASD Adolescents; High-Functioning ASD Adults; High-Functioning ASD Children; Low-Functioning ASD Adolescents; Low-Functioning ASD Adults; Low-Functioning Autism Spectrum Disorder (ASD) Children

Eligibility Criteria

• Age: 5 Years - 45 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Males and females
• Availability of a parent or other reliable caregiver. The same person must agree to accompany the participant to all clinic visits and provide information about the participant's behavior and symptoms
• Age: 5-45 years
• Diagnosis of ASD based on the Diagnostic and Statistical Manual of Mental Disorders (DSM-5), and the Autism Diagnostic Observation Schedule (ADOS-2).
• Children's Yale-Brown Obsessive Compulsive Scale modified for ASD (CY-BOCS-ASD) total score of at least 12
• Clinical Global Impression-Severity (CGI-S) scale of at least 4 about participant's current autism severity
• Intelligence quotient (IQ) score of 50 or above as assessed by the Abbreviated Intelligence Quotient (ABIQ) SB5 scale
• All medications and treatments are expected to be stable for the duration of the study
• TD participants aged 5-45 years

You may not qualify if:

• Participation in an in investigational drug or device study within 4 weeks or 5 times the half-life of the investigational molecule prior to screening, and participant is not expected to enroll in any other trial during the study
• Co-occurring disease, condition, or treatment that might interfere with the conduct of the study or pose an unacceptable risk to the participant
• Unstable or uncontrolled clinically significant psychiatric and/or neurological disorder that may interfere with study objectives
• TD healthy participants with a first-degree relative with ASD

Sponsors & Collaborators

• Hoffmann-La Roche: lead

Study Sites (18)

Harmonex Neuroscience Research

Dothan, Alabama, 36303, United States

Location

Southwest Autism Research and Resource Center

Phoenix, Arizona, 85006, United States

Location

PCSD Feighner Research

San Diego, California, 92108, United States

Location

Stanford University School of Medicine

Stanford, California, 94305, United States

Location

Clinical Neuroscience Solutions Inc; Jacksonville Clinic

Jacksonville, Florida, 32256, United States

Location

University of Minnesota; Clin. Neuro Research Unit

Minneapolis, Minnesota, 55414, United States

Location

Nathan S. Kline Institute for Psychiatric Research

Orangeburg, New York, 10962, United States

Location

Albert Einstein College of Medicine

The Bronx, New York, 10461, United States

Location

University Hospitals Case Medical Center

Cleveland, Ohio, 44106, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

BioBehavioral Research of Austin, PC

Austin, Texas, 78759, United States

Location

Red Oak Psychiatry Associates, PA

Houston, Texas, 77090, United States

Location

Road Runner Research Ltd

San Antonio, Texas, 78258, United States

Location

Northwest Clinical Research Center - ClinEdge - PPDS

Bellevue, Washington, 98007, United States

Location

Seattle Children's Hospital - PIN; Investigational Drug Services

Seattle, Washington, 98105, United States

Location

Holland Bloorview Kids Rehabilitation Hospital; Autism Research Centre

East York, Ontario, M4G 1R8, Canada

Location

Gartnavel Royal Hospital; Mental Health & Wellbeing

Glasgow, G12 0XH, United Kingdom

Location

Kings College London

London, WC2R 2LS, United Kingdom

Location

MeSH Terms

Conditions

Autism Spectrum Disorder

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Results Point of Contact

• Title: Medical Communications
• Organization: Hoffmann-La Roche

global-roche-genentech-trials@gene.com

800-821-8590

Study Officials

• Clinical Trials

  Hoffmann-La Roche

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: OTHER
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 11, 2018
• First Posted: August 2, 2018
• Study Start: August 13, 2018
• Primary Completion: May 21, 2020
• Study Completion: May 21, 2020
• Last Updated: June 21, 2022
• Results First Posted: July 23, 2021

Record last verified: 2022-06

Locations

US (15); GB (2); CA (1)