Autism Spectrum Disorder
Conditions
Keywords
Autism Spectrum Disorder, Cariprazine, VRAYLAR, AGN-241780
Brief summary
Autism spectrum disorder (ASD) consists of deficits in social, communication, and cognitive development, repetitive and stereotypic behaviors. Many ASD patients show notably high levels of irritability, including verbal and physical aggression, self injury, and/or property destruction. Autistic infants tend to avoid eye contact and show little interest in others. This study will assess how safe and effective cariprazine is in treating pediatric participants (5 to 17 years of age) with ASD. Adverse events and change in disease activity will be assessed. Cariprazine is an investigational drug being developed for the treatment of irritability due to ASD. This study is double-blinded means that neither the participants nor the study doctors will know who will be given cariprazine and who will be given placebo (does not contain treatment drug). Study doctors put the participants in 1 of the 2 groups, called treatment arms. Each group receives a different treatment. There is a 1 in 2 chance that participants will be assigned to placebo. Approximately 152 participants diagnosed with ASD will be enrolled in approximately 40 sites globally. Participants will receive oral capsules or oral solution of cariprazine or placebo once daily for 8-weeks and will undergo a 4-week safety follow-up period. There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, checking for side effects and completing questionnaires.
Interventions
DRUGCariprazine
Oral Capsules or Oral Solution
DRUGPlacebo
Oral capsules or Oral Solution
Sponsors
AbbVie
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
5 Years to 17 Years
Healthy volunteers
No
Inclusion criteria
* Participants at the time of screening must have a Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-5) diagnosis of ASD, confirmed by Kiddie Schedule for Affective Disorders and Schizophrenia-Present and Lifetime (K-SADS-PL) administered at screening (Visit 1) by a trained clinician. * Participants must have an Aberrant Behavior Checklist, 2nd edition - Community Version - Irritability (ABC-I) subscale score ≥ 18 (Visits 1 and 2). * Participants must have a Clinical Global Impressions - Severity - Irritability (CGI-S Irritability) score ≥ 4 at screening (Visits 1 and 2).
Exclusion criteria
* Participants with diagnosis of intellectual disability (Intelligence quotient \< 25). * No history of major depressive disorder, bipolar disorder, schizophrenia, schizoaffective disorder, schizophreniform disorder, brief psychotic disorder, or psychotic disorder due to another medical condition, as excluded by administration of Kiddie Schedule for Affective Disorders and Schizophrenia-Present and Lifetime (K-SADS-PL). * History of ASD that is associated with Rett Disorder, Fragile-X Syndrome, or Childhood Disintegrative Disorder.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change from Baseline in Aberrant Behavior Checklist, 2nd edition - Community Version - Irritability (ABC-I) Subscale Score | Baseline (Week 0) to Week 8 | The Aberrant Behavior Checklist (ABC) - Community version is a 58-item, caregiver-rated scale designed to measure inappropriate and maladaptive behavior of people with developmental disabilities (including intellectual disability and ASD). The ABC has 5 subscales (Irritability, Social Withdrawal, Stereotypic Behavior, Hyperactivity and Inappropriate Speech). The caregiver rates the child's behavior from 0=not at all a problem to 3= the problem is severe in degree. The range for Irritability is 0 to 45. Higher scores indicate greater severity. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants with responder status of Very Much Improved or Much Improved on the Clinical Global Impression- Change Irritability (CGI-C Irritability) Scale | Week 8 | The Clinical Global Impression - Change-Irritability (CGI-C Irritability) is a single-item rating scale that assesses change in the severity of a participant's irritability from the clinician's perspective. The CGI-C Irritability 7-point graded response scale (1 = Very much improved, 2 = Much improved, 3 = Minimally improved, 4 = No change, 5 = Minimally worse, 6 = Much worse, or 7 = Very much worse). |
| Change from Baseline in Parent-Rated Anxiety Scale for Youth with Autism Spectrum Disorder (PRAS-ASD) Total Score | Baseline (Week 0) to Week 8 | PRAS-ASD is a 25-item caregiver-reported measure of anxiety in participants aged 3 to 17 with ASD. All items use a 4-point response scale: 0 = None (not present); 1 = Mild (present sometimes, not a real problem); 2 = Moderate (often present and a problem); 3 = Severe (very frequent and a major problem). Total score ranges from 0 to 75, with higher scores indicating greater severity of anxiety. |
| Change from Baseline in Caregiver Strain Questionnaire Short Form 7-Item (CGSQ SF-7) total score | Baseline (Week 0) to Week 8 | CGSQ SF-7 is a caregiver-reported 7-item measure of self-reported strain that focuses on the impact of caring for a child with emotional and behavioral problems. Responses are scored on a 5-point scale (1 = Not at all, 2 = A little; 3 = Somewhat; 4 = Quite a bit; and 5 Very much) with higher scores indicating greater caregiver strain. |
Countries
Puerto Rico, United States
Outcome results
None listed