Clinical trial NCT05439616: Study of Cariprazine Oral Capsules or Solution to Assess Adverse Events and Change in Irritability Due to Autism Spectrum Disorder (ASD) in Participants Aged 5-17 Years With ASD
Official title: An 8-Week, Multicenter, Randomized, Double-Blind, Parallel-Group, Placebo-Controlled, Phase 3 Study of the Efficacy and Safety of Cariprazine in the Treatment of Pediatric Subjects (5 to 17 Years of Age) With Autism Spectrum Disorder
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Autism Spectrum Disorder
Keywords: Autism Spectrum Disorder
Cariprazine
VRAYLAR
AGN-241780
Lead sponsor: AbbVie
Sponsor class: INDUSTRY
First posted: 2022-06-30
Enrollment: 161
Sex: ALL
Minimum age (years): 5
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Change from Baseline in Aberrant Behavior Checklist, 2nd edition - Community Version - Irritability (ABC-I) Subscale Score
timeFrame: Baseline (Week 0) to Week 8
Secondary outcomes:
measure: Percentage of Participants with responder status of "Very Much Improved" or "Much Improved" on the Clinical Global Impression- Change Irritability (CGI-C Irritability) Scale
timeFrame: Week 8
measure: Change from Baseline in Parent-Rated Anxiety Scale for Youth with Autism Spectrum Disorder (PRAS-ASD) Total Score
timeFrame: Baseline (Week 0) to Week 8
measure: Change from Baseline in Caregiver Strain Questionnaire Short Form 7-Item (CGSQ SF-7) total score
timeFrame: Baseline (Week 0) to Week 8
Other outcomes:
Locations:
city: Anaheim
country: United States
facility: Advanced Research Center /ID# 245049
state: California
city: Imperial
country: United States
facility: Sun Valley Research Center /ID# 243133
state: California
city: Orange
country: United States
facility: CHOC Children's Hospital /ID# 245577
state: California
city: Upland
country: United States
facility: Pacific Clinical Research Management Group /ID# 243019
state: California
city: Doral
country: United States
facility: D&H Doral Research Center-Doral /ID# 255457
state: Florida
city: Gainesville
country: United States
facility: Sarkis Clinical Trials /ID# 242868
state: Florida
city: Homestead
country: United States
facility: Advanced Research Institute of Miami /ID# 243179
state: Florida
city: Lake Mary
country: United States
facility: Sandhill Research LLC /ID# 245079
state: Florida
city: Loxahatchee Groves
country: United States
facility: Axcess Medical Research /ID# 244952
state: Florida
city: Miami
country: United States
facility: G+C Research Group, LLC /ID# 252016
state: Florida
city: Orange City
country: United States
facility: Medical Research Group of Central Florida /ID# 243170
state: Florida
city: Orlando
country: United States
facility: K2 Medical Research - Orlando - South Orlando Avenue /ID# 248694
state: Florida
city: Orlando
country: United States
facility: APG Research, LLC /ID# 243130
state: Florida
city: Orlando
country: United States
facility: Nona Pediatric Center /ID# 245078
state: Florida
city: Spring Hill
country: United States
facility: Asclepes Research Centers - Spring Hill /ID# 248682
state: Florida
city: Tamarac
country: United States
facility: D&H Tamarac Research Center /ID# 250436
state: Florida
city: Atlanta
country: United States
facility: Atlanta Center for Medical Research /ID# 243124
state: Georgia
city: Dunwoody
country: United States
facility: Atlanta Behavioral Research, LLC /ID# 243082
state: Georgia
city: Savannah
country: United States
facility: iResearch Savannah /ID# 254582
state: Georgia
city: Lincoln
country: United States
facility: Alivation Research /ID# 242915
state: Nebraska
city: Jackson Heights
country: United States
facility: Dr. Hosneara, M.D. LLC /ID# 252129
state: New York
city: Staten Island
country: United States
facility: Richmond Behavioral Associates An Evolution Research Group Portfolio Company /ID# 259578
state: New York
city: Kinston
country: United States
facility: New Dawn Psychiatric Services PLLC /ID# 243697
state: North Carolina
city: Avon Lake
country: United States
facility: Quest Therapeutics of Avon Lake /ID# 252013
state: Ohio
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital /ID# 243968
state: Ohio
city: West Chester
country: United States
facility: CincyScience /ID# 242993
state: Ohio
city: Austin
country: United States
facility: BioBehavioral Research of Austin /ID# 255460
state: Texas
city: Dallas
country: United States
facility: Relaro Medical Trials /ID# 243126
state: Texas
city: Dallas
country: United States
facility: Cedar Health Research /ID# 248683
state: Texas
city: Plano
country: United States
facility: AIM Trials /ID# 243120
state: Texas
city: The Woodlands
country: United States
facility: Family Psychiatry of The Woodlands /ID# 242867
state: Texas
city: Ogden
country: United States
facility: Advanced Research Institute /ID# 243098
state: Utah
city: Bellevue
country: United States
facility: Northwest Clinical Research Center /ID# 243083
state: Washington
city: Everett
country: United States
facility: Core Clinical Research /ID# 243084
state: Washington
city: Caguas
country: Puerto Rico
facility: Dr. Samuel Sanchez PSC /ID# 245739
city: San Juan
country: Puerto Rico
facility: GCM Medical Group PSC /ID# 245735
Study officials:
affiliation: AbbVie
name: ABBVIE INC.
role: STUDY_DIRECTOR
References: