Clinical trial NCT02069977: Study to Evaluate the Efficacy and Safety of Aripiprazole
Official title: A Multinational, Multicenter, Open-label, Single-arm Study to Evaluate the Efficacy and Safety of Aripiprazole in Asian Pediatric Patients With Autism Spectrum Disorder and Behavior Problems
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE4
Conditions: Autism
Keywords: Autism
Aripiprazole
Abilify
Lead sponsor: Samsung Medical Center
Sponsor class: OTHER
Collaborators: name: Korea Otsuka International Asia Arab
First posted: 2014-02-24
Enrollment: 79
Sex: ALL
Minimum age (years): 6
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: * To evaluate the efficacy of flexibly dosed aripiprazole in reducing serious behavioral problems in Asian children and adolescents with a diagnosis of autism spectrum disorder, as measured by change from baseline to endpoint on the irritability subscale of the aberrant behavior checklist (ABC-I).
* To evaluate the long-term safety and tolerability of flexibly dosed aripiprazole in the treatment of serious behavioral problems in Asian children and adolescents with a diagnosis of autism spectrum disorder.

Detailed description: 1. Study design

- Multi-national, Multi-center, 52 weeks open label, single arm design

Eligibility criteria: Inclusion Criteria:

* 6 to 17 years of age.
* Meeting the diagnostic criteria for autistic disorder specified by the Diagnostic and Statistical Manual of Mental Disorders, Fourth Edition, Text Revision (DSM-IV-TR), with a diagnosis corroborated by the Autism Diagnostic Interview-Revised (ADI-R) diagnostic instrument and demonstrated behaviors such as tantrums, aggression, self-injurious behavior, or a combination of these.
* Clinical Global Impressions (CGI) Severity subscale score ≥ 4 at screening and baseline.
* Aberrant Behavior Checklist (ABC) Irritability subscale score ≥18 at screening and baseline.
* Mental age ≥ 18 months.
* Women of childbearing potential (WOCBP) have to use an adequate method of contraception
* WOCBP must have had a negative serum or urine pregnancy test.
* The patient and/or the designated guardian(s) or caregiver(s) who are able to comprehend and comply with the protocol requirements, in the opinion of the investigator and have consented to participate by signing an informed consent form.

Exclusion Criteria:

* Current diagnosis of bipolar disorder, schizophrenia, major depressive disorder, Rett's disorder, or Fragile-X syndrome.
* History of neuroleptic malignant syndrome.
* Significant risk of committing suicide based on history or routine psychiatric status examination.
* History of seizure in the past 1 year.
* History of severe head trauma or stroke
* History or current evidence of any unstable medical conditions
* Patient considered treatment resistant to neuroleptic medication
* Patient considered treatment resistant to aripiprazole
* Woman who is pregnant or breastfeeding
* ECG: QTc \> 475 msec
* Platelets ≤ 75,000/μL
* Hemoglobin ≤ 9 g/dL
* Neutrophils ≤ 1.0x10\^3/μL
* Aspartate or alanine transaminase (AST or ALT) \> 3xULN
* Serum creatinine ≥ 2 mg/dL
* Patient weighed \< 15 kg
* Patient who participated in any other clinical trial within 4 weeks
* Patient determined to require the administration of the prohibited medications during the study period
* Patient with other conditions determined by the investigator to be inappropriate for this clinical study

Primary outcomes:
measure: irritability subscale of the aberrant behavior checklist
timeFrame: Week 12
Secondary outcomes:
measure: Change from baseline in ABC-subscale of lethargy/social withdrawal, stereotypic behavior, hyperactivity, inappropriate speech
timeFrame: Week 1, 2, 4, 8, 12, 24, 36, 52
measure: Change of CGI-S, CGI-I from baseline
timeFrame: Week 1, 2, 4, 8, 12, 24, 36, 52
measure: Change of Behavioral problems from baseline
timeFrame: Week 12, 52
measure: Change of Adaptive Skills from baseline
timeFrame: week 12, 52
measure: Change of Parental stress from baseline
timeFrame: Weekk 12, 52
Other outcomes:
measure: Adverse events (AEs)
timeFrame: Week 1, 2, 4, 8, 12, 24, 36, 52
Locations:
city: Ilsan
country: Korea, Republic of
facility: Inje University Ilsan Paik Hospital
state: Gyeonggi-do
city: Seoul
country: Korea, Republic of
facility: Samsung Medical Center
city: Seoul
country: Korea, Republic of
facility: Asan Medical Center
city: Pasig
country: Philippines
facility: Medical City
city: Quezon City
country: Philippines
facility: Philippine Children's Medical Center
city: Bangkok
country: Thailand
facility: Siriraj Hospital Mahidol University
Study officials:
affiliation: Sumsung Medical Center
name: Yoo-Sook Joung, MD. PhD.
role: PRINCIPAL_INVESTIGATOR
References:
citation: Kim BU, Kim HW, Park EJ, Kim JH, Boon-Yasidhi V, Tarugsa J, Reyes A, Manalo SG, Joung YS. Long-Term Improvement and Safety of Aripiprazole for Irritability and Adaptive Function in Asian Children and Adolescents with Autistic Disorder: A 52-Week, Multinational, Multicenter, Open-Label Study. J Child Adolesc Psychopharmacol. 2022 Sep;32(7):390-399. doi: 10.1089/cap.2022.0004.