A Study of the Effectiveness and Safety of Risperidone Versus Placebo in the Treatment of Children With Autistic Disorder and Other Pervasive Developmental Disorders (PDD)
Condition: DCild Development Disorders, Pervasive; Autistic Disorder; Developmental Disabilities; Asperger Syndrome; Rett Syndrome
MeSH terms: Rett Syndrome; Syndrome; Autistic Disorder; Developmental Disabilities; Autism Spectrum Disorder; Asperger Syndrome
Study type: Interventional
Phase: Phase 3

Summary: 
      The purpose of the study is to evaluate the effectiveness of an oral solution of risperidone
      (an antipsychotic medication) versus placebo in the treatment of behavioral symptoms in
      children with Pervasive Developmental Disorders (PDD).
    

Detailed description: 
      The Pervasive Developmental Disorders (PDD) are a group of neuropsychiatric disorders,
      including autistic disorder, characterized by specific delays and deviance in social,
      communicative, and cognitive development with an onset typically in the first years in life.
      Children with PDD may show multiple, serious symptoms including hyperactivity, inattention,
      impulsive and aggressive behavior, repetitive movements or speech patterns, sleep disorders,
      screaming, and self-injurious behavior. Drug studies to date have suggested that different
      behavioral symptoms of PDDs respond to neuroleptics, such as risperidone. This is a
      randomized, double-blind, placebo-controlled study to evaluate the effectiveness of
      risperidone (0.02 to 0.06 mg/kg/day) compared with placebo in the treatment of behavioral
      symptoms in children 5 to 12 years of age with Pervasive Developmental Disorders (PDD).
      Patients receive study medication (risperidone or placebo) to be taken orally once a day at
      gradually increasing doses up to a maximum of 0.06 mg/kg, adjusted at weekly intervals to
      achieve optimal effectiveness while minimizing any intolerance to the drug. Treatment
      continues at the optimal dose through Week 8. A parent or caregiver evaluates the child's
      behavior and symptoms at scheduled office visits during the course of treatment. The primary
      measure of effectiveness is the change in the Irritability Subscale of the Aberrant Behavior
      Checklist (ABC) and other ABC subscales at end of treatment compared with baseline.
      Additional assessments of effectiveness include: the Nisonger Child Behavior Rating Form
      (N-CBRF); the Visual Analogue Scale (VAS), a measure of the patient's most disturbing
      symptom; and the Clinical Global Impression (CGI) of the overall severity of the disorder.
      Safety assessments include the incidence of adverse events throughout the study; measurement
      of vital signs (pulse, temperature, blood pressure), body weight, and evaluation of the
      presence and severity of extrapyramidal symptoms by the Extrapyramidal Symptom Rating Scale
      (ESRS) at specified intervals; clinical laboratory tests (hematology, biochemistry,
      urinalysis) before study initiation and at the end of treatment. The study hypothesis is that
      risperidone is more effective than placebo for the treatment of behavioral symptoms in
      children aged 5 to 12 years with Pervasive Developmental Disorders (PDD). Risperidone oral
      solution 1 mg/mL or placebo, taken orally, once daily. Days 1 - 2, dose is 0.01 mg/kg body
      weight. Days 3 - 7 dose is 0.02 mg/kg, increasing on Days 8 - 14 to 0.04 mg/kg (maximum), and
      0.06 mg/kg (maximum) through 8 weeks. Dosage may be adjusted at investigator's discretion.
    

Intervention name(s): risperidone
Intervention type(s): Drug
Intervention MeSH terms: Risperidone
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double
Primary purpose: Treatment
Keywords: Risperidone; child development disorders; pervasive development disorders; autistic disorder; autism; developmental disabilities; antipsychotropic agents