Clinical trial NCT01238575: Guanfacine for the Treatment of Hyperactivity in Pervasive Developmental Disorder
Official title: Guanfacine for the Treatment of Hyperactivity in Pervasive Developmental Disorder
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Pervasive Development Disorders
Keywords: Pervasive Developmental Disorders
PDD
PDD-NOS
Autistic Disorder
Autism
Asperger's Disorder
Hyperactivity
Attention Deficit Hyperactivity Disorder
ADHD
Medication
Drug
Guanfacine
Intuniv
Placebo
Methylphenidate
Lead sponsor: Yale University
Sponsor class: OTHER
Collaborators: name: Emory University
name: Massachusetts General Hospital
name: Seattle Children's Hospital
name: University of California, Los Angeles
name: National Institute of Mental Health (NIMH)
First posted: 2010-11-10
Enrollment: 62
Sex: ALL
Minimum age (years): 5
Maximum age (years): 14
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to determine whether guanfacine (trade name Intuniv) by itself or in combination with methylphenidate (also known as Ritalin) is helpful for treating hyperactivity in children and adolescents with a Pervasive Developmental Disorders (PDDs).

Detailed description: Pervasive Developmental Disorders (PDDs) are a group of conditions that includes Autistic Disorder, Asperger's disorder and so called Pervasive Developmental Disorder - Not Otherwise Specified. Children with PDD show delays in speech and language and reduced social interaction. Some children with PDD have also have problems with overactivity, impulsiveness and distractability. These behaviors are seen in children with Attention Deficit Hyperactivity Disorder (ADHD). Extended release guanfacine (Intuniv) is FDA-approved for the treatment of children with ADHD. The purpose of this study is to evaluate whether Intuniv is an effective treatment for ADHD symptoms in children with PDD.

This study has four parts: an 8-week double-blind trial, an 8-week blinded extension phase (for positive responders only), an 8-week open-label trial, and a 4-week add-on study. Following confirmation of eligibility, participants will be randomly assigned to receive either guanfacine or placebo in the 8-week double-blind trial. Children who show improvement after 8 weeks of treatment will continue on their assigned treatment for an additional 8 weeks (blinded extension phase). Children who show partial improvement with guanfacine will be offered 4 weeks of treatment with guanfacine plus methylphenidate (add-on study). Children who show no improvement on placebo will be offered 8 weeks of treatment with guanfacine (open-label trial). Children who show no improvement on guanfacine will exit the study.

Side effects and treatment response will be assessed at regularly scheduled visits.

The study protocol was formally revised with the Yale University IRB in May 2013 to address an early close to enrollment due to a reduction in funding. The original anticipated enrollment numbers of 112 subjects was reduced to 60 subjects. The study statistician was consulted prior to enrollment closure to address any issues related to statistical power and the adjustments made to the final statiscal analysis plan.

Eligibility criteria: Inclusion Criteria:

* Diagnosis of PDD (PDD-NOS, Asperger's Disorder, Autistic Disorder)
* Hyperactivity
* Between ages 5 years 0 months and 13 years 11 months.
* Weight \>/= 15 kg (33 lb)
* A mental age of at least 18 months

Exclusion Criteria:

* Prior failed treatment with an adequate trial of guanfacine in the last 2 years
* Concurrent treatment with another psychoactive medication

Primary outcomes:
measure: Aberrant Behavior Checklist Hyperactivity Subscale
timeFrame: Week 8
Secondary outcomes:
measure: ADHD Rating Scale - Total
timeFrame: Week 8
measure: Aberrant Behavior Checklist Irritability Subscale
timeFrame: 8 weeks
measure: Aberrant Behavior Checklist Social Withdrawal Subscale
timeFrame: 8 weeks
measure: Aberrant Behavior Checklist Sterotypy Subscale
timeFrame: 8 weeks
measure: Aberrant Behavior Checklist Inappropriate Speech Subscale
timeFrame: 8 weeks
measure: ADHD Rating Scale - Inattention Subscale
timeFrame: 8 weeks
measure: ADHD Rating Scale - Hyperactivity Subscale
timeFrame: 8 weeks
measure: Aberrant Behavior Checklist Hyperactivity Subscale
timeFrame: Baseline
measure: Aberrant Behavior Checklist Irritability Subscale
timeFrame: Baseline
measure: Aberrant Behavior Checklist Social Withdrawal Subscale
timeFrame: Baseline
measure: Aberrant Behavior Checklist Sterotypy Subscale
timeFrame: Baseline
measure: Aberrant Behavior Checklist Inappropriate Speech Subscale
timeFrame: Baseline
measure: ADHD Rating Scale - Inattention Subscale
timeFrame: Baseline
measure: ADHD Rating Scale - Hyperactivity Subscale
timeFrame: Baseline
measure: ADHD Rating Scale - Total
timeFrame: Baseline
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: University of California, Los Angeles
state: California
city: New Haven
country: United States
facility: Yale University
state: Connecticut
city: Atlanta
country: United States
facility: Emory University
state: Georgia
city: Lexington
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
Study officials:
affiliation: Emory University
name: Lawrence Scahill, MSN, PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: University of California, Los Angeles
name: James McCracken, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Seattle Children's Hospital
name: Bryan King, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Massachusetts General Hospital
name: Christopher McDougle, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Yale University
name: James Dziura, MPH, PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Scahill L, Aman MG, McDougle CJ, McCracken JT, Tierney E, Dziura J, Arnold LE, Posey D, Young C, Shah B, Ghuman J, Ritz L, Vitiello B. A prospective open trial of guanfacine in children with pervasive developmental disorders. J Child Adolesc Psychopharmacol. 2006 Oct;16(5):589-98. doi: 10.1089/cap.2006.16.589.