A Randomized, Placebo-controlled Trial of Fluoxetine in Preschool Children
Trial Snapshot
- Phase
- Phase 2
- Status
- Completed
- Enrollment
- 18
- Locations
- 2
- Primary Endpoint
- Rate of Recruitment
Study Overview
Brief Summary
This study is a pilot study to evaluate the feasibility and safety of conducting a year long, double-blind, placebo-controlled trial of fluoxetine in pre-school children to enhance developmental processes in core areas impacted by autism.
Detailed Description
Autism, a brain disorder that affects a small percentage of Americans, often results in a lifetime of impaired thinking, feeling, and social functioning. The disorder generally becomes apparent in children by the age of 3. Autism typically affects a person's ability to communicate, form relationships with others, and respond appropriately to the external world. Some people with autism can function at a relatively high level, with speech and intelligence intact. Others have serious cognitive impairments and language delays, and some never speak. This study will assess the safety and effectiveness of treating autistic children with fluoxetine to enhance developmental processes in core areas impacted by autism.
Each participant was randomly assigned to treatment with double-blinded placebo or fluoxetine for 12 months. After initial screening and randomization, participants were assessed every two weeks for approximately the first 3 months, or until the dose of medication is stabilized. After this initial period, they were assessed on a monthly basis. Dosing was flexible as determined by the adverse and beneficial responses to treatment although there was a suggested titration schedule.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Eligibility Criteria
- Ages
- 30 Months to 58 Months (Child)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Diagnosis of autism
Exclusion Criteria
- •Diagnosis of Asperger Syndrome, Rett Syndrome, Childhood Disintegrative Disorder, or Pervasive Development Disorder-Not Otherwise Specified
- •Informed that treatment with a selective serotonin reuptake inhibitor (SSRI) is medically inadvisable
- •Need for ongoing psychotropic medication (except for diphenhydramine, clonidine, or melatonin for sleep)
- •Recent use of stimulants within 5 days prior to enrollment
- •Ongoing need for or recent use of most psychotropic medications within 14 days of enrollment
- •Recent initiation of specialized educational, behavioral, or diet intervention for autism in the month prior to enrollment
Arms & Interventions
fluoxetine
Fluoxetine, 20mg/5ml solution, flexible dose 0.5 to 5ml every AM
Intervention: Fluoxetine (Drug)
Placebo
Placebo, liquid solution flexible dose 0.5 to 5ml every morning (AM)
Intervention: Placebo (Drug)
Outcomes
Primary Outcomes
Rate of Recruitment
Time Frame: 19 months
In order for a larger trial with similar design to be feasible a number of factors needed to be examined. The first was whether families would enroll very young children with ASD into a year long blinded medication study. To determine this we examined the average number of months to randomize 1 participant per site. We calculated this (as total # months required for recruitment\* 2sites ) /\[ # participants randomized \] and compared it to the typical # of months required to recruit an older child with ASD for a double-blind 12 week placebo controlled medication study, which is typically about 1.2 months at each of the sites involved in the study.
Secondary Outcomes
- Rate of Attrition(Measured at Month 12)
- Change From Baseline to 12 Months in Total Score on Caregiver Strain Questionnaire(12 months)
- Change From Baseline to Month 12 in Aberrant Behavior Checklist Irritability Subscale Score (ABC-I)(12 months)
Investigators
Linmarie Sikich, MD
Associate Professor
University of North Carolina, Chapel Hill