A Randomized, Double-Blind, Placebo-Controlled, Cross-Over Pilot Study of Riluzole for Drug-Refractory Irritability in Autism Spectrum Disorders
Overview
- Phase
- Early Phase 1
- Status
- Completed
- Enrollment
- 8
- Locations
- 1
- Primary Endpoint
- Clinical Global Impression-Improvement
Overview
Brief Summary
Determine if riluzole shows evidence of efficacy, safety, and tolerability targeting drug-refractory irritability (DRI) in persons with autism spectrum disorders (ASD).
Detailed Description
A randomized, double blind, placebo-controlled, 12-week cross-over study (5-week treatment periods with 2-week washout) of adjunctive riluzole in 12 persons with ASD and DRI between the ages of 12 and 25 years.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Crossover
- Primary Purpose
- Basic Science
- Masking
- Triple (Participant, Care Provider, Investigator)
Eligibility Criteria
- Ages
- 12 Years to 25 Years (Child, Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Age ≥ 12 and \< 26 years.
- •Weight greater than 50 kg.
- •Diagnosis of ASD
- •Drug-refractory irritability, as defined as screening ABC Irritability subscale (ABC-I) score of ≥18 AND 1) failure of clinically adequate treatment trials of both aripiprazole and risperidone or 2) failure of at least three previous clinically adequate drug trials targeting irritability (one trial must include aripiprazole or risperidone), as confirmed by caregiver reports and medical record review when available.
- •Stable dosing of all concomitant psychotropic medications (including those targeting irritability) for four weeks prior to screening visit and during the study.
- •Presence of parent/guardian willing to serve as informant for behavioral outcome measures and shipping control sample for Extracellular Signal-Related Kinase biomarker (ERK) assay.
Exclusion Criteria
- •Current use of more than two concomitant psychotropic drugs targeting irritability.
- •Current use of valproic acid.
- •Current use of drugs with known interaction with riluzole
- •Current use of drugs with concomitant glutamatergic or glutamatergic- modulating action medications.
- •For female subjects of child bearing potential, a positive serum pregnancy test.
- •History of pancreatitis.
- •Hemoglobin less than or equal to 8.0 gm/dL.
- •Neutropenia with absolute neutrophil count less than or equal to 1.0 K/mcL.
- •Problems with kidney functioning, as assessed by lab work
- •Any major chronic medical or chronic respiratory illness considered to be uncontrolled by the Principal Investigator.
Arms & Interventions
Riluzole
The maximum dose of riluzole to be used in this study is 200 mg per day divided BID
Intervention: Riluzole (Drug)
Placebo
Placebo will be administered in the same manner as the riluzole group, in order to maintain subject assignment throughout the study.
Intervention: placebo (Other)
Outcomes
Primary Outcomes
Clinical Global Impression-Improvement
Time Frame: Change from baseline to end of week 12
A clinician-rated global assessment of symptom change rated on a scale from 1 to 7
Aberrant Behavior Checklist- Irritability
Time Frame: Change between baseline and end of each phase
A parent questionnaire measuring five behavioral domains
Secondary Outcomes
No secondary outcomes reported