Clinical trial NCT02081027: Pilot Study of Riluzole for Drug-Refractory Irritability in Autism Spectrum Disorders
Official title: A Randomized, Double-Blind, Placebo-Controlled, Cross-Over Pilot Study of Riluzole for Drug-Refractory Irritability in Autism Spectrum Disorders
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: EARLY_PHASE1
Conditions: Autism Spectrum Disorders
Lead sponsor: Children's Hospital Medical Center, Cincinnati
Sponsor class: OTHER
First posted: 2014-03-07
Enrollment: 8
Sex: ALL
Minimum age (years): 12
Maximum age (years): 25
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: BASIC_SCIENCE

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Clinical Global Impression-Improvement
timeFrame: Change from baseline to end of week 12
measure: Aberrant Behavior Checklist- Irritability
timeFrame: Change between baseline and end of each phase
Secondary outcomes:
Other outcomes:
Locations:
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
Study officials:
affiliation: Children's Hospital Medical Center, Cincinnati
name: Logan Wink, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Wink LK, Adams R, Horn PS, Tessier CR, Bantel AP, Hong M, Shaffer RC, Pedapati EV, Erickson CA. A Randomized Placebo-Controlled Cross-Over Pilot Study of Riluzole for Drug-Refractory Irritability in Autism Spectrum Disorder. J Autism Dev Disord. 2018 Sep;48(9):3051-3060. doi: 10.1007/s10803-018-3562-5.