Pilot Study of Riluzole for Drug-Refractory Irritability in Autism Spectrum Disorders

NCT02081027 | Completed Early Phase 1 DMC FDA Drug

• 1 other identifier: CIN001- Riluzole in Autism
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 1

Brief Summary

Determine if riluzole shows evidence of efficacy, safety, and tolerability targeting drug-refractory irritability (DRI) in persons with autism spectrum disorders (ASD).

Conditions

Autism Spectrum Disorders

Outcome Measures

Primary Outcomes (2)

• Clinical Global Impression-Improvement

  A clinician-rated global assessment of symptom change rated on a scale from 1 to 7

  Change from baseline to end of week 12

• Aberrant Behavior Checklist- Irritability

  A parent questionnaire measuring five behavioral domains

  Change between baseline and end of each phase

Study Arms (2)

Riluzole

EXPERIMENTAL

The maximum dose of riluzole to be used in this study is 200 mg per day divided BID

Drug: Riluzole

Placebo

PLACEBO COMPARATOR

Placebo will be administered in the same manner as the riluzole group, in order to maintain subject assignment throughout the study.

Other: placebo

Interventions

Riluzole DRUG

Also known as: Other name: Rilutek

Riluzole

placebo OTHER

Placebo

Eligibility Criteria

• Age: 12 Years - 25 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Children's Hospital Medical Center, Cincinnati: lead

Study Sites (1)

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Related Publications (1)

• 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.

  PMID: 29644582 DERIVED

Related Links

• Journal of Autism and Developmental Disorders (2018) 48:3051-3060

MeSH Terms

Conditions

Autism Spectrum Disorder

Interventions

Riluzole

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

Thiazoles; Sulfur Compounds; Organic Chemicals; Benzothiazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Study Officials

• Logan Wink, MD

  Children's Hospital Medical Center, Cincinnati

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: BASIC SCIENCE
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 5, 2013
• First Posted: March 7, 2014
• Study Start: September 19, 2013
• Primary Completion: April 1, 2015
• Study Completion: May 1, 2015
• Last Updated: January 14, 2021

Record last verified: 2017-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)