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

Primary Purpose

Autism Spectrum Disorders

Status
Completed
Phase
Early Phase 1
Locations
United States
Study Type
Interventional
Intervention
Riluzole
placebo
Sponsored by
Children's Hospital Medical Center, Cincinnati
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional basic science trial for Autism Spectrum Disorders

Eligibility Criteria

12 Years - 25 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Cincinnati Children's Hospital Medical Center

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Riluzole

Placebo

Arm Description

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

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

Outcomes

Primary Outcome Measures

Clinical Global Impression-Improvement
A clinician-rated global assessment of symptom change rated on a scale from 1 to 7
Aberrant Behavior Checklist- Irritability
A parent questionnaire measuring five behavioral domains

Secondary Outcome Measures

Full Information

First Posted
August 5, 2013
Last Updated
January 12, 2021
Sponsor
Children's Hospital Medical Center, Cincinnati
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1. Study Identification

Unique Protocol Identification Number
NCT02081027
Brief Title
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

2. Study Status

Record Verification Date
April 2017
Overall Recruitment Status
Completed
Study Start Date
September 19, 2013 (Actual)
Primary Completion Date
April 2015 (Actual)
Study Completion Date
May 2015 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Children's Hospital Medical Center, Cincinnati

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Autism Spectrum Disorders

7. Study Design

Primary Purpose
Basic Science
Study Phase
Early Phase 1
Interventional Study Model
Crossover Assignment
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
8 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Riluzole
Arm Type
Experimental
Arm Description
The maximum dose of riluzole to be used in this study is 200 mg per day divided BID
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo will be administered in the same manner as the riluzole group, in order to maintain subject assignment throughout the study.
Intervention Type
Drug
Intervention Name(s)
Riluzole
Other Intervention Name(s)
Other name: Rilutek
Intervention Type
Other
Intervention Name(s)
placebo
Primary Outcome Measure Information:
Title
Clinical Global Impression-Improvement
Description
A clinician-rated global assessment of symptom change rated on a scale from 1 to 7
Time Frame
Change from baseline to end of week 12
Title
Aberrant Behavior Checklist- Irritability
Description
A parent questionnaire measuring five behavioral domains
Time Frame
Change between baseline and end of each phase

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Years
Maximum Age & Unit of Time
25 Years
Accepts Healthy Volunteers
No
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Logan Wink, MD
Organizational Affiliation
Children's Hospital Medical Center, Cincinnati
Official's Role
Principal Investigator
Facility Information:
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No
Citations:
PubMed Identifier
29644582
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.
Results Reference
derived
Links:
URL
https://doi.org/10.1007/s10803-018-3562-5
Description
Journal of Autism and Developmental Disorders (2018) 48:3051-3060

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

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