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

Cincinnati Children's Hospital Medical Center

Status and phase: Completed; Early Phase 1

Conditions: Autism Spectrum Disorders

Treatments: Other: placebo; Drug: Riluzole

Study type: Interventional

Funder types: Other

Identifiers

NCT02081027

CIN001- Riluzole in Autism

Details and patient eligibility

About

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

Full 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.

Enrollment: 8 patients

Sex: All

Ages: 12 to 25 years old

Volunteers: No 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.

Trial design

Primary purpose: Basic Science

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: Triple Blind

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8 participants in 2 patient groups, including a placebo group

Riluzole

Experimental group

Description:

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

Treatment:

Drug: Riluzole

Placebo

Placebo Comparator group

Description:

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

Treatment:

Other: placebo