A Study of RG7314 to Investigate Efficacy and Safety in Individuals With Autism Spectrum Disorders (ASD)

Hoffmann-La Roche223 enrolled

Overview

This multi-center, randomized, double-blind, parallel group, placebo-controlled, proof of concept study will investigate the efficacy and safety of RG7314 in adult participants with ASD. In Stage I of the study, participants will be randomized in 2:1 to receive daily oral doses of 1.5 milligrams (mg) RG7314 or placebo for 12 weeks. After an independent safety review, the study may proceed to Stage II. In Stage II of the study, additional participants will be randomized in 2:1 to receive daily oral doses of 4 mg RG7314 or placebo for 12 weeks. After an independent safety review, Stage III will be started wherein additional participants will be randomized in 2:1 to receive daily oral doses of 10 mg RG7314 or placebo for 12 weeks. During Stage III, safety will be reviewed by independent safety review twice and if no safety signal is observed, then additional participants will be randomized in 1:1:1 either to receive 1.5 milligrams per day (mg/day) or 10 mg/day RG7314 orally or placebo for 12 weeks in Stage IV.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Enrollment

223

Conditions

Autism Spectrum Disorder

Interventions

Eligibility

Sex: MALEMin age: 18 YearsMax age: 45 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Participants meet Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-5) criteria for ASD and Tenth Revision of the International Classification of Diseases and Related Health Problems (ICD-10) criteria for Autism diagnosis * SRS-2 (T-score) greater than or equal to (\>/=) 66 * CGI-S \>/=4 (moderately ill) * Participants have an Intelligence Quotient (IQ) \>/=70 (Wechsler Abbreviated State of Intelligence) * A body mass index (BMI) between 18 to 40 kilograms per square meter (kg/m\^2) inclusive * Language, hearing and vision compatible with the study measurements as judged by the investigator * Lives with (or has substantial periods of contact with) a caregiver who is willing and able to attend visits when required, oversee the participant's compliance with protocol-specified procedures and study medication dosing, and report on the participant's status via completion of study assessments. Any period of absence must be covered by another caregiver. Non-cohabitating caregiver(s) must spend sufficient time with the participant so that, in the opinion of the investigator, the caregiver(s) can reliably assess participant's mental status, activities and behavior. Exclusion Criteria: * Alcohol and/or substance abuse/dependence during the last 12 months * A significant risk for suicidal behavior, in the opinion of the investigator * Systolic blood pressure greater than (\>) 140 or less than (\<) 90 millimeters of mercury (mm Hg), and diastolic blood pressure \>90 or less than (\<) 50 mm Hg * Resting pulse rate \>90 or \<40 beats per minute * Use of prohibited medications or herbal remedies within 2 weeks prior to randomization, or 5 half-lives (whichever is longer) * Initiation of a new major change in psychological intervention within 4 weeks prior to randomization. Minor changes in ongoing treatment are not considered significant * Participation in an investigational drug or device study within 60 days prior to randomization

Locations (56)

Southwest Autism Research & Resource Center

Phoenix, Arizona, United States

Unnamed facility

Phoenix, Arizona, United States

University of California Davis

Sacramento, California, United States

Unnamed facility

Sacramento, California, United States

PCSD Feighner Research

San Diego, California, United States

Outcomes

Primary Outcomes

Change From Baseline in Social Communication Deficits as Measured by Social Responsiveness Scale 2 (SRS-2)

Time frame: Baseline, Week 12

Percentage of Participants with Suicidality, as Measured by the Columbia-Suicide Severity Rating Scale (C-SSRS)

Time frame: Baseline up to Week 12

Percentage of Participants With Adverse Events

Time frame: From Baseline up to Week 18

Secondary Outcomes

Change From Baseline in Behavior/Symptoms as Measured by Aberrant Behavior Checklist (ABC)

Time frame: Baseline, Week 12

Change From Baseline in Behavior/Symptoms as Measured by Repetitive Behavior Scale-Revised (RBS-R)

Time frame: Baseline, Week 12

Change From Baseline in Behavior/Symptoms as Measured by Anxiety, Depression and Mood Scale (ADAMS)

Time frame: Baseline, Week 12

Measure: Change From Baseline in Behavior/Symptoms as Measured by State-Trait Anxiety Inventory (STAI)

Time frame: Baseline, Week 12

Change From Baseline in Clinical Global Impressions as Measured by Clinical Global Impressions Improvement (CGI-I) Scale

Time frame: Baseline, Week 12

Change From Baseline in Adaptive Functioning and Skills as Measured by Vineland-II Adaptive Behavior Scale (VABS)

Time frame: Baseline, Week 12

Plasma Concentration of RG7314

Time frame: Pre-dose (0 hours [hr]), and 2, 4, 6 hrs post-dose on Day 1, 14, 84; pre-dose (0 hr) on Day 42; any time during early withdrawal visit (up to Week 12)

Plasma Concentration of RG7314 Metabolite M3