Overview

This trial has been completed.

Condition autism spectrum disorder
Treatments placebo, rg7314
Phase phase 2
Sponsor Hoffmann-La Roche
Start date September 2013
End date September 2016
Trial size 223 participants
Trial identifier NCT01793441, 2012-005597-55, BP28420

Summary

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.

United States No locations recruiting
Other countries No locations recruiting

Study Design

Allocation randomized
Intervention model parallel assignment
Primary purpose treatment
Masking participant, investigator
Arm
(Placebo Comparator)
Participants will receive placebo matching to RG7314 in each stage (Stage I, II, III and IV) for 12 weeks.
placebo
Placebo will be administered in different stages of the study for 12 weeks.
(Experimental)
Participants will receive RG7314 orally at a dose of 1.5 mg/day in Stage I, 4 mg/day in Stage II, 10 mg/day in Stage III and 1.5 mg/day or 10 mg/day in Stage IV for 12 weeks.
rg7314
RG7314 will be administered orally at a dose of 1.5 mg/day in Stage I, 4 mg/day in Stage II, 10 mg/day in Stage III and 1.5 mg/day or 10 mg/day in Stage IV for 12 weeks.

Primary Outcomes

Measure
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

Measure
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
time frame: Pre-dose (0 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)
Area Under the Plasma Concentration Versus Time Curve From Time Zero to Last Measurable Concentration (AUClast) of RG7314
time frame: Pre-dose (0 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)
AUClast of RG7314 Metabolite M3
time frame: Pre-dose (0 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)
Maximum Observed Plasma Concentration (Cmax) of RG7314
time frame: Pre-dose (0 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)
Cmax of RG7314 Metabolite M3
time frame: Pre-dose (0 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)
Percentage of Participants With Clinical Response, Defined as At Least 25% Improvement in the SRS-2 Score With a CGI-I Score of 1 or 2
time frame: From Baseline to Week 12

Eligibility Criteria

Male participants from 18 years up to 45 years old.

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

Additional Information

Official title A Multi-Center, Randomized, Double-Blind, 12-Week, Parallel Group, Placebo-Controlled Proof of Concept Study to Investigate the Efficacy and Safety of RG7314 in Individuals With Autism Spectrum Disorders (ASD)
Trial information was received from ClinicalTrials.gov and was last updated in February 2017.
Information provided to ClinicalTrials.gov by Hoffmann-La Roche.