Overview

This trial is active, not recruiting.

Condition autistic 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, 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 autism spectrum disorders (ASD). In Stage I of the study, participants will be randomized 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 2 of the study, additional participants will be randomized 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 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 either to receive 1.5 mg/day or 10 mg day RG7314 orally or placebo for 12 weeks in Stage IV. The anticipated time on study treatment is 12 weeks.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking double blind (subject, investigator)
Primary purpose treatment
Arm
(Placebo Comparator)
Participants will receive placebo 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 responsiveness scale (SRS-2)
time frame: From baseline to Week 12
Number of participants with adverse events
time frame: 12 weeks

Secondary Outcomes

Measure
Area under the curve from time zero to last measurable concentration (AUClast) of RG7314
time frame: 12 weeks
Maximum plasma concentration of RG7314
time frame: 12 weeks
Number of participants with clinical response
time frame: From baseline to Week 12
Change from baseline in Aberrant Behavior Checklist
time frame: From baseline to Week 12
Change from baseline in Repetitive Behavior Scale-Revised
time frame: From baseline to Week 12
Change from baseline in Anxiety, Depression and Mood Scale
time frame: From baseline to Week 12
Change from baseline in State-Trait Anxiety Inventory
time frame: From baseline to Week 12
Change from baseline in Clinical Global Impressions - Improvement
time frame: From baseline to Week 12
Change from baseline in Vineland-II Adaptive Behavior Scale
time frame: From baseline to Week 12
Plasma concentration of RG7314
time frame: 12 weeks

Eligibility Criteria

Male participants from 18 years up to 45 years old.

Inclusion Criteria: - Male adult participants, 18 to 45 years of age - 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 - Social Responsiveness Scale greater than or equal to (>=) 66 - Clinical Global Impressions-Severity (CGI-S) >=4 (moderately ill) - Participants have an intelligence quotient (IQ) >=70 - A body mass index (BMI) between 18 to 40 kilograms per meter square (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 greater than (>) 90 or <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 screening

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 September 2016.
Information provided to ClinicalTrials.gov by Hoffmann-La Roche.