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

Primary Purpose

Autism Spectrum Disorder

Status

Completed

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

Placebo

RG7314

About this trial

This is an interventional treatment trial for Autism Spectrum Disorder

Eligibility Criteria

18 Years - 45 Years (Adult)MaleDoes not accept healthy volunteers

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

Sites / Locations

Southwest Autism Research & Resource Center
University of California Davis
PCSD Feighner Research
University of California at San Francisco
DBA IMMUNOe Int'l Res Center
Yale University; Yale Child Study Center
Neurology offices of south florida
Research Centers of America, LLC
Compass Research
Emory University School of Medicine; Department of Human Genetics & Pediatrics
Rush University Medical Center
The Kennedy Krieger Institute
Neuroscientific Insights
Massachusetts General Hospital
University of Minnesota; Clin. Neuro Research Unit
Montefiore Medical Center; Departments of Psychiatry & Biobehavioral Science
Mount Sinai School of Medicine; Seaver Autism Center
Nathan S. Kline Institute for Psychiatric Research
DUKE SCHOOL OF MEDICINE;Duke Center for Autism and Brain Development
Cincinnati Children's Hospital Medical Center
University Hospitals
UPMC Western Psychiatric Institute and Clinic
Vanderbilt Medical Center
Road Runner Research
Northwest Clinical Research Center
Pacific Institute of Medical Sciences
Seattle Children's Research Institute

Arms of the Study

Arm 1

Arm 2

Arm Type

Placebo Comparator

Experimental

Arm Label

Placebo

RG7314

Arm Description

Participants will receive placebo matching to RG7314 in each stage (Stage I, II, III and IV) for 12 weeks.

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.

Outcomes

Primary Outcome Measures

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

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

Percentage of Participants With Adverse Events

Secondary Outcome Measures

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

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

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

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

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

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

Plasma Concentration of RG7314

Plasma Concentration of RG7314 Metabolite M3

Area Under the Plasma Concentration Versus Time Curve From Time Zero to Last Measurable Concentration (AUClast) of RG7314

AUClast of RG7314 Metabolite M3

Maximum Observed Plasma Concentration (Cmax) of RG7314

Cmax of RG7314 Metabolite M3

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

Full Information

NCT ID

NCT01793441

First Posted

February 14, 2013

Last Updated

February 2, 2017

Sponsor

Hoffmann-La Roche

1. Study Identification

Unique Protocol Identification Number

NCT01793441

Brief Title

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

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)

Study Type

Interventional

2. Study Status

Record Verification Date

January 2017

Overall Recruitment Status

Completed

Study Start Date

September 2013 (undefined)

Primary Completion Date

September 2016 (Actual)

Study Completion Date

September 2016 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Hoffmann-La Roche

4. Oversight

5. Study Description

Brief 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.

6. Conditions and Keywords

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

Autism Spectrum Disorder

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Masking

ParticipantInvestigator

Allocation

Randomized

Enrollment

223 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Placebo

Arm Type

Placebo Comparator

Arm Description

Participants will receive placebo matching to RG7314 in each stage (Stage I, II, III and IV) for 12 weeks.

Arm Title

RG7314

Arm Type

Experimental

Arm Description

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.

Intervention Type

Drug

Intervention Name(s)

Placebo

Intervention Description

Placebo will be administered in different stages of the study for 12 weeks.

Intervention Type

Drug

Intervention Name(s)

RG7314

Intervention Description

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 Outcome Measure Information:

Title

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

Time Frame

Baseline, Week 12

Title

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

Time Frame

Baseline up to Week 12

Title

Percentage of Participants With Adverse Events

Time Frame

From Baseline up to Week 18

Secondary Outcome Measure Information:

Title

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

Time Frame

Baseline, Week 12

Title

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

Time Frame

Baseline, Week 12

Title

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

Time Frame

Baseline, Week 12

Title

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

Time Frame

Baseline, Week 12

Title

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

Time Frame

Baseline, Week 12

Title

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

Time Frame

Baseline, Week 12

Title

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)

Title

Plasma Concentration of RG7314 Metabolite M3

Time Frame