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

NCT01793441 | Completed Phase 2

• 2 other identifiers: BP284202012-005597-55
• Study Type: interventional
• Target: 223
• Locations: 1 country
• Sites: 56

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.

Conditions

Autism Spectrum Disorder

Outcome Measures

Primary Outcomes (3)

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

  Baseline, Week 12

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

  Baseline up to Week 12

• Percentage of Participants With Adverse Events

  From Baseline up to Week 18

Secondary Outcomes (13)

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

  Baseline, Week 12

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

  Baseline, Week 12

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

  Baseline, Week 12

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

  Baseline, Week 12

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

  Baseline, Week 12

Study Arms (2)

Placebo

PLACEBO COMPARATOR

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

Drug: Placebo

RG7314

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.

Drug: RG7314

Interventions

Placebo DRUG

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

Placebo

RG7314 DRUG

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.

RG7314

Eligibility Criteria

• Age: 18 Years - 45 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Hoffmann-La Roche: lead

Study Sites (56)

Southwest Autism Research & Resource Center

Phoenix, Arizona, 85006, United States

Location

Unknown Facility

Phoenix, Arizona, 85006, United States

Location

University of California Davis

Sacramento, California, 95817, United States

Location

Unknown Facility

Sacramento, California, 95817, United States

Location

PCSD Feighner Research

San Diego, California, 92108, United States

Location

Unknown Facility

San Diego, California, 92108, United States

Location

University of California at San Francisco

San Francisco, California, 94115, United States

Location

Unknown Facility

San Francisco, California, 94115, United States

Location

Unknown Facility

Santa Ana, California, 92705, United States

Location

Unknown Facility

Stanford, California, 94305, United States

Location

DBA IMMUNOe Int'l Res Center

Centennial, Colorado, 80112, United States

Location

Unknown Facility

Centennial, Colorado, 80112, United States

Location

Yale University; Yale Child Study Center

New Haven, Connecticut, 06510, United States

Location

Unknown Facility

New Haven, Connecticut, 06510, United States

Location

Neurology offices of south florida

Delray Beach, Florida, 33445, United States

Location

Unknown Facility

Delray Beach, Florida, 33445, United States

Location

Research Centers of America, LLC

Oakland Park, Florida, 33334, United States

Location

Unknown Facility

Oakland Park, Florida, 33334, United States

Location

Compass Research

Orlando, Florida, 32806, United States

Location

Unknown Facility

Orlando, Florida, 32806, United States

Location

Emory University School of Medicine; Department of Human Genetics & Pediatrics

Decatur, Georgia, 30033, United States

Location

Unknown Facility

Decatur, Georgia, 30033, United States

Location

Rush University Medical Center

Chicago, Illinois, 60612, United States

Location

Unknown Facility

Chicago, Illinois, 60612, United States

Location

The Kennedy Krieger Institute

Baltimore, Maryland, 21205, United States

Location

Unknown Facility

Baltimore, Maryland, 21205, United States

Location

Neuroscientific Insights

Rockville, Maryland, 20852, United States

Location

Unknown Facility

Rockville, Maryland, 20852, United States

Location

Massachusetts General Hospital

Lexington, Massachusetts, 02421, United States

Location

Unknown Facility

Lexington, Massachusetts, 02421, United States

Location

University of Minnesota; Clin. Neuro Research Unit

Minneapolis, Minnesota, 55414, United States

Location

Unknown Facility

Minneapolis, Minnesota, 55414, United States

Location

Mount Sinai School of Medicine; Seaver Autism Center

New York, New York, 10029, United States

Location

Unknown Facility

New York, New York, 10029, United States

Location

Nathan S. Kline Institute for Psychiatric Research

Orangeburg, New York, 10962, United States

Location

Unknown Facility

Orangeburg, New York, 10962, United States

Location

Montefiore Medical Center; Departments of Psychiatry & Biobehavioral Science

The Bronx, New York, 10467, United States

Location

Unknown Facility

The Bronx, New York, 10467, United States

Location

DUKE SCHOOL OF MEDICINE;Duke Center for Autism and Brain Development

Durham, North Carolina, 27705, United States

Location

Unknown Facility

Durham, North Carolina, 27705, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Unknown Facility

Cincinnati, Ohio, 45229, United States

Location

University Hospitals

Cleveland, Ohio, 44106, United States

Location

Unknown Facility

Cleveland, Ohio, 44106, United States

Location

UPMC Western Psychiatric Institute and Clinic

Pittsburgh, Pennsylvania, 15203 1101, United States

Location

Unknown Facility

Pittsburgh, Pennsylvania, 15203 1101, United States

Location

Vanderbilt Medical Center

Nashville, Tennessee, 37232, United States

Location

Unknown Facility

Nashville, Tennessee, 37232, United States

Location

Road Runner Research

San Antonio, Texas, 78258, United States

Location

Unknown Facility

San Antonio, Texas, 78258, United States

Location

Northwest Clinical Research Center

Bellevue, Washington, 98007, United States

Location

Unknown Facility

Bellevue, Washington, 98007, United States

Location

Pacific Institute of Medical Sciences

Bothell, Washington, 98011, United States

Location

Unknown Facility

Bothell, Washington, 98011, United States

Location

Seattle Children's Research Institute

Seattle, Washington, 98145, United States

Location

Unknown Facility

Seattle, Washington, 98145, United States

Location

Related Publications (1)

• Iffland M, Livingstone N, Jorgensen M, Hazell P, Gillies D. Pharmacological intervention for irritability, aggression, and self-injury in autism spectrum disorder (ASD). Cochrane Database Syst Rev. 2023 Oct 9;10(10):CD011769. doi: 10.1002/14651858.CD011769.pub2.

  PMID: 37811711 DERIVED

MeSH Terms

Conditions

Autism Spectrum Disorder

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Study Officials

• Clinical Trials

  Hoffmann-La Roche

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 14, 2013
• First Posted: February 15, 2013
• Study Start: September 1, 2013
• Primary Completion: September 1, 2016
• Study Completion: September 1, 2016
• Last Updated: February 3, 2017

Record last verified: 2017-01

Locations

US (56)