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

NCT ID: NCT01793441

Last Updated: 2017-02-03

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

223 participants

Study Classification

INTERVENTIONAL

Study Start Date

2013-09-30

Study Completion Date

2016-09-30

Brief Summary

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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.

Detailed Description

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Conditions

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Autism Spectrum Disorder

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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Placebo

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

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

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

RG7314

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.

Group Type EXPERIMENTAL

RG7314

Intervention Type 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.

Interventions

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Placebo

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

Intervention Type DRUG

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.

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Maximum Eligible Age

45 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Hoffmann-La Roche

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Clinical Trials

Role: STUDY_DIRECTOR

Hoffmann-La Roche

Locations

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Southwest Autism Research & Resource Center

Phoenix, Arizona, United States

Site Status

Phoenix, Arizona, United States

Site Status

University of California Davis

Sacramento, California, United States

Site Status

Sacramento, California, United States

Site Status

PCSD Feighner Research

San Diego, California, United States

Site Status

San Diego, California, United States

Site Status

University of California at San Francisco

San Francisco, California, United States

Site Status

San Francisco, California, United States

Site Status

Santa Ana, California, United States

Site Status

Stanford, California, United States

Site Status

DBA IMMUNOe Int'l Res Center

Centennial, Colorado, United States

Site Status

Centennial, Colorado, United States

Site Status

Yale University; Yale Child Study Center

New Haven, Connecticut, United States

Site Status

New Haven, Connecticut, United States

Site Status

Neurology offices of south florida

Delray Beach, Florida, United States

Site Status

Delray Beach, Florida, United States

Site Status

Research Centers of America, LLC

Oakland Park, Florida, United States

Site Status

Oakland Park, Florida, United States

Site Status

Compass Research

Orlando, Florida, United States

Site Status

Orlando, Florida, United States

Site Status

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

Decatur, Georgia, United States

Site Status

Decatur, Georgia, United States

Site Status

Rush University Medical Center

Chicago, Illinois, United States

Site Status

Chicago, Illinois, United States

Site Status

The Kennedy Krieger Institute

Baltimore, Maryland, United States

Site Status

Baltimore, Maryland, United States

Site Status

Neuroscientific Insights

Rockville, Maryland, United States

Site Status

Rockville, Maryland, United States

Site Status

Massachusetts General Hospital

Lexington, Massachusetts, United States

Site Status

Lexington, Massachusetts, United States

Site Status

University of Minnesota; Clin. Neuro Research Unit

Minneapolis, Minnesota, United States

Site Status

Minneapolis, Minnesota, United States

Site Status

Mount Sinai School of Medicine; Seaver Autism Center

New York, New York, United States

Site Status

New York, New York, United States

Site Status

Nathan S. Kline Institute for Psychiatric Research

Orangeburg, New York, United States

Site Status

Orangeburg, New York, United States

Site Status

Montefiore Medical Center; Departments of Psychiatry & Biobehavioral Science

The Bronx, New York, United States

Site Status

The Bronx, New York, United States

Site Status

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

Durham, North Carolina, United States

Site Status

Durham, North Carolina, United States

Site Status

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

Cincinnati, Ohio, United States

Site Status

University Hospitals

Cleveland, Ohio, United States

Site Status

Cleveland, Ohio, United States

Site Status

UPMC Western Psychiatric Institute and Clinic

Pittsburgh, Pennsylvania, United States

Site Status

Pittsburgh, Pennsylvania, United States

Site Status

Vanderbilt Medical Center

Nashville, Tennessee, United States

Site Status

Nashville, Tennessee, United States

Site Status

Road Runner Research

San Antonio, Texas, United States

Site Status

San Antonio, Texas, United States

Site Status

Northwest Clinical Research Center

Bellevue, Washington, United States

Site Status

Bellevue, Washington, United States

Site Status

Pacific Institute of Medical Sciences

Bothell, Washington, United States

Site Status

Bothell, Washington, United States

Site Status

Seattle Children's Research Institute

Seattle, Washington, United States

Site Status

Seattle, Washington, United States

Site Status

Countries

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United States

References

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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.

Reference Type DERIVED
PMID: 37811711 (View on PubMed)

Other Identifiers

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2012-005597-55

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

BP28420

Identifier Type: -

Identifier Source: org_study_id

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