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



Status:Completed
Conditions:Neurology, Psychiatric, Psychiatric, Autism
Therapuetic Areas:Neurology, Psychiatry / Psychology
Healthy:No
Age Range:18 - 45
Updated:2/4/2017
Start Date:September 2013
End Date:September 2016

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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)

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.


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
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707 North Broadway
Baltimore, Maryland 21205
443-923-9200
Kennedy Krieger Institute While not officially part of Johns Hopkins Medicine, Kennedy Krieger Institute is...
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3333 Burnet Avenue # Mlc3008
Cincinnati, Ohio 45229
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100 West Gore St # 202
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