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.
Brief Title
A Study of RG7314 to Investigate Efficacy and Safety in Individuals With Autism Spectrum Disorders (ASD)
Categories
Completion Date
Completion Date Type
Actual
Conditions
Autism Spectrum Disorder
Eligibility Criteria
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
* 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
Inclusion Criteria
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.
* 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.
Gender
Male
Gender Based
false
Healthy Volunteers
No
Last Update Post Date
Last Update Post Date Type
Estimated
Last Update Submit Date
Maximum Age
45 Years
Minimum Age
18 Years
NCT Id
NCT01793441
Org Class
Industry
Org Full Name
Hoffmann-La Roche
Org Study Id
BP28420
Overall Status
Completed
Phases
Phase 2
Primary Completion Date
Primary Completion Date Type
Actual
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)
Primary Outcomes
Outcome Measure
Change From Baseline in Social Communication Deficits as Measured by Social Responsiveness Scale 2 (SRS-2)
Outcome Time Frame
Baseline, Week 12
Outcome Measure
Percentage of Participants with Suicidality, as Measured by the Columbia-Suicide Severity Rating Scale (C-SSRS)
Outcome Time Frame
Baseline up to Week 12
Outcome Measure
Percentage of Participants With Adverse Events
Outcome Time Frame
From Baseline up to Week 18
Secondary Ids
Secondary Id
2012-005597-55
Secondary Outcomes
Outcome Time Frame
Baseline, Week 12
Outcome Measure
Change From Baseline in Behavior/Symptoms as Measured by Aberrant Behavior Checklist (ABC)
Outcome Time Frame
Baseline, Week 12
Outcome Measure
Change From Baseline in Behavior/Symptoms as Measured by Repetitive Behavior Scale-Revised (RBS-R)
Outcome Time Frame
Baseline, Week 12
Outcome Measure
Change From Baseline in Behavior/Symptoms as Measured by Anxiety, Depression and Mood Scale (ADAMS)
Outcome Time Frame
Baseline, Week 12
Outcome Measure
Measure: Change From Baseline in Behavior/Symptoms as Measured by State-Trait Anxiety Inventory (STAI)
Outcome Time Frame
Baseline, Week 12
Outcome Measure
Change From Baseline in Clinical Global Impressions as Measured by Clinical Global Impressions Improvement (CGI-I) Scale
Outcome Time Frame
Baseline, Week 12
Outcome Measure
Change From Baseline in Adaptive Functioning and Skills as Measured by Vineland-II Adaptive Behavior Scale (VABS)
Outcome 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)
Outcome Measure
Plasma Concentration of RG7314
Outcome Time Frame
Pre-dose (0 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)
Outcome Measure
Plasma Concentration of RG7314 Metabolite M3
Outcome Time Frame
Pre-dose (0 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)
Outcome Measure
Area Under the Plasma Concentration Versus Time Curve From Time Zero to Last Measurable Concentration (AUClast) of RG7314
Outcome Time Frame
Pre-dose (0 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)
Outcome Measure
AUClast of RG7314 Metabolite M3
Outcome Time Frame
Pre-dose (0 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)
Outcome Measure
Maximum Observed Plasma Concentration (Cmax) of RG7314
Outcome Time Frame
Pre-dose (0 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)
Outcome Measure
Cmax of RG7314 Metabolite M3
Outcome Time Frame
From Baseline to Week 12
Outcome Measure
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
Start Date
Status Verified Date
First Post Date
First Post Date Type
Estimated
First Submit Date
First Submit QC Date
Std Ages
Adult
Maximum Age Number (converted to Years and rounded down)
45
Minimum Age Number (converted to Years and rounded down)
18
Investigators
Investigator Type
Principal Investigator
Investigator Name
Eric Hollander
Investigator Email
eholland@montefiore.org
Investigator Phone
718-920-4287