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Completed

NCT Number: NCT01793441

A Study of RG7314 to Investigate Efficacy and Safety 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.

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Key information

Conditions

Autism Spectrum Disorder Child Development Disorders, Pervasive Mental Disorders Neurodevelopmental Disorders

Age range

18 year–45 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 2

Primary location

Southwest Autism Research & Resource Center, Phoenix, Arizona, United States

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Placebo

Drug

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

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.

Primary outcomes

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

    Time frame: Baseline, Week 12

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

    Time frame: Baseline up to Week 12

  3. Percentage of Participants With Adverse Events

    Time frame: From Baseline up to Week 18

Secondary outcomes

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

    Time frame: Baseline, Week 12

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

    Time frame: Baseline, Week 12

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

    Time frame: Baseline, Week 12

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

    Time frame: Baseline, Week 12

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

    Time frame: Baseline, Week 12

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

    Time frame: Baseline, Week 12

  7. 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)

  8. Plasma Concentration of RG7314 Metabolite M3

    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)

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

    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)

  10. AUClast of RG7314 Metabolite M3

    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)

  11. Maximum Observed Plasma Concentration (Cmax) of RG7314

    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)

  12. Cmax of RG7314 Metabolite M3

    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)

  13. 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

    Time frame: From Baseline to Week 12

Sponsors and collaborators

Lead sponsor

Hoffmann-La Roche

Industry

Registry information

Official study 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)

Important dates

Study start
2013
Primary completion
2016
Study completion
2016
First posted
Feb 15, 2013
Registry last updated
Feb 3, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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