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

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ClinicalTrials.gov Identifier: NCT01793441
Recruitment Status : Completed
First Posted : February 15, 2013
Last Update Posted : February 3, 2017
Sponsor:
Information provided by (Responsible Party):
Hoffmann-La Roche

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.

Condition or disease Intervention/treatment Phase
Autism Spectrum Disorder Drug: Placebo Drug: RG7314 Phase 2

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 223 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
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)
Study Start Date : September 2013
Actual Primary Completion Date : September 2016
Actual Study Completion Date : September 2016

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Placebo Comparator: Placebo
Participants will receive placebo matching to RG7314 in each stage (Stage I, II, III and IV) for 12 weeks.
Drug: Placebo
Placebo will be administered in different stages of the study for 12 weeks.

Experimental: 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.
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.




Primary Outcome Measures :
  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 Outcome Measures :
  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 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 45 Years   (Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01793441


  Show 56 Study Locations
Sponsors and Collaborators
Hoffmann-La Roche
Investigators
Study Director: Clinical Trials Hoffmann-La Roche

Responsible Party: Hoffmann-La Roche
ClinicalTrials.gov Identifier: NCT01793441     History of Changes
Other Study ID Numbers: BP28420
2012-005597-55 ( EudraCT Number )
First Posted: February 15, 2013    Key Record Dates
Last Update Posted: February 3, 2017
Last Verified: January 2017

Additional relevant MeSH terms:
Disease
Autistic Disorder
Autism Spectrum Disorder
Child Development Disorders, Pervasive
Pathologic Processes
Neurodevelopmental Disorders
Mental Disorders