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Autism Spectrum DisorderPhase 2NCT01793441

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

A Phase 2 study testing RG7314 in people with Autism Spectrum Disorder, run by Hoffmann-La Roche at 56 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 to 45 years, men only
Conditions the study is for
Autism Spectrum Disorder
What is being tested?
RG7314
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
223 participants
Where
56 sitesUnited States
Study dates
Sep 2013to Sep 2016
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 45 years
  • Men only
  • No healthy volunteers
  • Adult

Who may be able to join

  • 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.

Who cannot join

  • 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

Trial locations and contacts

56 sites in 1 country.

56 sites in 1 country

United States56 sites

  • The Kennedy Krieger Institute

    Baltimore, Maryland, 21205

  • Study site

    Baltimore, Maryland, 21205

  • Northwest Clinical Research Center

    Bellevue, Washington, 98007

  • Study site

    Bellevue, Washington, 98007

  • Pacific Institute of Medical Sciences

    Bothell, Washington, 98011

  • Study site

    Bothell, Washington, 98011

  • DBA IMMUNOe Int'l Res Center

    Centennial, Colorado, 80112

  • Study site

    Centennial, Colorado, 80112

  • Rush University Medical Center

    Chicago, Illinois, 60612

  • Study site

    Chicago, Illinois, 60612

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229

  • Study site

    Cincinnati, Ohio, 45229

  • University Hospitals

    Cleveland, Ohio, 44106

  • Study site

    Cleveland, Ohio, 44106

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

    Decatur, Georgia, 30033

  • Study site

    Decatur, Georgia, 30033

  • Neurology offices of south florida

    Delray Beach, Florida, 33445

  • Study site

    Delray Beach, Florida, 33445

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

    Durham, North Carolina, 27705

  • Study site

    Durham, North Carolina, 27705

  • Massachusetts General Hospital

    Lexington, Massachusetts, 02421

  • Study site

    Lexington, Massachusetts, 02421

  • University of Minnesota; Clin. Neuro Research Unit

    Minneapolis, Minnesota, 55414

  • Study site

    Minneapolis, Minnesota, 55414

  • Vanderbilt Medical Center

    Nashville, Tennessee, 37232

  • Study site

    Nashville, Tennessee, 37232

  • Yale University; Yale Child Study Center

    New Haven, Connecticut, 06510

  • Study site

    New Haven, Connecticut, 06510

  • Mount Sinai School of Medicine; Seaver Autism Center

    New York, New York, 10029

  • Study site

    New York, New York, 10029

  • Research Centers of America, LLC

    Oakland Park, Florida, 33334

  • Study site

    Oakland Park, Florida, 33334

  • Nathan S. Kline Institute for Psychiatric Research

    Orangeburg, New York, 10962

  • Study site

    Orangeburg, New York, 10962

  • Compass Research

    Orlando, Florida, 32806

  • Study site

    Orlando, Florida, 32806

  • Southwest Autism Research & Resource Center

    Phoenix, Arizona, 85006

  • Study site

    Phoenix, Arizona, 85006

  • UPMC Western Psychiatric Institute and Clinic

    Pittsburgh, Pennsylvania, 15203 1101

  • Study site

    Pittsburgh, Pennsylvania, 15203 1101

  • Neuroscientific Insights

    Rockville, Maryland, 20852

  • Study site

    Rockville, Maryland, 20852

  • University of California Davis

    Sacramento, California, 95817

  • Study site

    Sacramento, California, 95817

  • Road Runner Research

    San Antonio, Texas, 78258

  • Study site

    San Antonio, Texas, 78258

  • PCSD Feighner Research

    San Diego, California, 92108

  • Study site

    San Diego, California, 92108

  • University of California at San Francisco

    San Francisco, California, 94115

  • Study site

    San Francisco, California, 94115

  • Study site

    Santa Ana, California, 92705

  • Seattle Children's Research Institute

    Seattle, Washington, 98145

  • Study site

    Seattle, Washington, 98145

  • Study site

    Stanford, California, 94305

  • Montefiore Medical Center; Departments of Psychiatry & Biobehavioral Science

    The Bronx, New York, 10467

  • Study site

    The Bronx, New York, 10467

Other trials in United States

About this study

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)

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.

Being tested
Placebo, RG7314
Medical subject headings
Autism Spectrum Disorder

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • PlaceboDrug

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

  • RG7314Drug

    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.

Study groups in NCT01793441
Study groupRoleWhat they receive
PlaceboPlacebo comparatorParticipants will receive placebo matching to RG7314 in each stage (Stage I, II, III and IV) for 12 weeks.
Receives: Drug: Placebo
RG7314ExperimentalParticipants 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.
Receives: Drug: RG7314

What is being measured

The primary outcome is the main question the study is designed to answer.

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

    Measured: Baseline, Week 12

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

    Measured: Baseline up to Week 12

  3. Percentage of Participants With Adverse Events

    Measured: From Baseline up to Week 18

Secondary outcomes (13)

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

    Measured: Baseline, Week 12

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

    Measured: Baseline, Week 12

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

    Measured: Baseline, Week 12

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

    Measured: Baseline, Week 12

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

    Measured: Baseline, Week 12

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

    Measured: Baseline, Week 12

  7. Plasma Concentration of RG7314

    Measured: 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

    Measured: 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

    Measured: 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

    Measured: 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

    Measured: 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

    Measured: 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

    Measured: From Baseline to Week 12

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted15 Feb 2013 (estimated)
  2. Study startSep 2013
  3. Primary completionSep 2016
  4. Study completionSep 2016

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Double blind
Who is masked
Participant, Investigator
Enrolment
223 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01793441
Sponsor's protocol ID
BP28420
Other IDs
2012-005597-55
Status last verified
Jan 2017
First submitted
14 Feb 2013
Last updated
3 Feb 2017
Results posted
No

Publications

  • 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.

    PubMed 37811711

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing RG7314 in people with Autism Spectrum Disorder, run by Hoffmann-La Roche at 56 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 3 Feb 2017. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 56 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change From Baseline in Social Communication Deficits as Measured by Social Responsiveness Scale 2 (SRS-2) (time frame: Baseline, Week 12). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01793441, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Feb 2017.

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