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Trial record 1 of 5 for:    roche and autism
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A Study to Investigate the Efficacy and Safety of RO5285119 in Participants With Autism Spectrum Disorder (ASD)

This study is currently recruiting participants.
Verified October 2017 by Hoffmann-La Roche
Sponsor:
ClinicalTrials.gov Identifier:
NCT02901431
First Posted: September 15, 2016
Last Update Posted: November 1, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
Hoffmann-La Roche
  Purpose
This is a Phase II multi-center, randomized, double-blind, 24-week, 3-arm, parallel group, placebo-controlled study to investigate the efficacy, safety, and pharmacokinetics of RO5285119 in children and adolescents aged 5-17 years with ASD who are high functioning (intelligence quotient [IQ] greater than or equal to [>=] 70). Enrollment will be staggered, starting first with adolescents (aged 13 to 17 years) and then with children (aged 5 to 12 years). Initially, a first cohort of approximately 24 adolescents will be enrolled together. Based on pharmacokinetic evaluation, safety, and tolerability in the first adolescent cohort and determined final doses, enrollment of adolescents will resume and the enrollment of a first cohort of approximately 24 children (aged 5 to 12 years) can commence. Based on acceptable safety and tolerability in the first children cohort and determined final doses, enrollment of children will resume.

Condition Intervention Phase
Autism Spectrum Disorder Drug: Placebo Drug: RO5285119 Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: A Phase II Multi-center, Randomized, Double-blind, 24-Week, 3-Arm, Parallel Group, Placebo-controlled Study to Investigate the Efficacy and Safety of RO5285119 in Children and Adolescents Age 5-17 With Autism Spectrum Disorder (ASD)

Resource links provided by NLM:


Further study details as provided by Hoffmann-La Roche:

Primary Outcome Measures:
  • Change From Baseline in Vineland-II Adaptive Behavior Scale Composite Standard Score at Week 24 [ Time Frame: Baseline, Week 24 ]
  • Number of Participants With Adverse events [ Time Frame: Baseline to Week 30 ]

Secondary Outcome Measures:
  • Change From Baseline in Vineland-II Adaptive Behavior Scale Communication, Socialization, and Daily Living Skills Domain Standard Scores at Weeks 12 and 24 [ Time Frame: Baseline, Weeks 12 and 24 ]
  • Change From Baseline in Clinical Global Impressions-Severity (CGI-S) Score at Weeks 12 and 24 [ Time Frame: Baseline, Weeks 12 and 24 ]
  • Change From Baseline in Ohio Autism Clinical Impressions Scale-Severity (OACIS-S) Score at Weeks 12 and 24 [ Time Frame: Baseline, Weeks 12 and 24 ]
  • Change From Baseline in Clinical Global Impressions- Improvement (CGI-I) Score at Weeks 12 and 24 [ Time Frame: Baseline, Weeks 12 and 24 ]
  • Change From Baseline in Ohio Autism Clinical Impressions Scale- Improvement (OACIS-I) Score at Weeks 12 and 24 [ Time Frame: Baseline, Weeks 12 and 24 ]
  • Change From Baseline in Aberrant Behavior Checklist (ABC) Lethargy/ Social Withdrawal Subscale Score at Weeks 12 and 24 [ Time Frame: Baseline, Weeks 12 and 24 ]
  • Change From Baseline in Repetitive Behavior Scale-Revised (RBS-R) Score at Weeks 12 and 24 [ Time Frame: Baseline, Weeks 12 and 24 ]
  • Change From Baseline in Vineland-II Adaptive Behavior Scale Composite Standard Score in Adolescents and Children Independently at Weeks 12 and 24 [ Time Frame: Baseline, Weeks 12 and 24 ]
  • Change From Baseline in Vineland-II Adaptive Behavior Scale Composite Standard Score at Week 12 [ Time Frame: Baseline, Week 12 ]
  • Apparent Clearance (CL) of RO5285119 [ Time Frame: 4 hours (hrs) postdose on Day 1; predose (0 hrs) at Weeks 1, 3 and on Day 10; predose (0 hrs), 2, 4, 6 hrs postdose at Week 2; end of treatment(up to Week 24); 8-10 hrs postdose in evening at Week 10; predose (0 hrs), 2, 4 hrs postdose at Weeks 12 and 24 ]
  • Volume of Distribution (VD) of RO5285119 [ Time Frame: 4 hrs postdose on Day 1; predose (0 hrs) at Weeks 1, 3 and on Day 10; predose (0 hrs), 2, 4, 6 hrs postdose at Week 2; end of treatment visit (up to Week 24); 8-10 hrs postdose in evening at Week 10; predose (0 hrs), 2, 4 hrs postdose at Weeks 12 and 24 ]
  • Area Under the Concentration-time Curve of RO5285119 in Plasma at Steady State Over 24 Hours (AUC0-24,ss) [ Time Frame: 4 hrs postdose on Day 1; predose (0 hrs) at Weeks 1, 3 and on Day 10; predose (0 hrs), 2, 4, 6 hrs postdose at Week 2; end of treatment visit (up to Week 24); 8-10 hrs postdose in evening at Week 10; predose (0 hrs), 2, 4 hrs postdose at Weeks 12 and 24 ]

Estimated Enrollment: 300
Actual Study Start Date: November 21, 2016
Estimated Study Completion Date: April 18, 2019
Estimated Primary Completion Date: April 18, 2019 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Placebo Comparator: Placebo
Participants will receive a matching placebo orally. Approximate treatment duration will be up to 24 weeks.
Drug: Placebo
Participants will receive a matching placebo orally. Approximate treatment duration will be up to 24 weeks.
Experimental: RO5285119 10 mg/d equivalent
Participants will receive age-adjusted total daily oral dose approximately equivalent to the adult dose of 10 milligrams per day (mg/d) of RO5285119. Approximate treatment duration will be up to 24 weeks.
Drug: RO5285119
Participants will receive age-adjusted total daily oral dose approximately equivalent to the adult doses of either 4 mg/d or 10 mg/d of RO5285119. Approximate treatment duration will be up to 24 weeks.
Experimental: RO5285119 4 mg/d equivalent
Participants will receive age-adjusted total daily oral dose approximately equivalent to the adult dose of 4 mg/d of RO5285119. Approximate treatment duration will be up to 24 weeks.
Drug: RO5285119
Participants will receive age-adjusted total daily oral dose approximately equivalent to the adult doses of either 4 mg/d or 10 mg/d of RO5285119. Approximate treatment duration will be up to 24 weeks.

  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   5 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Fluent in English
  • Diagnostic and Statistical Manual of Mental Disorders, 5th Edition (DSM-5) criteria for ASD or International Statistical Classification of Diseases and Related Health Problems, 10th revision (ICD10) criteria for Autism diagnosis confirmed by Autism Diagnostic Observational Schedule (ADOS-2) criteria
  • Social Responsiveness Scale, second edition (SRS-2) (T-score) >= 66
  • Clinical Global Impressions of Severity (CGI-S) >= 4 (moderately ill) at screening
  • IQ >= 70 as assessed by Wechsler Abbreviated Scale of Intelligence Scale: Second Edition (WASI-II) or Wechsler Preschool and Primary Scale of Intelligence: Fourth Edition (WPPSI-IV) intelligence test
  • Language, hearing, and vision compatible with the study measurements as judged by the investigator

Exclusion Criteria:

  • Initiation of a major change in psychosocial intervention (including investigational) within 4 weeks prior to screening
  • Unstable or uncontrolled clinically significant psychiatric and/or neurological disorder that may interfere with the safety or efficacy endpoints
  • Known personal or family history of cerebral aneurysm
  • Risk of suicidal behavior
  • Seizure within the past 6 months
  • Medical history of alcohol or substance abuse/dependence
  • Concurrent cardio-vascular disease not considered well controlled by the Investigator
  • Clinically significant abnormality on electrocardiogram at screening
  • Concomitant disease or condition (pulmonary, gastro-intestinal, hepatic, renal, metabolic, immunological system, or obesity that could interfere with the conduct of the study
  • Evidence for current gastro-intestinal bleeding, e.g., active stomach ulcer disease
  • History of coagulopathies, bleeding disorders, or blood dyscrasias
  • Positive serology for hepatitis B (HBV), hepatitis C (HCV), human immunodeficiency virus (HIV) 1, or HIV 2
  • Confirmed clinically significant abnormality in parameters of hematology, clinical chemistry, coagulation, or urinalysis
  • Medical history of malignancy if not considered cured
  • Participation in an investigational drug study within 90 days or 5 times the half-life of the investigational molecule (whichever is longer) prior to randomization
  • Loss of blood over 250 milliliters within three months prior to screening
  • Allowed medications have not been stable since 4 weeks before screening, and allowed medications for treatment of epilepsy have not been stable since 3 months before screening
  • Use of prohibited medications within 2 weeks prior to screening visit or 5 times the half-life prior to randomization (whichever is longer)
  Contacts and Locations
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): NCT02901431


Contacts
Contact: Reference Study ID Number: BP30153 www.roche.com/about_roche/roche_worldwide.htm 888-662-6728 (U.S. and Canada) global-roche-genentech-trials@gene.com

  Show 31 Study Locations
Sponsors and Collaborators
Hoffmann-La Roche
Investigators
Study Director: Clinical Trials Hoffmann-La Roche
  More Information

Responsible Party: Hoffmann-La Roche
ClinicalTrials.gov Identifier: NCT02901431     History of Changes
Other Study ID Numbers: BP30153
First Submitted: September 12, 2016
First Posted: September 15, 2016
Last Update Posted: November 1, 2017
Last Verified: October 2017

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