Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

A Study to Investigate the Efficacy and Safety of Balovaptan (RO5285119) in Participants With Autism Spectrum Disorder (ASD)

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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02901431
Recruitment Status : Active, not recruiting
First Posted : September 15, 2016
Last Update Posted : November 26, 2019
Sponsor:
Information provided by (Responsible Party):
Hoffmann-La Roche

Brief Summary:

For participants enrolled prior to Version 6 of the protocol: 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 balovaptan in children and adolescents aged 5-17 years with ASD who are high functioning (intelligence quotient [IQ] greater than or equal to [>=] 70).

For participants enrolled according to Version 6 of the protocol: This is a Phase II multi-center, randomized, double-blind, 24-week, parallel group, placebo-controlled, 2-arm study with participants assigned either to a 10 milligram (mg) or equivalent dose of balovaptan, or placebo. All other study parameters remain as stated above.

All participants that complete the 24-week treatment period will be eligible to participate in an optional 52-week open-label extension (OLE) during which they will receive balovaptan treatment.


Condition or disease Intervention/treatment Phase
Autism Spectrum Disorder Drug: Placebo Drug: RO5285119 Phase 2

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 340 participants
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, Parallel Group, Placebo-Controlled Study to Investigate the Efficacy and Safety of Balovaptan (RO5285119) in Children and Adolescents Age 5-17 With Autism Spectrum Disorder (ASD)
Actual Study Start Date : November 21, 2016
Estimated Primary Completion Date : July 6, 2021
Estimated Study Completion Date : July 6, 2021

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
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: Balovaptan (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 balovaptan (RO5285119). Approximate treatment duration will be up to 24 weeks (up to 52 additional weeks for those enrolled in the OLE).
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 balovaptan (RO5285119). Approximate treatment duration will be up to 24 weeks (up to 52 additional weeks for those enrolled in the OLE).

Experimental: Balovaptan (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 balovaptan (RO5285119). Approximate treatment duration will be up to 24 weeks. This arm is open only to those participants enrolled prior to Version 6 of the study protocol.
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 balovaptan (RO5285119). Approximate treatment duration will be up to 24 weeks (up to 52 additional weeks for those enrolled in the OLE).




Primary Outcome Measures :
  1. Change From Baseline in Vineland-II Adaptive Behavior Scale Two Domain Composite (2DC) Score at Week 24 [ Time Frame: Baseline, Week 24 ]
  2. Number of Participants With Adverse events [ Time Frame: Baseline to Week 30 ]

Secondary Outcome Measures :
  1. Change from Baseline in Vineland-II Composite Standard Score After 12 Weeks and 24 Weeks of Treatment [ Time Frame: Baseline, Weeks 12 and 24 ]
  2. 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 ]
  3. Change From Baseline in Clinical Global Impressions-Severity (CGI-S) Score at Weeks 12 and 24 [ Time Frame: Baseline, Weeks 12 and 24 ]
  4. 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 ]
  5. Change From Baseline in Clinical Global Impressions- Improvement (CGI-I) Score at Weeks 12 and 24 [ Time Frame: Baseline, Weeks 12 and 24 ]
  6. 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 ]
  7. Patient-Reported Pediatric Quality of Life (PedsQL) v4.0 Generic Core Scale after 12 Weeks and 24 Weeks of Treatment [ Time Frame: Weeks 12 and 24 ]
  8. 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 ]
  9. Change From Baseline in Vineland-II Adaptive Behavior Scale Composite Standard Score at Week 12 [ Time Frame: Baseline, Week 12 ]
  10. Apparent Clearance (CL) of Balovaptan (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 ]
  11. Volume of Distribution (VD) of Balovaptan (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 ]
  12. Area Under the Concentration-time Curve of Balovaptan (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 ]


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.


Layout table for eligibility information
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

Inclusion Criteria for the OLE:

  • Have completed the blinded treatment phase of the study OR were required to stop dosing at or before Week 8
  • Have no adverse events that would prohibit starting the OLE

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)

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


  Hide Study Locations
Locations
Layout table for location information
United States, Alabama
Harmonex Neuroscience Research
Dothan, Alabama, United States, 36303
United States, Arizona
Southwest Autism Research & Resource Center
Phoenix, Arizona, United States, 85006
United States, California
NRC Research Institute
Orange, California, United States, 92868
PCSD Feighner Research
San Diego, California, United States, 92108
University of California at San Francisco
San Francisco, California, United States, 94115
United States, Colorado
Children's Hospital of Colorado
Aurora, Colorado, United States, 80045
DBA IMMUNOe Int'l Res Center
Centennial, Colorado, United States, 80112
United States, Connecticut
Yale University / Yale-New Haven Hospital
New Haven, Connecticut, United States, 06519-1124
United States, Florida
Sarkis Clinical Trials
Gainesville, Florida, United States, 32607
Segal trials
North Miami, Florida, United States, 33161
Medical Research Group of Central Florida
Orange City, Florida, United States, 32763
APG- Advanced Psychiatric Group
Orlando, Florida, United States, 32803
University of South Florida
Saint Petersburg, Florida, United States, 33701
United States, Illinois
Rush University Medical Center
Chicago, Illinois, United States, 60612
Capstone Clinical Research
Libertyville, Illinois, United States, 60048
United States, Maryland
Kennedy Krieger Institute
Baltimore, Maryland, United States, 21205
United States, Massachusetts
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
Boston Childrens Hospital
Boston, Massachusetts, United States, 02115
Massachusetts General Hospital; Lurie Center for Autism
Lexington, Massachusetts, United States, 02421
UMASS Medical School
Worcester, Massachusetts, United States, 01655
United States, Minnesota
University of Minnesota; Clin. Neuro Research Unit
Minneapolis, Minnesota, United States, 55414
United States, Missouri
St. Charles Psychiatric Associates
Saint Charles, Missouri, United States, 63304
United States, Nebraska
Midwest Childrens Health Research Institute
Lincoln, Nebraska, United States, 68516
United States, New York
Albert Einstein College of Medicine
Bronx, New York, United States, 10461
Center for Autism and the Developing Brain
New York, New York, United States, 10032
Nathan S. Kline Institute for Psychiatric Research
Orangeburg, New York, United States, 10962
University of Rochester
Rochester, New York, United States, 14627 0001
Richmond Behavioral Associates
Staten Island, New York, United States, 10312
United States, North Carolina
DUKE SCHOOL OF MEDICINE;Duke Center for Autism and Brain Development
Durham, North Carolina, United States, 27705
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
University Hospitals
Cleveland, Ohio, United States, 44106
Ohio State University
Columbus, Ohio, United States, 43210
United States, Oklahoma
Cutting Edge Research Group
Oklahoma City, Oklahoma, United States, 73116
United States, Pennsylvania
Suburban Research Associates
Media, Pennsylvania, United States, 19063
Children's Hospital of Philadelphia;Allergy/Immunology Department
Philadelphia, Pennsylvania, United States, 19104
UPMC Western Psychiatric Institute and Clinic
Pittsburgh, Pennsylvania, United States, 15203 1101
United States, Tennessee
Vanderbilt University Medical Center
Nashville, Tennessee, United States, 37232
United States, Texas
BioBehavioral Research of Austin, PC
Austin, Texas, United States, 78759
Relaro Medical Trials
Dallas, Texas, United States, 75243
Red Oak Psychiatry Associates, PA
Houston, Texas, United States, 77090
Road Runner Research
San Antonio, Texas, United States, 78249
United States, Washington
Northwest Clinical Research Center
Bellevue, Washington, United States, 98007
Pacific Institute of Medical Sciences
Bothell, Washington, United States, 98011
Core Clinical Research
Everett, Washington, United States, 98201
Seattle Children's Research Institute; Psychiatry and Behavioral Medicine
Seattle, Washington, United States, 98121
Sponsors and Collaborators
Hoffmann-La Roche
Investigators
Layout table for investigator information
Study Director: Clinical Trials Hoffmann-La Roche

Layout table for additonal information
Responsible Party: Hoffmann-La Roche
ClinicalTrials.gov Identifier: NCT02901431     History of Changes
Other Study ID Numbers: BP30153
First Posted: September 15, 2016    Key Record Dates
Last Update Posted: November 26, 2019
Last Verified: November 2019
Additional relevant MeSH terms:
Layout table for MeSH terms
Autistic Disorder
Autism Spectrum Disorder
Child Development Disorders, Pervasive
Neurodevelopmental Disorders
Mental Disorders