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



Status:Recruiting
Conditions:Neurology, Psychiatric, Psychiatric, Autism
Therapuetic Areas:Neurology, Psychiatry / Psychology
Healthy:No
Age Range:5 - 17
Updated:4/4/2019
Start Date:November 21, 2016
End Date:July 6, 2021
Contact:Reference Study ID Number: BP30153 www.roche.com/about_roche/roche_worldwide.htm
Email:global-roche-genentech-trials@gene.com
Phone:888-662-6728 (U.S. and Canada)

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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)

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.


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)
We found this trial at
47
sites
3333 Burnet Avenue # Mlc3008
Cincinnati, Ohio 45229
 1-513-636-4200 
Cincinnati Children's Hospital Medical Center Patients and families from across the region and around the...
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707 North Broadway
Baltimore, Maryland 21205
443-923-9200
Kennedy Krieger Institute While not officially part of Johns Hopkins Medicine, Kennedy Krieger Institute is...
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185 Cambridge Street
Boston, Massachusetts 02114
617-724-5200
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1211 Medical Center Dr
Nashville, Tennessee 37232
(615) 322-5000
Vanderbilt Univ Med Ctr Vanderbilt University Medical Center (VUMC) is a comprehensive healthcare facility dedicated...
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60 Crittenden Blvd # 70
Rochester, New York 14642
(585) 275-2121
University of Rochester The University of Rochester is one of the country's top-tier research universities....
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13123 E 16th Ave
Aurora, Colorado 80045
(720) 777-1234
Children's Hospital Colorado At Children's Hospital Colorado, we see more, treat more and heal more...
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Austin, Texas 78759
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Bellevue, Washington 98007
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300 Longwood Ave
Boston, Massachusetts 02115
(617) 355-6000
Boston Children's Hospital Boston Children's Hospital is a 395-bed comprehensive center for pediatric health care....
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Bothell, Washington 98011
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1300 Morris Park Ave
Bronx, New York 10461
(718) 430-2000
Albert Einstein College of Medicine The Albert Einstein College of Medicine of Yeshiva University is...
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7286 South Yosemite Street
Centennial, Colorado 80112
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1653 W. Congress Parkway
Chicago, Illinois 60612
(312) 942-5000
Rush University Medical Center Rush University Medical Center encompasses a 664-bed hospital serving adults and...
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Cleveland, Ohio 44106
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281 W. Lane Ave
Columbus, Ohio 43210
(614) 292-6446
Ohio State University The Ohio State University’s main Columbus campus is one of America’s largest...
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12655 North Central Expressway
Dallas, Texas 75243
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Dothan, Alabama 36303
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2608 Erwin Road
Durham, North Carolina 27705
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Everett, Washington 98201
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Gainesville, Florida 32607
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Houston, Texas 17115
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Libertyville, Illinois 60048
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Media, Pennsylvania 19063
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720 Southeast Washington Avenue
Minneapolis, Minnesota 55414
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New Haven, Connecticut 06510
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North Miami, Florida 33161
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Oklahoma City, Oklahoma 73116
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Orange, California 92868
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Orange City, Florida 32763
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Richland, Washington 99352
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Rockville, Maryland 20852
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4801 Weldon Spring Parkway
Saint Charles, Missouri 63301
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Staten Island, New York 10312
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Worcester, Massachusetts 01655
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