Autism Spectrum Disorder MedDRA version: 21.1 Level: PT Classification code 10063844 Term: Autism spectrum disorder System Organ Class: 10037175 - Psychiatric disorders MedDRA version: 20.0 Level: LLT Classification code 10003805 Term: Autism System Organ Class: 10037175 - Psychiatric disorders MedDRA version: 21.1 Level: LLT Classification code 10034739 Term: Pervasive developmental disorder NOS System Organ Class: 10037175 - Psychiatric disorders
Conditions
Interventions
Product Name: Balovaptan
Product Code: RO5285119
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: Balovaptan
Current Sponsor code: RO5285119
Other descriptive name: v1a(2)
Concentration un
Sponsors
Roche Farma S.A(Soc. Unipersonal) que realiza el ensayo en España y actúa como representante de F. Hoffmann-La Roche LTD
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Males or females, age 18 years or older - Subject meets the Diagnostic and Statistical Manual of Mental Disorders, Version 5 (DSM-5) criteria for ASD for an autism diagnosis and is confirmed using Autism Diagnostic Observation Schedule (ADOS)-2 criteria - Social Responsiveness Scale (SRS)-2, proxy version, total t score >=66 at screening - A full scale IQ score >=70 on the Wechsler Abbreviated Scale of Intelligence®-II - Ability and willingness to fully comply with study visit schedule and regular assessments and fluency in the language of the site - Subject’s participation in the study or discontinuation of prohibited medication will not pose undue risks to the subject, in the investigator’s opinion - Subject has an appropriate study partner, in the opinion of the investigator - For women of childbearing potential: agreement to remain abstinent or use a contraceptive method with a failure rate of =65 years) yes F.1.3.1 Number of subjects for this age range 1
Exclusion criteria
Exclusion criteria: General Exclusion Criteria - Pregnancy or breastfeeding, or intention to become pregnant during the study Neurologic and Psychiatric Exclusion Criteria -Previous initiation of new or major change in psychosocial intervention within 6 weeks prior to screening - Unstable or uncontrolled clinically significant affective or psychotic disorders and/or neurologic disorder that may interfere with the assessment of safety or efficacy endpoints - Alcohol or substance abuse or dependence disorder during the last 12 months, as defined using the DSM-5 criteria - Significant risk for suicidal behavior, in the opinion of the investigator - Epilepsy or seizure disorder considered not well controlled within the past 6 months or changes in anticonvulsive therapy within the last 6 months - Clinical diagnosis of peripheral neuropathy Exclusions Related to Cardiovascular Disorders - Within the last 2 years, unstable or clinically significant cardiovascular disease - Uncontrolled hypertension Exclusions Related to Other Organ Systems - Positive serology results for hepatitis B surface antigen (HBsAg), hepatitis C virus (HCV) antibody, or human immunodeficiency virus (HIV) 1 or 2 - History of coagulopathies, bleeding disorders, blood dyscrasias, hematological malignancies, myelosuppression, or current major bleeding event - Concomitant disease or condition that could interfere with, or treatment of which might interfere with, the conduct of the study, or what would, in the opinion of the investigator, pose an unacceptable risk to the subject in this study - Confirmed clinically significant abnormality in parameters of hematology, clinical chemistry, coagulation, or urinalysis - Medical history of malignancy, if not considered cured Additional Exclusion Criteria - Allowed medications have not been stable for 12 weeks prior to screening - Previous treatment with prohibited medications or herbal remedies within 2 weeks prior to randomization or 5 half-lives - Blood donation or loss of blood > 500 mL within 3 months prior to randomization - Previous participation in an investigational drug or device study within 60 days prior to randomization or previous enrollment in investigational trials of balovaptan
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the efficacy of 10 mg of balovaptan compared with placebo based on the Vineland TM-II two-domain composite (2DC) score;Primary end point(s): 1. Change from baseline at Week 24 on the Vineland TM -II 2DC score;Secondary Objective: To evaluate the efficacy of 10 mg of balovaptan compared with placebo based on the Vineland-II 2DC score, Pediatric Quality of Life Inventory TM Generic Core Scales(PedsQLTM), Version 4.0, summary and total scores, Vineland-II composite standard score, Vineland-II Socialization, Communication, Daily Living Skills domain standard scores, Clinical Global Impressions(CGI) –Severity(S) and Improvement(I), Hamilton Anxiety Rating Scale (HAM-A) total and domain scores;Timepoint(s) of evaluation of this end point: 1. Baseline (Day 1) and Week 24 | — |
Secondary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: 1. Baseline and Week 12 2-6. Baseline, Week 12 and Week 24 7-8. Week 12 and Week 24 9-10. Baseline, Week 12 and Week 24;Secondary end point(s): 1. Change from baseline at Week 12 on the Vineland-II 2DC score 2. Change from baseline at Weeks 12 and 24 in the PedsQLTM Core module, Version 4.0, on summary and total scores 3. Change from baseline at Weeks 12 and 24 in the Vineland-II a composite standard score 4. Change from baseline at Weeks 12 and 24 in Vineland-II Socialization domain standard score 5. Change from baseline at Weeks 12 and 24 in Vineland-II Communication domain standard score 6. Change from baseline at Weeks 12 and 24 in Vineland-II Daily Living Skills domain standard score 7. Change from baseline in severity of clinical impressions as measured by CGI-S after 12 weeks and 24 weeks of treatment 8. Improvements in clinical impressions, as measured by CGI-I after 12 weeks and 24 weeks of treatment 9. Change from baseline in the HAM-A total and domain scores at Weeks 12 and 24 10. Proportion of subjects with a >=6-point improvement in Vineland-II 2DC score at Weeks 12 and 24 | — |
Countries
Canada, France, Germany, Italy, Spain, United Kingdom, United States
Contacts
Public ContactTrial Information Support Line-TISL
F. Hoffmann-La Roche Ltd
Outcome results
None listed