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Trial record 9 of 31 for:    Developmental Disabilities | ( Map: Alabama, United States )

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

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ClinicalTrials.gov Identifier: NCT02901431
Recruitment Status : Recruiting
First Posted : September 15, 2016
Last Update Posted : October 8, 2019
Sponsor:
Information provided by (Responsible Party):
Hoffmann-La Roche

Tracking Information
First Submitted Date  ICMJE September 12, 2016
First Posted Date  ICMJE September 15, 2016
Last Update Posted Date October 8, 2019
Actual Study Start Date  ICMJE November 21, 2016
Estimated Primary Completion Date July 6, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 12, 2018)
  • Change From Baseline in Vineland-II Adaptive Behavior Scale Two Domain Composite (2DC) Score at Week 24 [ Time Frame: Baseline, Week 24 ]
  • Number of Participants With Adverse events [ Time Frame: Baseline to Week 30 ]
Original Primary Outcome Measures  ICMJE
 (submitted: September 12, 2016)
  • 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 ]
Change History Complete list of historical versions of study NCT02901431 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: November 12, 2018)
  • Change from Baseline in Vineland-II Composite Standard Score After 12 Weeks and 24 Weeks of Treatment [ Time Frame: Baseline, Weeks 12 and 24 ]
  • 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 ]
  • 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 ]
  • 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 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 ]
  • 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 ]
  • 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 ]
Original Secondary Outcome Measures  ICMJE
 (submitted: September 12, 2016)
  • 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 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study to Investigate the Efficacy and Safety of Balovaptan (RO5285119) in Participants With Autism Spectrum Disorder (ASD)
Official Title  ICMJE 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)
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.

Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Condition  ICMJE Autism Spectrum Disorder
Intervention  ICMJE
  • Drug: Placebo
    Participants will receive a matching placebo orally. 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 balovaptan (RO5285119). Approximate treatment duration will be up to 24 weeks (up to 52 additional weeks for those enrolled in the OLE).
Study Arms  ICMJE
  • Placebo Comparator: Placebo
    Participants will receive a matching placebo orally. Approximate treatment duration will be up to 24 weeks.
    Intervention: Drug: Placebo
  • 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).
    Intervention: Drug: RO5285119
  • 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.
    Intervention: Drug: RO5285119
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: March 7, 2019)
340
Original Estimated Enrollment  ICMJE
 (submitted: September 12, 2016)
300
Estimated Study Completion Date  ICMJE July 6, 2021
Estimated Primary Completion Date July 6, 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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)
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 5 Years to 17 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
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
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02901431
Other Study ID Numbers  ICMJE BP30153
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Hoffmann-La Roche
Study Sponsor  ICMJE Hoffmann-La Roche
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Clinical Trials Hoffmann-La Roche
PRS Account Hoffmann-La Roche
Verification Date October 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP