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A Study of RO5186582 in Down Syndrome Among Children 6 to 11 Years of Age

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ClinicalTrials.gov Identifier: NCT02484703
Recruitment Status : Terminated (Study was terminated due to data from another study showing no efficacy. There were no safety issues of concern.)
First Posted : June 30, 2015
Last Update Posted : July 6, 2017
Sponsor:
Information provided by (Responsible Party):
Hoffmann-La Roche

Tracking Information
First Submitted Date  ICMJE June 16, 2015
First Posted Date  ICMJE June 30, 2015
Last Update Posted Date July 6, 2017
Actual Study Start Date  ICMJE October 28, 2015
Actual Primary Completion Date August 3, 2016   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 23, 2017)
  • Percentage of Participants With Adverse Events (AEs) [ Time Frame: Baseline up to Week 6 ]
  • Percentage of Participants With Epileptiform Abnormalities as Assessed Using Electroencephalogram (EEG) Analysis [ Time Frame: Baseline up to Week 6 ]
  • Percentage of Participants by Suicidality Classification as Assessed Using an Adapted Form of the Columbia Classification Algorithm for Suicide Assessment (C-CASA) [ Time Frame: Baseline up to Week 6 ]
  • Anxiety, Depression, and Mood Scale (ADAMS) Score [ Time Frame: Baseline up to Week 6 ]
  • Hyperactivity and Impulsivity as Assessed by the Short Version of Conners Third Edition Parent Short-Form (Conners-3) Score [ Time Frame: Baseline up to Week 6 ]
  • Sleep Disturbances as Assessed by the Children's Sleep Habits Questionnaire (CSHQ) Score [ Time Frame: Baseline up to Week 6 ]
  • Gamma Power at Posterior Electrodes as Assessed Using EEG Analysis [ Time Frame: Baseline up to Week 6 ]
  • Theta Power at Posterior Electrodes as Assessed Using EEG Analysis [ Time Frame: Baseline up to Week 6 ]
  • Cognition as Assessed by the Children's Memory Scale (CMS) Subtests Score [ Time Frame: Baseline up to Week 6 ]
Original Primary Outcome Measures  ICMJE
 (submitted: June 24, 2015)
  • Incidence of treatment discontinuation due to AEs [ Time Frame: Cumulative from each visit until up to 42 days after last dose ]
  • Incidence of adverse events (AEs) [ Time Frame: Cumulative from each visit until up to 42 days after last dose ]
  • Incidence of epileptiform abnormalities as assessed using electroencephalogram (EEG) analysis [ Time Frame: Cumulative at Screening, Baseline, and during Weeks 2, 6, and 26 ]
  • Change from Baseline in suicidality as assessed using an adapted form of the Columbia Classification Algorithm for Suicide Assessment (C-CASA) [ Time Frame: During each visit until up to 42 days after last dose ]
  • Change from Baseline in Anxiety, Depression, and Mood Scale (ADAMS) score [ Time Frame: Baseline, during Weeks 6 and 26, and up to 42 days after last dose ]
  • Change from Baseline in Conners Third Edition Parent Short-Form (Conners-3) score [ Time Frame: Baseline, during Weeks 6 and 26, and up to 42 days after last dose ]
  • Change from Baseline in Children's Sleep Habits Questionnaire (CSHQ) score [ Time Frame: Baseline, during Weeks 6 and 26, and up to 14 days after last dose ]
Change History Complete list of historical versions of study NCT02484703 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: March 23, 2017)
  • Percentage of Participants With AEs [ Time Frame: Baseline up to Week 26 ]
  • Percentage of Participants With Epileptiform Abnormalities as Assessed Using EEG Analysis [ Time Frame: Baseline up to Week 26 ]
  • Percentage of Participants by Suicidality Classification as Assessed Using an Adapted Form of the C-CASA [ Time Frame: Baseline up to Week 26 ]
  • ADAMS Score [ Time Frame: Baseline up to Week 26 ]
  • Hyperactivity and Impulsivity as Assessed by the Short Version of Conners-3 Score [ Time Frame: Baseline up to Week 26 ]
  • Sleep Disturbances as Assessed by the CSHQ Score [ Time Frame: Baseline up to Week 26 ]
  • Gamma Power at Posterior Electrodes as Assessed Using EEG Analysis [ Time Frame: Baseline up to Week 26 ]
  • Theta Power at Posterior Electrodes as Assessed Using EEG Analysis [ Time Frame: Baseline up to Week 26 ]
  • Cognition as Assessed by the CMS Subtests Score [ Time Frame: Baseline up to Week 26 ]
  • Adaptive Behavior as Assessed by the Vineland Adaptive Behavior Scales-II (VABS-II) Score [ Time Frame: Baseline up to Week 26 ]
  • Clinical Global Impression-Improvement (CGI-I) Scale Score [ Time Frame: Baseline up to Week 26 ]
  • Daily Functional Memory as Assessed by the Observer Memory Questionnaire-Parent Form (OMQ-PF) Score [ Time Frame: Baseline up to Week 26 ]
  • Intellectual Quotient (IQ) as Assessed by the Leiter 3 [ Time Frame: Baseline up to Week 26 ]
  • Plasma Concentration of RO5186582 [ Time Frame: Predose (2 predose samples separated by at least 1 hour) at Weeks 2 and 6; and predose or postdose (as convenient) during Weeks 10, 17, and 26 ]
Original Secondary Outcome Measures  ICMJE
 (submitted: June 24, 2015)
Plasma concentration of RG1662 [ Time Frame: Pre-dose at Weeks 2 and 6; and pre-dose or post-dose (as convenient) during Weeks 10, 17, and 26 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study of RO5186582 in Down Syndrome Among Children 6 to 11 Years of Age
Official Title  ICMJE A Randomized, Double-Blind, Placebo-Controlled, Parallel Group 26-Week Dose-Investigating Study to Explore the Pharmacokinetics, Pharmacodynamic Effects, Efficacy, Safety and Tolerability of RO5186582 in Children With Down Syndrome Aged 6-11 Years
Brief Summary This study will evaluate the safety, tolerability, efficacy, and pharmacokinetic and pharmacodynamic activity of 3 different dosages of RO5186582 compared with placebo. A total of approximately 46 participants will be enrolled, in order to have at least 32 evaluable, and will be randomly assigned to 1 of 4 treatments in a 1:1:1:1 ratio, with 9 children per treatment arm. The target ratio between 6-8 years and 9-11 years age groups is approximately 1:1 in each treatment arm, with a minimum of 3 children per age group in each treatment arm.
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 Down Syndrome
Intervention  ICMJE
  • Drug: Placebo
    Participants will receive matching placebo PO BID. Study medication will first be administered on Day 1, and only the morning dose will be given on the last day of treatment (Week 26).
  • Drug: RO5186582
    Participants will receive 1 of 3 dosages of RO5186582 PO BID, including 40 mg, 60 mg, or 120 mg. Study medication will first be administered on Day 1, and only the morning dose will be given on the last day of treatment (Week 26).
Study Arms  ICMJE
  • Placebo Comparator: Placebo
    Participants will receive matching placebo by mouth (PO) twice daily (BID) for up to 26 weeks.
    Intervention: Drug: Placebo
  • Experimental: RO5186582 120 mg BID
    Participants will receive RO5186582 at a dosage of 120 milligrams (mg) PO BID for up to 26 weeks.
    Intervention: Drug: RO5186582
  • Experimental: RO5186582 40 mg BID
    Participants will receive RO5186582 at a dosage of 40 mg PO BID for up to 26 weeks.
    Intervention: Drug: RO5186582
  • Experimental: RO5186582 60 mg BID
    Participants will receive RO5186582 at a dosage of 60 mg PO BID for up to 26 weeks.
    Intervention: Drug: RO5186582
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 Terminated
Actual Enrollment  ICMJE
 (submitted: August 1, 2016)
45
Original Estimated Enrollment  ICMJE
 (submitted: June 24, 2015)
36
Actual Study Completion Date  ICMJE August 3, 2016
Actual Primary Completion Date August 3, 2016   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Diagnosis of Down syndrome, except for mosaic Down syndrome
  • Available parent or caregiver to attend clinic visits and provide information about the participant's behavior and symptoms

Exclusion Criteria:

  • Any primary psychiatric comorbid disorder
  • History of infantile spasms, West syndrome, Lennox-Gastaut syndrome, early infantile epileptic encephalopathy, treatment-refractory epilepsy with cognitive/developmental regression, severe head trauma, or central nervous system (CNS) infection
  • Seizure event of any type within 12 months prior to Screening or relevant changes in anti-epileptic drugs 6 weeks prior to enrollment
  • Significant sleep disruption
  • Significant gastrointestinal, renal, hepatic, endocrine, or cardiovascular disease
  • New-onset or ongoing hematologic/oncologic disorder
  • Severe lactose intolerance
  • Participation in another clinical study within 1 month or 6 half-lives prior to first dose, or any extent of participation in Study BP29589 (NCT02451657)
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 6 Years to 11 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02484703
Other Study ID Numbers  ICMJE WP28760
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 July 2017

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