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

Primary Purpose

Down Syndrome

Status
Terminated
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Placebo
RO5186582
Sponsored by
Hoffmann-La Roche
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Down Syndrome

Eligibility Criteria

6 Years - 11 Years (Child)All SexesDoes not accept healthy volunteers

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)

Sites / Locations

  • Southwest Autism Research & Resource Center
  • Emory University School of Medicine; Department of Human Genetics & Pediatrics
  • Rush University Medical Center
  • Massachusette General Hospital; Medical Genetics
  • Boston Children's Hospital, Department of Neurology
  • Duke Clin Rsch Institute
  • UPMC Western Psychiatric Institute and Clinic
  • Vanderbilt University Medical Center
  • University of Wisconsin Hospital and Clinics

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Placebo Comparator

Experimental

Experimental

Experimental

Arm Label

Placebo

RO5186582 120 mg BID

RO5186582 40 mg BID

RO5186582 60 mg BID

Arm Description

Participants will receive matching placebo by mouth (PO) twice daily (BID) for up to 26 weeks.

Participants will receive RO5186582 at a dosage of 120 milligrams (mg) PO BID for up to 26 weeks.

Participants will receive RO5186582 at a dosage of 40 mg PO BID for up to 26 weeks.

Participants will receive RO5186582 at a dosage of 60 mg PO BID for up to 26 weeks.

Outcomes

Primary Outcome Measures

Percentage of Participants With Adverse Events (AEs)
Percentage of Participants With Epileptiform Abnormalities as Assessed Using Electroencephalogram (EEG) Analysis
Percentage of Participants by Suicidality Classification as Assessed Using an Adapted Form of the Columbia Classification Algorithm for Suicide Assessment (C-CASA)
Anxiety, Depression, and Mood Scale (ADAMS) Score
Hyperactivity and Impulsivity as Assessed by the Short Version of Conners Third Edition Parent Short-Form (Conners-3) Score
Sleep Disturbances as Assessed by the Children's Sleep Habits Questionnaire (CSHQ) Score
Gamma Power at Posterior Electrodes as Assessed Using EEG Analysis
Theta Power at Posterior Electrodes as Assessed Using EEG Analysis
Cognition as Assessed by the Children's Memory Scale (CMS) Subtests Score

Secondary Outcome Measures

Percentage of Participants With AEs
Percentage of Participants With Epileptiform Abnormalities as Assessed Using EEG Analysis
Percentage of Participants by Suicidality Classification as Assessed Using an Adapted Form of the C-CASA
ADAMS Score
Hyperactivity and Impulsivity as Assessed by the Short Version of Conners-3 Score
Sleep Disturbances as Assessed by the CSHQ Score
Gamma Power at Posterior Electrodes as Assessed Using EEG Analysis
Theta Power at Posterior Electrodes as Assessed Using EEG Analysis
Cognition as Assessed by the CMS Subtests Score
Adaptive Behavior as Assessed by the Vineland Adaptive Behavior Scales-II (VABS-II) Score
Clinical Global Impression-Improvement (CGI-I) Scale Score
Daily Functional Memory as Assessed by the Observer Memory Questionnaire-Parent Form (OMQ-PF) Score
Intellectual Quotient (IQ) as Assessed by the Leiter 3
Plasma Concentration of RO5186582

Full Information

First Posted
June 16, 2015
Last Updated
July 4, 2017
Sponsor
Hoffmann-La Roche
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1. Study Identification

Unique Protocol Identification Number
NCT02484703
Brief Title
A Study of RO5186582 in Down Syndrome Among Children 6 to 11 Years of Age
Official Title
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
Study Type
Interventional

2. Study Status

Record Verification Date
July 2017
Overall Recruitment Status
Terminated
Why Stopped
Study was terminated due to data from another study showing no efficacy. There were no safety issues of concern.
Study Start Date
October 28, 2015 (Actual)
Primary Completion Date
August 3, 2016 (Actual)
Study Completion Date
August 3, 2016 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Hoffmann-La Roche

4. Oversight

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Down Syndrome

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
45 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Participants will receive matching placebo by mouth (PO) twice daily (BID) for up to 26 weeks.
Arm Title
RO5186582 120 mg BID
Arm Type
Experimental
Arm Description
Participants will receive RO5186582 at a dosage of 120 milligrams (mg) PO BID for up to 26 weeks.
Arm Title
RO5186582 40 mg BID
Arm Type
Experimental
Arm Description
Participants will receive RO5186582 at a dosage of 40 mg PO BID for up to 26 weeks.
Arm Title
RO5186582 60 mg BID
Arm Type
Experimental
Arm Description
Participants will receive RO5186582 at a dosage of 60 mg PO BID for up to 26 weeks.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
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).
Intervention Type
Drug
Intervention Name(s)
RO5186582
Intervention Description
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).
Primary Outcome Measure Information:
Title
Percentage of Participants With Adverse Events (AEs)
Time Frame
Baseline up to Week 6
Title
Percentage of Participants With Epileptiform Abnormalities as Assessed Using Electroencephalogram (EEG) Analysis
Time Frame
Baseline up to Week 6
Title
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
Title
Anxiety, Depression, and Mood Scale (ADAMS) Score
Time Frame
Baseline up to Week 6
Title
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
Title
Sleep Disturbances as Assessed by the Children's Sleep Habits Questionnaire (CSHQ) Score
Time Frame
Baseline up to Week 6
Title
Gamma Power at Posterior Electrodes as Assessed Using EEG Analysis
Time Frame
Baseline up to Week 6
Title
Theta Power at Posterior Electrodes as Assessed Using EEG Analysis
Time Frame
Baseline up to Week 6
Title
Cognition as Assessed by the Children's Memory Scale (CMS) Subtests Score
Time Frame
Baseline up to Week 6
Secondary Outcome Measure Information:
Title
Percentage of Participants With AEs
Time Frame
Baseline up to Week 26
Title
Percentage of Participants With Epileptiform Abnormalities as Assessed Using EEG Analysis
Time Frame
Baseline up to Week 26
Title
Percentage of Participants by Suicidality Classification as Assessed Using an Adapted Form of the C-CASA
Time Frame
Baseline up to Week 26
Title
ADAMS Score
Time Frame
Baseline up to Week 26
Title
Hyperactivity and Impulsivity as Assessed by the Short Version of Conners-3 Score
Time Frame
Baseline up to Week 26
Title
Sleep Disturbances as Assessed by the CSHQ Score
Time Frame
Baseline up to Week 26
Title
Gamma Power at Posterior Electrodes as Assessed Using EEG Analysis
Time Frame
Baseline up to Week 26
Title
Theta Power at Posterior Electrodes as Assessed Using EEG Analysis
Time Frame
Baseline up to Week 26
Title
Cognition as Assessed by the CMS Subtests Score
Time Frame
Baseline up to Week 26
Title
Adaptive Behavior as Assessed by the Vineland Adaptive Behavior Scales-II (VABS-II) Score
Time Frame
Baseline up to Week 26
Title
Clinical Global Impression-Improvement (CGI-I) Scale Score
Time Frame
Baseline up to Week 26
Title
Daily Functional Memory as Assessed by the Observer Memory Questionnaire-Parent Form (OMQ-PF) Score
Time Frame
Baseline up to Week 26
Title
Intellectual Quotient (IQ) as Assessed by the Leiter 3
Time Frame
Baseline up to Week 26
Title
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

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
11 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Clinical Trials
Organizational Affiliation
Hoffmann-La Roche
Official's Role
Study Director
Facility Information:
Facility Name
Southwest Autism Research & Resource Center
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85006
Country
United States
Facility Name
Emory University School of Medicine; Department of Human Genetics & Pediatrics
City
Decatur
State/Province
Georgia
ZIP/Postal Code
30033
Country
United States
Facility Name
Rush University Medical Center
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Facility Name
Massachusette General Hospital; Medical Genetics
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Facility Name
Boston Children's Hospital, Department of Neurology
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Duke Clin Rsch Institute
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
UPMC Western Psychiatric Institute and Clinic
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15203 1101
Country
United States
Facility Name
Vanderbilt University Medical Center
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States
Facility Name
University of Wisconsin Hospital and Clinics
City
Madison
State/Province
Wisconsin
ZIP/Postal Code
53792
Country
United States

12. IPD Sharing Statement

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

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