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Trial record 24 of 176 for:    "Down syndrome"

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

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.

Condition or disease Intervention/treatment Phase
Down Syndrome Drug: Placebo Drug: RO5186582 Phase 2

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 45 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
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
Actual Study Start Date : October 28, 2015
Actual Primary Completion Date : August 3, 2016
Actual Study Completion Date : August 3, 2016

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Down Syndrome

Arm Intervention/treatment
Placebo Comparator: Placebo
Participants will receive matching placebo by mouth (PO) twice daily (BID) for up to 26 weeks.
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).

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

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

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




Primary Outcome Measures :
  1. Percentage of Participants With Adverse Events (AEs) [ Time Frame: Baseline up to Week 6 ]
  2. Percentage of Participants With Epileptiform Abnormalities as Assessed Using Electroencephalogram (EEG) Analysis [ Time Frame: Baseline up to Week 6 ]
  3. 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 ]
  4. Anxiety, Depression, and Mood Scale (ADAMS) Score [ Time Frame: Baseline up to Week 6 ]
  5. 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 ]
  6. Sleep Disturbances as Assessed by the Children's Sleep Habits Questionnaire (CSHQ) Score [ Time Frame: Baseline up to Week 6 ]
  7. Gamma Power at Posterior Electrodes as Assessed Using EEG Analysis [ Time Frame: Baseline up to Week 6 ]
  8. Theta Power at Posterior Electrodes as Assessed Using EEG Analysis [ Time Frame: Baseline up to Week 6 ]
  9. Cognition as Assessed by the Children's Memory Scale (CMS) Subtests Score [ Time Frame: Baseline up to Week 6 ]

Secondary Outcome Measures :
  1. Percentage of Participants With AEs [ Time Frame: Baseline up to Week 26 ]
  2. Percentage of Participants With Epileptiform Abnormalities as Assessed Using EEG Analysis [ Time Frame: Baseline up to Week 26 ]
  3. Percentage of Participants by Suicidality Classification as Assessed Using an Adapted Form of the C-CASA [ Time Frame: Baseline up to Week 26 ]
  4. ADAMS Score [ Time Frame: Baseline up to Week 26 ]
  5. Hyperactivity and Impulsivity as Assessed by the Short Version of Conners-3 Score [ Time Frame: Baseline up to Week 26 ]
  6. Sleep Disturbances as Assessed by the CSHQ Score [ Time Frame: Baseline up to Week 26 ]
  7. Gamma Power at Posterior Electrodes as Assessed Using EEG Analysis [ Time Frame: Baseline up to Week 26 ]
  8. Theta Power at Posterior Electrodes as Assessed Using EEG Analysis [ Time Frame: Baseline up to Week 26 ]
  9. Cognition as Assessed by the CMS Subtests Score [ Time Frame: Baseline up to Week 26 ]
  10. Adaptive Behavior as Assessed by the Vineland Adaptive Behavior Scales-II (VABS-II) Score [ Time Frame: Baseline up to Week 26 ]
  11. Clinical Global Impression-Improvement (CGI-I) Scale Score [ Time Frame: Baseline up to Week 26 ]
  12. Daily Functional Memory as Assessed by the Observer Memory Questionnaire-Parent Form (OMQ-PF) Score [ Time Frame: Baseline up to Week 26 ]
  13. Intellectual Quotient (IQ) as Assessed by the Leiter 3 [ Time Frame: Baseline up to Week 26 ]
  14. 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 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   6 Years to 11 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02484703


Locations
United States, Arizona
Southwest Autism Research & Resource Center
Phoenix, Arizona, United States, 85006
United States, Georgia
Emory University School of Medicine; Department of Human Genetics & Pediatrics
Decatur, Georgia, United States, 30033
United States, Illinois
Rush University Medical Center
Chicago, Illinois, United States, 60612
United States, Massachusetts
Massachusette General Hospital; Medical Genetics
Boston, Massachusetts, United States, 02114
Boston Children's Hospital, Department of Neurology
Boston, Massachusetts, United States, 02115
United States, North Carolina
Duke Clin Rsch Institute
Durham, North Carolina, United States, 27710
United States, Pennsylvania
UPMC Western Psychiatric Institute and Clinic
Pittsburgh, Pennsylvania, United States, 15203 1101
United States, Tennessee
Vanderbilt University Medical Center
Nashville, Tennessee, United States, 37232
United States, Wisconsin
University of Wisconsin Hospital and Clinics
Madison, Wisconsin, United States, 53792
Sponsors and Collaborators
Hoffmann-La Roche
Investigators
Study Director: Clinical Trials Hoffmann-La Roche

Responsible Party: Hoffmann-La Roche
ClinicalTrials.gov Identifier: NCT02484703     History of Changes
Other Study ID Numbers: WP28760
First Posted: June 30, 2015    Key Record Dates
Last Update Posted: July 6, 2017
Last Verified: July 2017

Additional relevant MeSH terms:
Down Syndrome
Syndrome
Disease
Pathologic Processes
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn