Overview

This trial is active, not recruiting.

Condition down syndrome
Treatments placebo, rg1662
Phase phase 2
Sponsor Hoffmann-La Roche
Start date October 2015
End date August 2016
Trial size 45 participants
Trial identifier NCT02484703, WP28760

Summary

This study will evaluate the safety, tolerability, efficacy, and pharmacokinetic (PK) and pharmacodynamic (PD) activity of 3 different dosages of RG1662 compared with placebo. Each arm will enroll approximately 9 children with Down syndrome between the ages of 6 and 11 years to receive treatment for up to 26 weeks.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking double blind (subject, investigator)
Primary purpose treatment
Arm
(Placebo Comparator)
Participants will receive matching placebo by mouth (PO) twice daily (BID) for up to 26 weeks.
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)
Participants will receive RG1662 at a dosage of 120 milligrams (mg) PO BID for up to 26 weeks.
rg1662
Participants will receive 1 of 3 dosages of RG1662 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)
Participants will receive RG1662 at a dosage of 40 mg PO BID for up to 26 weeks.
rg1662
Participants will receive 1 of 3 dosages of RG1662 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)
Participants will receive RG1662 at a dosage of 60 mg PO BID for up to 26 weeks.
rg1662
Participants will receive 1 of 3 dosages of RG1662 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 Outcomes

Measure
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

Secondary Outcomes

Measure
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

Eligibility Criteria

Male or female participants from 6 years up to 11 years old.

Inclusion Criteria: - Children 6 to 11 years of age - Diagnosis of Down syndrome, except for mosaic Down syndrome - Available parent or caregiver to attent 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 or change in antiepileptic regimen within 12 months prior to Screening - 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

Additional Information

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 RG1662 in Children With Down Syndrome Aged 6 to 11 Years
Trial information was received from ClinicalTrials.gov and was last updated in August 2016.
Information provided to ClinicalTrials.gov by Hoffmann-La Roche.