A MULTI-CENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED PHASE 2 STUDY OF THE EFFICACY, SAFETY AND TOLERABILITY OF RG1662 IN ADULTS AND ADOLESCENTS WITH DOWN SYNDROME (CLEMATIS)

Hoffmann-La Roche37 sites in 10 countries173 target enrollmentMay 5, 2014

ConditionsDown Syndrome

InterventionsPlacebo RG1662

DrugsPlacebo RG1662

Overview

Phase: Phase 2
Intervention: Placebo
Conditions: Down Syndrome
Sponsor: Hoffmann-La Roche
Enrollment: 173
Locations: 37
Primary Endpoint: Cognition as assessed by the Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) sub-tests
Status: Completed
Last Updated: 8 years ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

This multi-center, randomized, double-blind, 3-arm, parallel-group, placebo-controlled study will evaluate the efficacy and safety of RG1662 in adults and adolescents with Down syndrome. Subjects will be randomized to receive RG1662 either at low or high dose or placebo orally twice daily for 26 weeks.

Registry

clinicaltrials.gov

Start Date

May 5, 2014

End Date

May 4, 2016

Last Updated

8 years ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

Hoffmann-La Roche

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Individuals aged 12-30 years of age inclusive
•Clinical diagnosis of Down syndrome (trisomy 21) confirmed by chromosomal analysis (karyotyping)
•Males, or non-pregnant, non-lactating females. For females of childbearing potential, strict contraceptive prevention is required.
•Body-mass Index (BMI) 18-42 and 15-30 kg/m2 inclusive for adults and adolescents respectively
•Ability to complete the Clinical Evaluation of Language Fundamentals (CELF)-preschool 2 word classes task
•Subjects must have a parent, or other reliable caregiver who agrees to accompany the subject to all clinic visits, provide information about the subject as required by the protocol, and ensure compliance with the medication schedule
•Study participants must have sufficient language, vision and hearing to participate in study evaluations, as judged clinically by investigator

Exclusion Criteria

•Subjects with a current DSM 5 diagnosis of any primary psychiatric diagnosis (including ASD or MDD)
•Subjects with a history of infantile spasms, of West syndrome, Lennox-Gastaut syndrome, Early Infantile Epileptic Encephalopathy or any treatment-refractory epilepsy associated with cognitive or developmental regression, of severe head trauma or CNS infections (e.g. meningitis)
•Subjects with a known or suspected clinical seizure event of any type within 24 months prior to screening
•Clinically relevant ECG abnormalities at screening or baseline; QTcF above 450 ms; personal or family history (first degree relatives) of congenital long QT syndrome
•Inadequate renal or hepatic function

Arms & Interventions

Placebo

Intervention: Placebo

RG1662 120 mg bid

Intervention: RG1662

RG1662 240 mg bid

Intervention: RG1662

Outcomes

Primary Outcomes

Cognition as assessed by the Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) sub-tests

Time Frame: 26 weeks

Adaptive behavior as assessed by the Vineland Adaptive Behavior Scales-II (VABS-II) standard scores

Time Frame: 26 weeks

Clinical global impression as assessed by Clinician Rated Global Improvement (CGI-I) scale

Time Frame: 26 weeks

Secondary Outcomes

Abnormal ECG changes in adolescents as compared to baseline(from baseline to Week 26)
Safety: Incidence of adverse events(approximately 32 weeks)
RG1662 plasma concentrations(26 weeks)
Incidence of abnormal blood pressure(26 weeks)
Incidence of abnormal ECG changes(26 weeks)