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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT02024789




Registration number
NCT02024789
Ethics application status
Date submitted
27/12/2013
Date registered
31/12/2013
Date last updated
25/10/2017

Titles & IDs
Public title
A Study of RG1662 in Adults and Adolescents With Down Syndrome (CLEMATIS)
Scientific title
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)
Secondary ID [1] 0 0
2013-001263-23
Secondary ID [2] 0 0
BP27832
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Down Syndrome 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Down's syndrome
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Placebo
Treatment: Drugs - RG1662
Treatment: Drugs - RG1662

Placebo Comparator: Placebo -

Experimental: RG1662 120 mg bid -

Experimental: RG1662 240 mg bid -


Treatment: Drugs: Placebo
Orally twice daily, 26 weeks

Treatment: Drugs: RG1662
120 mg (80 mg for subjects 12 and 13 years of age) orally twice daily, 26 weeks

Treatment: Drugs: RG1662
240 mg (160 mg for subjects 12 and 13 years of age) orally twice daily, 26 weeks
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Cognition as assessed by the Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) sub-tests
Timepoint [1] 0 0
26 weeks
Primary outcome [2] 0 0
Adaptive behavior as assessed by the Vineland Adaptive Behavior Scales-II (VABS-II) standard scores
Timepoint [2] 0 0
26 weeks
Primary outcome [3] 0 0
Clinical global impression as assessed by Clinician Rated Global Improvement (CGI-I) scale
Timepoint [3] 0 0
26 weeks
Secondary outcome [1] 0 0
Incidence of abnormal ECG changes
Timepoint [1] 0 0
26 weeks
Secondary outcome [2] 0 0
Abnormal ECG changes in adolescents as compared to baseline
Timepoint [2] 0 0
from baseline to Week 26
Secondary outcome [3] 0 0
Safety: Incidence of adverse events
Timepoint [3] 0 0
approximately 32 weeks
Secondary outcome [4] 0 0
Incidence of abnormal blood pressure
Timepoint [4] 0 0
26 weeks
Secondary outcome [5] 0 0
RG1662 plasma concentrations
Timepoint [5] 0 0
26 weeks

Eligibility
Key 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
Minimum age
12 Years
Maximum age
30 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
5/05/2014
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
4/05/2016
Sample size
Target
Accrual to date
Final
173
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Georgia
Country [3] 0 0
United States of America
State/province [3] 0 0
Illinois
Country [4] 0 0
United States of America
State/province [4] 0 0
Maryland
Country [5] 0 0
United States of America
State/province [5] 0 0
Massachusetts
Country [6] 0 0
United States of America
State/province [6] 0 0
North Carolina
Country [7] 0 0
United States of America
State/province [7] 0 0
Utah
Country [8] 0 0
United States of America
State/province [8] 0 0
Wisconsin
Country [9] 0 0
Argentina
State/province [9] 0 0
Caba
Country [10] 0 0
Argentina
State/province [10] 0 0
Ciudad Autonoma de Bs As
Country [11] 0 0
Canada
State/province [11] 0 0
Nova Scotia
Country [12] 0 0
France
State/province [12] 0 0
Bron
Country [13] 0 0
France
State/province [13] 0 0
Montpellier
Country [14] 0 0
France
State/province [14] 0 0
Paris
Country [15] 0 0
France
State/province [15] 0 0
St Etienne
Country [16] 0 0
Italy
State/province [16] 0 0
Lazio
Country [17] 0 0
Italy
State/province [17] 0 0
Sardegna
Country [18] 0 0
Mexico
State/province [18] 0 0
Jalisco
Country [19] 0 0
Mexico
State/province [19] 0 0
Aguascalientes
Country [20] 0 0
Mexico
State/province [20] 0 0
Monterrey
Country [21] 0 0
Mexico
State/province [21] 0 0
Queretaro
Country [22] 0 0
New Zealand
State/province [22] 0 0
Auckland
Country [23] 0 0
New Zealand
State/province [23] 0 0
Dunedin
Country [24] 0 0
New Zealand
State/province [24] 0 0
Wellington
Country [25] 0 0
Singapore
State/province [25] 0 0
Singapore
Country [26] 0 0
Spain
State/province [26] 0 0
Girona
Country [27] 0 0
Spain
State/province [27] 0 0
La Coruña
Country [28] 0 0
Spain
State/province [28] 0 0
Barcelona
Country [29] 0 0
Spain
State/province [29] 0 0
Madrid
Country [30] 0 0
United Kingdom
State/province [30] 0 0
Blackpool
Country [31] 0 0
United Kingdom
State/province [31] 0 0
Dartford, Kent
Country [32] 0 0
United Kingdom
State/province [32] 0 0
Doncaster
Country [33] 0 0
United Kingdom
State/province [33] 0 0
Redruth

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Hoffmann-La Roche
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT02024789
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Clinical Trials
Address 0 0
Hoffmann-La Roche
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT02024789