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RG1662

Phase 2

Down Syndrome | Small molecule | Rare Disease |Roche Holding AG|Trials Updated: Oct 25, 2017

RG1662 development status

Highest phase Phase 2
Registered trials 6 across 1 sponsor since Sep 2011

RG1662 target and mechanism

ModalitySmall molecule

Also known as RG1662 tablet

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials3
Total Enrollment221

FDA Designations

No designations recorded

RG1662 clinical trials

RG1662 · 3 trials · 2 indications

Phase 2 1Phase 1 2
NCT02024789A Study of RG1662 in Adults and Adolescents With Down Syndrome (CLEMATIS)Down Syndrome
COMPLETED173 Analytics
PHASE2COMPLETED
A Study of RG1662 in Adults and Adolescents With Down Syndrome (CLEMATIS)
Down Syndrome
NCT02024789173
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Study Endpoints

Primary Endpoints

Cognition as assessed by the Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) sub-tests
26 weeks
Adaptive behavior as assessed by the Vineland Adaptive Behavior Scales-II (VABS-II) standard scores
26 weeks
Clinical global impression as assessed by Clinician Rated Global Improvement (CGI-I) scale
26 weeks
Brain GABAA receptor alpha5 subunit localization/density assessed by positron emission tomography (PET)
approximately 1 day
Brain GABAA receptor alpha5 subunit occupancy following single dose of RG1662
approximately 1 day
Correlation of RG1662 plasma concentrations and GABAAalpha5 receptor occupancy
approximately 12 months
Safety: Incidence of adverse events
16 weeks

Secondary Endpoints

Incidence of abnormal ECG changes
26 weeks
Abnormal ECG changes in adolescents as compared to baseline
from baseline to Week 26
Safety: Incidence of adverse events
approximately 32 weeks
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Study Design & Arms

AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
PlaceboPLACEBO_COMPARATOR -
RG1662 120 mg bidEXPERIMENTAL -
RG1662 240 mg bidEXPERIMENTAL -
RG1662EXPERIMENTAL -
AEXPERIMENTAL -
BPLACEBO_COMPARATOR -

Interventions

NameTypeDescription
PlaceboDRUGOrally twice daily, 26 weeks
RG1662DRUG120 mg (80 mg for subjects 12 and 13 years of age) orally twice daily, 26 weeks
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Eligibility Criteria

Age Range12 Years to 30 Years
SexALL
Healthy VolunteersNo
Study Sites37

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....

Countries:United StatesArgentinaCanadaFranceItalyMexicoNew ZealandSingaporeSpainUnited Kingdom
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Competitive Landscape -Acute Respiratory Distress Syndrome 27 trials (matched to "Down Syndrome")

Top 2 of 7 competitors

CompanyTickerTrialsLead PhaseDrugs
Akebia Therapeutics, Inc.AKBA1PHASE2Vadadustat
Rigel Pharmaceuticals, Inc.RIGL1PHASE2Fostamatinib
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Frequently asked questions about RG1662

What is RG1662?

RG1662 is an investigational small molecule developed by Roche Holding AG for Down Syndrome. It is a tablet formulation that has been studied in Phase 2 clinical trials. RG1662 is not approved for any indication and remains in clinical development.

What is RG1662 used for in Down Syndrome?

RG1662 is being studied as a potential treatment for Down Syndrome. It is not approved for this or any other use. Clinical trials have evaluated RG1662 in individuals with Down Syndrome, including adolescents and adults, to assess its effects.

Who makes RG1662?

RG1662 is developed by Roche Holding AG, which trades under the ticker RHHBY. Roche is the sponsor of the clinical trials investigating RG1662 for Down Syndrome.

What phase is RG1662 in?

RG1662 is in Phase 2 clinical development. It has not been approved by the FDA or any regulatory agency. The Phase 2 trial, known as CLEMATIS, has been completed, and no active trials are currently listed.

What clinical trials is RG1662 in?

RG1662 has been evaluated in three completed clinical trials: NCT02024789 (Phase 2 CLEMATIS), NCT01667367 (Phase 1 imaging study), and NCT01436955 (Phase 1 study). These trials enrolled a total of 221 participants across multiple countries.

Is RG1662 the same as RG1662 tablet?

Yes, RG1662 tablet is another name for RG1662. Both refer to the same investigational small molecule developed by Roche Holding AG for Down Syndrome.

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