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RG1662

Phase 2

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

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials2
Total Enrollment208

FDA Designations

No designations recorded

Clinical trial landscape

RG1662 · 6 trials · 4 indications

Phase 2 1Phase 1 5
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 SyndromeUnlock trial analytics

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
Area under the plasma concentration-time curve from 0-t (AUC0-t)
Within 29 days
Maximum observed plasma concentration (Cmax)
Within 29 days
Time to maximum observed plasma concentration (Tmax)
Within 29 days
Pharmacokinetic parameters of metformin derived from plasma and urine concentrations of metformin: area under the concentration-time curve (AUC), maximum concentration (Cmax), renal clearance (CLr) [composite outcome measure]
Up to 9 weeks
Pharmacokinetics: Area under the concentration time curve (AUC)
Selected Days between 1 and 28
Pharmacokinetics: maximum serum concentration
Selected Days between 1 and 28
Pharmacokinetics: Clearance of 13C-RG1662
Days 1 and 28
Pharmacokinetics: Bioavailability of 13C-RG1662
Days 1 and 28
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 -
Combination therapy RG1662 + itraconazoleEXPERIMENTALDays 20-29: Oral administration RG1662 twice daily within 30 minutes of a meal + 2 x 100 mg itraconazole once daily with food
RG1662 MonotherapyEXPERIMENTALDays 1-10: RG1662 120 mg twice daily (b.i.d.) within 30 minutes of a meal for 10 days (Days 1 to 9, Day 10 only a.m. dose). (Cohort A subjects will receive 1 x 120 mg RG1662 tablets twice daily. In Cohorts B and C the dose of RG1662 will be decided upon following review of the interim safety and pharmacokinetic data of the 4 subjects in Cohort A.)
itraconazole MonotherapyEXPERIMENTALDays 15-19: 200 mg of itraconazole twice daily for 5 days (Days 15 to 18, Day 19 only a.m. dose)
RG1662 plus metforminEXPERIMENTAL -
metformin aloneEXPERIMENTAL -
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
itraconazoleDRUG -
Omnipaque 300OTHER647 mg/mL of iohexol, equivalent to 300 mg/mL of iodine, used for assessment of glomerular function and other measure of renal function
metforminDRUGSingle 850 mg dose of metformin
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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 KingdomNetherlands
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Frequently asked questions about RG1662

What is RG1662 used for?

RG1662 is an investigational small molecule being studied for the treatment of cognitive impairment associated with Down Syndrome. It has been evaluated in clinical trials involving adults and adolescents with Down Syndrome, as well as in healthy volunteers for safety and pharmacokinetic studies.

Who is developing RG1662?

RG1662 is being developed by Roche Holding AG, which trades under the ticker RHHBY on the OTC market. Roche has sponsored multiple clinical trials of RG1662 in Down Syndrome and healthy volunteer populations.

What phase is RG1662 in?

RG1662 has completed Phase 2 clinical development. The Phase 2 trial, known as CLEMATIS (NCT02024789), was completed with 173 participants. Earlier Phase 1 trials have also been completed. RG1662 is not approved and remains an investigational agent.

What clinical trials has RG1662 been in?

RG1662 has been studied in four completed trials: NCT01436955 and NCT01667367 (Phase 1 in Down Syndrome and healthy volunteers), NCT02024789 (Phase 2 CLEMATIS in Down Syndrome), and NCT02470832 (Phase 1 drug interaction study in healthy subjects).

Is RG1662 the same as basmisanil?

RG1662 is also known as basmisanil. The Phase 2 CLEMATIS trial (NCT02024789) evaluated RG1662 in adults and adolescents with Down Syndrome, and the drug has been referenced under both names in clinical research.