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lumacaftor q12h + ivacaftor q12h

Phase 1

Hepatic Impairment | Small molecule | Gastrointestinal |Vertex Pharmaceuticals Incorporated|Last Updated: Nov 19, 2013

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
CONTROLLEDDMC
Total Trials1
Total Enrollment23
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01888393Study of Lumacaftor in Combination With Ivacaftor in Subjects With Moderate Hepatic Impairment and Healthy SubjectsPHASE1 COMPLETED 23 — — Jun 1, 2013Nov 1, 2013Nov 19, 20132 Czechia, Slovakia
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Study Endpoints
Primary Endpoints
Lumacaftor pharmacokinetic parameters, including Cmax, tmax, AUC, Vd, CL/F
16 days
Ivacaftor pharmacokinetic parameters, including Cmax, tmax, AUC, Vd, CL/F
16 days
Secondary Endpoints
Lumacaftor metabolites pharmacokinetic parameters, including Cmax and AUC
16 days
Ivacaftor metabolites pharmacokinetic parameters including Cmax, tmax, AUC
16 days
Safety and tolerability as mentioned by adverse events, clinical laboratory values, standard electrocardiograms, Vital signs and pulse oximetry
21 days
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelPARALLEL
Treatment Arms
ArmTypeDescription
Group AEXPERIMENTALApproximately 12 subjects (male and female) with moderate hepatic impairment
Group BEXPERIMENTALApproximately 12 healthy subjects (male and female)
Interventions
NameTypeDescription
lumacaftor 200 mg q12h + ivacaftor 250 mg q12hDRUG -
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Eligibility Criteria
Age Range18 Years — 65 Years
SexALL
Healthy VolunteersYes
Study Sites2

Inclusion Criteria: Group A: Subjects with Moderate Hepatic Impairment * Male and female 18 to 65 years of age (inclusive) * Satisfy the criteria for moderate hepatic impairment defined as a Child Pugh total score of 7 to 9 (Child Pugh Class B) at the Screening Visit * Willing and able to comply w...

Countries:CzechiaSlovakia
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