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Vanzacaftor

Phase 1

Cystic Fibrosis | Small molecule | Respiratory |Vertex Pharmaceuticals Incorporated|Last Updated: Jul 14, 2023

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
RandomizedDouble-BlindACTIVE_CONTROLLEDBiomarker
Total Trials1
Total Enrollment56
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05867147An Electrocardiogram Study to Evaluate the Effect of Vanzacaftor on the QT/QTc Interval in Healthy ParticipantsPHASE1 COMPLETED 56Apr 26, 2023Jun 14, 2023Jul 14, 20231 United States
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Study Endpoints
Primary Endpoints
Change in QT interval corrected by Fridericia's formula (QTcF)
From Baseline up to Day 23
Secondary Endpoints
Change in Heart Rate (HR)
From Baseline up to Day 23
Change in PR interval, segment
From Baseline up to Day 23
Change in QRS duration
From Baseline up to Day 23
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Study Design & Arms
AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Group 1EXPERIMENTALParticipants will receive VNZ-matching placebo, moxifloxacin-matching placebo and VNZ at different time points.
Group 2AACTIVE_COMPARATORParticipants will receive VNZ-matching placebo, moxifloxacin, and moxifloxacin-matching placebo at different time points.
Group 2BACTIVE_COMPARATORParticipants will receive VNZ-matching placebo, moxifloxacin-matching placebo and moxifloxacin at different time points.
Interventions
NameTypeDescription
VanzacaftorDRUGTablets for oral administration.
Vanzacaftor PlaceboDRUGPlacebo for oral administration.
MoxifloxacinDRUGTablets for oral administration.
Moxifloxacin PlaceboDRUGTablets for oral administration.
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Eligibility Criteria
Age Range18 Years — 45 Years
SexALL
Healthy VolunteersYes
Study Sites1

Key Inclusion Criteria: * Body mass index (BMI) of 18.0 to 32.0 kilogram per meter square (Kg/m\^2), both inclusive * Male and female participants of age 18 to 45 years, both inclusive * Serum potassium, calcium, and magnesium values within normal ranges Key Exclusion Criteria: * Median QTcF\>450...

Countries:United States
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