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VX-152

Phase 2

Cystic Fibrosis | Small molecule | Respiratory |Vertex Pharmaceuticals Incorporated|Last Updated: Jan 28, 2021

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-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment80
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02951195A Study Evaluating the Safety of VX-152 Combination Therapy in Adults With Cystic FibrosisPHASE2 COMPLETED 80Nov 1, 2016Jan 1, 2018Jan 28, 202116 United States
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Study Endpoints
Primary Endpoints
Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Day 1 Through Safety Follow-up Visit (Up to Day 43 for Part 1 and Day 71 for Part 2)
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 15 for Part 1 and Part 2 Cohort 2A
From Baseline at Day 15

FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

Absolute Change in ppFEV1 Through Day 29 for Part 2 Cohort 2B
From Baseline Through Day 29

FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

Secondary Endpoints
Absolute Change in Sweat Chloride Concentrations at Day 15 for Part 1 and Part 2 Cohort 2A
From Baseline at Day 15
Absolute Change in Sweat Chloride Concentrations Through Day 29 for Part 2 Cohort 2B
From Baseline Through Day 29
Relative Change in ppFEV1 at Day 15 for Part 1 and Part 2 Cohort 2A
From Baseline at Day 15
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Study Design & Arms
AllocationRANDOMIZED
MaskingTRIPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Part 1: PlaceboPLACEBO_COMPARATOR -
Part 1 Cohort 1A: TCEXPERIMENTAL -
Part 1 Cohort 1B: TCEXPERIMENTAL -
Part 1 Cohort 1C: TCEXPERIMENTAL -
Part 2 Cohort 2A: TEZ/IVAACTIVE_COMPARATOR -
Part 2 Cohort 2A: TCEXPERIMENTAL -
Part 2 Cohort 2B: TEZ/IVAACTIVE_COMPARATOR -
Part 2 Cohort 2B: TCEXPERIMENTAL -
Interventions
NameTypeDescription
VX-152DRUGTablet for oral administration.
TEZ/IVADRUGFixed-dose combination tablet for oral administration.
IVADRUGTablet for oral administration.
PlaceboDRUGPlacebo matched to VX-152.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites16

Inclusion Criteria: * Willing and able to comply with scheduled visits, treatment pan, study restrictions, laboratory tests, contraceptive guidelines, and other study procedures. * Body weight ≥35 kg. * Sweat chloride value ≥ 60 mmol/L from test results obtained during screening. * Subjects must ha...

Countries:United States
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