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AZLI three times a day

Phase 3

Cystic Fibrosis | Small molecule | Respiratory |Gilead Sciences, Inc.|Last Updated: Apr 21, 2011

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 Enrollment166
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00112359International Safety and Efficacy Study of Aztreonam for Inhalation Solution (AZLI) in Cystic Fibrosis Patients With P. AeruginosaPHASE3 COMPLETED 166May 1, 2005Apr 1, 2007Apr 21, 201153 United States, Australia +2
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Study Endpoints
Primary Endpoints
Change in CFQ-R Respiratory Symptoms Scale (RSS) Score
Day 0 to Day 28

The CFQ-R was administered at baseline and every visit thereafter. The endpoint was change in respiratory symptoms from baseline, assessed with the CFQ-R respiratory symptoms scale (RSS; range of scores: 0-100; higher scores indicate fewer symptoms).

Secondary Endpoints
Change in CFQ-R RSS Score
Day 0 to Day 14
Percent Change in FEV1 (L)
Day 0 to Day 28
Change From Baseline in Pseudomonas Aeruginosa (PA) Log10 Colony Forming Units (CFU) Per Gram of Sputum
Day 0 to Day 28
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Placebo three times a day (TID)PLACEBO_COMPARATOR -
AZLI 75 mg three times a day (TID)EXPERIMENTAL -
Interventions
NameTypeDescription
AZLI 75 mg three times a day (TID)DRUG -
Placebo three times a day (TID)DRUG -
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Eligibility Criteria
Age Range6 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites53

Inclusion Criteria: * Documentation of CF diagnosis as evidenced by one or more clinical features consistent with the CF phenotype and one or more of the following criteria: * Sweat chloride greater than or equal to 60 mEq/L by quantitative pilocarpine iontophoresis test (QPIT); * Two well-cha...

Countries:United StatesAustraliaCanadaNew Zealand
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