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QAU145

Phase 2

Cystic Fibrosis | Small molecule | Respiratory |Novartis AG|Last Updated: Dec 19, 2020

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_CONTROLLED
Total Trials1
Total Enrollment9
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00506792Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of QAU145 in Patients With Cystic FibrosisPHASE2 COMPLETED 9Jul 1, 2007Nov 1, 2009Dec 19, 20201 United States
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Study Endpoints
Primary Endpoints
Pharmacodynamic response to single intranasal doses of QAU145 in cystic fibrosis patients using nasal potential difference measurements up to 6 hours post-dose.
Part I: Baseline, 2 hr post dose, after each dose, end of Part I. Part II: Pre dose and 2 hours post each dose
Secondary Endpoints
Safety and tolerability of single ascending doses assessed by nasal examinations, adverse events, medications, and a nasal symptom questionnaire. Pharmacokinetics up to 6 hours post-dose
Part I: Throughout study (about 8 weeks) Part II: Throughout study (about 9 weeks)
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelCROSSOVER
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
1EXPERIMENTALQAU145
2PLACEBO_COMPARATORPlacebo
Interventions
NameTypeDescription
QAU145DRUG -
PlaceboDRUG -
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Eligibility Criteria
Age Range18 Years — 50 Years
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: * Male and female (women must be post-menopausal or surgically sterile) subjects with cystic fibrosis ages 18 to 50. Exclusion Criteria: * Any presence of seasonal or non-seasonal allergies affecting the nose, nasal passages, throat or sinuses within 2 weeks prior to dosing. *...

Countries:United States
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