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TOBI

Phase 3

Treatment of Early Pulmonary Infections With P. Aeruginosa in Cystic Fibrosis Patients | Small molecule | Respiratory |Novartis AG|Last Updated: Jul 15, 2016

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_CONTROLLEDBiomarker
Total Trials1
Total Enrollment50
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01082367Randomized, Controlled Study of CF Patients Between 3 Months and Less Than 7 YearsPHASE3 COMPLETED 50Apr 1, 2010Jun 1, 2015Jul 15, 201619 Canada, Egypt +8
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Study Endpoints
Primary Endpoints
Percentage of Participants P Aeruginosa-free After Completion of the First Treatment Cycle
Day 29

Sputum/throat swab cultures were assessed.

Secondary Endpoints
Percentage of Participants Free From P. Aeruginosa 28 Days After Termination of the Second Treatment Cycle
Day 91
Percentage of Participants P Aeruginosa-free at Termination of the Double Blind Period
Day 91
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelCROSSOVER
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
TOBI (tobramycin inhaled solution)/PlaceboEXPERIMENTALParticipants randomized to TOBI received the investigational treatment for 28 days twice daily (bi)d in the first treatment cycle. At the end of first treatment cycle, participants who were positive for P. aeruginosa entered the open label (OL) phase of the study and received TOBI for 28 days bid. Participants who were negative for P. aeruginosa at the end of first treatment cycle and agreed to participate in the cross-over treatment period received placebo for 28 days bid (second treatment cycle).
Placebo/TOBIPLACEBO_COMPARATORParticipants randomized to placebo group received 0.9 % saline (NaCl) for 28 days bid in the first treatment cycle. At the end of first treatment cycle, participants who were positive for P. aeruginosa entered the OL phase of the study and received TOBI for 28 days bid. Participants who were negative for P. aeruginosa at the end of first treatment cycle and agreed to participate in the cross-over treatment period received TOBI for 28 days bid (second treatment cycle).
Interventions
NameTypeDescription
TOBIDRUGTOBI (tobramycin inhaled solution)
PlaceboDRUGPlacebo
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Eligibility Criteria
Age Range3 Months — 6 Years
SexALL
Healthy VolunteersNo
Study Sites19

Inclusion Criteria: * Diagnosis of cystic fibrosis * Early lower respiratory tract infection with P. aeruginosa, Exclusion Criteria: * Known local or systemic hypersensitivity to aminoglycosides or inhaled antibiotics. * Administration of loop diuretics within 7 days prior to study drug administr...

Countries:CanadaEgyptFranceGermanyGreeceHungaryItalyRomaniaRussiaSwitzerland
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