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ARIKACE

Phase 1

Cystic Fibrosis | Small molecule | Respiratory |Insmed Incorporated|Last Updated: Jul 30, 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
RandomizedPLACEBO_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment66
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00777296Multidose Safety and Tolerability Study of Dose Escalation of Liposomal Amikacin for Inhalation (ARIKACE™)PHASE1 COMPLETED 66Feb 22, 2007Feb 27, 2008Jul 30, 202011 Belgium, Hungary +5
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Study Endpoints
Primary Endpoints
Clinically Significant Laboratory Abnormalities.
28 Days

Changes in chemistry and hematology lab tests (clinically significant value of CTCAE grade ≥ 3).

Secondary Endpoints
Pharmacokinetics (PK) of Arikace™ in Serum.
Day 1, Day 14 and Day 28
Pharmacokinetic (PK) of Arikace in Serum (Cmax).
Day 1, Day 14 and Day 28
Pharmacokinetics (PK) of Arikace™ in Sputum (AUC).
28 days
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Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Cohort 1 - 280 mg ARIKACE™EXPERIMENTALSubjects in this cohort will receive 280 mg of ARIKACE™
Cohort 1 - PlaceboPLACEBO_COMPARATORSubjects in this arm of cohort 1 will receive matching placebo
Cohort 2 - 560 mg ARIKACE™EXPERIMENTALSubjects in this cohort will receive 560 mg of ARIKACE™
Cohort 2 - PlaceboPLACEBO_COMPARATORSubjects in this arm of cohort 2 will receive matching placebo
Interventions
NameTypeDescription
ARIKACE™DRUGStudy start date is before Jan 18, 2017.
PlaceboDRUGStudy start date is before Jan 18, 2017.
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Eligibility Criteria
Age Range6 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites11

Inclusion Criteria: * Written informed consent obtained from patient or designated legal guardian prior to the performance of any study related procedures. * Male or female study subjects ≥6 years of age or older * Confirmed diagnosis of CF * History of chronic infection with P. aeruginosa * Study ...

Countries:BelgiumHungaryNorth MacedoniaPolandSerbiaSlovakiaUkraine
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