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E004 HFA-MDI

Phase 3

Asthma | Small molecule | Respiratory |Amphastar Pharmaceuticals, Inc.|Last Updated: Jul 31, 2018

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 Enrollment70
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01460511Evaluation of Efficacy and Safety of E004 in Children With AsthmaPHASE3 COMPLETED 70Oct 1, 2011Jul 1, 2012Jul 31, 20188 United States
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Study Endpoints
Primary Endpoints
Primary Efficacy Endpoint AUC of FEV1's relative change
Visit 1 and Visit 3 at 5, 30, 60, 120, 180, 240, and 360 minutes post-dose

bronchodilator effect expressed as AUC of FEV1's relative change (from the same day baseline) versus time, defined as AUC of ΔFEV1%.

Secondary Endpoints
AUC of FEV1 volume changes (AUC of change in FEV1)
Visit 1 and Visit 3 at 5, 30, 60, 120, 180, 240, and 360 minutes post-dose
Maximum of change in FEV1% (Fmax)
Visit 1 and Visit 3 at 5, 30, 60, 120, 180, 240, and 360 minutes post-dose
Curves of change in FEV1, and change in FEV1%, versus time
Visit 1 and Visit 3 at 5, 30, 60, 120, 180, 240, and 360 minutes post-dose
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
P - Placebo-HFAPLACEBO_COMPARATORPlacebo-HFA, 0 mcg/inhalation, 2 inhalations QID
T - E004 (Epinephrine Inhalation Aerosol) HFA-MDIEXPERIMENTALE004 (Epinephrine Inhalation Aerosol) HFA-MDI, 125 mcg/inhalation, 2 inhalations QID
Interventions
NameTypeDescription
E004 (Epinephrine Inhalation Aerosol) HFA-MDIDRUGE004 (Epinephrine Inhalation Aerosol) HFA-MDI, 125 mcg/inhalation, 2 inhalations QID
Placebo-HFADRUGPlacebo-HFA, 0 mcg/inhalation, 2 inhalations QID
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Eligibility Criteria
Age Range4 Years — 11 Years
SexALL
Healthy VolunteersNo
Study Sites8

Inclusion Criteria: * Generally healthy male, and premenarchal female, children aged 4 - 11 years upon Screening. * With documented asthma, requiring inhaled epinephrine or beta2-agonist treatment, with or without concurrent anti-inflammatory therapies for at least 6-months prior to Screening. * Be...

Countries:United States
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