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AVI-6003

Phase 1

Marburg Hemorrhagic Fever | Small molecule | Other |Sarepta Therapeutics, Inc.|Last Updated: May 7, 2012

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 Enrollment30
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01353040Safety Study of Single Administration Post-exposure Prophylaxis Treatment for Marburg VirusPHASE1 COMPLETED 30May 1, 2011Dec 1, 2011May 7, 20121 United States
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Study Endpoints
Primary Endpoints
Number of subjects experiencing adverse events
28 days
Secondary Endpoints
Plasma drug concentration
28 days
Urine drug concentration
28 days
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
AVI-6003EXPERIMENTALPhosphorodiamidate morpholino antisense oligomer with positive charges on selected subunits (PMOplus™)
PlaceboPLACEBO_COMPARATORNormal saline
Interventions
NameTypeDescription
AVI-6003DRUGSingle intravenous administration
PlaceboDRUGSingle intravenous administration
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Eligibility Criteria
Age Range18 Years — 50 Years
SexALL
Healthy VolunteersYes
Study Sites1

Inclusion Criteria: * Male or female and between the ages of 18 and 50 years in good general health * Volunteers must be willing to use barrier methods of contraception or be of non-childbearing potential * Volunteers must be willing to undergo a urine screen for drugs of abuse Exclusion Criteria:...

Countries:United States
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