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AVI-7288

Phase 1

Marburg Hemorrhagic Fever | Small molecule | Other |Sarepta Therapeutics, Inc.|Last Updated: May 7, 2014

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 Enrollment40
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01566877A Study to Assess the Safety, Tolerability and Pharmacokinetics of AVI-7288 in Healthy Adult VolunteersPHASE1 COMPLETED 40May 1, 2013Jan 1, 2014May 7, 20141 United States
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Study Endpoints
Primary Endpoints
The outcome measure is safety, as assessed through adverse event evaluation and serial clinical assessments.
daily during dosing period with follow up on Day 21 and Day 42
Secondary Endpoints
The outcome measures are Serial PK Day 1 over 24 hours(predose, 10minutes post, 30 minutes post, 1 hour 1.5, 2, 4, 6,. 8, 12,16,24 hours post dose), daily trough level and on Day 14 (last day of dosing) serial PK until 48 hours post last dose.
2 Weeks
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
AVI-7288ACTIVE_COMPARATORAVI-7288 is a phosphorodiamidate morpholino oligomer with positive charges (PMOplus™) that targets Marburg virus nucleoprotein (NP). AVI-7288 is supplied in 5 mL vials containing 5 mL AVI-7288 at a concentration of 50 mg/mL. The dose levels of AVI-7288 will vary in four cohort's.
PlaceboPLACEBO_COMPARATORPlacebo control consists of approximately 150 mL normal saline solution administered by IV infusion over 30 minutes once a day for 14 days.
Interventions
NameTypeDescription
AVI-7288DRUGCohort 1: AVI-7288 at 1 mg/kg IV; Cohort 2: AVI-7288 at 4 mg/kg IV;Cohort 3: AVI-7288 at 8 mg/kg IV; Cohort 4: AVI-7288 at 12 mg/kg IV The amount of AVI-7288 required to administer the required dose will be diluted to a volume of approximately 150 mL with normal saline solution (NSS) and given by IV infusion over 30 minutes once a day for 14 days. Infusions will be administered at approximately the same time each day.
PlaceboOTHERNormal Saline Solution (NSS)
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Eligibility Criteria
Age Range18 Years — 50 Years
SexALL
Healthy VolunteersYes
Study Sites1

Inclusion Criteria: A subject must meet all of the following criteria to be eligible for this study. 1.Man or woman 18 to 50 years of age, inclusive, at the time of screening. 2.Body mass index 18 kg/m2 to 35 kg/m2, inclusive, at the time of screening and Check-in (Day -1).3.Good general health (n...

Countries:United States
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