A Randomized, Double-Blind, Placebo-Controlled, Single-Dose, Dose-Escalation Study to Assess the Safety, Tolerability and Pharmacokinetics of AVI-6003 in Healthy Adult Volunteers
Trial Snapshot
- Phase
- Phase 1
- Status
- Completed
- Sponsor
- Sarepta Therapeutics, Inc.
- Enrollment
- 30
- Locations
- 1
- Primary Endpoint
- Number of subjects experiencing adverse events
Study Overview
Brief Summary
The purpose of this study is to characterize the safety and pharmacology of single administrations of AVI-6003, a post-exposure prophylaxis candidate treatment for Marburg virus.
Detailed Description
Marburg hemorrhagic fever is a rare human disease caused by the Marburg virus, a filamentous, single-stranded, negative-sense RNA virus. In general, the mortality rate associated with Marburg virus outbreaks has ranged from 23% to 88%, with the highest death rates seen in more recent epidemics. No vaccine or effective therapy is available for Marburg hemorrhagic fever. AVI-6003 is an experimental combination of 2 phosphorodiamidate morpholino antisense oligomers with positive charges on selected subunits (PMOplus™). These oligomers specifically target viral messenger RNA encoding 2 Marburg virus proteins thought to be important in viral replication and host immune suppression. The present study is designed to characterize the safety, tolerability and pharmacokinetics of escalating single-administration doses of AVI-6003 in healthy human subjects.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Eligibility Criteria
- Ages
- 18 Years to 50 Years (Adult)
- Sex
- All
- Accepts Healthy Volunteers
- Yes
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
- •Pregnancy or breastfeeding.
- •Any clinically relevant abnormalities in physical examinations, vital signs, ECG, clinical chemistry, hematology or urinalysis
- •Positive test for human immunodeficiency virus, hepatitis B or hepatitis C or known history of HIV infection
Arms & Interventions
AVI-6003
Phosphorodiamidate morpholino antisense oligomer with positive charges on selected subunits (PMOplus™)
Intervention: AVI-6003 (Drug)
Placebo
Normal saline
Intervention: Placebo (Drug)
Outcomes
Primary Outcomes
Number of subjects experiencing adverse events
Time Frame: 28 days
Secondary Outcomes
- Plasma drug concentration(28 days)
- Urine drug concentration(28 days)