Safety Study of Single Administration Post-exposure Prophylaxis Treatment for Marburg Virus

Phase 1

Completed

• Conditions: Marburg Hemorrhagic Fever
• Interventions: Drug: AVI-6003; Drug: Placebo

• Registration Number: NCT01353040
• Lead Sponsor: Sarepta Therapeutics, Inc.

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 Timeline

• Study First Submitted: 2011-04-22
• Study Start: 2011-05
• Study First Submitted QC: 2011-05-11
• Study First Posted: 2011-05-12
• Primary Completion: 2011-12
• Study Completion: 2011-12
• Status Verified: 2012-05
• Last Update Submitted: 2012-05-04
• Last Update Posted: 2012-05-07

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 30

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

Read More

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

Read More

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
AVI-6003	AVI-6003	Phosphorodiamidate morpholino antisense oligomer with positive charges on selected subunits (PMOplus™)
Placebo	Placebo	Normal saline

Primary Outcome Measures

Name	Time	Method
Number of subjects experiencing adverse events	28 days

Secondary Outcome Measures

Name	Time	Method
Plasma drug concentration	28 days
Urine drug concentration	28 days

Trial Locations

Locations (1)

West Coast Clinical Trials; 🇺🇸 Cypress, California, United States