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

Sarepta Therapeutics

Status and phase: Completed; Phase 1

Conditions: Marburg Hemorrhagic Fever

Treatments: Drug: AVI-6003; Drug: Placebo

Study type: Interventional

Funder types: Industry; Other U.S. Federal agency

Identifiers

NCT01353040

6003-us-101

W9113M-10-C-0056 (Other Grant/Funding Number)

Details and patient eligibility

About

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.

Full 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.

Enrollment: 30 estimated patients

Sex: All

Ages: 18 to 50 years old

Volunteers: Accepts Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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30 participants in 2 patient groups, including a placebo group

AVI-6003

Experimental group

Description:

Phosphorodiamidate morpholino antisense oligomer with positive charges on selected subunits (PMOplus™)

Treatment:

Drug: AVI-6003

Placebo

Placebo Comparator group

Description:

Normal saline

Treatment:

Drug: Placebo