Safety Study of Single Administration Post-Exposure Prophylaxis Treatment for Ebola Virus

Sarepta Therapeutics

Status and phase: Completed; Phase 1

Conditions: Ebola Hemorrhagic Fever

Treatments: Drug: Placebo; Drug: AVI-6002

Study type: Interventional

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

Identifiers

NCT01353027

6002-us-101

Details and patient eligibility

About

The purpose of this study is to characterize the safety and pharmacology of single administrations of AVI-6002, a post-exposure prophylaxis candidate treatment for Ebolavirus.

Full description

Ebola hemorrhagic fever (EHF) is a rare human disease caused by Ebola Virus (EBOV), a filamentous single-stranded, negative-sense RNA virus. Since 1976 several Ebolavirus outbreaks have occurred with fatality rates ranging from 57% to 90%, with most of these outbreaks traced to single EBOV species; EBOV-Z. No effective therapy is currently available for Ebolavirus.

AVI-6002 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 Ebolavirus 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-6002 in healthy human subjects.

Enrollment: 30 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

Placebo

Placebo Comparator group

Treatment:

Drug: Placebo

AVI-6002

Experimental group

Description:

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

Treatment:

Drug: AVI-6002