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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01353040
Recruitment Status : Completed
First Posted : May 12, 2011
Last Update Posted : May 7, 2012
United States Department of Defense
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment Phase
Marburg Hemorrhagic Fever Drug: AVI-6003 Drug: Placebo Phase 1

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: 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
Study Start Date : May 2011
Actual Primary Completion Date : December 2011
Actual Study Completion Date : December 2011

Arm Intervention/treatment
Experimental: AVI-6003
Phosphorodiamidate morpholino antisense oligomer with positive charges on selected subunits (PMOplus™)
Drug: AVI-6003
Single intravenous administration

Placebo Comparator: Placebo
Normal saline
Drug: Placebo
Single intravenous administration

Primary Outcome Measures :
  1. Number of subjects experiencing adverse events [ Time Frame: 28 days ]

Secondary Outcome Measures :
  1. Plasma drug concentration [ Time Frame: 28 days ]
  2. Urine drug concentration [ Time Frame: 28 days ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 50 Years   (Adult)
Sexes Eligible for Study:   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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01353040

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United States, California
West Coast Clinical Trials
Cypress, California, United States, 90630
Sponsors and Collaborators
Sarepta Therapeutics, Inc.
United States Department of Defense
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Study Director: Alison Heald, MD Sarepta Therapeutics, Inc.
Principal Investigator: Apinya B Vutikullird, MD West Coast Clinical Trials
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Responsible Party: Sarepta Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT01353040    
Other Study ID Numbers: 6003-us-101
W9113M-10-C-0056 ( Other Grant/Funding Number: Transformational Medical Technologies, US Dept. of Defense )
First Posted: May 12, 2011    Key Record Dates
Last Update Posted: May 7, 2012
Last Verified: May 2012
Additional relevant MeSH terms:
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Hemorrhagic Fevers, Viral
Marburg Virus Disease
RNA Virus Infections
Virus Diseases
Filoviridae Infections
Mononegavirales Infections