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

This study has been completed.
Sponsor:
Collaborator:
Information provided by (Responsible Party):
Sarepta Therapeutics
ClinicalTrials.gov Identifier:
NCT01353040
First received: April 22, 2011
Last updated: May 4, 2012
Last verified: May 2012
  Purpose

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 Intervention Phase
Marburg Hemorrhagic Fever
Drug: AVI-6003
Drug: Placebo
Phase 1

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, 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

Resource links provided by NLM:


Further study details as provided by Sarepta Therapeutics:

Primary Outcome Measures:
  • Number of subjects experiencing adverse events [ Time Frame: 28 days ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Plasma drug concentration [ Time Frame: 28 days ] [ Designated as safety issue: No ]
  • Urine drug concentration [ Time Frame: 28 days ] [ Designated as safety issue: No ]

Estimated Enrollment: 30
Study Start Date: May 2011
Study Completion Date: December 2011
Primary Completion Date: December 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

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.

  Eligibility

Ages Eligible for Study:   18 Years to 50 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   Yes
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01353040

Locations
United States, California
West Coast Clinical Trials
Cypress, California, United States, 90630
Sponsors and Collaborators
Sarepta Therapeutics
Investigators
Study Director: Alison Heald, MD Sarepta Therapeutics
Principal Investigator: Apinya B Vutikullird, MD West Coast Clinical Trials
  More Information

No publications provided

Responsible Party: Sarepta Therapeutics
ClinicalTrials.gov Identifier: NCT01353040     History of Changes
Other Study ID Numbers: 6003-us-101, W9113M-10-C-0056
Study First Received: April 22, 2011
Last Updated: May 4, 2012
Health Authority: United States: Food and Drug Administration
United States: Institutional Review Board
United States: Federal Government

Additional relevant MeSH terms:
Hemorrhagic Fevers, Viral
Marburg Virus Disease
Filoviridae Infections
Mononegavirales Infections
RNA Virus Infections
Virus Diseases

ClinicalTrials.gov processed this record on October 21, 2014