A Multicenter, Open-label Study of CMX001 Treatment of Serious Diseases or Conditions Caused by dsDNA Viruses

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. Identifier: NCT01143181
Recruitment Status : Completed
First Posted : June 14, 2010
Last Update Posted : August 29, 2013
Information provided by (Responsible Party):

Brief Summary:
CMX001 is an orally administered lipid conjugate of the synthetic nucleotide analog cidofovir (CDV). The conjugate is believed to be absorbed in the small intestine then delivered to target organs throughout the body where it crosses cell membranes by facilitated and passive diffusion. Inside the cell, CMX001 is cleaved by intracellular phospholipases to release CDV which is converted to the active antiviral agent, CDV-diphosphate (CDV-PP), by intracellular anabolic kinases. Adults and adolescents, regardless of viral infection/disease, will have a maximum weekly dose of 200 mg i.e., 200 mg once weekly OR 100 mg twice weekly; not to exceed 4mg/kg total weekly dose. Pediatric subjects (< 12 years), regardless of viral infection/disease, will have a maximum weekly dose of 4 mg/kg i.e., 4 mg/kg once weekly OR 2 mg/kg twice weekly.

Condition or disease Intervention/treatment Phase
Male or Female Patients With a Serious or Immediately Life-threatening Disease or Condition Caused by CMV, ADV, HSV, VAVC, VARV or Monkeypox Viruses(s) Who Have a Life Expectancy of ≥ 2 Weeks and for Whom no Comparable or Satisfactory Alternative Therapy is Available Drug: CMX001 Phase 2 Phase 3

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 232 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multicenter, Open-label Study of CMX001 Treatment of Serious Diseases or Conditions Caused by dsDNA Viruses
Study Start Date : December 2010
Actual Primary Completion Date : December 2012
Actual Study Completion Date : December 2012

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Intervention Details:
    Drug: CMX001
    CMX001 administered orally twice weekly for up to 3 months. Treatment may be extended for up to 6 months depending on the patient's clinical response.

Information from the National Library of Medicine

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Ages Eligible for Study:   1 Month and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Patients with an immediately life-threatening or serious disease or condition caused by CMV, ADV, HSV, VAVC, VARV or monkeypox viruses(s).
  2. Life expectancy of at least 2 weeks and commitment to continuation of supportive care for at least 4 weeks.
  3. Able to ingest and absorb oral medication (in the judgment of the investigator and based on lack of significant GI pathology such as small bowel resection or ileus). [Note: Use of TPN (total parenteral nutrition) is not in and of itself exclusionary as long as the reason for use would not disqualify the patient based on this criterion.]
  4. Willing and able to understand and provide written informed consent. For minors or those incapable of providing written informed consent (i.e., incapacitated), understood, written and informed consent must be provided by a parent or legal guardian or representative.
  5. To the best of his or her (or parent/guardian) knowledge, willing and able to participate in all required study activities for the duration of the study.
  6. In the judgment of the investigator, patients for whom no comparable or satisfactory therapeutic alternative is available

Exclusion Criteria:

  1. Females who are pregnant or currently nursing.
  2. Patients with hypersensitivity to cidofovir or CMX001.
  3. Patients whose long-term prognosis includes a poor likelihood of survival due to irreversible organ failure including, for example, patients with frank hepatic failure and adults with Grade 4 GVHD of the GI tract.
  4. Patients who are eligible for enrollment and able to participate in a clinical trial evaluating CMX001.
  5. Patients with any other condition that would, in the judgment of the investigator, put the patient at increased risk during participation in the study, or interfere with the conduct of the study.

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 identifier (NCT number): NCT01143181

  Hide Study Locations
United States, California
Loma Linda University Hospital
Loma Linda, California, United States, 92354
Children's Hospital of LA
Los Angeles, California, United States, 90027
UCLA Department of Medicine
Los Angeles, California, United States, 90095
CHOC Children's
Orange, California, United States, 92868
Univeristy of San Francisco
San Francisco, California, United States, 94143
Lucile Packard Children's Hospital
Stanford, California, United States, 94304
United States, Colorado
Children's Hospital of Colorado
Aurora, Colorado, United States, 80045
United States, District of Columbia
Children's National Medical Center
Washington, District of Columbia, United States, 20010
United States, Illinois
University of Chicago
Chicago, Illinois, United States, 60637
United States, Iowa
University of Iowa
Iowa City, Iowa, United States, 52242
United States, Louisiana
Childrens Hospital LSU
New Orleans, Louisiana, United States, 70118
United States, Maryland
Bethesda, Maryland, United States, 20892
United States, Massachusetts
Tufts Medical Center
Boston, Massachusetts, United States, 02111
Brigham and Womens Hospital
Boston, Massachusetts, United States, 02115
United States, Michigan
University of Michigan
Ann Arbor, Michigan, United States, 48109
United States, Minnesota
University of Minnesota
Minneapolis, Minnesota, United States, 55455
United States, Missouri
St. Louis Children's Hospital
St. Louis, Missouri, United States, 63110
United States, Nebraska
University of Nebraska Medical Center
Omaha, Nebraska, United States, 68198-5130
United States, New Jersey
Hackensack University Medical Center
Hackensack, New Jersey, United States, 07601
United States, New York
Mt. Sinai
New York, New York, United States, 10029
Columbia University
New York, New York, United States, 10032
Memorial Sloan Kettering Cancer Institute
New York, New York, United States, 10065
United States, North Carolina
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States, 27599
Levine Children's Hospital Carolina Medical Center
Charlotte, North Carolina, United States, 28203
Duke University Medical Center
Durham, North Carolina, United States, 27710
United States, Ohio
Cincinnati Childrens Hospital
Cincinnati, Ohio, United States, 45229
Cleveland Clinic
Cleveland, Ohio, United States, 44195
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
United States, Oregon
Oregon Health and Science University
Portland, Oregon, United States, 97239
United States, Pennsylvania
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Pittsburgh, Pennsylvania, United States, 15213
Childrens Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15224
United States, Texas
Cook Children's Medical Center
Fort Worth, Texas, United States, 76104-2796
UT MD Anderson Cancer Center
Houston, Texas, United States, 77030
United States, Utah
Intermountain BMT program LDS Hospital
Salt Lake City, Utah, United States, 84143
United States, Washington
University of Washington-Fred Hutchinson Cancer Center
Seattle, Washington, United States, 98109
United States, Wisconsin
Medical College of Wisconsin
Milwaukee, Wisconsin, United States, 53226
Sponsors and Collaborators

Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: Chimerix Identifier: NCT01143181     History of Changes
Other Study ID Numbers: CMX001-350
First Posted: June 14, 2010    Key Record Dates
Last Update Posted: August 29, 2013
Last Verified: August 2013

Keywords provided by Chimerix:
life threatening

Additional relevant MeSH terms:
Pathologic Processes
Poxviridae Infections
DNA Virus Infections
Virus Diseases