Study to Assess Brincidofovir Treatment of Serious Diseases or Conditions Caused by Double-stranded DNA Viruses
A Multicenter, Open-label Study of CMX001 Treatment of Serious Diseases or Conditions Caused by Double-stranded DNA Viruses
1 other identifier
interventional
210
1 country
37
Brief Summary
This was a multicenter, open-label study of oral brincidofovir (BCV) treatment of serious disease or conditions caused by double-stranded DNA (dsDNA) virus(es). Subjects received either a weight-based or a fixed dose of oral BCV once weekly (QW) or twice weekly (BIW) for up to 3 months until clinical disease was resolved or stabilized and/or viral DNA by polymerase chain reaction testing was negative for 4 consecutive weeks, whichever was longer. Under the first protocol amendment, adults and adolescents (≥13 years) received 200 mg or 300 mg BCV BIW (not to exceed 4 mg/kg total weekly dose) depending on the difficulty of treating their disease (i.e., Group 1 or Group 2, respectively), and pediatric subjects (≤12 years) received 4 mg/kg BCV BIW. Under the second protocol amendment, adults and adolescents (≥13 years), regardless of viral infection/disease, had a maximum weekly dose of 200 mg, i.e., 200 mg QW or 100 mg BIW; not to exceed 4mg/kg total weekly dose. Pediatric subjects (≤12 years), regardless of viral infection/disease, had a maximum weekly dose of 4 mg/kg, i.e., 4 mg/kg QW or 2 mg/kg BIW; not to exceed 200 mg.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_3
Started Dec 2010
37 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
June 11, 2010
CompletedFirst Posted
Study publicly available on registry
June 14, 2010
CompletedStudy Start
First participant enrolled
December 1, 2010
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 1, 2012
CompletedStudy Completion
Last participant's last visit for all outcomes
December 1, 2012
CompletedResults Posted
Study results publicly available
August 12, 2021
CompletedAugust 12, 2021
July 1, 2021
2 years
June 11, 2010
June 28, 2021
July 19, 2021
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Number of Subjects Who Had a Sustained and Significant Reduction in Plasma Viral Load of Primary dsDNA Virus
Proportion of subjects who achieved a confirmed reduction in viral load for the primary dsDNA virus of ≥1 log10 copies/mL from baseline or to an undetectable level. Confirmation required the reduction in viral load (i.e., decrease of ≥ 1 log10 copies/mL from baseline or to undetectable levels) to be maintained at the next assessment for the subject to be considered a success.
3 months
Study Arms (1)
Brincidofovir
EXPERIMENTALSubjects received either a weight-based or a fixed-dose of oral brincidofovir (BCV) once weekly or twice weekly for up to 3 months until clinical disease was resolved or stabilized and/or viral DNA polymerase chain reaction testing was negative for 4 consecutive weeks, whichever was longer.
Interventions
Brincidofovir (BCV) was administered orally either once or twice weekly for up to 3 months. Treatment may have been extended for an additional 3 months depending a satisfactory review of safety parameters. Subjects could not receive more than a total of 6 months of treatment with BCV without prior approval.
Eligibility Criteria
You may qualify if:
- Had an immediately life-threatening or serious disease or condition caused by infection with a double-stranded DNA virus (including subjects with recurrent viral disease). \[Note: During the course of the study, the viral disease indications were narrow to focus on indications that were under study in controlled clinical trials of brincidofovir (BCV) and on viral diseases that had few, if any, options for treatment, including cytomegalovirus (CMV), adenovirus (AdV), herpes simplex virus (HSV), vaccinia virus (VAVC), variola virus (VARV) or monkeypox viruses(s).\]
- Had a life expectancy of at least 2 weeks and commitment to continuation of supportive care for at least 4 weeks.
- Were willing and able to understand and provide written informed consent. \[Note: For minors or those incapable of providing written informed consent (i.e., incapacitated), consent was provided by a parent or legal guardian or representative who could understand and provide written informed consent.\]
- Were willing and able, to the best of his or her (or parent/guardian) knowledge, to participate in all required study activities for the duration of the study.
- If female of reproductive potential, agreed to use 2 acceptable methods of birth control throughout the study with at least 1 being a barrier method.
- In the judgment of the investigator, subjects for whom no comparable or satisfactory therapeutic alternative was available
You may not qualify if:
- Were pregnant or currently nursing.
- Had hypersensitivity to cidofovir or BCV.
- Had a long-term prognosis that included a poor likelihood of survival due to irreversible organ failure including, for example, subjects with frank hepatic failure and adults with Grade 4 graft versus host disease of the GI tract.
- Were eligible for enrollment and able to participate in a clinical trial evaluating BCV. \[Note: Per the FDA guidance, subjects eligible and able to participate in a controlled clinical study evaluating BCV were not eligible for participation in this study. Subjects who did not meet eligibility criteria for a controlled BCV clinical study or who were unable to participate because, for example, of logistical or other issues were eligible to participate in this study. The investigator verified that his/her subjects met this criterion on the Eligibility electronic case report form. A subject simply preferring enrollment in this study over a BCV controlled clinical trial did not qualify for enrollment in this study.\]
- Had any other condition that would have, in the judgment of the investigator, put the subject at increased risk during participation in the study or interfered with the conduct of the study.
- Subjects with acute or chronic renal impairment, pediatric and adolescent subjects, and subjects aged 65 years and older were included in this study. Subjects with hepatic impairment were included unless the investigator judged that the subject had irreversible hepatic compromise.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (37)
Loma Linda University Hospital
Loma Linda, California, 92354, United States
Children's Hospital of LA
Los Angeles, California, 90027, United States
UCLA Department of Medicine
Los Angeles, California, 90095, United States
CHOC Children's
Orange, California, 92868, United States
Univeristy of San Francisco
San Francisco, California, 94143, United States
Lucile Packard Children's Hospital
Stanford, California, 94304, United States
Children's Hospital of Colorado
Aurora, Colorado, 80045, United States
Children's National Medical Center
Washington D.C., District of Columbia, 20010, United States
University of Chicago
Chicago, Illinois, 60637, United States
University of Iowa
Iowa City, Iowa, 52242, United States
Childrens Hospital LSU
New Orleans, Louisiana, 70118, United States
NIH
Bethesda, Maryland, 20892, United States
Tufts Medical Center
Boston, Massachusetts, 02111, United States
Brigham and Womens Hospital
Boston, Massachusetts, 02115, United States
University of Michigan
Ann Arbor, Michigan, 48109, United States
University of Minnesota
Minneapolis, Minnesota, 55455, United States
St. Louis Children's Hospital
St Louis, Missouri, 63110, United States
University of Nebraska Medical Center
Omaha, Nebraska, 68198-5130, United States
Hackensack University Medical Center
Hackensack, New Jersey, 07601, United States
Mt. Sinai
New York, New York, 10029, United States
Columbia University
New York, New York, 10032, United States
Memorial Sloan Kettering Cancer Institute
New York, New York, 10065, United States
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27599, United States
Levine Children's Hospital Carolina Medical Center
Charlotte, North Carolina, 28203, United States
Duke University Medical Center
Durham, North Carolina, 27710, United States
Cincinnati Childrens Hospital
Cincinnati, Ohio, 45229, United States
Cleveland Clinic
Cleveland, Ohio, 44195, United States
Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
Oregon Health and Science University
Portland, Oregon, 97239, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
UPMC
Pittsburgh, Pennsylvania, 15213, United States
Childrens Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224, United States
Cook Children's Medical Center
Fort Worth, Texas, 76104-2796, United States
UT MD Anderson Cancer Center
Houston, Texas, 77030, United States
Intermountain BMT program LDS Hospital
Salt Lake City, Utah, 84143, United States
University of Washington-Fred Hutchinson Cancer Center
Seattle, Washington, 98109, United States
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226, United States
Related Publications (1)
Tippin TK, Morrison ME, Brundage TM, Mommeja-Marin H. Brincidofovir Is Not a Substrate for the Human Organic Anion Transporter 1: A Mechanistic Explanation for the Lack of Nephrotoxicity Observed in Clinical Studies. Ther Drug Monit. 2016 Dec;38(6):777-786. doi: 10.1097/FTD.0000000000000353.
PMID: 27851688DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Chief Medical Officer
- Organization
- Chimerix, Inc.
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
June 11, 2010
First Posted
June 14, 2010
Study Start
December 1, 2010
Primary Completion
December 1, 2012
Study Completion
December 1, 2012
Last Updated
August 12, 2021
Results First Posted
August 12, 2021
Record last verified: 2021-07