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Study of CMX001 to Prevent/Control Cytomegalovirus Infection in R+ Hematopoietic Stem Cell Transplant Recipients

Phase 2
Completed
Conditions
Cytomegalovirus Infection
Interventions
Drug: Placebo
Registration Number
NCT00942305
Lead Sponsor
Chimerix
Brief Summary

This was a multicenter, randomized, double-blind, placebo-controlled, dose-escalation study of brincidofovir (BCV) administered orally once or twice weekly for up to 11 weeks. Dosing was initiated immediately following engraftment (between Days 14-30 post-transplant) to prevent/control cytomegalovirus (CMV) infection or prevent disease in R+ hematopoietic stem cell transplant (HCT) recipients.

Detailed Description

This was to be a 2-part study. Part 1 was a randomized, double-blind, placebo-controlled, dose-escalation study of multiple doses of oral brincidofovir (BCV) in R+ hematopoietic stem cell transplant (HCT) recipients. In Part 2, one of the dose levels administered in Part 1 was to be tested against placebo to evaluate the statistical significance of BCV as a therapy for preventing progression of cytomegalovirus (CMV) infection or preventing CMV disease. The first 3 dose-escalating cohorts in Part 1 were to include 32 subjects within each cohort (24 randomized to receive oral BCV and 8 randomized to receive placebo in a 3:1 ratio) for a total of 96 planned subjects. Following completion of the first 3 cohorts and subsequent safety review of the data, up to an additional 3 cohorts of 32 subjects each could have been enrolled. Two additional cohorts (labeled Cohort 4 and Cohort 4A) beyond the initial 3 cohorts were actually enrolled in Part 1 of the study. Following the safety and antiviral activity analyses of the 5 cohorts in Part 1 and the number of subjects enrolled in each of those cohorts, Part 2 of the study was not conducted.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
239
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
SEQUENTIAL
Arm && Interventions
GroupInterventionDescription
PlaceboPlacebo* Cohort 1 = 40 mg of matching placebo administered once weekly (QW) * Cohort 2 = 100 mg of matching placebo administered QW * Cohort 3 = 200 mg of matching placebo administered QW * Cohort 4 = 200 mg of matching placebo administered twice weekly (BIW) * Cohort 4A = 100 mg of matching placebo administered BIW
BrincidofovirBrincidofovir* Cohort 1 = 40 mg brincidofovir (BCV) administered once weekly (QW) * Cohort 2 = 100 mg BCV administered QW * Cohort 3 = 200 mg BCV administered QW * Cohort 4 = 200 mg BCV administered twice weekly (BIW) * Cohort 4A = 100 mg BCV administered BIW
Primary Outcome Measures
NameTimeMethod
Number of Participants With Clinically Significant CMV InfectionRandomization to Week 8 post-treatment (~19 weeks)

The primary efficacy endpoint was a binomial outcome of failure to prevent cytomegalovirus (CMV) infection defined as CMV DNAemia \>200 copies/mL obtained at the time of the last treatment with study drug or diagnosis of CMV disease at some point during the treatment phase.

Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (26)

UCLA Medical Center

🇺🇸

Los Angeles, California, United States

Winship Cancer Institute at Emory University

🇺🇸

Atlanta, Georgia, United States

UT Southwestern Medical Center at Dallas

🇺🇸

Dallas, Texas, United States

Brigham and Womens Hospital, Division of Infectious Disease

🇺🇸

Boston, Massachusetts, United States

University of Minnesota Medical Center

🇺🇸

Minneapolis, Minnesota, United States

Duke University Medical Center

🇺🇸

Durham, North Carolina, United States

The Cleveland Clinic

🇺🇸

Cleveland, Ohio, United States

Utah Cancer Specialists - Intermountain Healthcare

🇺🇸

Salt Lake City, Utah, United States

University of Texas, MD Anderson Cancer Center

🇺🇸

Houston, Texas, United States

University of Chicago Medical Center

🇺🇸

Chicago, Illinois, United States

Fred Hutchinson Cancer Research Center

🇺🇸

Seattle, Washington, United States

Moores UCSD Cancer Center

🇺🇸

La Jolla, California, United States

Harper University Hospital

🇺🇸

Detroit, Michigan, United States

Mt. Sinai School of Medicine

🇺🇸

New York, New York, United States

Montefiore Medical Center Oncology

🇺🇸

Bronx, New York, United States

University of Rochester Medical Center

🇺🇸

Rochester, New York, United States

Memorial Sloan Kettering Cancer Center

🇺🇸

New York, New York, United States

Hackensack University Medical Center

🇺🇸

Hackensack, New Jersey, United States

Baylor University Medical Center

🇺🇸

Dallas, Texas, United States

The University of Alabama at Birmingham

🇺🇸

Birmingham, Alabama, United States

University of Colorado Denver

🇺🇸

Aurora, Colorado, United States

University of Michigan Medical School

🇺🇸

Ann Arbor, Michigan, United States

Nebraska Medical Center

🇺🇸

Omaha, Nebraska, United States

UNC Health Care Center

🇺🇸

Chapel Hill, North Carolina, United States

Oregon Health and Science University

🇺🇸

Portland, Oregon, United States

Wake Forest University School of Medicine

🇺🇸

Winston-Salem, North Carolina, United States

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