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

Phase 2

Completed

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

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	* 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
Brincidofovir	Brincidofovir	* 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

Name	Time	Method
Number of Participants With Clinically Significant CMV Infection	Randomization 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

Name	Time	Method

Locations (26): The University of Alabama at Birmingham
🇺🇸
Birmingham, Alabama, United States
Moores UCSD Cancer Center
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La Jolla, California, United States
UCLA Medical Center
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Los Angeles, California, United States
University of Colorado Denver
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Aurora, Colorado, United States
Winship Cancer Institute at Emory University
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Atlanta, Georgia, United States
University of Chicago Medical Center
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Chicago, Illinois, United States
Brigham and Womens Hospital, Division of Infectious Disease
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Boston, Massachusetts, United States
University of Michigan Medical School
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Ann Arbor, Michigan, United States
Harper University Hospital
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Detroit, Michigan, United States
University of Minnesota Medical Center
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Minneapolis, Minnesota, United States
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The University of Alabama at Birmingham
🇺🇸Birmingham, Alabama, United States