Conditions: Cytomegalovirus Infection
Keywords: Allogeneic stem cell transplant CMV seropositive (R+) transplant
Allocation: RANDOMIZED | Intervention model: SEQUENTIAL
Enrollment: 239
Healthy volunteers: no

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.

Arms:
arm 1: * 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 arm 2: * 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

Interventions: intervention 1: Brincidofovir intervention 2: Placebo
Intervention descriptions: intervention 1: Subjects received their first dose of study drug of brincidofovir (BCV) within 30 (+5) days post-transplant and were treated through Week 13 post-transplant. intervention 2: Subjects received their first dose of study drug within 30 (+5) days post-transplant and were treated through Week 13 post-transplant.

Matching placebo administered for each cohort.

Protocol document: