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Phase 2 Study to Evaluate Brincidofovir for the Prevention of Adenovirus Disease
Trial ID: NCT01241344
This study was designed to assess the safety and efficacy of preemptive treatment with oral brincidofovir (BCV), as compared to placebo, for the prevention of adenovirus (AdV) disease in recipients of hematopoietic stem cell transplantation (HCT) with asymptomatic AdV viremia.
A Randomized, Placebo-controlled, Multi-site Phase 2 Study Evaluating the Safety and Efficacy of Preemptive Treatment With CMX001 for the Prevention of Adenovirus Disease Following Hematopoietic Stem Cell Transplantation
For inclusion into the study, subjects were required to fulfill all of the following
1. Were males or female aged ≥3 months to ≤75 years.
2. Received an allogeneic hematopoietic stem cell transplant (HCT).
3. Had positive serum adenovirus (AdV) PCR (>100 copies/mL) as measured by the central
laboratory (unless the subject developed AdV disease while participating in the
prescreening activities and after concurrence from the Chimerix medical monitor or
4. Was on dialysis during treatment if he/she had an estimated glomerular filtration rate
(eGFR) ≤30 mL/minute.
5. Subject or guardian(s) were willing to comply with the protocol.
6. Subject or guardian(s) were willing and able to understand the informed
7. Female subjects of child-bearing potential must have had a negative pregnancy test and
must have agreed to use 2 acceptable methods of birth control throughout the study
with at least 1 being a barrier method. Sexually active males of procreation potential
must have been able and willing to se a reliable and medically approved contraceptive
method throughout the study. At least 1 barrier method of contraception must have been
Subjects meeting any of the following exclusion criteria were excluded from participation
in the study:
1. Had possible, probable, or definitive AdV disease (unless the subject developed
probable or definitive AdV disease while participating in prescreening activities and
after concurrence from he Chimerix medical monitor or designee).
2. Had suspected gut graft versus host disease that was not biopsy-proven (subjects with
a biopsy performed were included in the study).
3. Had an eGFR ≤30 mL/minute and was not currently on dialysis.
4. Had an alanine aminotransferase or aspartate aminotransferase >5 x the upper limit of
normal (ULN), total bilirubin ≥2 x the ULN, or conjugated (direct) bilirubin ≥1.5 x
5. Had a condition that prevented oral dosing of study drug.
6. Was HIV antibody positive, based upon available medical records.
7. Had ocular hypotony, uveitis, or retinitis or any other intraocular pathology that
would have predisposed the subject to 1 of these conditions.
8. Had participated in another clinical study of an investigational drug/biologic or was
exposed to an investigational drug/biologic within 30 days of enrollment without the
prior written approval of the Chimerix medical monitor or designee. For subjects who
were participating in any clinical study involving non-investigational drugs and/or
biologics, the investigator must have obtained approval from the Chimerix medical
monitor or designee prior to enrolling the subject.
9. Was pregnant or breast-feeding or intended to conceive during the course of the study,
including the follow-up period after drug discontinuation.
10. Had known immunologic hypersensitivity to cidofovir (CDV) or brincidofovir (BCV) drug
or any of its excipients.
11. Had a history of illicit drug use or alcohol abuse within the previous 6 months.
12. Had any medical condition that, in the opinion of the investigator, might have
interfered with the subject's participation in the study, posed an added risk for the
subject, or confounded the assessment of the subject (e.g. severe cardiovascular,
central nervous system or pulmonary disease).
13. Received BCV, CDV, ribavirin, or leflunomide within the previous 14 days.
14. Was receiving or was anticipated to need treatment with digoxin that could not have
been withheld for the duration of BCV therapy.
Any exemptions to the protocol inclusion or exclusion criteria, as applicable, for subjects
who developed probably or definitive AdV disease while participating in prescreening
activities must have been discussed with and approved by the Chimerix medical monitor or
designee before the subject was allowed to receive open-label BCV therapy.
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Pediatric Stem Cell Transplantation