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The primary objective of this study is to evaluate the safety and efficacy of preemptive treatment with CMX001 versus placebo for the prevention of AdV disease in recipients of HSCT with asymptomatic AdV viremia.
A Randomized, Placebo-Controlled Multi-Site Phase 2 Study Evaluation the Safety and Efficacy of Preemptive Treatment With CMX001 for the Prevention of Adenovirus Disease Following Hematopoietic Stem Cell Transplantation in Adults and Children
The primary objective of this study is to evaluate the safety and efficacy of preemptive treatment with CMX001 versus placebo for the prevention of AdV disease in recipients of HSCT with asymptomatic AdV viremia
The outcome measure for the primary endpoint will be "treatment failure". Treatment failure is a composite endpoint consisting of:
Progression to probable or definitive AdV disease.
OR increasing AdV viremia during randomized therapy (defined as increase from baseline in AdV viremia by ≥ 1 log10, confirmed on a second measurement, at least one week apart) AND requiring discontinuation from randomized therapy
Secondary Outcome Measures
1. To compare the safety and efficacy of two dosing regimens of CMX001 versus each other and versus placebo in this indication.
2. To compare the incidence of treatment emergent dsDNA viral infections (other than those caused by AdV), in subjects treated with CMX001 QW versus CMX001 BIW versus placebo, initially, for the preemption of adenoviral disease.
3. To characterize the safety and efficacy of CMX001 open-label therapy in patients who meet the primary endpoint of treatment failure during randomized therapy.
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Ages Eligible for Study:
3 Months to 75 Years (Child, Adult, Senior)
Sexes Eligible for Study:
Accepts Healthy Volunteers:
Males and females ≥ 3 months and ≤ 75 years of age.
Subjects have received an allogeneic HSCT.
Positive serum AdV PCR (> 100 copies/mL) as measured by the central laboratory.
Subject or guardian(s) are willing to comply with the protocol.
Subject or guardian(s) are willing and able to understand the informed consent/assent.
Females of child-bearing potential must have a negative pregnancy test (serum beta-hCG) and sexually active females must use a reliable and medically approved method of contraception throughout the study. Sexually active males of procreation potential must be able and willing to use a reliable and medically approved contraceptive method throughout the study. At least one barrier method of contraception must be used, in subjects of procreation potential.
Subject has possible, probable, or definitive AdV disease. See Appendix 2Error! Reference source not found. for a definition and criteria outlining possible, probable or definitive AdV disease.
Suspected gut graft versus host disease (GVHD) that is not biopsy-proven (subjects with a biopsy performed may be included in the study).
Subject has an eGFR ≤ 30 mL/minute and is not currently on dialysis.
Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 10 times the ULN, conjugated bilirubin > 5 times the upper limit of normal (ULN).
Subject has mucositis preventing ingestion of oral medication.
Subject is Human Immunodeficiency Virus (HIV) antibody positive, based upon available medical records.
Subject has ocular hypotony, uveitis, or retinitis or any other intraocular pathology that would predispose the subject to one of these conditions.
Subject has participated in any other investigational drug study or was exposed to an investigational drug within 14 days of enrollment.
Is pregnant or breast-feeding or intending to conceive during the course of the study, including the follow-up period after drug discontinuation.
Known immunologic hypersensitivity to CDV or CMX001 drug or any of its excipients.
History of illicit drug use or alcohol abuse within the previous 6 months.
Any medical condition that, in the opinion of the Investigator, might interfere with the subject's participation in the study, poses an added risk for the subject, or confounds the assessment of the subject (e.g. severe cardiovascular, central nervous system (CNS) or pulmonary disease).
Subject has received CMX001, CDV, ribavirin, or leflunomide within the previous 14 days.