The phase II AdV HALT trial is a randomized, placebo-controlled study intended to evaluate the safety and efficacy of preemptive treatment with CMX001 to prevent immunocompromised pediatric and adult patients with asymptomatic adenovirus infection from developing disease. The adenoviruses area family of double-stranded DNA (dsDNA) viruses is responsible for respiratory diseases, including pneumonia and bronchitis, as well as other infections including gastroenteritis and acute diarrheal diseases. In immunocompromised patients, including those who have undergone HSCT, adenovirus infections are recognized as a significant cause of morbidity and mortality. Immunocompromised pediatric HSCT patients are particularly susceptible to serious and/or fatal adenovirus infections.
The study, which will be conducted at approximately 30 centers, is expected to enroll 48 pediatric and adult patients who have undergone hematopoietic stem cell transplant and have evidence of infection but no symptoms of disease. Subjects will be randomized to receive CMX001 or placebo once or twice weekly for at least six, but no more than 12, weeks. The dose of CMX001 or placebo given during the randomized treatment phase will be based on the age and weight of the subject at the time of enrollment.
The primary endpoint will be "treatment failure," consisting of progression to AdV disease or increasing AdV viremia. Secondary endpoints include safety and tolerability of CMX001, as well as other measurements that include the percentage of subjects who have emergence or progression of cytomegalovirus(CMV), Epstein-Barr virus (EBV), or BK virus (BKV) viremia or disease during therapy. Drug pharmacokinetics and the development of viral resistance will also be assessed.