Clinical Trial Details
— Status: Completed
Administrative data
| NCT number |
NCT00031512 |
| Other study ID # |
99-018 |
| Secondary ID |
|
| Status |
Completed |
| Phase |
Phase 2
|
| First received |
|
| Last updated |
|
| Start date |
June 27, 2001 |
| Est. completion date |
September 15, 2012 |
Study information
| Verified date |
August 9, 2012 |
| Source |
National Institute of Allergy and Infectious Diseases (NIAID) |
| Contact |
n/a |
| Is FDA regulated |
No |
| Health authority |
|
| Study type |
Interventional
|
Clinical Trial Summary
A common group of viruses that infect humans are enteroviruses. Enteroviruses produce
illnesses in children which may range from very mild (summer colds) to severe (infections of
the brain, liver, and heart). The purpose of this study is to determine if a new drug called
pleconaril helps treat babies with enteroviral sepsis. In addition, researchers are
attempting to determine a safe and effective dose of pleconaril to help babies with this
disease. Infants who are 15 days or younger when diagnosed with enteroviral disease are
eligible for this study. Two out of 3 babies will be randomly assigned to receive Pleconaril
and the other one out of three will receive a placebo (inactive substitute). Participants
will be hospitalized while receiving study medication. Babies will receive standard treatment
care for their symptoms and will be observed for their medical progress. Participants may be
in the study for up to 2 years.
Description:
Enteroviral infection is a serious health problem in the newborn infant. Approximately 60-70%
of infants diagnosed with enteroviral disease within the first 10 days of life acquire their
infection by transmission from the mother at the time of delivery. Congenital infection is
rare but often fatal. Perinatal transmission of enteroviral infections in newborn nurseries
has also been implicated as an important route of spread of the disease in newborn infants
and postnatal transmission of enteroviral infections during seasonal peaks of enterovirus
activity occurs commonly. Thus, during periods of high prevalence of enterovirus infection in
the community, there are many potential sources of infection both during and after discharge
from the nursery, including the mother, other family members, and hospital staff.
Approximately 75% of cases of neonatal enteroviral disease carry a benign outcome, with
diagnosis and symptomatic treatment in non-intensive care unit settings. For the remainder of
patients, more serious consequences can result from systemic enteroviral infection, including
meningoencephalitis, cardiovascular collapse, myocarditis, or hepatitis. These last two
organ-specific complications carry high mortality rates. Historically, symptom management and
supportive care have been the rule in the management of these patients. No specific
therapeutic intervention is currently available for the management of these gravely ill
neonates. The current study will evaluate the antiviral drug pleconaril as a treatment for
enterovial sepsis syndrome. This trial is a multi-center, randomized, placebo-controlled
study to evaluate the virologic efficacy, safety, and pharmacokinetics of pleconaril in the
treatment of severe enteroviral sepsis syndrome. Patients will be randomized 2:1 to drug or
placebo. For enrollment into this trial, infants must have evidence of severe hepatic
involvement, myocardial involvement, and/or consumptive coagulopathy. Their age must be 15
days or less at the time of the onset of disease symptoms. Enrollment will continue until 45
subjects with confirmed enteroviral disease have been enrolled. The primary objective of this
investigation is to determine if administration of pleconaril to critically ill neonates with
enteroviral sepsis syndrome results in more rapid clearance of virus from various body sites.
Other objectives of this study are to assess the safety and pharmacokinetics of this drug in
this patient population. The effects of pleconaril on measures of clinical outcome also will
be evaluated. These include the degree of inotropic and blood product support required during
the acute illness; duration of hospitalization; the time to resolution of residual organ
injury; and short-term (at 2 months of age) and long-term (at 1 year of age) survival. The
primary endpoint will be the percentage of patients shedding virus (as detected by viral
culture) from the oropharynx (i.e. throat) 5 days after beginning study drug. The secondary
endpoints will include: duration (in days) of shedding of virus (as detected by viral
culture) from the oropharynx, rectum, urine, and serum; change in baseline laboratory
abnormalities [aspartate aminotransferase (AST), alanine aminotransferase (ALT), bilirubin,
platelets, creatinine), reflecting either resolution or progression of enteroviral disease;
pleconaril pharmacokinetics; safety; duration (in days) of total hospitalization; survival at
2 months of age; time (in days) to resolution of residual organ-related abnormalities
following acute disease; and survival at 1 year of age.
