Clinical Trial: Assessment of valganciclovir in neonates with CMV

This study is currently recruiting patients.

Sponsored by: National Institute of Allergy and Infectious Diseases (NIAID)
Information provided by: National Institute of Allergy and Infectious Diseases (NIAID)



o To determine the pharmacokinetics of ganciclovir following administration of oral valganciclovir syrup in neonates and young infants with symptomatic congenital CMV disease

o To identify a dose of valganciclovir to provide comparable ganciclovir plasma concentrations in neonates and young infants with symptomatic congenital CMV disease.


o To evaluate the safety and tolerability of valganciclovir syrup in the neonatal and infantile populations

o To determine the pharmacodynamics of ganciclovir following administration of oral valganciclovir syrup in neonates and young infants with symptomatic congenital CMV disease.

Condition Treatment or Intervention Phase
Cytomegalovirus Infections
 Drug: Ganciclovir
 Drug: Valganciclovir (Oral)
Phase I
Phase II

MedlinePlus related topics:  Cytomegalovirus Infections

Study Type: Interventional
Study Design: Treatment, Non-Randomized, Uncontrolled, Factorial Assignment

Official Title: A Phase I/II Pharmacokinetic and Pharmacodynamic Evaluation of Oral Valganciclovir in Neonates with Symptomatic Congenital Cytomegalovirus (CMV) Infection Involving the Central Nervous System (CASG 109)

Further Study Details: 

Expected Total Enrollment:  24

Study start: July 2002

Recent trials have demonstrated that ganciclovir treatment of neonates with symptomatic congenital CMV disease involving the CNS results in improved hearing function (or maintenance of normal hearing function) and prevents hearing deterioration at 6 months. Furthermore, ganciclovir therapy may prevent hearing deterioration at 1 year. Ganciclovir recipients also have a more rapid resolution of their transaminase elevations and a greater degree of short term growth in weight and head circumference compared with untreated patients.

Ganciclovir therapy must be administered intravenously and often requires the establishment of a central line in these babies. Valganciclovir, the oral prodrug of ganciclovir, has been developed as a syrup formulation and presents the opportunity to treat longer without the requirement for a central line, but pharmacokinetic data are needed in infants first to assure the correct dose is being utilized.

This Phase I/II, multi-center, open-label trial will assess the safety/tolerability and pharmacokinetics (ganciclovir concentrations) following administration of oral valganciclovir to neonates with symptomatic congenital CMV disease. A total of 24 patients will be evaluated.

Two different dose determination strategies will be applied in this protocol. The first is an individual patient approach. The second is a group dose modification strategy.

The primary endpoint is pharmacokinetics of ganciclovir following administration of oral valganciclovir syrup. The pharmacokinetics will be assessed by a population approach to PK data analysis. Secondary endpoints are: the pharmacokinetics of valganciclovir following administration of oral valganciclovir; the correlation of ganciclovir plasma concentrations following intravenous ganciclovir or oral valganciclovir syrup with CMV whole blood viral load; the incidence of emesis following oral valganciclovir administration (tolerability); safety as assessed by neutropenia incidence


Ages Eligible for Study:  up to  1 Month,  Genders Eligible for Study:  Both



  • Signed informed consent from parent(s) or legal guardian(s).
  • Culture confirmation of cytomegalovirus from urine or throat swab specimens.
  • Less than or equal to 30 days of age at study enrollment.
  • Weight at study enrollment > or equal to 1800 grams
  • Gestational age greater than or equal to 32 weeks.

Symptomatic congenital CMV disease, as manifest by one or more of the following:

  • Thrombocytopenia
  • Petechiae
  • Hepatomegaly
  • Splenomegaly
  • Intrauterine growth restriction
  • Hepatitis (elevated transaminases and/or bilirubin
  • Central nervous system involvement of the CMV disease (such as microcephaly, radiographic abnormalities indicative of CMV CNS disease, abnormal CSF indices for age, chorioretinitis, hearing deficits as detected by brainstem evoked response, and/or positive CMV PCR from CSF)


  • Imminent demise.
  • Patients receiving other antiviral agents or immune globulin.
  • Gastrointestinal abnormality which might preclude absorption of an oral medication (e.g., a history of necrotizing enterocolitis).
  • Creatinine clearance less than 10 mL/min at time of study enrollment.
  • Infants known to be born to women who are HIV positive (but HIV testing is not required for study entry).

Location and Contact Information

Penny Jester      877-975-7280

      University of Alabama at Birmingham (CASG), Birmingham,  Alabama,  35294,  United States; Recruiting

      Arkansas Children's Hospital, Little Rock,  Arkansas,  72202,  United States; Recruiting

      Stanford University, Stanford,  California,  94305-52,  United States; Recruiting

      Children's Hospital of Orange County, Orange,  California,  92868,  United States; Recruiting

      University of Florida HSC - Jacksonville, Jacksonville,  Florida,  32209,  United States; Recruiting

      University of Kansas, Kansas City,  Kansas,  66160,  United States; Recruiting

      Via Christi Regional Medical Center, Wichita,  Kansas,  67214,  United States; Recruiting

      University of Louisville Health Sciences Center, Louisville,  Kentucky,  40292,  United States; Recruiting

      Tulane University Health Sciences Center, New Orleans,  Louisiana,  70112,  United States; Recruiting

      Johns Hopkins University, Baltimore,  Maryland,  21287-25,  United States; Recruiting

      St. Louis University, St. Louis,  Missouri,  63104,  United States; Recruiting

      Washington University, St. Louis,  Missouri,  63110,  United States; Recruiting

      Creighton University, Omaha,  Nebraska,  68178,  United States; Recruiting

New York
      State University New York Stony Brook, Stony Brook,  New York,  11794,  United States; Recruiting

      SUNY Upstate Medical University, Syracuse,  New York,  13210,  United States; Recruiting

      North Shore - Long Island Jewish Health System, Manhasset,  New York,  11030,  United States; Recruiting

      MetroHealth Medical Center of Cleveland, Cleveland,  Ohio,  44109,  United States; Recruiting

      Thomas Jefferson University, Philadelphia,  Pennsylvania,  19107,  United States; Recruiting

      University of Texas Hlth Sci Ctr San Ant, San Antonio,  Texas,  78284-78,  United States; Recruiting

      Cook - Fort Worth Children's Medical Center, Fort Worth,  Texas,  76104,  United States; Recruiting

      University of Texas Southwestern Medical Center, Dallas,  Texas,  75235,  United States; Recruiting

Canada, Alberta
      University of Alberta, Edmonton,  Alberta,  T6G2B7,  Canada; Recruiting

Canada, Manitoba
      University of Manitoba - Winnipeg, Winnipeg,  Manitoba,  R3T 2N2,  Canada; Recruiting

More Information

Study ID Numbers:  01-595
Record last reviewed:  November 2004
Last Updated:  December 29, 2004
Record first received:  March 6, 2002 Identifier:  NCT00031434
Health Authority: United States: Food and Drug Administration processed this record on 2005-04-08

Cache Date: April 9, 2005