Phase 3
N=96
Short-Term vs. Long-Term Valganciclovir Therapy for Symptomatic Congenital CMV Infections
Cytomegalovirus Infection
Bottom Line
View on ClinicalTrials.gov: NCT00466817 ↗Enrolled (actual)
96
Serious AEs
28.4%
Results posted
Nov 2013
Primary outcome: Primary: Change in Best Ear Hearing Assessments at 6 Months. — 3; 5; 37; 36 participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Placebo (Other); Valganciclovir (Drug)
- Age
- Pediatric
- Sex
- All
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- Primary completion
- Dec 2011
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in Best Ear Hearing Assessments at 6 Months. |
3; 5; 37; 36; 3; 2 | — |
| SECONDARY Adverse Events Which Lead to Permanent Discontinuation of Valganciclovir Therapy or Lead to Irreversible Outcome of the Adverse Event. |
0; 0; 0; 0 | — |
| SECONDARY Change in Best Ear Hearing Assessments at 12 Months. |
2; 2; 23; 30; 10; 6 | — |
| SECONDARY Change in Best Ear Hearing Assessments at 24 Months. |
2; 2; 20; 30; 7; 2 | — |
| SECONDARY Number of Ears With Improvement or Protected Hearing in Hearing Assessments Over Left and Right Ears at 6 Months.(Based on 84 Ears From 43 Placebo Subjects and 82 Ears From 43 Valganciclovir Subjects) |
46; 52; 38; 30 | — |
| SECONDARY Number of Ears With Improvement or Protected Hearing in Hearing Assessments Over Left and Right Ears at 12 Months.(Based on 77 Ears From 40 Placebo Subjects and 79 Ears From 41 Valganciclovir Subjects) |
44; 58; 33; 21 | — |
| SECONDARY Number of Ears With Improvement or Protected Hearing Assessments Over Left and Right Ears at 24 Months.(Based on 58 Ears From 31 Placebo Subjects and 70 Ears From 37 Valganciclovir Subjects) |
37; 54; 21; 16 | — |
| SECONDARY Number of Ears With Hearing Deterioration Over Left and Right Ears at 6 Months.(Based on 84 Ears From 43 Placebo Subjects and 82 Ears From 43 Valganciclovir Subjects) |
75; 71; 9; 11 | — |
| SECONDARY Number of Ears With Hearing Deterioration Over Left and Right Ears at 12 Months.(Based on 77 Ears From 40 Placebo Subjects and 79 Ears From 41 Valganciclovir Subjects) |
67; 73; 10; 6 | — |
| SECONDARY Number of Ears With Hearing Deterioration Over Left and Right Ears at 24 Months.(Based on 58 Ears From 31 Placebo Subjects and 70 Ears From 37 Valganciclovir Subjects) |
53; 62; 5; 8 | — |
| SECONDARY Neurological Impairment at 12 Months of Life Utilizing the Bayley Scales of Infant and Toddler Development (Cognitive Composite Score). |
76.5; 84.2 | — |
| SECONDARY Neurological Impairment at 12 Months of Age Utilizing the Bayley Scales of Infant and Toddler Development (Receptive Communication Scaled Score). |
5.8; 6.8 | — |
| SECONDARY Neurological Impairment at 12 Months of Age Utilizing the Bayley Scales of Infant and Toddler Development (Expressive Communication Scaled Score). |
6.1; 7.3 | — |
| SECONDARY Neurological Impairment at 12 Months of Life Utilizing the Bayley Scales of Infant and Toddler Development (Language Composite Score). |
74.8; 82.9 | — |
| SECONDARY Neurological Impairment at 12 Months of Life Utilizing the Bayley Scales of Infant and Toddler Development (Fine Motor Scaled Score). |
5.5; 6.5 | — |
| SECONDARY Neurological Impairment at 12 Months of Life Utilizing the Bayley Scales of Infant and Toddler Development (Gross Motor Scaled Score). |
4.9; 5.6 | — |
| SECONDARY Neurological Impairment at 12 Months of Life Utilizing the Bayley Scales of Infant and Toddler Development (Motor Composite Score). |
70.0; 76.5 | — |
| SECONDARY Neurological Impairment at 24 Months Utilizing the Bayley Scales of Infant and Toddler Development (Receptive Communication Scaled Score). |
4.9; 6.4 | — |
| SECONDARY Neurological Impairment at 24 Months of Life Utilizing the Bayley Scales of Infant and Toddler Development (Cognitive Composite Score). |
74.9; 79.6 | — |
| SECONDARY Neurological Impairment at 24 Months of Life, Utilizing the Bayley Scales of Infant and Toddler Development (Expressive Communication Scaled Score). |
5.3; 6.5 | — |
| SECONDARY Neurologic Impairment at 24 Months of Life Utilizing the Bayley Scales of Infant and Toddler Development (Language Composite Score). |
71.2; 79.5 | — |
| SECONDARY Neurological Impairment at 24 Months, Utilizing the Bayley Scales of Infant and Toddler Development (Fine Motor Scaled Score). |
6.0; 6.9 | — |
| SECONDARY Neurological Impairment at 24 Months of Life, Utilizing the Bayley Scales of Infant and Toddler Development (Gross Motor Scaled Score). |
4.9; 6.1 | — |
| SECONDARY Neurological Impairment at 24 Months of Life, Utilizing the Bayley Scales of Infant and Toddler Development (Motor Composite Score). |
71.8; 80.1 | — |
Summary
Cytomegalovirus (CMV) infection is known to cause hearing loss and mental retardation. The purpose of this study is to compare a 6-week course to a 6-month course of the drug valganciclovir in babies born with CMV to assess the safety and efficacy of this treatment. Participants will include 104 infants (30 days old or younger) born with CMV disease. All infants will take valganciclovir by mouth for 6 weeks. At the end of the 6 week period, subjects will be assigned by chance to receive either valganciclovir or placebo (inactive substance) to complete the 6 months of antiviral treatment. Patients will be followed for the study related evaluations of safety, changes to hearing, and developmental milestones for up to 2 years. Patients will be followed by telephone contact for an additional 3 years. Thus, participants may be involved in study related procedures for approximately 5 years.
Eligibility Criteria
Inclusion Criteria
- Signed informed consent from parent(s) or legal guardian(s)
- Confirmation of cytomegalovirus (CMV) from urine or throat swab specimens by culture, shell vial, or polymerase chain reaction (PCR) tests
- 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 (CNS) involvement of the CMV disease [such as microcephaly, radiographic abnormalities indicative of CMV CNS disease, abnormal cerebrospinal fluid (CSF) indices for age, chorioretinitis, hearing deficits as detected by formal brainstem evoked response (not a screening auditory brainstem response {ABR}), and/or positive CMV PCR from CSF]
- Less than or equal to 30 days of age at study enrollment
- Weight at study enrollment greater than or equal to 1800 grams
- Gestational age greater than or equal to 32 weeks at birth
Exclusion Criteria
- 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)
- Documented renal insufficiency, as noted by a creatinine clearance less than 10 mL/min/1.73m^2 at time of study enrollment
- Breastfeeding from mother who is receiving ganciclovir, valganciclovir, foscarnet, cidofovir, or maribivir
- Infants known to be born to women who are human immunodeficiency virus (HIV) positive (but HIV testing is not required for study entry)
- Current receipt of other investigational drugs
Data sourced from ClinicalTrials.gov (NCT00466817). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.