Valganciclovir Therapy in Infants and Children With Congenital CMV Infection and Hearing Loss

• ClinicalTrials.gov Identifier: NCT01649869
• Recruitment Status: Completed
• First Posted: July 25, 2012
• Results First Posted: June 2, 2021
• Last Update Posted: June 2, 2021
• Sponsor: National Institute of Allergy and Infectious Diseases (NIAID)
• Information provided by (Responsible Party): National Institute of Allergy and Infectious Diseases (NIAID)

• Study Type: Interventional
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Triple (Participant, Care Provider, Investigator); Primary Purpose: Treatment
• Condition: Cytomegalovirus Infection
• Interventions: Other: Placebo; Drug: Valganciclovir
• Enrollment: 54

Participant Flow

Recruitment Details	For this study, subjects were enrolled once they signed the informed consent form (ICF). It was not until after hearing loss was confirmed by audiology testing and confirmation of congenital CMV by dried blood spot that a subject was randomized and started on study drug. This reduced protocol enrollment from 54 to 35.
Pre-assignment Details

Arm/Group Title	Placebo	Active
Arm/Group Description	27 Children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive placebo orally twice a day for 6 weeks Placebo: Simple Syrup as 60-90% sucrose in purified water: given orally twice a day for 6 weeks	27 Children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive valganciclovir HCl 16.0 mg/kg orally twice a day for 6 weeks Valganciclovir: Valcyte (valganciclovir hydrochloride) 50 mg of valganciclovir free base per 1 mL, oral solution: given at 16.0 mg/kg, twice a day for 6 weeks.
Period Title: Overall Study
Started	18	17
Completed	16	16
Not Completed	2	1
Reason Not Completed
Family member illness	1	0
Required treatment outside protocol	1	0
Severe hearing loss borderline eligible	0	1

Baseline Characteristics

Arm/Group Title		Placebo	Active	Total
Arm/Group Description		27 Children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive placebo orally twice a day for 6 weeks Placebo: Simple Syrup as 60-90% sucrose in purified water: given orally twice a day for 6 weeks	27 Children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive valganciclovir HCl 16.0 mg/kg orally twice a day for 6 weeks Valganciclovir: Valcyte (valganciclovir hydrochloride) 50 mg of valganciclovir free base per 1 mL, oral solution: given at 16.0 mg/kg, twice a day for 6 weeks.	Total of all reporting groups
Overall Number of Baseline Participants		18	17	35
Baseline Analysis Population Description		[Not Specified]
Age, Continuous; Median (Standard Deviation); Unit of measure: MONTHS
	Number Analyzed	18 participants	17 participants	35 participants
		19.5 (13.1)	17.8 (15.8)	18.7 (14.3)
Sex: Female, Male; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	18 participants	17 participants	35 participants
	Female	8 44.4%	6 35.3%	14 40.0%
	Male	10 55.6%	11 64.7%	21 60.0%
Ethnicity (NIH/OMB); Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	18 participants	17 participants	35 participants
	Hispanic or Latino	0 0.0%	0 0.0%	0 0.0%
	Not Hispanic or Latino	17 94.4%	15 88.2%	32 91.4%
	Unknown or Not Reported	1 5.6%	2 11.8%	3 8.6%
Race (NIH/OMB); Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	18 participants	17 participants	35 participants
	American Indian or Alaska Native	0 0.0%	0 0.0%	0 0.0%
	Asian	1 5.6%	2 11.8%	3 8.6%
	Native Hawaiian or Other Pacific Islander	0 0.0%	0 0.0%	0 0.0%
	Black or African American	2 11.1%	2 11.8%	4 11.4%
	White	15 83.3%	12 70.6%	27 77.1%
	More than one race	0 0.0%	1 5.9%	1 2.9%
	Unknown or Not Reported	0 0.0%	0 0.0%	0 0.0%
Region of Enrollment; Measure Type: Number; Unit of measure: Participants	Number Analyzed	18 participants	17 participants	35 participants
United States		7	4	11
United Kingdom		11	13	24

Outcome Measures

1. Primary Outcome

Title	Number of Ears That Had (1) Improved Hearing or no Change in Hearing (2) Worsened Hearing.
Description	A single, independent study audiologist who is masked (blinded) to treatment assignment will assess the audiology test battery for each subject and assign the classifications of normal hearing, mild hearing loss, moderate hearing loss, or severe hearing loss based upon their hearing thresholds (in decibels). The classifications will be assigned by ear (one for the left ear and one for the right ear), giving "total ear" classifications. At the analyses stage, the "best ear" classification for the subject at that study visit will be determined; for example, if a subject had mild hearing loss in their left ear and severe hearing loss in their right ear, then the "best ear" classification will be mild hearing loss. Not both ears are evaluable for all subjects. In some subjects, only one ear is evaluable.
Time Frame	Day 1 through Day 180

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Placebo	Active
Arm/Group Description:	27 Children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive placebo orally twice a day for 6 weeks Placebo: Simple Syrup as 60-90% sucrose in purified water: given orally twice a day for 6 weeks	27 Children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive valganciclovir HCl 16.0 mg/kg orally twice a day for 6 weeks Valganciclovir: Valcyte (valganciclovir hydrochloride) 50 mg of valganciclovir free base per 1 mL, oral solution: given at 16.0 mg/kg, twice a day for 6 weeks.
Overall Number of Participants Analyzed	16	15
Overall Number of Units Analyzed; Type of Units Analyzed: Ears	28	26
Measure Type: Number; Unit of Measure: Ears
Improve + no change	27	20
Worsened	1	6

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Placebo, Active
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	0.0859
	Comments	[Not Specified]
	Method	Generalized linear model
	Comments	Generalized linear model for binary outcome based on generalized estimating equations.

2. Secondary Outcome

Title	Number of Best Ear That Had (1) Improved Hearing or no Change in Hearing (2) Worsened Hearing [ex. Improved+ no Change (Normal to Normal) Versus Other].
Description	A single, independent study audiologist who is masked (blinded) to treatment assignment will assess the audiology test battery for each subject and assign the classifications of normal hearing, mild hearing loss, moderate hearing loss, or severe hearing loss based upon their hearing thresholds (in decibels). The classifications will be assigned by ear (one for the left ear and one for the right ear), giving "total ear" classifications. At the analyses stage, the "best ear" classification for the subject at that study visit will be determined; for example, if a subject had mild hearing loss in their left ear and severe hearing loss in their right ear, then the "best ear" classification will be mild hearing loss. For this outcome, we combine the improved hearing and no change for the special case only of normal to normal. Other category include worsened and no change from (1) mild to mild hearing loss, (2) moderate to moderate hearing loss, or (3) severe to severe hearing loss.
Time Frame	Day 1 through Day 180

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Placebo	Active
Arm/Group Description:	27 Children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive placebo orally twice a day for 6 weeks Placebo: Simple Syrup as 60-90% sucrose in purified water: given orally twice a day for 6 weeks	27 Children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive valganciclovir HCl 16.0 mg/kg orally twice a day for 6 weeks Valganciclovir: Valcyte (valganciclovir hydrochloride) 50 mg of valganciclovir free base per 1 mL, oral solution: given at 16.0 mg/kg, twice a day for 6 weeks.
Overall Number of Participants Analyzed	15	12
Measure Type: Number; Unit of Measure: Participants
Improved + normal to normal	9	6
No change abnormal or worsened	6	6

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Placebo, Active
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	0.7068
	Comments	[Not Specified]
	Method	Fisher Exact
	Comments	[Not Specified]

3. Secondary Outcome

Title	Change in Best Ear Hearing Assessments [Improved Versus Other] Between Baseline and Study Month 6.
Description	A single, independent study audiologist who is masked (blinded) to treatment assignment will assess the audiology test battery for each subject and assign the classifications of normal hearing, mild hearing loss, moderate hearing loss, or severe hearing loss based upon their hearing thresholds (in decibels). The classifications will be assigned by ear (one for the left ear and one for the right ear), giving "total ear" classifications. At the analyses stage, the "best ear" classification for the subject at that study visit will be determined; for example, if a subject had mild hearing loss in their left ear and severe hearing loss in their right ear, then the "best ear" classification will be mild hearing loss.
Time Frame	Day 1 through Day 180

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Placebo	Active
Arm/Group Description:	27 Children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive placebo orally twice a day for 6 weeks Placebo: Simple Syrup as 60-90% sucrose in purified water: given orally twice a day for 6 weeks	27 Children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive valganciclovir HCl 16.0 mg/kg orally twice a day for 6 weeks Valganciclovir: Valcyte (valganciclovir hydrochloride) 50 mg of valganciclovir free base per 1 mL, oral solution: given at 16.0 mg/kg, twice a day for 6 weeks.
Overall Number of Participants Analyzed	15	12
Measure Type: Number; Unit of Measure: Participants
Improved	0	0
No change or worsened	15	12

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Placebo, Active
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	1
	Comments	[Not Specified]
	Method	Fisher Exact
	Comments	[Not Specified]

4. Secondary Outcome

Title	Change in Best Ear Hearing Assessments [Worse + no Change (Abnormal to Abnormal) Versus Other] Between Baseline and Study Month 6.
Description	A single, independent study audiologist who is masked (blinded) to treatment assignment will assess the audiology test battery for each subject and assign the classifications of normal hearing, mild hearing loss, moderate hearing loss, or severe hearing loss based upon their hearing thresholds (in decibels). The classifications will be assigned by ear (one for the left ear and one for the right ear), giving "total ear" classifications. At the analyses stage, the "best ear" classification for the subject at that study visit will be determined; for example, if a subject had mild hearing loss in their left ear and severe hearing loss in their right ear, then the "best ear" classification will be mild hearing loss.
Time Frame	Day 1 through Day 180

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Placebo	Active
Arm/Group Description:	27 Children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive placebo orally twice a day for 6 weeks Placebo: Simple Syrup as 60-90% sucrose in purified water: given orally twice a day for 6 weeks	27 Children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive valganciclovir HCl 16.0 mg/kg orally twice a day for 6 weeks Valganciclovir: Valcyte (valganciclovir hydrochloride) 50 mg of valganciclovir free base per 1 mL, oral solution: given at 16.0 mg/kg, twice a day for 6 weeks.
Overall Number of Participants Analyzed	15	12
Measure Type: Number; Unit of Measure: Participants
No change abnormal to abnormal + worsened	6	6
Improved + normal to normal	9	6

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Placebo, Active
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	0.7068
	Comments	[Not Specified]
	Method	Fisher Exact
	Comments	[Not Specified]

5. Secondary Outcome

Title	Change in Best Ear Hearing Assessments [Worse Versus Other] Between Baseline and Study Month 6.
Description	A single, independent study audiologist who is masked (blinded) to treatment assignment will assess the audiology test battery for each subject and assign the classifications of normal hearing, mild hearing loss, moderate hearing loss, or severe hearing loss based upon their hearing thresholds (in decibels). The classifications will be assigned by ear (one for the left ear and one for the right ear), giving "total ear" classifications. At the analyses stage, the "best ear" classification for the subject at that study visit will be determined; for example, if a subject had mild hearing loss in their left ear and severe hearing loss in their right ear, then the "best ear" classification will be mild hearing loss.
Time Frame	Day 1 through Day 180

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Placebo	Active
Arm/Group Description:	27 Children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive placebo orally twice a day for 6 weeks Placebo: Simple Syrup as 60-90% sucrose in purified water: given orally twice a day for 6 weeks	27 Children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive valganciclovir HCl 16.0 mg/kg orally twice a day for 6 weeks Valganciclovir: Valcyte (valganciclovir hydrochloride) 50 mg of valganciclovir free base per 1 mL, oral solution: given at 16.0 mg/kg, twice a day for 6 weeks.
Overall Number of Participants Analyzed	15	12
Measure Type: Number; Unit of Measure: Participants
Worsened	0	3
Other	15	9

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Placebo, Active
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	0.0752
	Comments	[Not Specified]
	Method	Fisher Exact
	Comments	[Not Specified]

6. Secondary Outcome

Title	Change in Total Ear Hearing Assessments [Improved Versus Other] Between Baseline and Study Month 6.
Description	A single, independent study audiologist who is masked (blinded) to treatment assignment will assess the audiology test battery for each subject and assign the classifications of normal hearing, mild hearing loss, moderate hearing loss, or severe hearing loss based upon their hearing thresholds (in decibels). The classifications will be assigned by ear (one for the left ear and one for the right ear), giving "total ear" classifications. At the analyses stage, the "best ear" classification for the subject at that study visit will be determined; for example, if a subject had mild hearing loss in their left ear and severe hearing loss in their right ear, then the "best ear" classification will be mild hearing loss.
Time Frame	Day 1 through Day 180

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Placebo	Active
Arm/Group Description:	27 Children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive placebo orally twice a day for 6 weeks Placebo: Simple Syrup as 60-90% sucrose in purified water: given orally twice a day for 6 weeks	27 Children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive valganciclovir HCl 16.0 mg/kg orally twice a day for 6 weeks Valganciclovir: Valcyte (valganciclovir hydrochloride) 50 mg of valganciclovir free base per 1 mL, oral solution: given at 16.0 mg/kg, twice a day for 6 weeks.
Overall Number of Participants Analyzed	16	15
Overall Number of Units Analyzed; Type of Units Analyzed: Ears	28	26
Measure Type: Number; Unit of Measure: Ears
Improved	0	0
Other	28	26

7. Secondary Outcome

Title	Change in Total Ear Hearing Assessments [Worse+ no Change (Abnormal to Abnormal) Versus Other] Between Baseline and Study Month 6.
Description	A single, independent study audiologist who is masked (blinded) to treatment assignment will assess the audiology test battery for each subject and assign the classifications of normal hearing, mild hearing loss, moderate hearing loss, or severe hearing loss based upon their hearing thresholds (in decibels). The classifications will be assigned by ear (one for the left ear and one for the right ear), giving "total ear" classifications. At the analyses stage, the "best ear" classification for the subject at that study visit will be determined; for example, if a subject had mild hearing loss in their left ear and severe hearing loss in their right ear, then the "best ear" classification will be mild hearing loss.
Time Frame	Day 1 through Day 180

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Placebo	Active
Arm/Group Description:	27 Children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive placebo orally twice a day for 6 weeks Placebo: Simple Syrup as 60-90% sucrose in purified water: given orally twice a day for 6 weeks	27 Children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive valganciclovir HCl 16.0 mg/kg orally twice a day for 6 weeks Valganciclovir: Valcyte (valganciclovir hydrochloride) 50 mg of valganciclovir free base per 1 mL, oral solution: given at 16.0 mg/kg, twice a day for 6 weeks.
Overall Number of Participants Analyzed	16	15
Overall Number of Units Analyzed; Type of Units Analyzed: Ears	28	26
Measure Type: Number; Unit of Measure: Ears
worse + no change (abnormal to abnormal)	19	20
Other	9	6

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Placebo, Active
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	0.4823
	Comments	[Not Specified]
	Method	Generalized linear model
	Comments	Generalized linear model for binary outcome using generalized estimating equations.

8. Secondary Outcome

Title	Change in Total Ear Hearing Assessments [Worse Versus Other] Between Baseline and Study Month 6.
Description	A single, independent study audiologist who is masked (blinded) to treatment assignment will assess the audiology test battery for each subject and assign the classifications of normal hearing, mild hearing loss, moderate hearing loss, or severe hearing loss based upon their hearing thresholds (in decibels). The classifications will be assigned by ear (one for the left ear and one for the right ear), giving "total ear" classifications. At the analyses stage, the "best ear" classification for the subject at that study visit will be determined; for example, if a subject had mild hearing loss in their left ear and severe hearing loss in their right ear, then the "best ear" classification will be mild hearing loss.
Time Frame	Day 1 through Day 180

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Placebo	Active
Arm/Group Description:	27 Children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive placebo orally twice a day for 6 weeks Placebo: Simple Syrup as 60-90% sucrose in purified water: given orally twice a day for 6 weeks	27 Children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive valganciclovir HCl 16.0 mg/kg orally twice a day for 6 weeks Valganciclovir: Valcyte (valganciclovir hydrochloride) 50 mg of valganciclovir free base per 1 mL, oral solution: given at 16.0 mg/kg, twice a day for 6 weeks.
Overall Number of Participants Analyzed	16	15
Overall Number of Units Analyzed; Type of Units Analyzed: Ears	28	26
Measure Type: Number; Unit of Measure: Ears
Worsened	1	6
Other	27	20

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Placebo, Active
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	0.0859
	Comments	[Not Specified]
	Method	Generalized linear model
	Comments	Generalized linear model for binary outcome using generalized estimating equations.

9. Secondary Outcome

Title	Association of Change in Viral Load (Blood) With Change in Total Ear Hearing at 6 Months
Description	Analysis of actual viral load was done using log base 10 transformation. Undetectable viral load value was replaced by a value of 10. A summary measure of the viral load over time considers all time points available by calculating the average area under the curve (AUC) (trapezoidal rule) applied to the log base 10 viral load. Average is based on the maximum period of time with viral load data for a given subject. The average or standardize AUC units is therefore the original AUC units of log 10 copies/ml*days divided by days in study which equals log 10 copies/ml.
Time Frame	At 6 months

Outcome Measure Data

Analysis Population Description

Analysis was stratified by hearing outcome--total analyzed is the sum of the number analyzed for 2 rows below

Arm/Group Title		Randomized Group
Arm/Group Description:		Randomized children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive placebo orally twice a day for 6 weeks
Overall Number of Participants Analyzed		31
Overall Number of Units Analyzed; Type of Units Analyzed: Ears		54
Mean (Standard Deviation); Unit of Measure: log 10 copies/ml
Improved + normal to normal	Number Analyzed	15 Ears
		1.396 (0.542)
No change abnormal + worsened	Number Analyzed	39 Ears
		1.326 (0.566)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Randomized Group
	Comments	[Not Specified]
	Type of Statistical Test	Other
	Comments	Association of binary outcome and continuous outcome
Statistical Test of Hypothesis	P-Value	0.8212
	Comments	[Not Specified]
	Method	Generalized linear model
	Comments	For binary outcome using generalized estimating equations

10. Secondary Outcome

Title	Association of Change in Viral Load (Saliva) With Change in Total Ear Hearing at 6 Months
Description	Analysis of actual viral load was done using log base 10 transformation. Undetectable viral load value was replaced by a value of 10. A summary measure of the viral load over time considers all time points available by calculating the average area under the curve (AUC) (trapezoidal rule) applied to the log base 10 viral load. Average is based on the maximum period of time with viral load data for a given subject. The average or standardize AUC units is therefore the original AUC units or log 10 copies/ml*days divided by days in study which equals log 10 copies/ml.
Time Frame	At 6 months

Outcome Measure Data

Analysis Population Description

Analysis stratified by hearing outcome--total analyzed is the sum of the number analyzed for 2 rows belos

Arm/Group Title		Randomized Group
Arm/Group Description:		Randomized children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive placebo orally twice a day for 6 weeks
Overall Number of Participants Analyzed		31
Overall Number of Units Analyzed; Type of Units Analyzed: Ears		54
Mean (Standard Deviation); Unit of Measure: log 10 copies/ml
Improved + normal to normal	Number Analyzed	15 Ears
		2.447 (1.715)
No change abnormal + worsened	Number Analyzed	39 Ears
		2.290 (1.349)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Randomized Group
	Comments	[Not Specified]
	Type of Statistical Test	Other
	Comments	Association of binary outcome and continuous outcome and continuous outcome
Statistical Test of Hypothesis	P-Value	0.8356
	Comments	[Not Specified]
	Method	Generalized linear model
	Comments	Generalized linear model for binary outcome using generalized estimating equations.

11. Secondary Outcome

Title	Association of Change in Viral Load (Urine) With Change in Total Ear Hearing at 6 Months
Description	Analysis of actual viral load was done using log base 10 transformation. Undetectable viral load value was replaced by a value of 10. A summary measure of the viral load over time considers all time points available by calculating the average area under the curve (AUC) (trapezoidal rule) applied to the log base 10 viral load. Average is based on the maximum period of time with viral load data for a given subject. The average or standardize AUC units is therefore the original AUC units of log 10 copies/ml*days divided by days in study which equals log 10 copies/ml.
Time Frame	At 6 months

Outcome Measure Data

Analysis Population Description

Analysis stratified by hearing outcome--total analyzed is the sum of the number analyzed for 2 rows below

Arm/Group Title		Randomized Group
Arm/Group Description:		Randomized children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive placebo orally twice a day for 6 weeks
Overall Number of Participants Analyzed		29
Overall Number of Units Analyzed; Type of Units Analyzed: Ears		50
Mean (Standard Deviation); Unit of Measure: log 10 copies/ml
Improved + normal to normal	Number Analyzed	15 Ears
		3.562 (1.139)
No change abnormal + worsened	Number Analyzed	35 Ears
		3.583 (1.370)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Randomized Group
	Comments	[Not Specified]
	Type of Statistical Test	Other
	Comments	Association of binary outcome and continuous outcome
Statistical Test of Hypothesis	P-Value	0.7961
	Comments	[Not Specified]
	Method	Generalized linear model
	Comments	Generalized linear model for binary outcome using generalized estimating equations

12. Secondary Outcome

Title	Association of Change in Viral Load (Blood) With Change in Best Ear Hearing at 6 Months
Description	Analysis of actual viral load was done using log base 10 transformation. Undetectable viral load value was replaced by a value of 10. A summary measure of the viral load over time considers all time points available by calculating the average area under the curve (AUC) (trapezoidal rule) applied to the log base 10 viral load. Average is based on the maximum period of time with viral load data for a given subject. The average or standardize AUC units is therefore the original AUC units of log 10 copies/ml*days divided by days in study which equals log 10 copies/ml.
Time Frame	At 6 months

Outcome Measure Data

Analysis Population Description

Analysis stratified by hearing outcome--total analyzed is the sum of the number analyzed for 2 rows below

Arm/Group Title		Randomized Group
Arm/Group Description:		Randomized children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive placebo orally twice a day for 6 weeks
Overall Number of Participants Analyzed		27
Mean (Standard Deviation); Unit of Measure: log 10 copies/ml
Improved + normal to normal	Number Analyzed	15 participants
		1.396 (0.542)
No change abnormal + worsened	Number Analyzed	12 participants
		1.359 (0.668)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Randomized Group
	Comments	[Not Specified]
	Type of Statistical Test	Other
	Comments	Association of binary outcome and continuous outcome
Statistical Test of Hypothesis	P-Value	0.8675
	Comments	[Not Specified]
	Method	Generalized linear model
	Comments	Generalized linear model for binary outcome

13. Secondary Outcome

Title	Association of Change in Viral Load (Saliva) With Change in Best Ear Hearing at 6 Months
Description	Analysis of actual viral load was done using log base 10 transformation. Undetectable viral load value was replaced by a value of 10. A summary measure of the viral load over time considers all time points available by calculating the average area under the curve (AUC) (trapezoidal rule) applied to the log base 10 viral load. Average is based on the maximum period of time with viral load data for a given subject. The average or standardize AUC units is therefore the original AUC units of log 10 copies/ml*days divided by days in study which equals log 10 copies/ml.
Time Frame	At 6 months

Outcome Measure Data

Analysis Population Description

Analysis stratified by hearing outcome--total analyzed is the sum of the number analyzed for 2 rows below

Arm/Group Title		Randomized Group
Arm/Group Description:		Randomized children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive placebo orally twice a day for 6 weeks
Overall Number of Participants Analyzed		27
Mean (Standard Deviation); Unit of Measure: log 10 copies/ml
Improved + normal to normal	Number Analyzed	15 participants
		2.447 (1.715)
No change abnormal + worsened	Number Analyzed	12 participants
		2.423 (1.484)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Randomized Group
	Comments	[Not Specified]
	Type of Statistical Test	Other
	Comments	Association of binary outcome and continuous outcome
Statistical Test of Hypothesis	P-Value	0.9682
	Comments	[Not Specified]
	Method	Generalized linear model
	Comments	Generalized linear model for binary outcome

14. Secondary Outcome

Title	Association of Change in Viral Load (Urine) With Change in Best Ear Hearing at 6 Months
Description	Analysis of actual viral load was done using log base 10 transformation. Undetectable viral load value was replaced by a value of 10. A summary measure of the viral load over time considers all time points available by calculating the average area under the curve (AUC) (trapezoidal rule) applied to the log base 10 viral load. Average is based on the maximum period of time with viral load data for a given subject. The average or standardize AUC units is therefore the original AUC units of log 10 copies/ml*days divided by days in study which equals log 10 copies/ml.
Time Frame	At 6 months

Outcome Measure Data

Analysis Population Description

Analysis stratified by hearing outcome--total analyzed is the sum of the number of analyzed for 2 rows below

Arm/Group Title		Randomized Group
Arm/Group Description:		Randomized children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive placebo orally twice a day for 6 weeks
Overall Number of Participants Analyzed		25
Mean (Standard Deviation); Unit of Measure: log 10 copies/ml
Improved + normal to normal	Number Analyzed	15 participants
		3.562 (1.139)
No change abnormal + worsened	Number Analyzed	10 participants
		3.831 (1.570)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Randomized Group
	Comments	[Not Specified]
	Type of Statistical Test	Other
	Comments	Association of binary outcome and continuous outcome
Statistical Test of Hypothesis	P-Value	0.6063
	Comments	[Not Specified]
	Method	Generalized linear model
	Comments	Generalized linear model for binary outcome

15. Secondary Outcome

Title	Detection of Viruria (Urine) by PCR Six Weeks After Trial Entry
Description	Each subject either has positive or negative PCR results. Virus is detected if the PCR is positive.
Time Frame	At 6 weeks (Day 42)

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Placebo	Active
Arm/Group Description:	27 Children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive placebo orally twice a day for 6 weeks Placebo: Simple Syrup as 60-90% sucrose in purified water: given orally twice a day for 6 weeks	27 Children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive valganciclovir HCl 16.0 mg/kg orally twice a day for 6 weeks Valganciclovir: Valcyte (valganciclovir hydrochloride) 50 mg of valganciclovir free base per 1 mL, oral solution: given at 16.0 mg/kg, twice a day for 6 weeks.
Overall Number of Participants Analyzed	12	12
Measure Type: Number; Unit of Measure: Participants
Positive	11	1
Negative	1	11

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Placebo, Active
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	0.0001
	Comments	[Not Specified]
	Method	Fisher Exact
	Comments	[Not Specified]

16. Secondary Outcome

Title	Detection of Viruria (Urine) by PCR Six Month After Trial Entry
Description	Each subject either has positive or negative PCR results. Virus is detected if the PCR is positive.
Time Frame	At 6 months

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Placebo	Active
Arm/Group Description:	27 Children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive placebo orally twice a day for 6 weeks Placebo: Simple Syrup as 60-90% sucrose in purified water: given orally twice a day for 6 weeks	27 Children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive valganciclovir HCl 16.0 mg/kg orally twice a day for 6 weeks Valganciclovir: Valcyte (valganciclovir hydrochloride) 50 mg of valganciclovir free base per 1 mL, oral solution: given at 16.0 mg/kg, twice a day for 6 weeks.
Overall Number of Participants Analyzed	11	14
Measure Type: Number; Unit of Measure: Participants
Positive	10	11
Negative	1	3

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Placebo, Active
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	0.6043
	Comments	[Not Specified]
	Method	Fisher Exact
	Comments	[Not Specified]

17. Secondary Outcome

Title	Detection of Viremia (Blood) by PCR Six Weeks After Trial Entry
Description	Each subject either has positive or negative PCR results. Virus is detected if the PCR is positive.
Time Frame	At 6 weeks (Day 42)

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Placebo	Active
Arm/Group Description:	27 Children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive placebo orally twice a day for 6 weeks Placebo: Simple Syrup as 60-90% sucrose in purified water: given orally twice a day for 6 weeks	27 Children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive valganciclovir HCl 16.0 mg/kg orally twice a day for 6 weeks Valganciclovir: Valcyte (valganciclovir hydrochloride) 50 mg of valganciclovir free base per 1 mL, oral solution: given at 16.0 mg/kg, twice a day for 6 weeks.
Overall Number of Participants Analyzed	15	14
Measure Type: Number; Unit of Measure: Participants
Positive	4	2
Negative	11	12

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Placebo, Active
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	0.6513
	Comments	[Not Specified]
	Method	Fisher Exact
	Comments	[Not Specified]

18. Secondary Outcome

Title	Detection of Viremia (Blood) by PCR Six Month After Trial Entry
Description	Each subject either has positive or negative PCR results. Virus is detected if the PCR is positive.
Time Frame	At 6 months

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Placebo	Active
Arm/Group Description:	27 Children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive placebo orally twice a day for 6 weeks Placebo: Simple Syrup as 60-90% sucrose in purified water: given orally twice a day for 6 weeks	27 Children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive valganciclovir HCl 16.0 mg/kg orally twice a day for 6 weeks Valganciclovir: Valcyte (valganciclovir hydrochloride) 50 mg of valganciclovir free base per 1 mL, oral solution: given at 16.0 mg/kg, twice a day for 6 weeks.
Overall Number of Participants Analyzed	15	15
Measure Type: Number; Unit of Measure: Participants
Positive	4	3
Negative	11	12

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Placebo, Active
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	1.0
	Comments	[Not Specified]
	Method	Fisher Exact
	Comments	[Not Specified]

19. Secondary Outcome

Title	Detection of CMV in Saliva by PCR Six Weeks After Trial Entry
Description	Each subject either has positive or negative PCR results. Virus is detected if the PCR is positive.
Time Frame	At 6 weeks (Day 42)

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Placebo	Active
Arm/Group Description:	27 Children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive placebo orally twice a day for 6 weeks Placebo: Simple Syrup as 60-90% sucrose in purified water: given orally twice a day for 6 weeks	27 Children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive valganciclovir HCl 16.0 mg/kg orally twice a day for 6 weeks Valganciclovir: Valcyte (valganciclovir hydrochloride) 50 mg of valganciclovir free base per 1 mL, oral solution: given at 16.0 mg/kg, twice a day for 6 weeks.
Overall Number of Participants Analyzed	16	16
Measure Type: Number; Unit of Measure: Participants
Positive	9	3
Negative	7	13

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Placebo, Active
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	0.0659
	Comments	[Not Specified]
	Method	Fisher Exact
	Comments	[Not Specified]

20. Secondary Outcome

Title	Detection of CMV in Saliva PCR Six Month After Trial Entry
Description	Each subject either has positive or negative PCR results. Virus is detected if the PCR is positive.
Time Frame	At 6 months

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Placebo	Active
Arm/Group Description:	27 Children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive placebo orally twice a day for 6 weeks Placebo: Simple Syrup as 60-90% sucrose in purified water: given orally twice a day for 6 weeks	27 Children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive valganciclovir HCl 16.0 mg/kg orally twice a day for 6 weeks Valganciclovir: Valcyte (valganciclovir hydrochloride) 50 mg of valganciclovir free base per 1 mL, oral solution: given at 16.0 mg/kg, twice a day for 6 weeks.
Overall Number of Participants Analyzed	16	16
Measure Type: Number; Unit of Measure: Participants
Positive	8	7
Negative	8	9

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Placebo, Active
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	1.0
	Comments	[Not Specified]
	Method	Fisher Exact
	Comments	[Not Specified]

21. Secondary Outcome

Title	The Quantitative Log Change in Viremia From Baseline to Month 6.
Description	The quantitative change (Month 6 minus baseline) in viremia (blood) Quantitative viral load by PCR in log 10 units measured in urine after 6 weeks of therapy; if undetectable, viral load is assigned a value of 10 (1 in log 10 units).
Time Frame	Baseline to month 6

Outcome Measure Data

Analysis Population Description

Overall number of participants analyzed=34: 17 active and 17 placebo

Arm/Group Title		Randomized Group
Arm/Group Description:		Randomized children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive placebo orally twice a day for 6 weeks
Overall Number of Participants Analyzed		34
Least Squares Mean (95% Confidence Interval); Unit of Measure: log10 IU/ml
Active	Number Analyzed	17 participants
		0.4908; (0.08108 to 0.9005)
Placebo	Number Analyzed	17 participants
		-0.1528; (-0.5721 to 0.2664)

22. Secondary Outcome

Title	The Quantitative Log Reduction in Viruria Detected After 6 Weeks of Therapy
Description	The quantitative log reduction in viruria (urine) detected after 6 weeks of therapy. Quantitative viral load by PCR in log 10 units measured in urine after 6 weeks of therapy; if undetectable, viral load is assigned a value of 10 (1 in log 10 units)
Time Frame	Baseline thru months 6

Outcome Measure Data

Analysis Population Description

Overall number of participants analyzed=28: 15 active and 13 placebo

Arm/Group Title		Randomized Group
Arm/Group Description:		Randomized children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive placebo orally twice a day for 6 weeks
Overall Number of Participants Analyzed		28
Least Squares Mean (95% Confidence Interval); Unit of Measure: log10 IU/ml
Active	Number Analyzed	15 participants
		1.2152; (0.5274 to 1.9029)
Placebo	Number Analyzed	13 participants
		0.8390; (-0.05057 to 1.7286)

23. Secondary Outcome

Title	The Quantitative Log Reduction in CMV in Saliva Detected After 6 Weeks of Therapy
Description	The quantitative log reduction in CMV in saliva (urine) detected after 6 weeks of therapy. Quantitative viral load by PCR in log 10 units measured in urine after 6 weeks of therapy; if undetectable, viral load is assigned a value of 10 (1 in log 10 units)
Time Frame	Baseline thru months 6

Outcome Measure Data

Analysis Population Description

Overall number of participants analyzed=34

Arm/Group Title		Randomized Group
Arm/Group Description:		Randomized children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive placebo orally twice a day for 6 weeks
Overall Number of Participants Analyzed		34
Least Squares Mean (95% Confidence Interval); Unit of Measure: log10 IU/ml
Active	Number Analyzed	17 participants
		1.3202; (0.6894 to 1.9509)
Placebo	Number Analyzed	17 participants
		0.0057; (-0.6395 to 0.6508)

24. Secondary Outcome

Title	Number of Adverse Events in the Active Group That Resulted in Discontinuation of Valganciclovir
Description	AE resulting in discontinuation of valganciclovir (active group only). This outcome summarizes the number of adverse events (AEs) that resulted in the discontinuation of valganciclovir in the active group only.
Time Frame	Day 1 thru day 70

Outcome Measure Data

Analysis Population Description

No participants in the active group were discontinued due to adverse event.

Arm/Group Title	Randomized Group
Arm/Group Description:	Randomized children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive valganciclovir orally twice a day for 6 weeks
Overall Number of Participants Analyzed	17
Measure Type: Number; Unit of Measure: Participants
No	17
Yes	0

25. Secondary Outcome

Title	Adverse Event (AE) Resulting in Unresolved Outcome
Description	Adverse event resulting in unresolved outcome. This outcome summarizes the number of adverse events (AEs) that resulted in unresolved outcome of that AE.
Time Frame	Day 1 thru day 70

Outcome Measure Data

Analysis Population Description

Overall number of participants analyzed=18

Arm/Group Title	Placebo	Active
Arm/Group Description:	27 Children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive placebo orally twice a day for 6 weeks Placebo: Simple Syrup as 60-90% sucrose in purified water: given orally twice a day for 6 weeks	27 Children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive valganciclovir HCl 16.0 mg/kg orally twice a day for 6 weeks Valganciclovir: Valcyte (valganciclovir hydrochloride) 50 mg of valganciclovir free base per 1 mL, oral solution: given at 16.0 mg/kg, twice a day for 6 weeks.
Overall Number of Participants Analyzed	18	17
Measure Type: Number; Unit of Measure: Participants
No	18	17
Yes	0	0

26. Secondary Outcome

Title	Adverse Event (AE) Resulting in Unanticipated Medically Attended Visit
Description	Adverse event resulting in unanticipated medically attended visit. This outcome summarizes the number of adverse events (AEs) that resulted in the unanticipated medically attended visit.
Time Frame	Day 1 thru day 70

Outcome Measure Data

Analysis Population Description

Overall number of participants analyzed=18

Arm/Group Title	Placebo	Active
Arm/Group Description:	27 Children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive placebo orally twice a day for 6 weeks Placebo: Simple Syrup as 60-90% sucrose in purified water: given orally twice a day for 6 weeks	27 Children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive valganciclovir HCl 16.0 mg/kg orally twice a day for 6 weeks Valganciclovir: Valcyte (valganciclovir hydrochloride) 50 mg of valganciclovir free base per 1 mL, oral solution: given at 16.0 mg/kg, twice a day for 6 weeks.
Overall Number of Participants Analyzed	18	17
Measure Type: Number; Unit of Measure: Participants
No	15	17
Yes	3	0

Adverse Events

Time Frame	All AEs and SAEs from Study Day 1 through four weeks following the last dose of study drug. All AEs and SAEs will be followed until resolution, even if this extends beyond the study reporting period. Resolution of an AE is defined as the return to pretreatment status or stabilization of the condition with the expectation that it will remain chronic.
Adverse Event Reporting Description	Any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of medicinal (investigational) product whether or not considered drug related. The occurrence of an AE may come to the attention of study personnel during study visits and interviews of a study recipient presenting for medical care, or upon review by a study monitor.
Arm/Group Title	Placebo		Active
Arm/Group Description	27 Children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive placebo orally twice a day for 6 weeks Placebo: Simple Syrup as 60-90% sucrose in purified water: given orally twice a day for 6 weeks		27 Children between 1 month and 3 years of age (up to 4th birthday) with sensoneural hearing loss and documented CMV infection will receive valganciclovir HCl 16.0 mg/kg orally twice a day for 6 weeks Valganciclovir: Valcyte (valganciclovir hydrochloride) 50 mg of valganciclovir free base per 1 mL, oral solution: given at 16.0 mg/kg, twice a day for 6 weeks.
All-Cause Mortality
	Placebo		Active
	Affected / at Risk (%)		Affected / at Risk (%)
Total	0/18 (0.00%)		0/17 (0.00%)
Serious Adverse Events
	Placebo		Active
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/18 (0.00%)		1/17 (5.88%)
Respiratory, thoracic and mediastinal disorders
Respiratory Distress † 1	0/18 (0.00%)	0	1/17 (5.88%)	1
1 Term from vocabulary, MedDRA (10.0); † Indicates events were collected by systematic assessment
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	0%
	Placebo		Active
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	18/35 (51.43%)		17/35 (48.57%)
Blood and lymphatic system disorders
Decrease in hemoglobin † 1	1/18 (5.56%)	1	0/17 (0.00%)	0
Neutropenia † 1	2/18 (11.11%)	4	1/17 (5.88%)	1
Monocytopenia † 1	0/18 (0.00%)	0	1/17 (5.88%)	1
Ear and labyrinth disorders
Bilateral ear infection † 1	1/18 (5.56%)	1	0/17 (0.00%)	0
Otitis Media † 1	1/18 (5.56%)	1	1/17 (5.88%)	1
Ear Infection † 1	0/18 (0.00%)	0	1/17 (5.88%)	1
Gastrointestinal disorders
Constipation † 1	2/18 (11.11%)	3	1/17 (5.88%)	1
Diarrhoea † 1	0/18 (0.00%)	0	1/17 (5.88%)	1
Emesis † 1	0/18 (0.00%)	0	1/17 (5.88%)	1
Gastroenteritis † 1	0/18 (0.00%)	0	1/17 (5.88%)	1
Loose stools † 1	1/18 (5.56%)	2	0/17 (0.00%)	0
Reduced Appetite † 1	0/18 (0.00%)	0	1/17 (5.88%)	1
Reflux † 1	0/18 (0.00%)	0	1/17 (5.88%)	1
Teething † 1	2/18 (11.11%)	2	2/17 (11.76%)	2
Viral Gastroenteritis † 1	0/18 (0.00%)	0	1/17 (5.88%)	1
Vomiting † 1	2/18 (11.11%)	5	4/17 (23.53%)	4
Weight Loss † 1	0/18 (0.00%)	0	1/17 (5.88%)	1
General disorders
Fall on chin † 1	1/18 (5.56%)	1	0/17 (0.00%)	0
Fever † 1	3/18 (16.67%)	3	1/17 (5.88%)	1
Hyperkalemia † 1	0/18 (0.00%)	0	1/17 (5.88%)	1
Long Sighted † 1	0/18 (0.00%)	0	1/17 (5.88%)	1
Nose Bleed † 1	1/18 (5.56%)	1	0/17 (0.00%)	0
Pyrexia † 1	0/18 (0.00%)	0	1/17 (5.88%)	1
Raised Temperature † 1	1/18 (5.56%)	1	0/17 (0.00%)	0
Infections and infestations
Chicken Pox † 1	1/18 (5.56%)	1	0/17 (0.00%)	0
Hand, Foot and Mouth Disease † 1	1/18 (5.56%)	1	1/17 (5.88%)	1
Infection at Surgical Incision Site † 1	0/18 (0.00%)	0	1/17 (5.88%)	1
Rhinitis † 1	1/18 (5.56%)	1	0/17 (0.00%)	0
Tonsillitis † 1	1/18 (5.56%)	1	1/17 (5.88%)	1
Viral Illness † 1	2/18 (11.11%)	3	1/17 (5.88%)	1
Injury, poisoning and procedural complications
Head, Eye and Right Arm Injury--Suspected trauma to lacrimal sac. † 1	0/18 (0.00%)	0	1/17 (5.88%)	1
Minor Injury--Bruise Right Eye and Cheek and Left Forehead † 1	0/18 (0.00%)	0	1/17 (5.88%)	1
Musculoskeletal and connective tissue disorders
Buckle Fracture of Right Tibia and Fibula † 1	0/18 (0.00%)	0	1/17 (5.88%)	1
Osteopenia † 1	0/18 (0.00%)	0	1/17 (5.88%)	1
Psychiatric disorders
Agitation † 1	1/18 (5.56%)	1	0/17 (0.00%)	0
Crying since first dose † 1	1/18 (5.56%)	1	0/17 (0.00%)	0
Fussiness † 1	1/18 (5.56%)	1	0/17 (0.00%)	0
Hallucination-Nightmare † 1	0/18 (0.00%)	0	1/17 (5.88%)	1
Intermittent Hyperactivity † 1	1/18 (5.56%)	1	0/17 (0.00%)	0
Lethargic and Sleeping more than usual † 1	0/18 (0.00%)	0	1/17 (5.88%)	1
Screaming--Unsettled † 1	0/18 (0.00%)	0	1/17 (5.88%)	1
Renal and urinary disorders
Blood in Urine † 1	0/18 (0.00%)	0	1/17 (5.88%)	1
Protein in Urine † 1	0/18 (0.00%)	0	1/17 (5.88%)	1
Urinary Incontinence † 1	1/18 (5.56%)	1	0/17 (0.00%)	0
Respiratory, thoracic and mediastinal disorders
Asthma † 1	0/18 (0.00%)	0	1/17 (5.88%)	1
Blocked Nose † 1	0/18 (0.00%)	0	1/17 (5.88%)	1
Bronchiolitis † 1	0/18 (0.00%)	0	2/17 (11.76%)	2
Coryzal Symptoms † 1	1/18 (5.56%)	2	2/17 (11.76%)	2
Cough † 1	2/18 (11.11%)	5	1/17 (5.88%)	1
Cough and Runny Nose † 1	0/18 (0.00%)	0	1/17 (5.88%)	1
Cough and Cold † 1	1/18 (5.56%)	1	0/17 (0.00%)	0
Exacerbation † 1	0/18 (0.00%)	0	1/17 (5.88%)	1
Upper Respiratory Tract Infection † 1	2/18 (11.11%)	2	1/17 (5.88%)	1
Rhinos Virus Infection † 1	0/18 (0.00%)	0	1/17 (5.88%)	1
Runny Nose † 1	0/18 (0.00%)	0	1/17 (5.88%)	1
Skin and subcutaneous tissue disorders
Fine Erythematous, raised rash on Chest and Abdomen † 1	0/18 (0.00%)	0	1/17 (5.88%)	1
Glass in Foot † 1	0/18 (0.00%)	0	1/17 (5.88%)	1
Impetigo † 1	0/18 (0.00%)	0	1/17 (5.88%)	1
Rash † 1	0/18 (0.00%)	0	2/17 (11.76%)	4
White Discolouration of Lips and Throat † 1	1/18 (5.56%)	1	0/17 (0.00%)	0
1 Term from vocabulary, MedDRA (10.0); † Indicates events were collected by systematic assessment

Limitations and Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

Results Point of Contact

• Name/Title: David Kimberlin, MD
• Organization: University of Alabama at Birmingham
• Phone: (205) 638-2530
• EMail: DKimberlin@peds.uab.edu

• Responsible Party: National Institute of Allergy and Infectious Diseases (NIAID)
• ClinicalTrials.gov Identifier: NCT01649869
• Other Study ID Numbers: 11-0069; HHSN272201100035C
• First Submitted: July 20, 2012
• First Posted: July 25, 2012
• Results First Submitted: December 23, 2020
• Results First Posted: June 2, 2021
• Last Update Posted: June 2, 2021