Vfend Special Investigation For Pediatric - Observational

• Sponsor: Pfizer (Industry)
• Overall Status: Completed
• CT.gov ID: NCT02554656
• Collaborator: (none)
• Enrollment: 89
• Location: 1
• Actual Duration (Months): 35
• Patients Per Site Per Month: 2.5

Study Details

Study Description

Brief Summary

Examine the safety and effectiveness of Vfend [voriconazole] for pediatric under general clinical practices.

Condition or Disease	Intervention/Treatment	Phase
Pediatric Safety and Effectiveness

Study Design

• Study Type: Observational
• Actual Enrollment: 89 participants
• Observational Model: Cohort
• Time Perspective: Prospective
• Official Title: VFEND SPECIAL INVESTIGATION- INVESTIGATION FOR TREATMENT OF INVASIVE FUNGAL INFECTIONS IN PEDIATRIC PATIENTS -
• Actual Study Start Date: Oct 27, 2015
• Actual Primary Completion Date: Sep 25, 2018
• Actual Study Completion Date: Sep 25, 2018

Outcome Measures

Primary Outcome Measures

1. Number of Participants With Adverse Reactions [16 weeks at maximum]

   An adverse drug reaction (ADR) was any untoward medical occurrence attributed to VFEND in a participant who received VFEND. A serious ADR was an ADR resulting in any of the following outcomes or deemed significant for any other reason: death; life-threatening experience (immediate risk of dying); initial or prolonged inpatient hospitalization; persistent or significant disability/incapacity; congenital anomaly. Relatedness to VFEND was assessed by the physician.

Secondary Outcome Measures

1. Number of Participants With Adverse Drug Reactions Not Expected From the LPD (Unknown Adverse Drug Reaction) [16 weeks at maximum]

   An adverse drug reaction (ADR) was any untoward medical occurrence attributed to VFEND in a participant who received VFEND. Expectedness of the adverse event was determined according to the Japanese package insert. Relatedness to VFEND was assessed by the physician.

2. Incidence of Aadverse Reactions by Diagnosis (Infection) [16 weeks at maximum]

   An ADR was any untoward medical occurrence attributed to VFEND in a participant who received VFEND. Relatedness to VFEND was assessed by the physician. Participants with ADRs were counted by diagnosis (infection) to assess whether it was a risk factor for the occurrence of ADRs.

3. Overall Clinical Response [16 weeks at maximum]

   Clinical effectiveness rate, which was defined as the percentage of participants who achieved clinical effectiveness over the total number of assessable effectiveness analysis population, was presented along with the corresponding 2-sided 95% CI. Overall clinical effectiveness of VFEND was assessed as "effective", "not effective", or "indeterminate" by the physician based on the clinical course at the end of the observation period or at the time of treatment discontinuation.

4. Clinical Response Rate by Diagnostic Name (Name of Infection) [16 weeks at maximum]

   Clinical effectiveness rate, which was defined as the percentage of participants who achieved clinical effectiveness over the total number of assessable effectiveness analysis population, was presented along with the corresponding 2-sided 95% CI. Overall clinical effectiveness of VFEND was assessed as "effective", "not effective", or "indeterminate" by the physician based on the clinical course at the end of the observation period or at the time of treatment discontinuation. Overall effectiveness of VFEND was determined by the physician based on the clinical course. Participants achieved clinical effectiveness by Diagnosis (Infection) were counted to assess whether it contributes to the clinical effectiveness.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 0 Years to 15 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Patients who is under 15 years old and deep mycosis infection.

Exclusion Criteria:

• Patients who have been previously enrolled in this study. -

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	XXXXXXX	Osaka		Japan

Sponsors and Collaborators

• Pfizer

Investigators

• Study Director: Pfizer CT.gov Call Center, Pfizer

Study Documents (Full-Text)

• Statistical Analysis Plan - Sep 20, 2018
• Study Protocol - May 12, 2015

More Information

Additional Information:

• To obtain contact information for a study center near you, click here.

Publications

None provided.

• Responsible Party: Pfizer
• ClinicalTrials.gov Identifier: NCT02554656
• Other Study ID Numbers: A1501100
• First Posted: Sep 18, 2015
• Last Update Posted: Apr 14, 2021
• Last Verified: Mar 1, 2021

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No

Study Results

Participant Flow

Arm/Group Title	VFEND (Voriconazole)
Arm/Group Description	Participants who received VFEND as indicated in the approved local product document were observed for a period of 16 weeks at maximum. The dosage can be adjusted as per physician's discretion.
Period Title: Overall Study
STARTED	89
COMPLETED	86
NOT COMPLETED	3

Baseline Characteristics

Arm/Group Title	VFEND (Voriconazole)
Arm/Group Description	Participants who received VFEND as indicated in the approved local product document were observed for a period of 16 weeks at maximum. The dosage can be adjusted as per physician's discretion.
Overall Participants	86
Age, Customized (Number) [Number]
<1 month after birth	0 0%
≥1 month and <1 year	1 1.2%
≥1 to <7 years	40 46.5%
≥7 to <15 years	45 52.3%
Sex: Female, Male (Count of Participants)
Female	37 43%
Male	49 57%
Race and Ethnicity Not Collected (Count of Participants)
Diagnostic Name (Name of Infection) (Number) [Number]
Invasive aspergillosis	34 39.5%
Pulmonary aspergilloma	6 7%
Chronic necrotic pulmonary aspergillosis	1 1.2%
Candidemia	7 8.1%
Esophageal candidiasis	2 2.3%
Candida peritonitis	0 0%
Bronchopulmonary candidiasis	2 2.3%
Cryptococcal meningitis	0 0%
Pulmonary cryptococcosis	0 0%
Fusariosis	0 0%
Scedosporiosis	0 0%
Other invasive fungal infections	20 23.3%
Invasive fungal infections (multiple infections)	2 2.3%
Other than invasive fungal infections	12 14%

Outcome Measures

1. Primary Outcome

Title	Number of Participants With Adverse Reactions
Description	An adverse drug reaction (ADR) was any untoward medical occurrence attributed to VFEND in a participant who received VFEND. A serious ADR was an ADR resulting in any of the following outcomes or deemed significant for any other reason: death; life-threatening experience (immediate risk of dying); initial or prolonged inpatient hospitalization; persistent or significant disability/incapacity; congenital anomaly. Relatedness to VFEND was assessed by the physician.
Time Frame	16 weeks at maximum

Outcome Measure Data

Analysis Population Description

The safety analysis set comprised of participants who satisfied the inclusion criteria and had received VFEND at least once.

Arm/Group Title	VFEND (Voriconazole)
Arm/Group Description	Participants who received VFEND as indicated in the approved local product document were observed for a period of 16 weeks at maximum. The dosage can be adjusted as per physician's discretion.
Measure Participants	86
ADRs	23 26.7%
Serious ADRs	3 3.5%

2. Secondary Outcome

Title	Number of Participants With Adverse Drug Reactions Not Expected From the LPD (Unknown Adverse Drug Reaction)
Description	An adverse drug reaction (ADR) was any untoward medical occurrence attributed to VFEND in a participant who received VFEND. Expectedness of the adverse event was determined according to the Japanese package insert. Relatedness to VFEND was assessed by the physician.
Time Frame	16 weeks at maximum

Outcome Measure Data

Analysis Population Description

The safety analysis set comprised of participants who satisfied the inclusion criteria and had received VFEND at least once.

Arm/Group Title	VFEND (Voriconazole)
Arm/Group Description	Participants who received VFEND as indicated in the approved local product document were observed for a period of 16 weeks at maximum. The dosage can be adjusted as per physician's discretion.
Measure Participants	86
Number [Participants]	2 2.3%

3. Secondary Outcome

Title	Incidence of Aadverse Reactions by Diagnosis (Infection)
Description	An ADR was any untoward medical occurrence attributed to VFEND in a participant who received VFEND. Relatedness to VFEND was assessed by the physician. Participants with ADRs were counted by diagnosis (infection) to assess whether it was a risk factor for the occurrence of ADRs.
Time Frame	16 weeks at maximum

Outcome Measure Data

Analysis Population Description

The safety analysis set comprised of participants who satisfied the inclusion criteria and had received VFEND at least once.

Arm/Group Title	VFEND (Voriconazole)
Arm/Group Description	Participants who received VFEND as indicated in the approved local product document were observed for a period of 16 weeks at maximum. The dosage can be adjusted as per physician's discretion.
Measure Participants	86
Invasive aspergillosis	35.29 41%
Pulmonary aspergilloma	33.33 38.8%
Chronic necrotic pulmonary aspergillosis	100.00 116.3%
Candidemia	0.00 0%
Esophageal candidiasis	0.00 0%
Bronchopulmonary candidiasis	50.00 58.1%
Other invasive fungal infections	20.00 23.3%
Invasive fungal infections (multiple infections)	50.00 58.1%
Other than invasive fungal infections	16.67 19.4%

4. Secondary Outcome

Title	Overall Clinical Response
Description	Clinical effectiveness rate, which was defined as the percentage of participants who achieved clinical effectiveness over the total number of assessable effectiveness analysis population, was presented along with the corresponding 2-sided 95% CI. Overall clinical effectiveness of VFEND was assessed as "effective", "not effective", or "indeterminate" by the physician based on the clinical course at the end of the observation period or at the time of treatment discontinuation.
Time Frame	16 weeks at maximum

Outcome Measure Data

Analysis Population Description

The efficacy analysis set comprised of participants in the safety analysis set who had effectiveness evaluation at the end of the observation period or at the time of treatment discontinuation. The efficacy analysis sets were 74 participants. Participants assessed as "indeterminate (n=7)" at the final observation were excluded from the calculation.

Arm/Group Title	VFEND (Voriconazole)
Arm/Group Description	Participants who received VFEND as indicated in the approved local product document were observed for a period of 16 weeks at maximum. The dosage can be adjusted as per physician's discretion.
Measure Participants	67
Number (95% Confidence Interval) [Parcentage of Participants]	80.6 93.7%

5. Secondary Outcome

Title	Clinical Response Rate by Diagnostic Name (Name of Infection)
Description	Clinical effectiveness rate, which was defined as the percentage of participants who achieved clinical effectiveness over the total number of assessable effectiveness analysis population, was presented along with the corresponding 2-sided 95% CI. Overall clinical effectiveness of VFEND was assessed as "effective", "not effective", or "indeterminate" by the physician based on the clinical course at the end of the observation period or at the time of treatment discontinuation. Overall effectiveness of VFEND was determined by the physician based on the clinical course. Participants achieved clinical effectiveness by Diagnosis (Infection) were counted to assess whether it contributes to the clinical effectiveness.
Time Frame	16 weeks at maximum

Outcome Measure Data

Analysis Population Description

The efficacy analysis set comprised of participants in the safety analysis set who had effectiveness evaluation at the end of the observation period or at the time of treatment discontinuation. The efficacy analysis sets were 74 participants. Participants assessed as "indeterminate (n=7)" at the final observation were excluded from the calculation.

Arm/Group Title	VFEND (Voriconazole)
Arm/Group Description	Participants who received VFEND as indicated in the approved local product document were observed for a period of 16 weeks at maximum. The dosage can be adjusted as per physician's discretion.
Measure Participants	67
Invasive aspergillosis	97.0 112.8%
Pulmonary aspergilloma	75.0 87.2%
Chronic necrotic pulmonary aspergillosis	0.0 0%
Candidemia	100.0 116.3%
Esophageal candidiasis	100.0 116.3%
Bronchopulmonary candidiasis	100.0 116.3%
Other invasive fungal infections	47.4 55.1%
Invasive fungal infections (multiple infections)	100.0 116.3%

Adverse Events

Time Frame	16 weeks at maximum
Adverse Event Reporting Description	The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Arm/Group Title	VFEND (Voriconazole)
Arm/Group Description	Participants who received VFEND as indicated in the approved local product document were observed for a period of 16 weeks at maximum. The dosage can be adjusted as per physician's discretion.
All Cause Mortality
	VFEND (Voriconazole)
	Affected / at Risk (%)	# Events
Total	6/86 (7%)
Serious Adverse Events
	VFEND (Voriconazole)
	Affected / at Risk (%)	# Events
Total	11/86 (12.8%)
Gastrointestinal disorders
Melaena	1/86 (1.2%)
Pancreatitis acute	1/86 (1.2%)
General disorders
Multiple organ dysfunction syndrome	2/86 (2.3%)
Pyrexia	1/86 (1.2%)
Hepatobiliary disorders
Venoocclusive liver disease	1/86 (1.2%)
Drug-induced liver injury	1/86 (1.2%)
Immune system disorders
Cytokine storm	1/86 (1.2%)
Infections and infestations
Cytomegalovirus viraemia	1/86 (1.2%)
Pneumonia bacterial	1/86 (1.2%)
Pneumonia	1/86 (1.2%)
Pulmonary mycosis	1/86 (1.2%)
Investigations
Lymphocyte count decreased	1/86 (1.2%)
Nervous system disorders
Haemorrhage intracranial	1/86 (1.2%)
Respiratory, thoracic and mediastinal disorders
Obliterative bronchiolitis	1/86 (1.2%)
Other (Not Including Serious) Adverse Events
	VFEND (Voriconazole)
	Affected / at Risk (%)	# Events
Total	41/86 (47.7%)
Eye disorders
Photophobia	2/86 (2.3%)
Hepatobiliary disorders
Hepatic function abnormal	2/86 (2.3%)
Liver disorder	3/86 (3.5%)
Investigations
Aspartate aminotransferase increased	13/86 (15.1%)
Alanine aminotransferase increased	12/86 (14%)
Gamma-glutamyltransferase increased	13/86 (15.1%)
Platelet count decreased	2/86 (2.3%)
Blood bilirubin increased	4/86 (4.7%)
Blood alkaline phosphatase increased	4/86 (4.7%)

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

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

Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.