Trial Outcomes & Findings for Vfend Special Investigation For Pediatric - Observational (NCT NCT02554656)
NCT ID: NCT02554656
Last Updated: 2021-04-14
Results Overview
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.
Recruitment status
COMPLETED
Target enrollment
89 participants
Primary outcome timeframe
16 weeks at maximum
Results posted on
2021-04-14
Participant Flow
Participant milestones
| Measure |
VFEND (Voriconazole)
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 Study
STARTED
|
89
|
|
Overall Study
COMPLETED
|
86
|
|
Overall Study
NOT COMPLETED
|
3
|
Reasons for withdrawal
| Measure |
VFEND (Voriconazole)
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 Study
Protocol Violation
|
3
|
Baseline Characteristics
Race and Ethnicity were not collected from any participant.
Baseline characteristics by cohort
| Measure |
VFEND (Voriconazole)
n=86 Participants
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.
|
|---|---|
|
Age, Customized
<1 month after birth
|
0 Participants
n=86 Participants
|
|
Age, Customized
≥1 month and <1 year
|
1 Participants
n=86 Participants
|
|
Age, Customized
≥1 to <7 years
|
40 Participants
n=86 Participants
|
|
Age, Customized
≥7 to <15 years
|
45 Participants
n=86 Participants
|
|
Sex: Female, Male
Female
|
37 Participants
n=86 Participants
|
|
Sex: Female, Male
Male
|
49 Participants
n=86 Participants
|
|
Diagnostic Name (Name of Infection)
Invasive aspergillosis
|
34 Participants
n=86 Participants
|
|
Diagnostic Name (Name of Infection)
Pulmonary aspergilloma
|
6 Participants
n=86 Participants
|
|
Diagnostic Name (Name of Infection)
Chronic necrotic pulmonary aspergillosis
|
1 Participants
n=86 Participants
|
|
Diagnostic Name (Name of Infection)
Candidemia
|
7 Participants
n=86 Participants
|
|
Diagnostic Name (Name of Infection)
Esophageal candidiasis
|
2 Participants
n=86 Participants
|
|
Diagnostic Name (Name of Infection)
Candida peritonitis
|
0 Participants
n=86 Participants
|
|
Diagnostic Name (Name of Infection)
Bronchopulmonary candidiasis
|
2 Participants
n=86 Participants
|
|
Diagnostic Name (Name of Infection)
Cryptococcal meningitis
|
0 Participants
n=86 Participants
|
|
Diagnostic Name (Name of Infection)
Pulmonary cryptococcosis
|
0 Participants
n=86 Participants
|
|
Diagnostic Name (Name of Infection)
Fusariosis
|
0 Participants
n=86 Participants
|
|
Diagnostic Name (Name of Infection)
Scedosporiosis
|
0 Participants
n=86 Participants
|
|
Diagnostic Name (Name of Infection)
Other invasive fungal infections
|
20 Participants
n=86 Participants
|
|
Diagnostic Name (Name of Infection)
Invasive fungal infections (multiple infections)
|
2 Participants
n=86 Participants
|
|
Diagnostic Name (Name of Infection)
Other than invasive fungal infections
|
12 Participants
n=86 Participants
|
PRIMARY outcome
Timeframe: 16 weeks at maximumPopulation: The safety analysis set comprised of participants who satisfied the inclusion criteria and had received VFEND at least once.
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.
Outcome measures
| Measure |
VFEND (Voriconazole)
n=86 Participants
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.
|
|---|---|
|
Number of Participants With Adverse Reactions
ADRs
|
23 Participants
|
|
Number of Participants With Adverse Reactions
Serious ADRs
|
3 Participants
|
SECONDARY outcome
Timeframe: 16 weeks at maximumPopulation: The safety analysis set comprised of participants who satisfied the inclusion criteria and had received VFEND at least once.
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.
Outcome measures
| Measure |
VFEND (Voriconazole)
n=86 Participants
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.
|
|---|---|
|
Number of Participants With Adverse Drug Reactions Not Expected From the LPD (Unknown Adverse Drug Reaction)
|
2 Participants
|
SECONDARY outcome
Timeframe: 16 weeks at maximumPopulation: The safety analysis set comprised of participants who satisfied the inclusion criteria and had received VFEND at least once.
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.
Outcome measures
| Measure |
VFEND (Voriconazole)
n=86 Participants
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.
|
|---|---|
|
Incidence of Aadverse Reactions by Diagnosis (Infection)
Invasive aspergillosis
|
35.29 Percentage of Participants
Interval 19.746 to 53.511
|
|
Incidence of Aadverse Reactions by Diagnosis (Infection)
Pulmonary aspergilloma
|
33.33 Percentage of Participants
Interval 4.327 to 77.722
|
|
Incidence of Aadverse Reactions by Diagnosis (Infection)
Chronic necrotic pulmonary aspergillosis
|
100.00 Percentage of Participants
Interval 2.5 to 100.0
|
|
Incidence of Aadverse Reactions by Diagnosis (Infection)
Candidemia
|
0.00 Percentage of Participants
Interval 0.0 to 40.962
|
|
Incidence of Aadverse Reactions by Diagnosis (Infection)
Esophageal candidiasis
|
0.00 Percentage of Participants
Interval 0.0 to 84.189
|
|
Incidence of Aadverse Reactions by Diagnosis (Infection)
Bronchopulmonary candidiasis
|
50.00 Percentage of Participants
Interval 1.258 to 98.742
|
|
Incidence of Aadverse Reactions by Diagnosis (Infection)
Other invasive fungal infections
|
20.00 Percentage of Participants
Interval 5.733 to 43.661
|
|
Incidence of Aadverse Reactions by Diagnosis (Infection)
Invasive fungal infections (multiple infections)
|
50.00 Percentage of Participants
Interval 1.258 to 98.742
|
|
Incidence of Aadverse Reactions by Diagnosis (Infection)
Other than invasive fungal infections
|
16.67 Percentage of Participants
Interval 2.086 to 48.414
|
SECONDARY outcome
Timeframe: 16 weeks at maximumPopulation: 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.
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.
Outcome measures
| Measure |
VFEND (Voriconazole)
n=67 Participants
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 Clinical Response
|
80.6 Parcentage of Participants
Interval 69.11 to 89.24
|
SECONDARY outcome
Timeframe: 16 weeks at maximumPopulation: 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.
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.
Outcome measures
| Measure |
VFEND (Voriconazole)
n=67 Participants
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.
|
|---|---|
|
Clinical Response Rate by Diagnostic Name (Name of Infection)
Invasive aspergillosis
|
97.0 Percentage of Participants
Interval 84.24 to 99.92
|
|
Clinical Response Rate by Diagnostic Name (Name of Infection)
Pulmonary aspergilloma
|
75.0 Percentage of Participants
Interval 19.41 to 99.37
|
|
Clinical Response Rate by Diagnostic Name (Name of Infection)
Chronic necrotic pulmonary aspergillosis
|
0.0 Percentage of Participants
Interval 0.0 to 97.5
|
|
Clinical Response Rate by Diagnostic Name (Name of Infection)
Candidemia
|
100.0 Percentage of Participants
Interval 47.82 to 100.0
|
|
Clinical Response Rate by Diagnostic Name (Name of Infection)
Esophageal candidiasis
|
100.0 Percentage of Participants
Interval 15.81 to 100.0
|
|
Clinical Response Rate by Diagnostic Name (Name of Infection)
Bronchopulmonary candidiasis
|
100.0 Percentage of Participants
Interval 2.5 to 100.0
|
|
Clinical Response Rate by Diagnostic Name (Name of Infection)
Other invasive fungal infections
|
47.4 Percentage of Participants
Interval 24.45 to 71.14
|
|
Clinical Response Rate by Diagnostic Name (Name of Infection)
Invasive fungal infections (multiple infections)
|
100.0 Percentage of Participants
Interval 15.81 to 100.0
|
Adverse Events
VFEND (Voriconazole)
Serious events: 11 serious events
Other events: 41 other events
Deaths: 6 deaths
Serious adverse events
| Measure |
VFEND (Voriconazole)
n=86 participants at risk
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.
|
|---|---|
|
Infections and infestations
Cytomegalovirus viraemia
|
1.2%
1/86 • 16 weeks at maximum
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.
|
|
Infections and infestations
Pneumonia bacterial
|
1.2%
1/86 • 16 weeks at maximum
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.
|
|
Infections and infestations
Pneumonia
|
1.2%
1/86 • 16 weeks at maximum
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.
|
|
Infections and infestations
Pulmonary mycosis
|
1.2%
1/86 • 16 weeks at maximum
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.
|
|
Immune system disorders
Cytokine storm
|
1.2%
1/86 • 16 weeks at maximum
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.
|
|
Nervous system disorders
Haemorrhage intracranial
|
1.2%
1/86 • 16 weeks at maximum
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.
|
|
Respiratory, thoracic and mediastinal disorders
Obliterative bronchiolitis
|
1.2%
1/86 • 16 weeks at maximum
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.
|
|
Gastrointestinal disorders
Melaena
|
1.2%
1/86 • 16 weeks at maximum
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.
|
|
Gastrointestinal disorders
Pancreatitis acute
|
1.2%
1/86 • 16 weeks at maximum
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.
|
|
Hepatobiliary disorders
Venoocclusive liver disease
|
1.2%
1/86 • 16 weeks at maximum
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.
|
|
Hepatobiliary disorders
Drug-induced liver injury
|
1.2%
1/86 • 16 weeks at maximum
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.
|
|
General disorders
Multiple organ dysfunction syndrome
|
2.3%
2/86 • 16 weeks at maximum
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.
|
|
General disorders
Pyrexia
|
1.2%
1/86 • 16 weeks at maximum
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.
|
|
Investigations
Lymphocyte count decreased
|
1.2%
1/86 • 16 weeks at maximum
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.
|
Other adverse events
| Measure |
VFEND (Voriconazole)
n=86 participants at risk
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.
|
|---|---|
|
Eye disorders
Photophobia
|
2.3%
2/86 • 16 weeks at maximum
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.
|
|
Hepatobiliary disorders
Hepatic function abnormal
|
2.3%
2/86 • 16 weeks at maximum
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.
|
|
Hepatobiliary disorders
Liver disorder
|
3.5%
3/86 • 16 weeks at maximum
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.
|
|
Investigations
Aspartate aminotransferase increased
|
15.1%
13/86 • 16 weeks at maximum
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.
|
|
Investigations
Alanine aminotransferase increased
|
14.0%
12/86 • 16 weeks at maximum
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.
|
|
Investigations
Gamma-glutamyltransferase increased
|
15.1%
13/86 • 16 weeks at maximum
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.
|
|
Investigations
Platelet count decreased
|
2.3%
2/86 • 16 weeks at maximum
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.
|
|
Investigations
Blood bilirubin increased
|
4.7%
4/86 • 16 weeks at maximum
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.
|
|
Investigations
Blood alkaline phosphatase increased
|
4.7%
4/86 • 16 weeks at maximum
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.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee 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.
- Publication restrictions are in place
Restriction type: OTHER