Trial Outcomes & Findings for A Phase 1 Dose-escalation Study to Evaluate the Safety and Pharmacokinetics (PK) of Palifermin in Subjects With Acute Leukemias Undergoing HSCT (NCT NCT00460421)
NCT ID: NCT00460421
Last Updated: 2014-12-05
Results Overview
A DLT is appearance of side effects during treatment severe enough to prevent further increase in dosage or strength of treatment agent, or to prevent continuation of treatment at any dosage level. A DLT was defined as: Grade 3 or 4 AE \[based on Cancer Therapy Evaluation Program Common Terminology Criteria for Adverse Events (CTCAE) v3.0\] considered by the investigator to be related to palifermin with the exceptions: Grade 3 erythema, pruritus or rash that resolves within 7 days of the last dose of palifermin. The percentage of particiapnts with a DLT during the study was assessed.
Recruitment status
COMPLETED
Study phase
PHASE1
Target enrollment
27 participants
Primary outcome timeframe
Approximately 1 month duration (Day -10 through Day +16)
Results posted on
2014-12-05
Participant Flow
This phase 1 dose-escalation study to evaluate the safety and pharmacokinetics (PK) of palifermin in pediatric subjects with acute leukemias undergoing myeloblative therapy and allogeneic hematopoietic stem cell transplant (HSCT) was performed in 7 centers in USA between 2006 and 2011.
Participant milestones
| Measure |
Palifermin 40 µg/kg/Day
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
Palifermin 60 µg/kg/Day
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
Palifermin 80 µg/kg/Day
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
|---|---|---|---|
|
Overall Study
STARTED
|
9
|
9
|
9
|
|
Overall Study
COMPLETED
|
9
|
9
|
9
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Phase 1 Dose-escalation Study to Evaluate the Safety and Pharmacokinetics (PK) of Palifermin in Subjects With Acute Leukemias Undergoing HSCT
Baseline characteristics by cohort
| Measure |
Palifermin 40 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
Palifermin 60 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
Palifermin 80 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
Total
n=27 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
9 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
27 Participants
n=4 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Age, Continuous
|
7.4 years
STANDARD_DEVIATION 6.3 • n=5 Participants
|
7.4 years
STANDARD_DEVIATION 5.6 • n=7 Participants
|
8.0 years
STANDARD_DEVIATION 6.6 • n=5 Participants
|
7.6 years
STANDARD_DEVIATION 5.9 • n=4 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
12 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
6 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
15 Participants
n=4 Participants
|
|
Region of Enrollment
United States
|
9 participants
n=5 Participants
|
9 participants
n=7 Participants
|
9 participants
n=5 Participants
|
27 participants
n=4 Participants
|
PRIMARY outcome
Timeframe: Approximately 1 month duration (Day -10 through Day +16)Population: Safety Analysis Set. This set consisted of subjects who received at least one dose of palifermin.
A DLT is appearance of side effects during treatment severe enough to prevent further increase in dosage or strength of treatment agent, or to prevent continuation of treatment at any dosage level. A DLT was defined as: Grade 3 or 4 AE \[based on Cancer Therapy Evaluation Program Common Terminology Criteria for Adverse Events (CTCAE) v3.0\] considered by the investigator to be related to palifermin with the exceptions: Grade 3 erythema, pruritus or rash that resolves within 7 days of the last dose of palifermin. The percentage of particiapnts with a DLT during the study was assessed.
Outcome measures
| Measure |
Palifermin 40 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
Palifermin 60 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
Palifermin 80 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
All Subjects
All subjects from each age group (1-2, 3-11, 12-16 years)
|
|---|---|---|---|---|
|
Incidence of Dose Limiting Toxicities (DLTs)
|
0 percentage of participants
|
0 percentage of participants
|
0 percentage of participants
|
—
|
SECONDARY outcome
Timeframe: Approximately 4 month duration (Through Day + 100 (+/- 40 days))Population: Safety Analysis Set. This set consisted of subjects who received at least one dose of palifermin.
The percentage of participants developing palifermin antibodies during the study was assessed.
Outcome measures
| Measure |
Palifermin 40 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
Palifermin 60 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
Palifermin 80 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
All Subjects
All subjects from each age group (1-2, 3-11, 12-16 years)
|
|---|---|---|---|---|
|
Incidence of Serum Palifermin Antibody Formation
|
0 percentage of participants
|
0 percentage of participants
|
0 percentage of participants
|
—
|
SECONDARY outcome
Timeframe: Approximately 1 1/2 months duration (Through Day +30/End of Treatment)Population: Safety Analysis Set. This set consisted of subjects who received at least one dose of palifermin.
The percentage of participants with a severe AE during the study was assessed.
Outcome measures
| Measure |
Palifermin 40 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
Palifermin 60 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
Palifermin 80 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
All Subjects
All subjects from each age group (1-2, 3-11, 12-16 years)
|
|---|---|---|---|---|
|
Incidence of Severe Adverse Events (AEs)
|
100 percentage of participants
|
100 percentage of participants
|
100 percentage of participants
|
—
|
SECONDARY outcome
Timeframe: Approximately 1 1/2 months duration (Through Day +30/End of Treatment)Population: Safety Analysis Set. This set consisted of subjects who received at least one dose of palifermin.
The percentage of participants with a laboratory value outside the normal ranges during the study.
Outcome measures
| Measure |
Palifermin 40 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
Palifermin 60 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
Palifermin 80 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
All Subjects
All subjects from each age group (1-2, 3-11, 12-16 years)
|
|---|---|---|---|---|
|
Incidence of Laboratory Abnormalities
|
100 percentage of participants
|
100 percentage of participants
|
100 percentage of participants
|
—
|
SECONDARY outcome
Timeframe: Day -10Population: Pharmacokinetic Analysis Set. This set consists of all subjects who receive at least one dose of palifermin and who have a sufficient number of serum concentration data points to allow calculation of the pharmacokinetic variables.
Clearence was estimated as dose divided by the area under serum concentration-time curve from time zero to infinity where the dose was given in amount palifermin actually administered.
Outcome measures
| Measure |
Palifermin 40 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
Palifermin 60 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
Palifermin 80 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
All Subjects
All subjects from each age group (1-2, 3-11, 12-16 years)
|
|---|---|---|---|---|
|
Pharmacokinetics of Palifermin, Clearence (CL) After the 1st Intravenous (IV) Bolus Injection for Multiple Dose Levels
|
1954.84 mL/hr/kg
Interval 570.83 to 3629.06
|
2164.87 mL/hr/kg
Interval 419.39 to 5152.07
|
3932.30 mL/hr/kg
Interval 1268.22 to 18182.41
|
—
|
SECONDARY outcome
Timeframe: Day -10Population: Pharmacokinetic Analysis Set. This set consists of all subjects who receive at least one dose of palifermin and who have a sufficient number of serum concentration data points to allow calculation of the pharmacokinetic variables.
Outcome measures
| Measure |
Palifermin 40 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
Palifermin 60 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
Palifermin 80 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
All Subjects
All subjects from each age group (1-2, 3-11, 12-16 years)
|
|---|---|---|---|---|
|
Pharmacokinetics of Palifermin, Volume of Distribution at Steady State (Vss) After the 1st IV Bolus Injection for Multiple Dose Levels
|
2552.79 mL/kg
Interval 369.31 to 4862.29
|
5134.84 mL/kg
Interval 1033.25 to 28349.41
|
8580.91 mL/kg
Interval 1165.08 to 70085.13
|
—
|
SECONDARY outcome
Timeframe: Day -10Population: Pharmacokinetic Analysis Set. This set consists of all subjects who receive at least one dose of palifermin and who have a sufficient number of serum concentration data points to allow calculation of the pharmacokinetic variables.
The terminal half-life was calculated as ln(2)/lambda,z where lambda,z was estimated using at least three quantifiable serum concentrations of the terminal log-linear phase.
Outcome measures
| Measure |
Palifermin 40 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
Palifermin 60 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
Palifermin 80 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
All Subjects
All subjects from each age group (1-2, 3-11, 12-16 years)
|
|---|---|---|---|---|
|
Pharmacokinetics of Palifermin, Terminal Half-life (t½,z) After the 1st IV Bolus Injection for Multiple Dose Levels
|
2.91 hour
Interval 1.43 to 5.81
|
3.05 hour
Interval 2.19 to 8.36
|
3.68 hour
Interval 0.97 to 4.88
|
—
|
SECONDARY outcome
Timeframe: Day -8Population: Pharmacokinetic Analysis Set. This set consists of all subjects who receive at least one dose of palifermin and who have a sufficient number of serum concentration data points to allow calculation of the pharmacokinetic variables.
The terminal half-life was calculated as ln(2)/lambda,z where lambda,z was estimated using at least three quantifiable serum concentrations of the terminal log-linear phase.
Outcome measures
| Measure |
Palifermin 40 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
Palifermin 60 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
Palifermin 80 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
All Subjects
All subjects from each age group (1-2, 3-11, 12-16 years)
|
|---|---|---|---|---|
|
Pharmacokinetics of Palifermin, t½,z After the 3rd IV Bolus Injection for Multiple Dose Levels
|
3.40 hour
Interval 0.84 to 5.71
|
3.89 hour
Interval 3.21 to 11.84
|
2.99 hour
Interval 2.06 to 4.03
|
—
|
SECONDARY outcome
Timeframe: Day -10Population: Pharmacokinetic Analysis Set. This set consists of all subjects who receive at least one dose of palifermin and who have a sufficient number of serum concentration data points to allow calculation of the pharmacokinetic variables.
The AUC was estimated using the linear/log trapezoidal method for AUC0-tau from time zero to the end of the dosing interval (24 hours (hrs) post-dose) Data collected at time points: 0, 2 minutes (min), 15 min, 30 min, 60 min, 2 hrs, 4 hrs, 6, hrs, 10, hrs and 24 hrs post-dose.
Outcome measures
| Measure |
Palifermin 40 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
Palifermin 60 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
Palifermin 80 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
All Subjects
All subjects from each age group (1-2, 3-11, 12-16 years)
|
|---|---|---|---|---|
|
Pharmacokinetics of Palifermin, Area Under the Concentration Time Curve From Zero to the End of the Dosing Interval (AUCtau) After the 1st IV Bolus Injection for Multiple Dose Levels
|
16.54 ng*hr/mL
Interval 4.69 to 70.57
|
18.14 ng*hr/mL
Interval 12.35 to 142.71
|
38.44 ng*hr/mL
Interval 4.53 to 185.35
|
—
|
SECONDARY outcome
Timeframe: Day -8Population: Pharmacokinetic Analysis Set. This set consists of all subjects who receive at least one dose of palifermin and who have a sufficient number of serum concentration data points to allow calculation of the pharmacokinetic variables.
The AUC was estimated using the linear/log trapezoidal method for AUC0-tau from time zero to the end of the dosing interval (24 hours (hrs) post-dose) Data collected at time points: 0, 2 minutes (min), 15 min, 30 min, 60 min, 2 hrs, 4 hrs, 6, hrs, 10, hrs and 24 hrs post-dose.
Outcome measures
| Measure |
Palifermin 40 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
Palifermin 60 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
Palifermin 80 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
All Subjects
All subjects from each age group (1-2, 3-11, 12-16 years)
|
|---|---|---|---|---|
|
Pharmacokinetics of Palifermin, AUCtau After the 3rd IV Bolus Injection for Multiple Dose Levels
|
18.32 ng*hr/mL
Interval 1.73 to 63.65
|
17.97 ng*hr/mL
Interval 2.45 to 325.96
|
34.74 ng*hr/mL
Interval 3.14 to 295.11
|
—
|
SECONDARY outcome
Timeframe: Up to 4 years duration (Assessments performed on months 6, 9, 12 (+/- 30 Days) for the first year and then annually)Population: Safety Analysis Set. This set consisted of subjects who received at least one dose of palifermin.
Outcome measures
| Measure |
Palifermin 40 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
Palifermin 60 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
Palifermin 80 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
All Subjects
All subjects from each age group (1-2, 3-11, 12-16 years)
|
|---|---|---|---|---|
|
Long-Term Follow-Up: Incidence of Secondary Malignancies
|
0 percentage of participants
|
11 percentage of participants
|
0 percentage of participants
|
—
|
SECONDARY outcome
Timeframe: Up to 4 years duration (Assessments performed on months 6, 9, 12 (+/- 30 Days) for the first year and then annually)Population: Safety Analysis Set. This set consisted of subjects who received at least one dose of palifermin.
Progression free survival (PFS) was defined as the number of days between the date of first investigational product administration and the date when physical or radiological evidence of disease progression is determined or death (regardless of cause)
Outcome measures
| Measure |
Palifermin 40 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
Palifermin 60 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
Palifermin 80 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
All Subjects
n=27 Participants
All subjects from each age group (1-2, 3-11, 12-16 years)
|
|---|---|---|---|---|
|
Long-Term Follow-Up: Progression Free Survival
|
NA months
Reported for the entire study population
|
NA months
Reported for the entire study population
|
NA months
Reported for the entire study population
|
36 months
Interval 6.0 to 48.0
|
SECONDARY outcome
Timeframe: Up to 4 years duration (Assessments performed on months 6, 9, 12 (+/- 30 Days) for the first year and then annually)Population: Safety Analysis Set. This set consisted of subjects who received at least one dose of palifermin.
Overall survival was defined as the number of days from the date of first investigational product administration to the date of death (regardless of cause)
Outcome measures
| Measure |
Palifermin 40 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
Palifermin 60 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
Palifermin 80 µg/kg/Day
n=9 Participants
Three subjects from each age group (1-2, 3-11, 12-16 years) were sequentially enrolled into the 3 planned dosing cohorts
|
All Subjects
n=27 Participants
All subjects from each age group (1-2, 3-11, 12-16 years)
|
|---|---|---|---|---|
|
Long-Term Follow-Up: Overall Survival
|
NA months
Reported for the entire study population
|
NA months
Reported for the entire study population
|
NA months
Reported for the entire study population
|
36 months
Interval 6.0 to 48.0
|
Adverse Events
All Subjects
Serious events: 5 serious events
Other events: 27 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
All Subjects
n=27 participants at risk
All subjects from each age group (1-2, 3-11, 12-16 years)
|
|---|---|
|
Gastrointestinal disorders
Diarrhoea
|
7.4%
2/27 • Number of events 2 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Gastrointestinal disorders
Vomiting
|
3.7%
1/27 • Number of events 1 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Cardiac disorders
Cardiac arrest
|
3.7%
1/27 • Number of events 1 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Gastrointestinal disorders
Ascites
|
3.7%
1/27 • Number of events 1 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Gastrointestinal disorders
Gastrointestinal haemorrhage
|
3.7%
1/27 • Number of events 1 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
General disorders
Pyrexia
|
7.4%
2/27 • Number of events 2 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Infections and infestations
Enterococcal infection
|
3.7%
1/27 • Number of events 1 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Infections and infestations
Septic chock
|
3.7%
1/27 • Number of events 1 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Investigations
Urine output decreased
|
3.7%
1/27 • Number of events 1 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Nervous system disorders
Cerebral haemorrhage
|
3.7%
1/27 • Number of events 1 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Renal and urinary disorders
Renal failure
|
7.4%
2/27 • Number of events 2 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
3.7%
1/27 • Number of events 1 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Vascular disorders
Hypotension
|
3.7%
1/27 • Number of events 1 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Vascular disorders
Venoocclusive disease
|
7.4%
2/27 • Number of events 2 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Nervous system disorders
Convulsion
|
3.7%
1/27 • Number of events 1 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Nervous system disorders
Haemorrhage intracranial
|
3.7%
1/27 • Number of events 1 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
Other adverse events
| Measure |
All Subjects
n=27 participants at risk
All subjects from each age group (1-2, 3-11, 12-16 years)
|
|---|---|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
11.1%
3/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Cardiac disorders
Pericardial effusion
|
7.4%
2/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Cardiac disorders
Tachycardia
|
25.9%
7/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Ear and labyrinth disorders
Ear pain
|
7.4%
2/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Eye disorders
Dry eye
|
7.4%
2/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Gastrointestinal disorders
Abdominal pain
|
55.6%
15/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Gastrointestinal disorders
Ascites
|
7.4%
2/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Gastrointestinal disorders
Constipation
|
18.5%
5/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Gastrointestinal disorders
Diarrhoea
|
70.4%
19/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Gastrointestinal disorders
Dyspepsia
|
7.4%
2/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Gastrointestinal disorders
Flatulence
|
7.4%
2/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Gastrointestinal disorders
Gastritis
|
7.4%
2/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Gastrointestinal disorders
Gingival hyperplasia
|
7.4%
2/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Gastrointestinal disorders
Haematemesis
|
7.4%
2/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Gastrointestinal disorders
Lip dry
|
7.4%
2/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Gastrointestinal disorders
Mouth haemorrhage
|
7.4%
2/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Gastrointestinal disorders
Nausea
|
77.8%
21/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Gastrointestinal disorders
Oesophagitis
|
7.4%
2/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Gastrointestinal disorders
Oral pain
|
7.4%
2/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Gastrointestinal disorders
Perianal erythema
|
7.4%
2/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Gastrointestinal disorders
Proctalgia
|
14.8%
4/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Gastrointestinal disorders
Vomiting
|
85.2%
23/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
General disorders
Catheter related complication
|
22.2%
6/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
General disorders
Catheter site pain
|
18.5%
5/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
General disorders
Chills
|
11.1%
3/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
General disorders
Face oedema
|
11.1%
3/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
General disorders
Fatigue
|
25.9%
7/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
General disorders
Irritability
|
14.8%
4/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
General disorders
Oedema
|
14.8%
4/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
General disorders
Oedema peripheral
|
18.5%
5/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
General disorders
Pain
|
14.8%
4/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
General disorders
Pyrexia
|
81.5%
22/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Hepatobiliary disorders
Hepatomegaly
|
7.4%
2/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Hepatobiliary disorders
Hyperbilirubinaemia
|
18.5%
5/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Immune system disorders
Acute graft versus host disease in skin
|
18.5%
5/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Infections and infestations
Alpha haemolytic streptococcal infection
|
11.1%
3/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Infections and infestations
Bacteraemia
|
7.4%
2/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Infections and infestations
Clostridium difficile colitis
|
11.1%
3/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Infections and infestations
Enterococcal sepsis
|
11.1%
3/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Infections and infestations
Fungal infection
|
7.4%
2/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Infections and infestations
Parotitis
|
11.1%
3/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Infections and infestations
Pneumonia
|
7.4%
2/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Infections and infestations
Sinusitis
|
11.1%
3/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Infections and infestations
Staphylococcal infection
|
14.8%
4/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Infections and infestations
Urinary tract infection enterococcal
|
7.4%
2/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Injury, poisoning and procedural complications
Contusion
|
7.4%
2/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Injury, poisoning and procedural complications
Excoriation
|
14.8%
4/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Injury, poisoning and procedural complications
Head injury
|
7.4%
2/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Investigations
Blood bilirubin increased
|
7.4%
2/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Investigations
Occult blood positive
|
7.4%
2/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Investigations
Weight decreased
|
7.4%
2/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Investigations
Weight increased
|
14.8%
4/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Metabolism and nutrition disorders
Anorexia
|
66.7%
18/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Metabolism and nutrition disorders
Decreased appetite
|
7.4%
2/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Metabolism and nutrition disorders
Fluid retention
|
18.5%
5/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
18.5%
5/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Metabolism and nutrition disorders
Hyperkalaemia
|
11.1%
3/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Metabolism and nutrition disorders
Hypoalbuminaemia
|
11.1%
3/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Metabolism and nutrition disorders
Hypocalcaemia
|
18.5%
5/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
33.3%
9/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Metabolism and nutrition disorders
Hypomagnesaemia
|
37.0%
10/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Metabolism and nutrition disorders
Hyponatraemia
|
18.5%
5/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Metabolism and nutrition disorders
Hypophosphataemia
|
11.1%
3/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
14.8%
4/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Musculoskeletal and connective tissue disorders
bone pain
|
7.4%
2/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
29.6%
8/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Musculoskeletal and connective tissue disorders
Pain in jaw
|
7.4%
2/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Nervous system disorders
Dizziness
|
7.4%
2/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Nervous system disorders
Headache
|
40.7%
11/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Nervous system disorders
Lethargy
|
7.4%
2/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Psychiatric disorders
Agitation
|
14.8%
4/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Psychiatric disorders
Anxiety
|
22.2%
6/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Psychiatric disorders
Insomnia
|
14.8%
4/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Renal and urinary disorders
Dysuria
|
7.4%
2/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Renal and urinary disorders
Haematuria
|
11.1%
3/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Renal and urinary disorders
Renal tubular acidosis
|
7.4%
2/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Reproductive system and breast disorders
Scrotal erythema
|
7.4%
2/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Reproductive system and breast disorders
Testicular pain
|
11.1%
3/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Respiratory, thoracic and mediastinal disorders
Atelectasis
|
7.4%
2/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
44.4%
12/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
11.1%
3/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
25.9%
7/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
51.9%
14/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
7.4%
2/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
7.4%
2/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Respiratory, thoracic and mediastinal disorders
Tachypnoea
|
14.8%
4/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Respiratory, thoracic and mediastinal disorders
Upper respiratory tract congestion
|
11.1%
3/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
11.1%
3/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Skin and subcutaneous tissue disorders
Dermatitis diaper
|
7.4%
2/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
7.4%
2/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Skin and subcutaneous tissue disorders
Erythema
|
11.1%
3/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Skin and subcutaneous tissue disorders
Ingrowing nail
|
7.4%
2/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Skin and subcutaneous tissue disorders
Petechiae
|
7.4%
2/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
59.3%
16/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Skin and subcutaneous tissue disorders
Rash
|
55.6%
15/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Skin and subcutaneous tissue disorders
Skin disorder
|
11.1%
3/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Skin and subcutaneous tissue disorders
Skin lesion
|
7.4%
2/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
7.4%
2/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Vascular disorders
Hypertension
|
59.3%
16/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
|
Vascular disorders
Hypotension
|
25.9%
7/27 • Adverse events collected from signed informed consent to Day 30, i.e. a period of up to 71 days.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The Clinical Trial Agreement generally does not restrict an investigator's discussion of trial results after completion. The Agreement permits sponsor a limited period of time to review material discussing trial results (approximately up to 90 days). Sponsor may remove confidential information, but authors have final control and approval of publication content. For multi-center studies, investigator agrees not to publish any results before the first multi-center publication.
- Publication restrictions are in place
Restriction type: OTHER