Trial Outcomes & Findings for Study to Assess Brincidofovir Treatment of Serious Diseases or Conditions Caused by Double-stranded DNA Viruses (NCT NCT01143181)
NCT ID: NCT01143181
Last Updated: 2021-08-12
Results Overview
Proportion of subjects who achieved a confirmed reduction in viral load for the primary dsDNA virus of ≥1 log10 copies/mL from baseline or to an undetectable level. Confirmation required the reduction in viral load (i.e., decrease of ≥ 1 log10 copies/mL from baseline or to undetectable levels) to be maintained at the next assessment for the subject to be considered a success.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
210 participants
Primary outcome timeframe
3 months
Results posted on
2021-08-12
Participant Flow
This was an expanded access study with the primary objective of providing brincidofovir (BCV) to subjects with serious or life-threatening conditions caused by double-stranded DNA viral infections. All subjects enrolled received BCV for up to 3 months until their clinical disease was resolved or stabilized and/or viral DNA testing was negative for 4 consecutive weeks, whichever was longer.
Participant milestones
| Measure |
BCV (≤4 mg/kg/Week)
Pediatric subjects (≤12 years) who received BCV once or twice weekly.
|
BCV (>4 mg/kg/Week)
Pediatric subjects (≤12 years) who received BCV once or twice weekly.
|
BCV (≤200 mg/Week)
Adult/adolescent subjects (≥13 years) who received BCV once or twice weekly.
|
BCV (>200 mg/Week)
Adult/adolescent subjects (≥13 years) who received BCV once or twice weekly.
|
|---|---|---|---|---|
|
Overall Study
STARTED
|
30
|
38
|
102
|
40
|
|
Overall Study
COMPLETED
|
14
|
22
|
58
|
17
|
|
Overall Study
NOT COMPLETED
|
16
|
16
|
44
|
23
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Study to Assess Brincidofovir Treatment of Serious Diseases or Conditions Caused by Double-stranded DNA Viruses
Baseline characteristics by cohort
| Measure |
BCV (≤4 mg/kg/Week)
n=30 Participants
Pediatric subjects (≤12 years) who received BCV once or twice weekly.
|
BCV (>4 mg/kg/Week)
n=38 Participants
Pediatric subjects (≤12 years) who received BCV once or twice weekly.
|
BCV (≤200 mg/Week)
n=102 Participants
Adult/adolescent subjects (≥13 years) who received BCV once or twice weekly.
|
BCV (>200 mg/Week)
n=40 Participants
Adult/adolescent subjects (≥13 years) who received BCV once or twice weekly.
|
Total
n=210 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|
|
Age, Continuous
|
5.2 years
STANDARD_DEVIATION 2.68 • n=99 Participants
|
5.9 years
STANDARD_DEVIATION 3.70 • n=107 Participants
|
45.3 years
STANDARD_DEVIATION 17.21 • n=206 Participants
|
45.7 years
STANDARD_DEVIATION 17.57 • n=7 Participants
|
32.5 years
STANDARD_DEVIATION 23.51 • n=31 Participants
|
|
Sex: Female, Male
Female
|
14 Participants
n=99 Participants
|
14 Participants
n=107 Participants
|
41 Participants
n=206 Participants
|
18 Participants
n=7 Participants
|
87 Participants
n=31 Participants
|
|
Sex: Female, Male
Male
|
16 Participants
n=99 Participants
|
24 Participants
n=107 Participants
|
61 Participants
n=206 Participants
|
22 Participants
n=7 Participants
|
123 Participants
n=31 Participants
|
PRIMARY outcome
Timeframe: 3 monthsPopulation: Only subjects who achieved a confirmed reduction in viral load for the primary dsDNA virus of ≥1 log10 copies/mL from baseline or to an undetectable level were included in this analysis.
Proportion of subjects who achieved a confirmed reduction in viral load for the primary dsDNA virus of ≥1 log10 copies/mL from baseline or to an undetectable level. Confirmation required the reduction in viral load (i.e., decrease of ≥ 1 log10 copies/mL from baseline or to undetectable levels) to be maintained at the next assessment for the subject to be considered a success.
Outcome measures
| Measure |
BCV (≤4 mg/kg/Week)
n=25 Participants
Pediatric subjects (≤12 years) who received BCV once or twice weekly.
|
BCV (>4 mg/kg/Week)
n=28 Participants
Pediatric subjects (≤12 years) who received BCV once or twice weekly.
|
BCV (≤200 mg/Week)
n=79 Participants
Adult/adolescent subjects (≥13 years) who received BCV once or twice weekly.
|
BCV (>200 mg/Week)
n=28 Participants
Adult/adolescent subjects (≥13 years) who received BCV once or twice weekly.
|
|---|---|---|---|---|
|
Number of Subjects Who Had a Sustained and Significant Reduction in Plasma Viral Load of Primary dsDNA Virus
|
15 Participants
|
18 Participants
|
39 Participants
|
13 Participants
|
Adverse Events
BCV (≤4 mg/kg/Week)
Serious events: 27 serious events
Other events: 30 other events
Deaths: 0 deaths
BCV (>4 mg/kg/Week)
Serious events: 32 serious events
Other events: 37 other events
Deaths: 0 deaths
BCV (≤200 mg/Week)
Serious events: 80 serious events
Other events: 101 other events
Deaths: 0 deaths
BCV (>200 mg/Week)
Serious events: 35 serious events
Other events: 40 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
BCV (≤4 mg/kg/Week)
n=30 participants at risk
Pediatric subjects (≤12 years) who received BCV once or twice weekly.
|
BCV (>4 mg/kg/Week)
n=38 participants at risk
Pediatric subjects (≤12 years) who received BCV once or twice weekly.
|
BCV (≤200 mg/Week)
n=102 participants at risk
Adult/adolescent subjects (≥13 years) who received BCV once or twice weekly.
|
BCV (>200 mg/Week)
n=40 participants at risk
Adult/adolescent subjects (≥13 years) who received BCV once or twice weekly.
|
|---|---|---|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
0.00%
0/30
|
2.6%
1/38
|
0.00%
0/102
|
2.5%
1/40
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
6.7%
2/30
|
5.3%
2/38
|
3.9%
4/102
|
0.00%
0/40
|
|
Blood and lymphatic system disorders
Leukocytosis
|
3.3%
1/30
|
0.00%
0/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Blood and lymphatic system disorders
Leukopenia
|
0.00%
0/30
|
0.00%
0/38
|
2.9%
3/102
|
0.00%
0/40
|
|
Blood and lymphatic system disorders
Lymphopenia
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Blood and lymphatic system disorders
Neutropenia
|
0.00%
0/30
|
0.00%
0/38
|
8.8%
9/102
|
0.00%
0/40
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
0.00%
0/30
|
0.00%
0/38
|
4.9%
5/102
|
0.00%
0/40
|
|
Blood and lymphatic system disorders
Thrombotic microangiopathy
|
0.00%
0/30
|
2.6%
1/38
|
0.00%
0/102
|
2.5%
1/40
|
|
Blood and lymphatic system disorders
Thrombotic thrombocytopenic purpura
|
0.00%
0/30
|
2.6%
1/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Cardiac disorders
Acute myocardial infarction
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Cardiac disorders
Bradycardia
|
0.00%
0/30
|
2.6%
1/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Cardiac disorders
Cardiac arrest
|
0.00%
0/30
|
0.00%
0/38
|
2.9%
3/102
|
0.00%
0/40
|
|
Cardiac disorders
Cardiac failure acute
|
0.00%
0/30
|
2.6%
1/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Cardiac disorders
Cardiogenic shock
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Cardiac disorders
Cardiopulmonary failure
|
3.3%
1/30
|
0.00%
0/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Cardiac disorders
Pericardial effusion
|
3.3%
1/30
|
2.6%
1/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Cardiac disorders
Tachycardia
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Cardiac disorders
Ventricular fibrillation
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Endocrine disorders
Adrenal insufficiency
|
0.00%
0/30
|
0.00%
0/38
|
0.00%
0/102
|
2.5%
1/40
|
|
Gastrointestinal disorders
Abdominal pain
|
10.0%
3/30
|
2.6%
1/38
|
3.9%
4/102
|
2.5%
1/40
|
|
Gastrointestinal disorders
Anal fistula
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Gastrointestinal disorders
Ascites
|
0.00%
0/30
|
2.6%
1/38
|
0.98%
1/102
|
2.5%
1/40
|
|
Gastrointestinal disorders
Colitis
|
0.00%
0/30
|
0.00%
0/38
|
2.0%
2/102
|
0.00%
0/40
|
|
Gastrointestinal disorders
Dental discomfort
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Gastrointestinal disorders
Diarrhoea haemorrhagic
|
0.00%
0/30
|
2.6%
1/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Gastrointestinal disorders
Diarrhoea
|
6.7%
2/30
|
7.9%
3/38
|
16.7%
17/102
|
15.0%
6/40
|
|
Gastrointestinal disorders
Dysphagia
|
0.00%
0/30
|
2.6%
1/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Gastrointestinal disorders
Gastrointestinal haemorrhage
|
0.00%
0/30
|
5.3%
2/38
|
0.98%
1/102
|
5.0%
2/40
|
|
Gastrointestinal disorders
Gastrointestinal necrosis
|
3.3%
1/30
|
0.00%
0/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Gastrointestinal disorders
Ileus
|
0.00%
0/30
|
2.6%
1/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Gastrointestinal disorders
Impaired gastric emptying
|
0.00%
0/30
|
0.00%
0/38
|
0.00%
0/102
|
2.5%
1/40
|
|
Gastrointestinal disorders
Intestinal ischaemia
|
3.3%
1/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Gastrointestinal disorders
Intestinal obstruction
|
0.00%
0/30
|
2.6%
1/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Gastrointestinal disorders
Lower gastrointestinal haemorrhage
|
10.0%
3/30
|
2.6%
1/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Gastrointestinal disorders
Malabsorption
|
3.3%
1/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/30
|
2.6%
1/38
|
8.8%
9/102
|
10.0%
4/40
|
|
Gastrointestinal disorders
Pancreatitis acute
|
3.3%
1/30
|
0.00%
0/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Gastrointestinal disorders
Pancreatitis
|
3.3%
1/30
|
5.3%
2/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Gastrointestinal disorders
Peritoneal haemorrhage
|
0.00%
0/30
|
2.6%
1/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Gastrointestinal disorders
Pneumatosis intestinalis
|
3.3%
1/30
|
5.3%
2/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Gastrointestinal disorders
Small intestinal obstruction
|
0.00%
0/30
|
2.6%
1/38
|
0.00%
0/102
|
2.5%
1/40
|
|
Gastrointestinal disorders
Stomatitis
|
0.00%
0/30
|
2.6%
1/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Gastrointestinal disorders
Upper gastrointestinal haemorrhage
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Gastrointestinal disorders
Vomiting
|
6.7%
2/30
|
5.3%
2/38
|
6.9%
7/102
|
10.0%
4/40
|
|
General disorders
Chest discomfort
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
General disorders
Chills
|
3.3%
1/30
|
0.00%
0/38
|
0.00%
0/102
|
0.00%
0/40
|
|
General disorders
Fatigue
|
0.00%
0/30
|
0.00%
0/38
|
0.00%
0/102
|
2.5%
1/40
|
|
General disorders
Generalised oedema
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
General disorders
Multi-organ failure
|
10.0%
3/30
|
2.6%
1/38
|
6.9%
7/102
|
15.0%
6/40
|
|
General disorders
Oedema peripheral
|
0.00%
0/30
|
0.00%
0/38
|
2.0%
2/102
|
0.00%
0/40
|
|
General disorders
Pyrexia
|
16.7%
5/30
|
15.8%
6/38
|
10.8%
11/102
|
0.00%
0/40
|
|
Hepatobiliary disorders
Cholestasis
|
0.00%
0/30
|
0.00%
0/38
|
0.00%
0/102
|
2.5%
1/40
|
|
Hepatobiliary disorders
Cholestatic liver injury
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Hepatobiliary disorders
Hepatic failure
|
3.3%
1/30
|
2.6%
1/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Hepatobiliary disorders
Hepatocellular injury
|
3.3%
1/30
|
0.00%
0/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Hepatobiliary disorders
Hyperbilirubinaemia
|
0.00%
0/30
|
0.00%
0/38
|
2.9%
3/102
|
0.00%
0/40
|
|
Hepatobiliary disorders
Venooclusive liver disease
|
3.3%
1/30
|
0.00%
0/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Immune system disorders
Acute graft versus host disease
|
0.00%
0/30
|
5.3%
2/38
|
12.7%
13/102
|
17.5%
7/40
|
|
Immune system disorders
Chronic graft versus host disease
|
10.0%
3/30
|
2.6%
1/38
|
3.9%
4/102
|
5.0%
2/40
|
|
Immune system disorders
Graft versus host disease
|
0.00%
0/30
|
0.00%
0/38
|
0.00%
0/102
|
2.5%
1/40
|
|
Immune system disorders
Immune reconstitution syndrome
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Immune system disorders
Lung transplant rejection
|
0.00%
0/30
|
0.00%
0/38
|
0.00%
0/102
|
2.5%
1/40
|
|
Infections and infestations
Adenoviral hepatitis
|
3.3%
1/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Infections and infestations
Adenovirus infection
|
3.3%
1/30
|
2.6%
1/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Infections and infestations
Aspergillosis
|
3.3%
1/30
|
0.00%
0/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Infections and infestations
BK virus infection
|
0.00%
0/30
|
2.6%
1/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Infections and infestations
Bacterial infection
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Infections and infestations
Bacterial sepsis
|
0.00%
0/30
|
2.6%
1/38
|
2.9%
3/102
|
2.5%
1/40
|
|
Infections and infestations
Bronchiectasis
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Infections and infestations
Bronchopulmonary aspergillosis
|
3.3%
1/30
|
0.00%
0/38
|
0.00%
0/102
|
2.5%
1/40
|
|
Infections and infestations
Candidiasis
|
0.00%
0/30
|
2.6%
1/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Infections and infestations
Cellulitis
|
0.00%
0/30
|
2.6%
1/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Infections and infestations
Central nervous system infection
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Infections and infestations
Citrobacter infection
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Infections and infestations
Clostridium difficile colitis
|
0.00%
0/30
|
0.00%
0/38
|
2.0%
2/102
|
7.5%
3/40
|
|
Infections and infestations
Corona virus infection
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Infections and infestations
Cytomegalovirus gastrointestinal infection
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Infections and infestations
Cytomegalovirus infection
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Infections and infestations
Cytomegalovirus viraemia
|
0.00%
0/30
|
0.00%
0/38
|
0.00%
0/102
|
2.5%
1/40
|
|
Infections and infestations
Disseminated cytomegaloviral infection
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Infections and infestations
Encephalitis herpes
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Infections and infestations
Enterobacter infection
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Infections and infestations
Enterobacter pneumonia
|
0.00%
0/30
|
0.00%
0/38
|
0.00%
0/102
|
2.5%
1/40
|
|
Infections and infestations
Enterobacter sepsis
|
3.3%
1/30
|
0.00%
0/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Infections and infestations
Enterobacter bacteraemia
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Infections and infestations
Enterococcal infection
|
0.00%
0/30
|
2.6%
1/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Infections and infestations
Enterococcal sepsis
|
0.00%
0/30
|
2.6%
1/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Infections and infestations
Enterococcal bacteraemia
|
0.00%
0/30
|
0.00%
0/38
|
2.0%
2/102
|
0.00%
0/40
|
|
Infections and infestations
Escherichia infection
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Infections and infestations
Escherichia bacteraemia
|
0.00%
0/30
|
2.6%
1/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Infections and infestations
Fungal sepsis
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Infections and infestations
Gastroenteritis viral
|
0.00%
0/30
|
2.6%
1/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Infections and infestations
Human herpesvirus 6 infection
|
3.3%
1/30
|
0.00%
0/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Infections and infestations
Influenza
|
0.00%
0/30
|
0.00%
0/38
|
0.00%
0/102
|
2.5%
1/40
|
|
Infections and infestations
JC virus infection
|
0.00%
0/30
|
0.00%
0/38
|
0.00%
0/102
|
2.5%
1/40
|
|
Infections and infestations
Klebsiella bacteraemia
|
3.3%
1/30
|
0.00%
0/38
|
2.0%
2/102
|
0.00%
0/40
|
|
Infections and infestations
Klebsiella infection
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Infections and infestations
Klebsiella sepsis
|
0.00%
0/30
|
0.00%
0/38
|
2.0%
2/102
|
0.00%
0/40
|
|
Infections and infestations
Legionella infection
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Infections and infestations
Lung infection
|
3.3%
1/30
|
0.00%
0/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Infections and infestations
Lung infection pseudomonal
|
0.00%
0/30
|
2.6%
1/38
|
2.0%
2/102
|
0.00%
0/40
|
|
Infections and infestations
Meningoencephalitis adenoviral
|
0.00%
0/30
|
0.00%
0/38
|
0.00%
0/102
|
2.5%
1/40
|
|
Infections and infestations
Osteomyelitis
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Infections and infestations
Parainfluenzae virus infection
|
0.00%
0/30
|
0.00%
0/38
|
0.00%
0/102
|
2.5%
1/40
|
|
Infections and infestations
Pneumococcal sepsis
|
0.00%
0/30
|
0.00%
0/38
|
2.0%
2/102
|
0.00%
0/40
|
|
Infections and infestations
Pneumocystis jiroveci pneumonia
|
0.00%
0/30
|
0.00%
0/38
|
0.00%
0/102
|
2.5%
1/40
|
|
Infections and infestations
Pneumonia adenoviral
|
3.3%
1/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Infections and infestations
Pneumonia bacterial
|
0.00%
0/30
|
0.00%
0/38
|
2.9%
3/102
|
0.00%
0/40
|
|
Infections and infestations
Pneumonia fungal
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Infections and infestations
Pneumonia herpes viral
|
0.00%
0/30
|
0.00%
0/38
|
0.00%
0/102
|
2.5%
1/40
|
|
Infections and infestations
Pneumonia necrotising
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Infections and infestations
Pneumonia pneumococcal
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Infections and infestations
Pneumonia primary atypical
|
3.3%
1/30
|
0.00%
0/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Infections and infestations
Pneumonia respiratory syncytial viral
|
0.00%
0/30
|
0.00%
0/38
|
0.00%
0/102
|
2.5%
1/40
|
|
Infections and infestations
Pneumonia
|
3.3%
1/30
|
2.6%
1/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Infections and infestations
Pneumonia cytomegaloviral
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
2.5%
1/40
|
|
Infections and infestations
Pneumonia staphylococcal
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
2.5%
1/40
|
|
Infections and infestations
Pneumonia streptococcal
|
0.00%
0/30
|
0.00%
0/38
|
0.00%
0/102
|
2.5%
1/40
|
|
Infections and infestations
Progressive multifocal leukoencephalopathy
|
0.00%
0/30
|
0.00%
0/38
|
2.0%
2/102
|
2.5%
1/40
|
|
Infections and infestations
Pseudomonal sepsis
|
0.00%
0/30
|
2.6%
1/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Infections and infestations
Pseudomonal bacteraemia
|
3.3%
1/30
|
0.00%
0/38
|
2.9%
3/102
|
0.00%
0/40
|
|
Infections and infestations
Rectal abscess
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Infections and infestations
Respiratory syncytial virus bronchiolitis
|
0.00%
0/30
|
0.00%
0/38
|
0.00%
0/102
|
2.5%
1/40
|
|
Infections and infestations
Respiratory syncytial virus infection
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Infections and infestations
Sepsis
|
0.00%
0/30
|
2.6%
1/38
|
2.0%
2/102
|
2.5%
1/40
|
|
Infections and infestations
Septic shock
|
0.00%
0/30
|
2.6%
1/38
|
2.0%
2/102
|
2.5%
1/40
|
|
Infections and infestations
Serratia infection
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Infections and infestations
Sinusitis fungal
|
3.3%
1/30
|
0.00%
0/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Infections and infestations
Sinusitis
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Infections and infestations
Staphylococcal sepsis
|
0.00%
0/30
|
0.00%
0/38
|
0.00%
0/102
|
2.5%
1/40
|
|
Infections and infestations
Staphylococcal bacteraemia
|
6.7%
2/30
|
2.6%
1/38
|
2.9%
3/102
|
2.5%
1/40
|
|
Infections and infestations
Staphylococcal infection
|
0.00%
0/30
|
0.00%
0/38
|
2.0%
2/102
|
0.00%
0/40
|
|
Infections and infestations
Stenotrophomonas infection
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Infections and infestations
Stenotrophomonas sepsis
|
3.3%
1/30
|
2.6%
1/38
|
2.0%
2/102
|
0.00%
0/40
|
|
Infections and infestations
Toxoplasmosis
|
3.3%
1/30
|
0.00%
0/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Infections and infestations
Upper respiratory tract infection
|
3.3%
1/30
|
0.00%
0/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Infections and infestations
Zygomycosis
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Injury, poisoning and procedural complications
Arteriovenous fistula thrombosis
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Injury, poisoning and procedural complications
Blood stem cell transplant failure
|
0.00%
0/30
|
2.6%
1/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Injury, poisoning and procedural complications
Transplant failure
|
3.3%
1/30
|
2.6%
1/38
|
0.00%
0/102
|
2.5%
1/40
|
|
Investigations
Alanine aminotransferase increased
|
0.00%
0/30
|
0.00%
0/38
|
2.9%
3/102
|
0.00%
0/40
|
|
Investigations
Aspartate aminotransferase increased
|
0.00%
0/30
|
0.00%
0/38
|
2.9%
3/102
|
0.00%
0/40
|
|
Investigations
Blood bilirubin increased
|
0.00%
0/30
|
0.00%
0/38
|
0.00%
0/102
|
2.5%
1/40
|
|
Investigations
Blood creatinine increased
|
0.00%
0/30
|
0.00%
0/38
|
2.9%
3/102
|
0.00%
0/40
|
|
Investigations
Neutrophil count decreased
|
3.3%
1/30
|
0.00%
0/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Investigations
Pancreatic enzymes increased
|
0.00%
0/30
|
2.6%
1/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Investigations
White blood cell count decreased
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Metabolism and nutrition disorders
Decreased appetite
|
3.3%
1/30
|
5.3%
2/38
|
2.0%
2/102
|
5.0%
2/40
|
|
Metabolism and nutrition disorders
Dehydration
|
3.3%
1/30
|
5.3%
2/38
|
5.9%
6/102
|
5.0%
2/40
|
|
Metabolism and nutrition disorders
Electrolyte imbalance
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Metabolism and nutrition disorders
Failure to thrive
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Metabolism and nutrition disorders
Fluid overload
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Metabolism and nutrition disorders
Fluid retention
|
0.00%
0/30
|
2.6%
1/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Metabolism and nutrition disorders
Hyperkalaemia
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
0.00%
0/30
|
2.6%
1/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Metabolism and nutrition disorders
Lactic acidosis
|
0.00%
0/30
|
2.6%
1/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Metabolism and nutrition disorders
Metabolic acidosis
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Musculoskeletal and connective tissue disorders
Myopathy
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Acute lymphocytic leukaemia recurrent
|
0.00%
0/30
|
2.6%
1/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Acute myeloid leukaemia
|
3.3%
1/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Adult T-cell lymphoma/leukaemia refractory
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Diffuse large B-cell lymphoma
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Epstein-Barr virus associated lymphoproliferative disorder
|
6.7%
2/30
|
2.6%
1/38
|
0.98%
1/102
|
5.0%
2/40
|
|
Nervous system disorders
Cerebral haemorrhage
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Nervous system disorders
Cerebrovascular accident
|
0.00%
0/30
|
0.00%
0/38
|
0.00%
0/102
|
2.5%
1/40
|
|
Nervous system disorders
Convulsion
|
3.3%
1/30
|
0.00%
0/38
|
0.00%
0/102
|
2.5%
1/40
|
|
Nervous system disorders
Embolic cerebral infarction
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Nervous system disorders
Encephalopathy
|
0.00%
0/30
|
2.6%
1/38
|
2.0%
2/102
|
2.5%
1/40
|
|
Nervous system disorders
Guillain-Barre syndrome
|
0.00%
0/30
|
0.00%
0/38
|
0.00%
0/102
|
5.0%
2/40
|
|
Nervous system disorders
Haemorrhage intracranial
|
0.00%
0/30
|
2.6%
1/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Nervous system disorders
Hemiparesis
|
0.00%
0/30
|
0.00%
0/38
|
0.00%
0/102
|
2.5%
1/40
|
|
Nervous system disorders
Status epilepticus
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Nervous system disorders
Syncope
|
0.00%
0/30
|
0.00%
0/38
|
2.0%
2/102
|
0.00%
0/40
|
|
Psychiatric disorders
Confusional state
|
0.00%
0/30
|
0.00%
0/38
|
2.9%
3/102
|
0.00%
0/40
|
|
Psychiatric disorders
Delirium
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Psychiatric disorders
Mental status changes
|
3.3%
1/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Renal and urinary disorders
Azotaemia
|
0.00%
0/30
|
0.00%
0/38
|
2.0%
2/102
|
0.00%
0/40
|
|
Renal and urinary disorders
Bladder spasm
|
0.00%
0/30
|
2.6%
1/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Renal and urinary disorders
Dysuria
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Renal and urinary disorders
Renal failure acute
|
0.00%
0/30
|
5.3%
2/38
|
3.9%
4/102
|
10.0%
4/40
|
|
Renal and urinary disorders
Renal failure chronic
|
0.00%
0/30
|
0.00%
0/38
|
2.0%
2/102
|
0.00%
0/40
|
|
Renal and urinary disorders
Renal failure
|
6.7%
2/30
|
0.00%
0/38
|
2.0%
2/102
|
2.5%
1/40
|
|
Renal and urinary disorders
Renal impairment
|
0.00%
0/30
|
0.00%
0/38
|
0.00%
0/102
|
2.5%
1/40
|
|
Reproductive system and breast disorders
Oedema genital
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Respiratory, thoracic and mediastinal disorders
Acute respiratory distress syndrome
|
10.0%
3/30
|
2.6%
1/38
|
0.98%
1/102
|
2.5%
1/40
|
|
Respiratory, thoracic and mediastinal disorders
Aspiration
|
0.00%
0/30
|
2.6%
1/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
0.00%
0/30
|
5.3%
2/38
|
3.9%
4/102
|
0.00%
0/40
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Respiratory, thoracic and mediastinal disorders
Haemothorax
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
6.7%
2/30
|
2.6%
1/38
|
4.9%
5/102
|
2.5%
1/40
|
|
Respiratory, thoracic and mediastinal disorders
Idiopathic pneumonia syndrome
|
0.00%
0/30
|
0.00%
0/38
|
2.0%
2/102
|
0.00%
0/40
|
|
Respiratory, thoracic and mediastinal disorders
Lung infiltration
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Respiratory, thoracic and mediastinal disorders
Organising pneumonia
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
0.00%
0/30
|
0.00%
0/38
|
2.0%
2/102
|
0.00%
0/40
|
|
Respiratory, thoracic and mediastinal disorders
Pleural haemorrhage
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Respiratory, thoracic and mediastinal disorders
Pneumomediastinum
|
3.3%
1/30
|
0.00%
0/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia aspiration
|
3.3%
1/30
|
0.00%
0/38
|
2.0%
2/102
|
0.00%
0/40
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
0.00%
0/30
|
0.00%
0/38
|
0.00%
0/102
|
2.5%
1/40
|
|
Respiratory, thoracic and mediastinal disorders
Pneumothorax
|
3.3%
1/30
|
0.00%
0/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary alveolar haemorrhage
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary congestion
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary haemorrhage
|
0.00%
0/30
|
2.6%
1/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary hypertension
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary oedema
|
0.00%
0/30
|
2.6%
1/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory acidosis
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory arrest
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory distress
|
3.3%
1/30
|
7.9%
3/38
|
2.0%
2/102
|
7.5%
3/40
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
20.0%
6/30
|
13.2%
5/38
|
8.8%
9/102
|
10.0%
4/40
|
|
Respiratory, thoracic and mediastinal disorders
Upper-airway cough syndrome
|
3.3%
1/30
|
0.00%
0/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Skin and subcutaneous tissue disorders
Decubitus ulcer
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Skin and subcutaneous tissue disorders
Skin haemorrhage
|
0.00%
0/30
|
2.6%
1/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Vascular disorders
Aortic thrombosis
|
3.3%
1/30
|
0.00%
0/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Vascular disorders
Capillary leak syndrome
|
3.3%
1/30
|
0.00%
0/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Vascular disorders
Deep vein thrombosis
|
0.00%
0/30
|
0.00%
0/38
|
2.0%
2/102
|
0.00%
0/40
|
|
Vascular disorders
Haematoma
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Vascular disorders
Hypertension
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
2.5%
1/40
|
|
Vascular disorders
Hypotension
|
3.3%
1/30
|
10.5%
4/38
|
6.9%
7/102
|
0.00%
0/40
|
|
Vascular disorders
Hypovolaemic shock
|
0.00%
0/30
|
5.3%
2/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Vascular disorders
Orthostatic hypotension
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Vascular disorders
Shock
|
0.00%
0/30
|
2.6%
1/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Vascular disorders
Venous stenosis
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
0.00%
0/40
|
Other adverse events
| Measure |
BCV (≤4 mg/kg/Week)
n=30 participants at risk
Pediatric subjects (≤12 years) who received BCV once or twice weekly.
|
BCV (>4 mg/kg/Week)
n=38 participants at risk
Pediatric subjects (≤12 years) who received BCV once or twice weekly.
|
BCV (≤200 mg/Week)
n=102 participants at risk
Adult/adolescent subjects (≥13 years) who received BCV once or twice weekly.
|
BCV (>200 mg/Week)
n=40 participants at risk
Adult/adolescent subjects (≥13 years) who received BCV once or twice weekly.
|
|---|---|---|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
6.7%
2/30
|
7.9%
3/38
|
4.9%
5/102
|
7.5%
3/40
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
6.7%
2/30
|
5.3%
2/38
|
4.9%
5/102
|
0.00%
0/40
|
|
Blood and lymphatic system disorders
Neutropenia
|
10.0%
3/30
|
5.3%
2/38
|
15.7%
16/102
|
20.0%
8/40
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
0.00%
0/30
|
2.6%
1/38
|
9.8%
10/102
|
7.5%
3/40
|
|
Cardiac disorders
Bradycardia
|
6.7%
2/30
|
7.9%
3/38
|
2.0%
2/102
|
2.5%
1/40
|
|
Cardiac disorders
Pericardial effusion
|
6.7%
2/30
|
5.3%
2/38
|
2.0%
2/102
|
2.5%
1/40
|
|
Cardiac disorders
Tachycardia
|
6.7%
2/30
|
2.6%
1/38
|
8.8%
9/102
|
2.5%
1/40
|
|
Endocrine disorders
Adrenal insufficiency
|
3.3%
1/30
|
5.3%
2/38
|
2.0%
2/102
|
5.0%
2/40
|
|
Eye disorders
Dry eye
|
3.3%
1/30
|
0.00%
0/38
|
3.9%
4/102
|
5.0%
2/40
|
|
Gastrointestinal disorders
Abdominal pain
|
20.0%
6/30
|
18.4%
7/38
|
25.5%
26/102
|
10.0%
4/40
|
|
Gastrointestinal disorders
Ascites
|
3.3%
1/30
|
7.9%
3/38
|
4.9%
5/102
|
7.5%
3/40
|
|
Gastrointestinal disorders
Colitis
|
0.00%
0/30
|
7.9%
3/38
|
4.9%
5/102
|
2.5%
1/40
|
|
Gastrointestinal disorders
Constipation
|
6.7%
2/30
|
2.6%
1/38
|
7.8%
8/102
|
5.0%
2/40
|
|
Gastrointestinal disorders
Diarrhoea
|
40.0%
12/30
|
57.9%
22/38
|
59.8%
61/102
|
47.5%
19/40
|
|
Gastrointestinal disorders
Dysphagia
|
0.00%
0/30
|
5.3%
2/38
|
2.9%
3/102
|
0.00%
0/40
|
|
Gastrointestinal disorders
Flatulence
|
0.00%
0/30
|
0.00%
0/38
|
5.9%
6/102
|
0.00%
0/40
|
|
Gastrointestinal disorders
Gastrointestinal haemorrhage
|
0.00%
0/30
|
10.5%
4/38
|
0.98%
1/102
|
7.5%
3/40
|
|
Gastrointestinal disorders
Haematochezia
|
3.3%
1/30
|
5.3%
2/38
|
2.0%
2/102
|
2.5%
1/40
|
|
Gastrointestinal disorders
Ileus
|
6.7%
2/30
|
2.6%
1/38
|
0.98%
1/102
|
5.0%
2/40
|
|
Gastrointestinal disorders
Lower gastrointestinal haemorrhage
|
10.0%
3/30
|
2.6%
1/38
|
0.00%
0/102
|
2.5%
1/40
|
|
Gastrointestinal disorders
Nausea
|
10.0%
3/30
|
15.8%
6/38
|
41.2%
42/102
|
22.5%
9/40
|
|
Gastrointestinal disorders
Pancreatitis
|
3.3%
1/30
|
5.3%
2/38
|
0.98%
1/102
|
2.5%
1/40
|
|
Gastrointestinal disorders
Pneumatosis
|
3.3%
1/30
|
5.3%
2/38
|
0.98%
1/102
|
2.5%
1/40
|
|
Gastrointestinal disorders
Small intestinal obstruction
|
0.00%
0/30
|
2.6%
1/38
|
0.00%
0/102
|
7.5%
3/40
|
|
Gastrointestinal disorders
Vomiting
|
26.7%
8/30
|
34.2%
13/38
|
29.4%
30/102
|
17.5%
7/40
|
|
General disorders
Asthenia
|
0.00%
0/30
|
0.00%
0/38
|
2.9%
3/102
|
7.5%
3/40
|
|
General disorders
Chest pain
|
0.00%
0/30
|
0.00%
0/38
|
0.00%
0/102
|
7.5%
3/40
|
|
General disorders
Chills
|
3.3%
1/30
|
2.6%
1/38
|
6.9%
7/102
|
5.0%
2/40
|
|
General disorders
Fatigue
|
3.3%
1/30
|
2.6%
1/38
|
12.7%
13/102
|
2.5%
1/40
|
|
General disorders
Generalized oedema
|
6.7%
2/30
|
2.6%
1/38
|
5.9%
6/102
|
0.00%
0/40
|
|
General disorders
Muscosal inflammation
|
6.7%
2/30
|
2.6%
1/38
|
0.00%
0/102
|
0.00%
0/40
|
|
General disorders
Multi-organ failure
|
10.0%
3/30
|
2.6%
1/38
|
6.9%
7/102
|
15.0%
6/40
|
|
General disorders
Oedema peripheral
|
0.00%
0/30
|
2.6%
1/38
|
16.7%
17/102
|
5.0%
2/40
|
|
General disorders
Pain
|
3.3%
1/30
|
5.3%
2/38
|
2.9%
3/102
|
0.00%
0/40
|
|
General disorders
Pyrexia
|
43.3%
13/30
|
23.7%
9/38
|
24.5%
25/102
|
10.0%
4/40
|
|
Hepatobiliary disorders
Cholelithiasis
|
0.00%
0/30
|
0.00%
0/38
|
0.00%
0/102
|
5.0%
2/40
|
|
Hepatobiliary disorders
Cholestasis
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
5.0%
2/40
|
|
Hepatobiliary disorders
Hepatic failure
|
3.3%
1/30
|
5.3%
2/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Hepatobiliary disorders
Hyperbilirubinaemia
|
3.3%
1/30
|
2.6%
1/38
|
6.9%
7/102
|
7.5%
3/40
|
|
Immune system disorders
Acute graft versus host disease
|
26.7%
8/30
|
28.9%
11/38
|
29.4%
30/102
|
27.5%
11/40
|
|
Immune system disorders
Chronic graft versus host disease
|
10.0%
3/30
|
5.3%
2/38
|
7.8%
8/102
|
7.5%
3/40
|
|
Infections and infestations
Adenovirus infection
|
6.7%
2/30
|
2.6%
1/38
|
2.0%
2/102
|
0.00%
0/40
|
|
Infections and infestations
BK virus infection
|
3.3%
1/30
|
5.3%
2/38
|
3.9%
4/102
|
5.0%
2/40
|
|
Infections and infestations
Bacteraemia
|
6.7%
2/30
|
5.3%
2/38
|
2.0%
2/102
|
2.5%
1/40
|
|
Infections and infestations
Candidiasis
|
0.00%
0/30
|
10.5%
4/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Infections and infestations
Clostridial infection
|
0.00%
0/30
|
7.9%
3/38
|
0.98%
1/102
|
2.5%
1/40
|
|
Infections and infestations
Clostridium difficile colitis
|
0.00%
0/30
|
7.9%
3/38
|
5.9%
6/102
|
12.5%
5/40
|
|
Infections and infestations
Cytomegalovirus colitis
|
6.7%
2/30
|
0.00%
0/38
|
0.98%
1/102
|
2.5%
1/40
|
|
Infections and infestations
Cytomegalovirus viraemia
|
3.3%
1/30
|
0.00%
0/38
|
2.9%
3/102
|
5.0%
2/40
|
|
Infections and infestations
Enterococcal sepsis
|
0.00%
0/30
|
2.6%
1/38
|
0.98%
1/102
|
5.0%
2/40
|
|
Infections and infestations
Epstein-Barr viraemia
|
0.00%
0/30
|
0.00%
0/38
|
5.9%
6/102
|
2.5%
1/40
|
|
Infections and infestations
Gastroenteritis norovirus
|
3.3%
1/30
|
5.3%
2/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Infections and infestations
Human herpesvirus 6 infection
|
6.7%
2/30
|
0.00%
0/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Infections and infestations
Lung infection
|
0.00%
0/30
|
2.6%
1/38
|
2.0%
2/102
|
5.0%
2/40
|
|
Infections and infestations
Oral candidiasis
|
3.3%
1/30
|
5.3%
2/38
|
5.9%
6/102
|
0.00%
0/40
|
|
Infections and infestations
Pneumonia
|
10.0%
3/30
|
7.9%
3/38
|
3.9%
4/102
|
0.00%
0/40
|
|
Infections and infestations
Pneumonia cytomegaloviral
|
10.0%
3/30
|
0.00%
0/38
|
2.0%
2/102
|
5.0%
2/40
|
|
Infections and infestations
Pseudomonal bacteraemia
|
3.3%
1/30
|
0.00%
0/38
|
3.9%
4/102
|
5.0%
2/40
|
|
Infections and infestations
Sinusitis
|
3.3%
1/30
|
5.3%
2/38
|
2.0%
2/102
|
5.0%
2/40
|
|
Infections and infestations
Staphylococcal bacteraemia
|
6.7%
2/30
|
2.6%
1/38
|
5.9%
6/102
|
5.0%
2/40
|
|
Infections and infestations
Systemic candida
|
0.00%
0/30
|
2.6%
1/38
|
0.00%
0/102
|
5.0%
2/40
|
|
Infections and infestations
Upper respiratory tract infection
|
3.3%
1/30
|
5.3%
2/38
|
2.9%
3/102
|
0.00%
0/40
|
|
Infections and infestations
Urinary tract infection enterococcal
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
7.5%
3/40
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/30
|
5.3%
2/38
|
2.0%
2/102
|
0.00%
0/40
|
|
Injury, poisoning and procedural complications
Transplant failure
|
3.3%
1/30
|
5.3%
2/38
|
0.00%
0/102
|
2.5%
1/40
|
|
Investigations
Alanine aminotransferase increased
|
20.0%
6/30
|
10.5%
4/38
|
16.7%
17/102
|
15.0%
6/40
|
|
Investigations
Aspartate aminotransferase increased
|
16.7%
5/30
|
7.9%
3/38
|
16.7%
17/102
|
10.0%
4/40
|
|
Investigations
Blood alkaline phosphatase increased
|
3.3%
1/30
|
0.00%
0/38
|
7.8%
8/102
|
0.00%
0/40
|
|
Investigations
Blood bilirubin increased
|
6.7%
2/30
|
10.5%
4/38
|
3.9%
4/102
|
2.5%
1/40
|
|
Investigations
Blood creatinine increased
|
0.00%
0/30
|
2.6%
1/38
|
7.8%
8/102
|
2.5%
1/40
|
|
Investigations
Clostridium test positive
|
3.3%
1/30
|
0.00%
0/38
|
2.0%
2/102
|
5.0%
2/40
|
|
Investigations
Neutrophil count decreased
|
13.3%
4/30
|
5.3%
2/38
|
3.9%
4/102
|
0.00%
0/40
|
|
Investigations
Weight decreased
|
3.3%
1/30
|
2.6%
1/38
|
5.9%
6/102
|
5.0%
2/40
|
|
Metabolism and nutrition disorders
Decreased appetite
|
3.3%
1/30
|
18.4%
7/38
|
16.7%
17/102
|
5.0%
2/40
|
|
Metabolism and nutrition disorders
Dehydration
|
6.7%
2/30
|
7.9%
3/38
|
7.8%
8/102
|
7.5%
3/40
|
|
Metabolism and nutrition disorders
Fluid overload
|
3.3%
1/30
|
0.00%
0/38
|
2.9%
3/102
|
7.5%
3/40
|
|
Metabolism and nutrition disorders
Fluid retention
|
6.7%
2/30
|
5.3%
2/38
|
0.98%
1/102
|
0.00%
0/40
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
13.3%
4/30
|
7.9%
3/38
|
8.8%
9/102
|
5.0%
2/40
|
|
Metabolism and nutrition disorders
Hyperkalaemia
|
3.3%
1/30
|
0.00%
0/38
|
2.9%
3/102
|
7.5%
3/40
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
23.3%
7/30
|
10.5%
4/38
|
8.8%
9/102
|
5.0%
2/40
|
|
Metabolism and nutrition disorders
Hypomagnesaemia
|
13.3%
4/30
|
2.6%
1/38
|
2.0%
2/102
|
0.00%
0/40
|
|
Metabolism and nutrition disorders
Malnutrition
|
0.00%
0/30
|
0.00%
0/38
|
0.00%
0/102
|
5.0%
2/40
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
0.00%
0/30
|
2.6%
1/38
|
7.8%
8/102
|
2.5%
1/40
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
6.7%
2/30
|
7.9%
3/38
|
7.8%
8/102
|
2.5%
1/40
|
|
Musculoskeletal and connective tissue disorders
Muscular weakness
|
0.00%
0/30
|
0.00%
0/38
|
7.8%
8/102
|
0.00%
0/40
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
3.3%
1/30
|
7.9%
3/38
|
5.9%
6/102
|
0.00%
0/40
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Epstein-Barr virus associated lymphoproliferative
|
6.7%
2/30
|
2.6%
1/38
|
2.9%
3/102
|
7.5%
3/40
|
|
Nervous system disorders
Convulsion
|
3.3%
1/30
|
0.00%
0/38
|
0.98%
1/102
|
5.0%
2/40
|
|
Nervous system disorders
Dizziness
|
0.00%
0/30
|
2.6%
1/38
|
5.9%
6/102
|
7.5%
3/40
|
|
Nervous system disorders
Encephalopathy
|
0.00%
0/30
|
2.6%
1/38
|
2.9%
3/102
|
7.5%
3/40
|
|
Nervous system disorders
Guillain-Barre syndrome
|
0.00%
0/30
|
0.00%
0/38
|
0.00%
0/102
|
5.0%
2/40
|
|
Nervous system disorders
Headache
|
3.3%
1/30
|
7.9%
3/38
|
11.8%
12/102
|
7.5%
3/40
|
|
Nervous system disorders
Tremor
|
0.00%
0/30
|
2.6%
1/38
|
6.9%
7/102
|
5.0%
2/40
|
|
Nervous system disorders
Neuropathy peripheral
|
0.00%
0/30
|
0.00%
0/38
|
5.9%
6/102
|
0.00%
0/40
|
|
Psychiatric disorders
Agitation
|
3.3%
1/30
|
0.00%
0/38
|
5.9%
6/102
|
10.0%
4/40
|
|
Psychiatric disorders
Anxiety
|
6.7%
2/30
|
5.3%
2/38
|
5.9%
6/102
|
7.5%
3/40
|
|
Psychiatric disorders
Confusional state
|
0.00%
0/30
|
0.00%
0/38
|
7.8%
8/102
|
2.5%
1/40
|
|
Psychiatric disorders
Depression
|
6.7%
2/30
|
0.00%
0/38
|
5.9%
6/102
|
0.00%
0/40
|
|
Psychiatric disorders
Insomnia
|
3.3%
1/30
|
7.9%
3/38
|
4.9%
5/102
|
7.5%
3/40
|
|
Psychiatric disorders
Mental status changes
|
6.7%
2/30
|
0.00%
0/38
|
7.8%
8/102
|
5.0%
2/40
|
|
Renal and urinary disorders
Haematuria
|
0.00%
0/30
|
5.3%
2/38
|
3.9%
4/102
|
2.5%
1/40
|
|
Renal and urinary disorders
Renal failure acute
|
0.00%
0/30
|
15.8%
6/38
|
5.9%
6/102
|
10.0%
4/40
|
|
Renal and urinary disorders
Renal failure
|
13.3%
4/30
|
7.9%
3/38
|
3.9%
4/102
|
10.0%
4/40
|
|
Respiratory, thoracic and mediastinal disorders
Acute respiratory distress syndrome
|
10.0%
3/30
|
2.6%
1/38
|
0.98%
1/102
|
2.5%
1/40
|
|
Respiratory, thoracic and mediastinal disorders
Atelectasis
|
0.00%
0/30
|
7.9%
3/38
|
2.0%
2/102
|
0.00%
0/40
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
3.3%
1/30
|
7.9%
3/38
|
15.7%
16/102
|
5.0%
2/40
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
3.3%
1/30
|
7.9%
3/38
|
13.7%
14/102
|
2.5%
1/40
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
10.0%
3/30
|
5.3%
2/38
|
6.9%
7/102
|
2.5%
1/40
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
10.0%
3/30
|
5.3%
2/38
|
7.8%
8/102
|
2.5%
1/40
|
|
Respiratory, thoracic and mediastinal disorders
Lung infiltration
|
0.00%
0/30
|
0.00%
0/38
|
4.9%
5/102
|
5.0%
2/40
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
3.3%
1/30
|
0.00%
0/38
|
7.8%
8/102
|
0.00%
0/40
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
0.00%
0/30
|
7.9%
3/38
|
2.9%
3/102
|
0.00%
0/40
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
6.7%
2/30
|
0.00%
0/38
|
9.8%
10/102
|
5.0%
2/40
|
|
Respiratory, thoracic and mediastinal disorders
Pneumomediastinum
|
6.7%
2/30
|
0.00%
0/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Respiratory, thoracic and mediastinal disorders
Pneumothorax
|
10.0%
3/30
|
2.6%
1/38
|
2.0%
2/102
|
5.0%
2/40
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory distress
|
13.3%
4/30
|
15.8%
6/38
|
2.9%
3/102
|
7.5%
3/40
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
23.3%
7/30
|
13.2%
5/38
|
9.8%
10/102
|
15.0%
6/40
|
|
Respiratory, thoracic and mediastinal disorders
Tachypnoea
|
6.7%
2/30
|
2.6%
1/38
|
2.0%
2/102
|
2.5%
1/40
|
|
Respiratory, thoracic and mediastinal disorders
Sleep apnoea syndrome
|
0.00%
0/30
|
5.3%
2/38
|
0.00%
0/102
|
0.00%
0/40
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
3.3%
1/30
|
5.3%
2/38
|
2.0%
2/102
|
2.5%
1/40
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
0.00%
0/30
|
2.6%
1/38
|
3.9%
4/102
|
7.5%
3/40
|
|
Skin and subcutaneous tissue disorders
Erythema
|
0.00%
0/30
|
0.00%
0/38
|
0.98%
1/102
|
5.0%
2/40
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
13.3%
4/30
|
2.6%
1/38
|
5.9%
6/102
|
2.5%
1/40
|
|
Skin and subcutaneous tissue disorders
Rash
|
13.3%
4/30
|
7.9%
3/38
|
9.8%
10/102
|
10.0%
4/40
|
|
Skin and subcutaneous tissue disorders
Skin lesion
|
3.3%
1/30
|
0.00%
0/38
|
0.98%
1/102
|
5.0%
2/40
|
|
Vascular disorders
Hypertension
|
26.7%
8/30
|
7.9%
3/38
|
6.9%
7/102
|
10.0%
4/40
|
|
Vascular disorders
Hypotension
|
20.0%
6/30
|
18.4%
7/38
|
15.7%
16/102
|
12.5%
5/40
|
|
Vascular disorders
Hypovolaemic shock
|
0.00%
0/30
|
5.3%
2/38
|
0.00%
0/102
|
0.00%
0/40
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Within 18 months after the end of the Study at all sites, if no publication of the overall multi-center results has been made, institutions are entitled to publish their locally obtained results, provided the Sponsor is given opportunity to review and comment. Institution publications may be delayed up to an additional 60 days to allow Sponsor to seek patent protection.
- Publication restrictions are in place
Restriction type: OTHER