Trial Outcomes & Findings for Tocilizumab for Patients With Giant Cell Arteritis (NCT NCT01450137)
NCT ID: NCT01450137
Last Updated: 2019-02-12
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
30 participants
Primary outcome timeframe
12 weeks
Results posted on
2019-02-12
Participant Flow
Participant milestones
| Measure |
Tocilizumab
Tocilizumab 8mg/kg every 4 weeks until week 52.
Tocilizumab + Glucocorticoids (GCs): Tocilizumab 8mg/kg every 4 weeks until week 52.
|
Placebo
Placebo every 4 weeks until week 52.
Placebo + Glucocorticoids (GCs): Placebo every 4 weeks until week 52.
|
|---|---|---|
|
Up to Week 12
STARTED
|
20
|
10
|
|
Up to Week 12
COMPLETED
|
18
|
7
|
|
Up to Week 12
NOT COMPLETED
|
2
|
3
|
|
Week 12 to Study End
STARTED
|
18
|
7
|
|
Week 12 to Study End
COMPLETED
|
18
|
5
|
|
Week 12 to Study End
NOT COMPLETED
|
0
|
2
|
Reasons for withdrawal
| Measure |
Tocilizumab
Tocilizumab 8mg/kg every 4 weeks until week 52.
Tocilizumab + Glucocorticoids (GCs): Tocilizumab 8mg/kg every 4 weeks until week 52.
|
Placebo
Placebo every 4 weeks until week 52.
Placebo + Glucocorticoids (GCs): Placebo every 4 weeks until week 52.
|
|---|---|---|
|
Up to Week 12
Adverse Event
|
2
|
1
|
|
Up to Week 12
Withdrawal by Subject
|
0
|
2
|
|
Week 12 to Study End
Death
|
0
|
1
|
|
Week 12 to Study End
Withdrawal by Subject
|
0
|
1
|
Baseline Characteristics
Missing data.
Baseline characteristics by cohort
| Measure |
Tocilizumab
n=20 Participants
Tocilizumab 8mg/kg every 4 weeks until week 52.
Tocilizumab + Glucocorticoids (GCs): Tocilizumab 8mg/kg every 4 weeks until week 52.
|
Placebo
n=10 Participants
Placebo every 4 weeks until week 52.
Placebo + Glucocorticoids (GCs): Placebo every 4 weeks until week 52.
|
Total
n=30 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
71.3 years
STANDARD_DEVIATION 8.9 • n=20 Participants
|
68.8 years
STANDARD_DEVIATION 16.9 • n=10 Participants
|
70.5 years
STANDARD_DEVIATION 11.9 • n=30 Participants
|
|
Sex: Female, Male
Female
|
13 Participants
n=20 Participants
|
8 Participants
n=10 Participants
|
21 Participants
n=30 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=20 Participants
|
2 Participants
n=10 Participants
|
9 Participants
n=30 Participants
|
|
Body Mass Index (BMI)
|
23.6 kg/m^2
STANDARD_DEVIATION 3.0 • n=20 Participants
|
27.9 kg/m^2
STANDARD_DEVIATION 3.7 • n=10 Participants
|
25.0 kg/m^2
STANDARD_DEVIATION 3.8 • n=30 Participants
|
|
New onset giant cell arteritis
|
16 Participants
n=20 Participants
|
7 Participants
n=10 Participants
|
23 Participants
n=30 Participants
|
|
Biopsy of the temporal artery
Normal
|
5 Participants
n=20 Participants
|
0 Participants
n=10 Participants
|
5 Participants
n=30 Participants
|
|
Biopsy of the temporal artery
Abnormal
|
13 Participants
n=20 Participants
|
8 Participants
n=10 Participants
|
21 Participants
n=30 Participants
|
|
Biopsy of the temporal artery
Not done
|
2 Participants
n=20 Participants
|
2 Participants
n=10 Participants
|
4 Participants
n=30 Participants
|
|
Thoracoabdominal MR angiography
Normal
|
9 Participants
n=20 Participants
|
2 Participants
n=10 Participants
|
11 Participants
n=30 Participants
|
|
Thoracoabdominal MR angiography
Abnormal
|
11 Participants
n=20 Participants
|
6 Participants
n=10 Participants
|
17 Participants
n=30 Participants
|
|
Thoracoabdominal MR angiography
Not done
|
0 Participants
n=20 Participants
|
2 Participants
n=10 Participants
|
2 Participants
n=30 Participants
|
|
Symptoms and signs of giant cell arteritis
Fever >= 38°C
|
1 Participants
n=20 Participants
|
1 Participants
n=10 Participants
|
2 Participants
n=30 Participants
|
|
Symptoms and signs of giant cell arteritis
Weight loss > 2kg within 4 weeks
|
6 Participants
n=20 Participants
|
3 Participants
n=10 Participants
|
9 Participants
n=30 Participants
|
|
Symptoms and signs of giant cell arteritis
Night sweats
|
3 Participants
n=20 Participants
|
2 Participants
n=10 Participants
|
5 Participants
n=30 Participants
|
|
Symptoms and signs of giant cell arteritis
Headache
|
13 Participants
n=20 Participants
|
5 Participants
n=10 Participants
|
18 Participants
n=30 Participants
|
|
Symptoms and signs of giant cell arteritis
Scalp tenderness
|
9 Participants
n=20 Participants
|
1 Participants
n=10 Participants
|
10 Participants
n=30 Participants
|
|
Symptoms and signs of giant cell arteritis
Claudication of tongue
|
2 Participants
n=20 Participants
|
0 Participants
n=10 Participants
|
2 Participants
n=30 Participants
|
|
Symptoms and signs of giant cell arteritis
Masseter muscle claudication
|
11 Participants
n=20 Participants
|
4 Participants
n=10 Participants
|
15 Participants
n=30 Participants
|
|
Symptoms and signs of giant cell arteritis
Claudication of upper limbs
|
4 Participants
n=20 Participants
|
2 Participants
n=10 Participants
|
6 Participants
n=30 Participants
|
|
Symptoms and signs of giant cell arteritis
Claudication of lower limbs
|
0 Participants
n=20 Participants
|
2 Participants
n=10 Participants
|
2 Participants
n=30 Participants
|
|
Symptoms and signs of giant cell arteritis
Visual impairment
|
5 Participants
n=20 Participants
|
2 Participants
n=10 Participants
|
7 Participants
n=30 Participants
|
|
Blood pressure
Systolic right arm
|
130.6 mmHg
STANDARD_DEVIATION 18.0 • n=20 Participants • Missing data.
|
137.7 mmHg
STANDARD_DEVIATION 16.4 • n=9 Participants • Missing data.
|
132.8 mmHg
STANDARD_DEVIATION 17.5 • n=29 Participants • Missing data.
|
|
Blood pressure
Diastolic right arm
|
74.3 mmHg
STANDARD_DEVIATION 11.8 • n=20 Participants • Missing data.
|
77.7 mmHg
STANDARD_DEVIATION 15.2 • n=9 Participants • Missing data.
|
75.3 mmHg
STANDARD_DEVIATION 12.8 • n=29 Participants • Missing data.
|
|
Blood pressure
Systolic left arm
|
131.3 mmHg
STANDARD_DEVIATION 16.3 • n=19 Participants • Missing data.
|
136.9 mmHg
STANDARD_DEVIATION 13.0 • n=8 Participants • Missing data.
|
132.9 mmHg
STANDARD_DEVIATION 15.3 • n=27 Participants • Missing data.
|
|
Blood pressure
Diastolic left arm
|
75.1 mmHg
STANDARD_DEVIATION 11.5 • n=19 Participants • Missing data.
|
82.8 mmHg
STANDARD_DEVIATION 8.5 • n=8 Participants • Missing data.
|
77.3 mmHg
STANDARD_DEVIATION 11.1 • n=27 Participants • Missing data.
|
|
Erythrocyte sedimentation rate
|
69.0 mm/h
n=20 Participants
|
40.0 mm/h
n=10 Participants
|
54.5 mm/h
n=30 Participants
|
|
C-reactive protein
|
25.5 mg/L
n=20 Participants
|
39.0 mg/L
n=10 Participants
|
31.5 mg/L
n=30 Participants
|
PRIMARY outcome
Timeframe: 12 weeksPopulation: All randomized patients, intention-to-treat
Outcome measures
| Measure |
Tocilizumab
n=20 Participants
Tocilizumab 8mg/kg every 4 weeks until week 52.
Tocilizumab + Glucocorticoids (GCs): Tocilizumab 8mg/kg every 4 weeks until week 52.
|
Placebo
n=10 Participants
Placebo every 4 weeks until week 52.
Placebo + Glucocorticoids (GCs): Placebo every 4 weeks until week 52.
|
|---|---|---|
|
Number of Patients That Have Achieved Complete Remission of Disease
|
17 Participants
|
4 Participants
|
SECONDARY outcome
Timeframe: 12 monthsPopulation: All randomized patients, intention-to-treat
Outcome measures
| Measure |
Tocilizumab
n=20 Participants
Tocilizumab 8mg/kg every 4 weeks until week 52.
Tocilizumab + Glucocorticoids (GCs): Tocilizumab 8mg/kg every 4 weeks until week 52.
|
Placebo
n=10 Participants
Placebo every 4 weeks until week 52.
Placebo + Glucocorticoids (GCs): Placebo every 4 weeks until week 52.
|
|---|---|---|
|
Number of Relapse Free Patients
|
17 Participants
|
2 Participants
|
SECONDARY outcome
Timeframe: 12 monthsPopulation: All randomized patients, intention-to-treat
cumulative weight-adapted prednisolone dose
Outcome measures
| Measure |
Tocilizumab
n=20 Participants
Tocilizumab 8mg/kg every 4 weeks until week 52.
Tocilizumab + Glucocorticoids (GCs): Tocilizumab 8mg/kg every 4 weeks until week 52.
|
Placebo
n=10 Participants
Placebo every 4 weeks until week 52.
Placebo + Glucocorticoids (GCs): Placebo every 4 weeks until week 52.
|
|---|---|---|
|
Cumulative Dose of GCs in mg/kg
|
43 mg/kg
Interval 39.0 to 52.0
|
110 mg/kg
Interval 88.0 to 150.0
|
SECONDARY outcome
Timeframe: 12 monthsPopulation: All randomized patients, intention-to-treat
Outcome measures
| Measure |
Tocilizumab
n=20 Participants
Tocilizumab 8mg/kg every 4 weeks until week 52.
Tocilizumab + Glucocorticoids (GCs): Tocilizumab 8mg/kg every 4 weeks until week 52.
|
Placebo
n=10 Participants
Placebo every 4 weeks until week 52.
Placebo + Glucocorticoids (GCs): Placebo every 4 weeks until week 52.
|
|---|---|---|
|
Restricted Mean Survival Time to First Relapse After Induction of Remission
|
50 Weeks
Interval 46.0 to 54.0
|
25 Weeks
Interval 11.0 to 38.0
|
Adverse Events
Tocilizumab
Serious events: 7 serious events
Other events: 11 other events
Deaths: 0 deaths
Placebo
Serious events: 5 serious events
Other events: 6 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Tocilizumab
n=20 participants at risk
Tocilizumab 8mg/kg every 4 weeks until week 52.
Tocilizumab + Glucocorticoids (GCs): Tocilizumab 8mg/kg every 4 weeks until week 52.
|
Placebo
n=10 participants at risk
Placebo every 4 weeks until week 52.
Placebo + Glucocorticoids (GCs): Placebo every 4 weeks until week 52.
|
|---|---|---|
|
Gastrointestinal disorders
Gastrointestinal disease
|
15.0%
3/20 • Number of events 3 • 1 year
|
10.0%
1/10 • Number of events 1 • 1 year
|
|
Cardiac disorders
Cardiovascular disease
|
5.0%
1/20 • Number of events 1 • 1 year
|
20.0%
2/10 • Number of events 3 • 1 year
|
|
Injury, poisoning and procedural complications
Osteoporotic fracture
|
0.00%
0/20 • 1 year
|
10.0%
1/10 • Number of events 2 • 1 year
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.00%
0/20 • 1 year
|
10.0%
1/10 • Number of events 2 • 1 year
|
|
Blood and lymphatic system disorders
Glucocorticoid related hyperglycaemia and myopathia
|
5.0%
1/20 • Number of events 1 • 1 year
|
20.0%
2/10 • Number of events 2 • 1 year
|
|
Infections and infestations
Infectious disease
|
5.0%
1/20 • Number of events 1 • 1 year
|
0.00%
0/10 • 1 year
|
|
Skin and subcutaneous tissue disorders
Skin disease
|
5.0%
1/20 • Number of events 1 • 1 year
|
0.00%
0/10 • 1 year
|
Other adverse events
| Measure |
Tocilizumab
n=20 participants at risk
Tocilizumab 8mg/kg every 4 weeks until week 52.
Tocilizumab + Glucocorticoids (GCs): Tocilizumab 8mg/kg every 4 weeks until week 52.
|
Placebo
n=10 participants at risk
Placebo every 4 weeks until week 52.
Placebo + Glucocorticoids (GCs): Placebo every 4 weeks until week 52.
|
|---|---|---|
|
Cardiac disorders
Cardiovascular disease
|
0.00%
0/20 • 1 year
|
20.0%
2/10 • Number of events 2 • 1 year
|
|
Reproductive system and breast disorders
Cystic lesion mamma
|
5.0%
1/20 • Number of events 1 • 1 year
|
0.00%
0/10 • 1 year
|
|
Gastrointestinal disorders
Gastrointestinal disease
|
5.0%
1/20 • Number of events 1 • 1 year
|
0.00%
0/10 • 1 year
|
|
Blood and lymphatic system disorders
Glucocorticoid-related hyperglycaemia and myopathia
|
5.0%
1/20 • Number of events 2 • 1 year
|
10.0%
1/10 • Number of events 1 • 1 year
|
|
Infections and infestations
Infectious disease
|
35.0%
7/20 • Number of events 9 • 1 year
|
10.0%
1/10 • Number of events 1 • 1 year
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal disease
|
20.0%
4/20 • Number of events 5 • 1 year
|
30.0%
3/10 • Number of events 6 • 1 year
|
|
Injury, poisoning and procedural complications
Osteoporotic fracture
|
5.0%
1/20 • Number of events 1 • 1 year
|
10.0%
1/10 • Number of events 1 • 1 year
|
|
Skin and subcutaneous tissue disorders
Skin disease
|
0.00%
0/20 • 1 year
|
20.0%
2/10 • Number of events 2 • 1 year
|
Additional Information
Prof Peter M Villiger
Department of Rheumatology, Immunology and Allergology, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place