Trial Outcomes & Findings for A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease (NCT NCT00376168)
NCT ID: NCT00376168
Last Updated: 2018-10-04
Results Overview
Calculated as percent change in spleen volume from Baseline to 9 months
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
32 participants
Primary outcome timeframe
Baseline and 9 months
Results posted on
2018-10-04
Participant Flow
Participant milestones
| Measure |
ELELYSO 30 Units/kg
Taliglucerase alfa 30 Units/kg by intravenous infusion every two weeks
|
ELELYSO 60 Units/kg
Taliglucerase alfa 60 Units/kg by intravenous infusion every two weeks
|
|---|---|---|
|
Overall Study
STARTED
|
16
|
16
|
|
Overall Study
COMPLETED
|
14
|
15
|
|
Overall Study
NOT COMPLETED
|
2
|
1
|
Reasons for withdrawal
| Measure |
ELELYSO 30 Units/kg
Taliglucerase alfa 30 Units/kg by intravenous infusion every two weeks
|
ELELYSO 60 Units/kg
Taliglucerase alfa 60 Units/kg by intravenous infusion every two weeks
|
|---|---|---|
|
Overall Study
Adverse Event
|
1
|
1
|
|
Overall Study
Pregnancy
|
1
|
0
|
Baseline Characteristics
A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease
Baseline characteristics by cohort
| Measure |
ELELYSO 30 Units/kg
n=16 Participants
Taliglucerase alfa 30 Units/kg by intravenous infusion every two weeks
|
ELELYSO 60 Units/kg
n=16 Participants
Taliglucerase alfa 60 Units/kg by intravenous infusion every two weeks
|
Total
n=32 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
15 Participants
n=5 Participants
|
16 Participants
n=7 Participants
|
31 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Age, Continuous
|
36.3 years
STANDARD_DEVIATION 11.8 • n=5 Participants
|
36.0 years
STANDARD_DEVIATION 12.2 • n=7 Participants
|
36.2 years
STANDARD_DEVIATION 11.8 • n=5 Participants
|
|
Sex: Female, Male
Female
|
8 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
16 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
8 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
16 Participants
n=5 Participants
|
|
Region of Enrollment
Canada
|
1 participants
n=5 Participants
|
1 participants
n=7 Participants
|
2 participants
n=5 Participants
|
|
Region of Enrollment
Spain
|
0 participants
n=5 Participants
|
1 participants
n=7 Participants
|
1 participants
n=5 Participants
|
|
Region of Enrollment
Chile
|
1 participants
n=5 Participants
|
1 participants
n=7 Participants
|
2 participants
n=5 Participants
|
|
Region of Enrollment
South Africa
|
1 participants
n=5 Participants
|
1 participants
n=7 Participants
|
2 participants
n=5 Participants
|
|
Region of Enrollment
Israel
|
6 participants
n=5 Participants
|
6 participants
n=7 Participants
|
12 participants
n=5 Participants
|
|
Region of Enrollment
Italy
|
1 participants
n=5 Participants
|
1 participants
n=7 Participants
|
2 participants
n=5 Participants
|
|
Region of Enrollment
United Kingdom
|
0 participants
n=5 Participants
|
1 participants
n=7 Participants
|
1 participants
n=5 Participants
|
|
Region of Enrollment
Mexico
|
4 participants
n=5 Participants
|
2 participants
n=7 Participants
|
6 participants
n=5 Participants
|
|
Region of Enrollment
Serbia
|
2 participants
n=5 Participants
|
2 participants
n=7 Participants
|
4 participants
n=5 Participants
|
|
Spleen Volume
|
2130.94 mL
STANDARD_DEVIATION 1154.72 • n=5 Participants
|
2117.38 mL
STANDARD_DEVIATION 1356.17 • n=7 Participants
|
2120 mL
STANDARD_DEVIATION 1200 • n=5 Participants
|
|
Liver Volume
|
2880.60 mL
STANDARD_DEVIATION 736.12 • n=5 Participants
|
2481.31 mL
STANDARD_DEVIATION 452.74 • n=7 Participants
|
2600 mL
STANDARD_DEVIATION 600 • n=5 Participants
|
|
Hemoglobin
|
12.2 g/dL
STANDARD_DEVIATION 1.7 • n=5 Participants
|
11.4 g/dL
STANDARD_DEVIATION 2.6 • n=7 Participants
|
12 g/dL
STANDARD_DEVIATION 2 • n=5 Participants
|
|
Platelet Count
|
75,320 count/mm^3
STANDARD_DEVIATION 40,861 • n=5 Participants
|
65,038 count/mm^3
STANDARD_DEVIATION 28,668 • n=7 Participants
|
70,000 count/mm^3
STANDARD_DEVIATION 32,000 • n=5 Participants
|
|
Chitotriosidase
|
28158 nmol/ml/hr
STANDARD_DEVIATION 11686 • n=5 Participants
|
24702 nmol/ml/hr
STANDARD_DEVIATION 17428 • n=7 Participants
|
26000 nmol/ml/hr
STANDARD_DEVIATION 14000 • n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline and 9 monthsPopulation: Intent to treat
Calculated as percent change in spleen volume from Baseline to 9 months
Outcome measures
| Measure |
ELELYSO 30 Units/kg
n=15 Participants
Taliglucerase alfa 30 Units/kg by intravenous infusion every two weeks
|
ELELYSO 60 Units/kg
n=16 Participants
Taliglucerase alfa 60 Units/kg by intravenous infusion every two weeks
|
|---|---|---|
|
Change From Baseline in Spleen Volume Measured by MRI.
|
-26.91 percentage of change
Standard Deviation 7.79
|
-38.01 percentage of change
Standard Deviation 9.38
|
SECONDARY outcome
Timeframe: Baseline and 9 monthsPopulation: Intent to treat
Calculated as percent change in liver volume from Baseline to 9 months
Outcome measures
| Measure |
ELELYSO 30 Units/kg
n=15 Participants
Taliglucerase alfa 30 Units/kg by intravenous infusion every two weeks
|
ELELYSO 60 Units/kg
n=16 Participants
Taliglucerase alfa 60 Units/kg by intravenous infusion every two weeks
|
|---|---|---|
|
Change From Baseline in Liver Volume
|
-10.48 percentage of change from baseline
Standard Deviation 11.27
|
-11.11 percentage of change from baseline
Standard Deviation 6.68
|
SECONDARY outcome
Timeframe: Baseline and Month 9Population: Intent to treat
Absolute change in Hemoglobin concentration from Baseline to Month 9
Outcome measures
| Measure |
ELELYSO 30 Units/kg
n=14 Participants
Taliglucerase alfa 30 Units/kg by intravenous infusion every two weeks
|
ELELYSO 60 Units/kg
n=16 Participants
Taliglucerase alfa 60 Units/kg by intravenous infusion every two weeks
|
|---|---|---|
|
Change in Hemoglobin
|
1.6 g/dL
Standard Deviation 1.4
|
2.2 g/dL
Standard Deviation 1.4
|
SECONDARY outcome
Timeframe: Baseline and Month 9Population: Intent to treat
Change in Platelet count from Baseline to Month 9
Outcome measures
| Measure |
ELELYSO 30 Units/kg
n=15 Participants
Taliglucerase alfa 30 Units/kg by intravenous infusion every two weeks
|
ELELYSO 60 Units/kg
n=16 Participants
Taliglucerase alfa 60 Units/kg by intravenous infusion every two weeks
|
|---|---|---|
|
Change in Platelet Count
|
11,427 count/mm^3
Standard Deviation 20,214
|
41,494 count/mm^3
Standard Deviation 47,063
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline and Month 9Population: Intent to treat
Change in Chitotriosidase from Baseline to Month 9
Outcome measures
| Measure |
ELELYSO 30 Units/kg
n=14 Participants
Taliglucerase alfa 30 Units/kg by intravenous infusion every two weeks
|
ELELYSO 60 Units/kg
n=15 Participants
Taliglucerase alfa 60 Units/kg by intravenous infusion every two weeks
|
|---|---|---|
|
Change in Chitotriosidase
|
-14,548 nmol/ml/hr
Standard Deviation 8,025.8
|
-12,538 nmol/ml/hr
Standard Deviation 14,489
|
Adverse Events
ELELYSO 30 Units/kg
Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths
ELELYSO 60 Units/kg
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
ELELYSO 30 Units/kg
n=16 participants at risk
Taliglucerase alfa 30 Units/kg by intravenous infusion every two weeks
|
ELELYSO 60 Units/kg
n=16 participants at risk
Taliglucerase alfa 60 Units/kg by intravenous infusion every two weeks
|
|---|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
6.2%
1/16 • 9 months
|
0.00%
0/16 • 9 months
|
|
General disorders
Feeling hot
|
6.2%
1/16 • 9 months
|
0.00%
0/16 • 9 months
|
|
Immune system disorders
Hypersensitivity
|
6.2%
1/16 • 9 months
|
6.2%
1/16 • 9 months
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
0.00%
0/16 • 9 months
|
6.2%
1/16 • 9 months
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
6.2%
1/16 • 9 months
|
0.00%
0/16 • 9 months
|
|
Nervous system disorders
Dizziness
|
6.2%
1/16 • 9 months
|
0.00%
0/16 • 9 months
|
|
Nervous system disorders
Headache
|
6.2%
1/16 • 9 months
|
6.2%
1/16 • 9 months
|
|
Renal and urinary disorders
Glycosuria
|
0.00%
0/16 • 9 months
|
6.2%
1/16 • 9 months
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place