Trial Outcomes & Findings for A Multicenter Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher Disease (NCT NCT01422187)
NCT ID: NCT01422187
Last Updated: 2023-04-19
Results Overview
Spleen volume measured by MRI
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
19 participants
Primary outcome timeframe
60 months
Results posted on
2023-04-19
Participant Flow
Participant milestones
| Measure |
Taliglucerase Alfa 30 Units/kg
Subjects randomized to receive 30 units/kg
Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months
|
Taliglucerase Alfa 60 Units/kg
Subjects randomized to 60 units/kg
Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months
|
Dose Adjusted
Subjects randomized to 30 units/kg but with dose increased
Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months
|
|---|---|---|---|
|
Overall Study
STARTED
|
8
|
9
|
2
|
|
Overall Study
COMPLETED
|
7
|
8
|
2
|
|
Overall Study
NOT COMPLETED
|
1
|
1
|
0
|
Reasons for withdrawal
| Measure |
Taliglucerase Alfa 30 Units/kg
Subjects randomized to receive 30 units/kg
Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months
|
Taliglucerase Alfa 60 Units/kg
Subjects randomized to 60 units/kg
Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months
|
Dose Adjusted
Subjects randomized to 30 units/kg but with dose increased
Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months
|
|---|---|---|---|
|
Overall Study
Personal reason
|
1
|
0
|
0
|
|
Overall Study
Lost to Follow-up
|
0
|
1
|
0
|
Baseline Characteristics
A Multicenter Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher Disease
Baseline characteristics by cohort
| Measure |
Taliglucerase Alfa 30 Units/kg
n=8 Participants
Subjects randomized to receive 30 units/kg
Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months
|
Taliglucerase Alfa 60 Units/kg
n=9 Participants
Subjects randomized to 60 units/kg
Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months
|
Dose Adjusted
n=2 Participants
Subjects randomized to 30 units/kg but with dose increased
Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months
|
Total
n=19 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
44.8 years
STANDARD_DEVIATION 13.4 • n=5 Participants
|
39.9 years
STANDARD_DEVIATION 12.0 • n=7 Participants
|
38.5 years
STANDARD_DEVIATION 0.7 • n=5 Participants
|
41.8 years
STANDARD_DEVIATION 11.8 • n=4 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
8 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
11 Participants
n=4 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
White
|
8 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
19 Participants
n=4 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Region of Enrollment
Israel
|
2 participants
n=5 Participants
|
2 participants
n=7 Participants
|
0 participants
n=5 Participants
|
4 participants
n=4 Participants
|
|
Region of Enrollment
South Africa
|
0 participants
n=5 Participants
|
0 participants
n=7 Participants
|
1 participants
n=5 Participants
|
1 participants
n=4 Participants
|
|
Region of Enrollment
Italy
|
0 participants
n=5 Participants
|
1 participants
n=7 Participants
|
0 participants
n=5 Participants
|
1 participants
n=4 Participants
|
|
Region of Enrollment
Serbia
|
2 participants
n=5 Participants
|
2 participants
n=7 Participants
|
0 participants
n=5 Participants
|
4 participants
n=4 Participants
|
|
Region of Enrollment
Mexico
|
3 participants
n=5 Participants
|
2 participants
n=7 Participants
|
0 participants
n=5 Participants
|
5 participants
n=4 Participants
|
|
Region of Enrollment
Chile
|
0 participants
n=5 Participants
|
1 participants
n=7 Participants
|
1 participants
n=5 Participants
|
2 participants
n=4 Participants
|
|
Region of Enrollment
Spain
|
1 participants
n=5 Participants
|
1 participants
n=7 Participants
|
0 participants
n=5 Participants
|
2 participants
n=4 Participants
|
PRIMARY outcome
Timeframe: 60 monthsSpleen volume measured by MRI
Outcome measures
| Measure |
Taliglucerase Alfa 30 Units/kg
n=8 Participants
Subjects randomized to receive 30 units/kg
Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months
|
Taliglucerase Alfa 60 Units/kg
n=9 Participants
Subjects randomized to 60 units/kg
Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months
|
Dose Adjusted
n=2 Participants
Subjects randomized to 30 units/kg but with dose increased
Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months
|
|---|---|---|---|
|
Spleen Volume
Baseline
|
2032.0 Milliliters
Standard Error 454.9
|
1960.0 Milliliters
Standard Error 451.7
|
2321.7 Milliliters
Standard Error 679.6
|
|
Spleen Volume
Month 12
|
1522.6 Milliliters
Standard Error 429.6
|
1063.7 Milliliters
Standard Error 280.7
|
1677.9 Milliliters
Standard Error 609.3
|
|
Spleen Volume
Month 24
|
1193.0 Milliliters
Standard Error 320.2
|
744.9 Milliliters
Standard Error 130.6
|
1536.3 Milliliters
Standard Error 506.7
|
|
Spleen Volume
Month 36
|
1068.7 Milliliters
Standard Error 264.6
|
634.0 Milliliters
Standard Error 71.0
|
1256.6 Milliliters
Standard Error 385.0
|
|
Spleen Volume
Month 48
|
1137.4 Milliliters
Standard Error 314.6
|
526.8 Milliliters
Standard Error 48.5
|
1070.0 Milliliters
Standard Error 280.6
|
|
Spleen Volume
Month 60
|
1019.2 Milliliters
Standard Error 285.3
|
516.1 Milliliters
Standard Error 54.5
|
919.9 Milliliters
Standard Error 180.9
|
SECONDARY outcome
Timeframe: 60 monthsLiver volume by MRI
Outcome measures
| Measure |
Taliglucerase Alfa 30 Units/kg
n=8 Participants
Subjects randomized to receive 30 units/kg
Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months
|
Taliglucerase Alfa 60 Units/kg
n=9 Participants
Subjects randomized to 60 units/kg
Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months
|
Dose Adjusted
n=2 Participants
Subjects randomized to 30 units/kg but with dose increased
Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months
|
|---|---|---|---|
|
Liver Volume
Month 48
|
2429.1 Milliliters
Standard Error 283.2
|
1971.2 Milliliters
Standard Error 179.3
|
1792.2 Milliliters
Standard Error 143.6
|
|
Liver Volume
Month 60
|
2290.9 Milliliters
Standard Error 233.6
|
2067.7 Milliliters
Standard Error 188.9
|
1820.8 Milliliters
Standard Error 196.5
|
|
Liver Volume
Baseline
|
3103.4 Milliliters
Standard Error 324.7
|
2536.1 Milliliters
Standard Error 170.0
|
2488.6 Milliliters
Standard Error 206.2
|
|
Liver Volume
Month 12
|
2619.3 Milliliters
Standard Error 266.4
|
2155.2 Milliliters
Standard Error 104.8
|
2048.0 Milliliters
Standard Error 87.8
|
|
Liver Volume
Month 24
|
2367.9 Milliliters
Standard Error 212.8
|
1939.5 Milliliters
Standard Error 101.5
|
2064.0 Milliliters
Standard Error 202.5
|
|
Liver Volume
Month 36
|
2429.6 Milliliters
Standard Error 229.6
|
2010.6 Milliliters
Standard Error 143.5
|
1959.0 Milliliters
Standard Error 140.5
|
SECONDARY outcome
Timeframe: 60 monthsPlatelet count measure annually
Outcome measures
| Measure |
Taliglucerase Alfa 30 Units/kg
n=8 Participants
Subjects randomized to receive 30 units/kg
Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months
|
Taliglucerase Alfa 60 Units/kg
n=9 Participants
Subjects randomized to 60 units/kg
Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months
|
Dose Adjusted
n=2 Participants
Subjects randomized to 30 units/kg but with dose increased
Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months
|
|---|---|---|---|
|
Platelet Count
Baseline
|
70975 platelets/mm^3
Standard Error 8700.6
|
70067 platelets/mm^3
Standard Error 7695.5
|
69500 platelets/mm^3
Standard Error 42500
|
|
Platelet Count
Month 12
|
86613 platelets/mm^3
Standard Error 11894
|
137556 platelets/mm^3
Standard Error 18358
|
88000 platelets/mm^3
Standard Error 65000
|
|
Platelet Count
Month 24
|
103250 platelets/mm^3
Standard Error 17008
|
168778 platelets/mm^3
Standard Error 23717
|
101000 platelets/mm^3
Standard Error 68000
|
|
Platelet Count
Month 36
|
106775 platelets/mm^3
Standard Error 13517
|
155667 platelets/mm^3
Standard Error 16060
|
94500 platelets/mm^3
Standard Error 64500
|
|
Platelet Count
Month 48
|
100643 platelets/mm^3
Standard Error 15314
|
184000 platelets/mm^3
Standard Error 16688
|
103000 platelets/mm^3
Standard Error 53000
|
|
Platelet Count
Month 60
|
104986 platelets/mm^3
Standard Error 18961
|
180625 platelets/mm^3
Standard Error 17062
|
103500 platelets/mm^3
Standard Error 60500
|
SECONDARY outcome
Timeframe: 60 monthsHemoglobin measure yearly
Outcome measures
| Measure |
Taliglucerase Alfa 30 Units/kg
n=8 Participants
Subjects randomized to receive 30 units/kg
Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months
|
Taliglucerase Alfa 60 Units/kg
n=9 Participants
Subjects randomized to 60 units/kg
Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months
|
Dose Adjusted
n=2 Participants
Subjects randomized to 30 units/kg but with dose increased
Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months
|
|---|---|---|---|
|
Hemoglobin
Baseline
|
12.3 mg/dL
Standard Error 0.7
|
11.6 mg/dL
Standard Error 0.9
|
12.5 mg/dL
Standard Error 0.9
|
|
Hemoglobin
Month 12
|
13.9 mg/dL
Standard Error 0.7
|
14.2 mg/dL
Standard Error 0.7
|
14.3 mg/dL
Standard Error 0.9
|
|
Hemoglobin
Month 24
|
13.5 mg/dL
Standard Error 0.8
|
14.1 mg/dL
Standard Error 0.7
|
14.2 mg/dL
Standard Error 0.2
|
|
Hemoglobin
Month 36
|
14.0 mg/dL
Standard Error 0.5
|
14.2 mg/dL
Standard Error 0.8
|
14.4 mg/dL
Standard Error 0.1
|
|
Hemoglobin
Month 48
|
13.7 mg/dL
Standard Error 0.6
|
14.0 mg/dL
Standard Error 0.8
|
14.5 mg/dL
Standard Error 0.1
|
|
Hemoglobin
Month 60
|
14.1 mg/dL
Standard Error 0.9
|
13.9 mg/dL
Standard Error 0.6
|
14.3 mg/dL
Standard Error 0.1
|
Adverse Events
Taliglucerase Alfa 30 Units/kg
Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths
Taliglucerase Alfa 60 Units/kg
Serious events: 2 serious events
Other events: 8 other events
Deaths: 0 deaths
Dose Adjusted
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Taliglucerase Alfa 30 Units/kg
n=8 participants at risk
Subjects randomized to receive 30 units/kg
Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months
|
Taliglucerase Alfa 60 Units/kg
n=9 participants at risk
Subjects randomized to 60 units/kg
Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months
|
Dose Adjusted
n=2 participants at risk
Subjects randomized to 30 units/kg but with dose increased
Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months
|
|---|---|---|---|
|
Musculoskeletal and connective tissue disorders
Osteoarthritis
|
0.00%
0/8 • 21 Months
|
11.1%
1/9 • Number of events 1 • 21 Months
|
0.00%
0/2 • 21 Months
|
|
Metabolism and nutrition disorders
Thyroid Carcinoma
|
0.00%
0/8 • 21 Months
|
11.1%
1/9 • Number of events 1 • 21 Months
|
0.00%
0/2 • 21 Months
|
Other adverse events
| Measure |
Taliglucerase Alfa 30 Units/kg
n=8 participants at risk
Subjects randomized to receive 30 units/kg
Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months
|
Taliglucerase Alfa 60 Units/kg
n=9 participants at risk
Subjects randomized to 60 units/kg
Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months
|
Dose Adjusted
n=2 participants at risk
Subjects randomized to 30 units/kg but with dose increased
Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months
|
|---|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
NASOPHARYNGITIS
|
25.0%
2/8 • Number of events 2 • 21 Months
|
22.2%
2/9 • Number of events 2 • 21 Months
|
0.00%
0/2 • 21 Months
|
|
Respiratory, thoracic and mediastinal disorders
PHARYNGITIS
|
25.0%
2/8 • Number of events 2 • 21 Months
|
0.00%
0/9 • 21 Months
|
0.00%
0/2 • 21 Months
|
|
Musculoskeletal and connective tissue disorders
ARTHRALGIA
|
0.00%
0/8 • 21 Months
|
33.3%
3/9 • Number of events 3 • 21 Months
|
0.00%
0/2 • 21 Months
|
|
Nervous system disorders
HYPOAESTHESIA
|
25.0%
2/8 • Number of events 2 • 21 Months
|
0.00%
0/9 • 21 Months
|
0.00%
0/2 • 21 Months
|
|
Musculoskeletal and connective tissue disorders
BONE PAIN
|
0.00%
0/8 • 21 Months
|
11.1%
1/9 • Number of events 1 • 21 Months
|
50.0%
1/2 • Number of events 1 • 21 Months
|
|
Injury, poisoning and procedural complications
CONTUSION
|
0.00%
0/8 • 21 Months
|
22.2%
2/9 • Number of events 2 • 21 Months
|
0.00%
0/2 • 21 Months
|
|
Gastrointestinal disorders
DYSPEPSIA
|
12.5%
1/8 • Number of events 1 • 21 Months
|
11.1%
1/9 • Number of events 1 • 21 Months
|
0.00%
0/2 • 21 Months
|
|
Gastrointestinal disorders
TOOTHACHE
|
12.5%
1/8 • Number of events 1 • 21 Months
|
11.1%
1/9 • Number of events 1 • 21 Months
|
0.00%
0/2 • 21 Months
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place