Trial Outcomes & Findings for A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease (NCT NCT01132690)
NCT ID: NCT01132690
Last Updated: 2018-10-05
Results Overview
median and interquartile range for change from baseline in haemoglobin
Recruitment status
COMPLETED
Study phase
PHASE4
Target enrollment
11 participants
Primary outcome timeframe
Every 3 months for 12 months
Results posted on
2018-10-05
Participant Flow
Participant milestones
| Measure |
30 Units/kg
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months
|
60 Units/kg
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months
|
|---|---|---|
|
Overall Study
STARTED
|
6
|
5
|
|
Overall Study
COMPLETED
|
6
|
5
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
Baseline characteristics by cohort
| Measure |
30 Units/kg
n=6 Participants
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months
|
60 Units/kg
n=5 Participants
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months
|
Total
n=11 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
9.5 years
STANDARD_DEVIATION 4.0 • n=5 Participants
|
6.6 years
STANDARD_DEVIATION 3.1 • n=7 Participants
|
8.2 years
STANDARD_DEVIATION 3.8 • n=5 Participants
|
|
Age, Categorical
<=18 years
|
6 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
11 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 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
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
6 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
3 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
3 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
Paraguay
|
3 participants
n=5 Participants
|
1 participants
n=7 Participants
|
4 participants
n=5 Participants
|
|
Region of Enrollment
South Africa
|
1 participants
n=5 Participants
|
2 participants
n=7 Participants
|
3 participants
n=5 Participants
|
|
Region of Enrollment
Israel
|
2 participants
n=5 Participants
|
2 participants
n=7 Participants
|
4 participants
n=5 Participants
|
|
Religion
Jewish - Ashkenazi
|
0 participants
n=5 Participants
|
2 participants
n=7 Participants
|
2 participants
n=5 Participants
|
|
Religion
Jewish - Non-Ashkenazi
|
0 participants
n=5 Participants
|
0 participants
n=7 Participants
|
0 participants
n=5 Participants
|
|
Religion
Non Jewish
|
6 participants
n=5 Participants
|
3 participants
n=7 Participants
|
9 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Every 3 months for 12 monthsmedian and interquartile range for change from baseline in haemoglobin
Outcome measures
| Measure |
30 Units/kg
n=6 Participants
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months
|
60 Units/kg
n=5 Participants
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months
|
|---|---|---|
|
Hemoglobin
Month 3
|
1.2 g/dL
Interval -0.3 to 1.6
|
0.4 g/dL
Interval -0.1 to 1.1
|
|
Hemoglobin
Month 6
|
1.1 g/dL
Interval 0.3 to 2.3
|
1.5 g/dL
Interval 1.0 to 1.7
|
|
Hemoglobin
Month 9
|
0.9 g/dL
Interval 0.5 to 1.9
|
1.5 g/dL
Interval 1.4 to 1.6
|
|
Hemoglobin
Month 12
|
1.4 g/dL
Interval 0.2 to 2.5
|
1.6 g/dL
Interval 1.4 to 2.0
|
SECONDARY outcome
Timeframe: Every 3 months for 12 monthsPercent change from baseline in chitotriosidase
Outcome measures
| Measure |
30 Units/kg
n=6 Participants
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months
|
60 Units/kg
n=5 Participants
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months
|
|---|---|---|
|
Chitotriosidase
Month 3
|
-32.4 Percent Change from Baseline
Standard Deviation 23.5
|
-32.7 Percent Change from Baseline
Standard Deviation 15.1
|
|
Chitotriosidase
Month 6
|
-49.6 Percent Change from Baseline
Standard Deviation 19.8
|
-50.2 Percent Change from Baseline
Standard Deviation 23.7
|
|
Chitotriosidase
Month 9
|
-57.2 Percent Change from Baseline
Standard Deviation 18.7
|
-58.9 Percent Change from Baseline
Standard Deviation 21.8
|
|
Chitotriosidase
Month 12
|
-58.5 Percent Change from Baseline
Standard Deviation 17.5
|
-66.1 Percent Change from Baseline
Standard Deviation 20.1
|
SECONDARY outcome
Timeframe: Baseline and Month 12Spleen volume measured by MRI
Outcome measures
| Measure |
30 Units/kg
n=6 Participants
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months
|
60 Units/kg
n=5 Participants
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months
|
|---|---|---|
|
Spleen Volume
Baseline
|
1218 mL
Standard Deviation 638.4
|
1023 mL
Standard Deviation 753.4
|
|
Spleen Volume
Month 12
|
811.6 mL
Standard Deviation 409.6
|
524.0 mL
Standard Deviation 281.1
|
SECONDARY outcome
Timeframe: Baseline and 12 monthsMean and standard deviation of platelet count per cubic mm
Outcome measures
| Measure |
30 Units/kg
n=6 Participants
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months
|
60 Units/kg
n=5 Participants
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months
|
|---|---|---|
|
Platelet Count
Baseline
|
162667 platelets per cubic mm
Standard Deviation 71838
|
99600 platelets per cubic mm
Standard Deviation 42899
|
|
Platelet Count
Month 12
|
208167 platelets per cubic mm
Standard Deviation 90747
|
172200 platelets per cubic mm
Standard Deviation 89290
|
SECONDARY outcome
Timeframe: Every 3 months for 12 monthsPercent change from baseline in CCL18
Outcome measures
| Measure |
30 Units/kg
n=6 Participants
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months
|
60 Units/kg
n=5 Participants
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months
|
|---|---|---|
|
Chemokine (C-C Motif) Ligand 18 (CCL18)
Month 3
|
-31.4 Percent Change from Baseline
Standard Deviation 15.3
|
-18.3 Percent Change from Baseline
Standard Deviation 16.3
|
|
Chemokine (C-C Motif) Ligand 18 (CCL18)
Month 6
|
-45.7 Percent Change from Baseline
Standard Deviation 11.9
|
-37.3 Percent Change from Baseline
Standard Deviation 28.8
|
|
Chemokine (C-C Motif) Ligand 18 (CCL18)
Month 9
|
-49.9 Percent Change from Baseline
Standard Deviation 13.0
|
-46.5 Percent Change from Baseline
Standard Deviation 22.4
|
|
Chemokine (C-C Motif) Ligand 18 (CCL18)
Month 12
|
-50.6 Percent Change from Baseline
Standard Deviation 19.4
|
-52.6 Percent Change from Baseline
Standard Deviation 22.5
|
SECONDARY outcome
Timeframe: Baseline and Month 12Liver volume measured by MRI
Outcome measures
| Measure |
30 Units/kg
n=6 Participants
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months
|
60 Units/kg
n=5 Participants
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months
|
|---|---|---|
|
Liver Volume
Baseline
|
1214 mL
Standard Deviation 424.7
|
991.7 mL
Standard Deviation 301.3
|
|
Liver Volume
Month 12
|
1116 mL
Standard Deviation 366.9
|
849.1 mL
Standard Deviation 271.9
|
Adverse Events
30 Units/kg
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
60 Units/kg
Serious events: 1 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
30 Units/kg
n=6 participants at risk
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months
|
60 Units/kg
n=5 participants at risk
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months
|
|---|---|---|
|
Gastrointestinal disorders
GASTROINTESTINAL INFLAMMATION
|
0.00%
0/6 • 1 year
|
20.0%
1/5 • Number of events 1 • 1 year
|
Other adverse events
| Measure |
30 Units/kg
n=6 participants at risk
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months
|
60 Units/kg
n=5 participants at risk
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months
|
|---|---|---|
|
Gastrointestinal disorders
VOMITING
|
33.3%
2/6 • Number of events 2 • 1 year
|
40.0%
2/5 • Number of events 2 • 1 year
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place