Trial Outcomes & Findings for A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease (NCT NCT01411228)
NCT ID: NCT01411228
Last Updated: 2018-09-07
Results Overview
Median and interquartile range. Baseline is the value obtained from the parent study: PB-06-005 for 30 and 60 Units/kg arms and PB-06-002 from Switchover arm.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
15 participants
Primary outcome timeframe
Baseline, months 9, 12 and 24
Results posted on
2018-09-07
Participant Flow
Participant milestones
| Measure |
60 Units/kg
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months
|
30 Units/kg
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months
|
Switchover
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months
|
|---|---|---|---|
|
Overall Study
STARTED
|
5
|
5
|
5
|
|
Overall Study
COMPLETED
|
4
|
5
|
4
|
|
Overall Study
NOT COMPLETED
|
1
|
0
|
1
|
Reasons for withdrawal
| Measure |
60 Units/kg
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months
|
30 Units/kg
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months
|
Switchover
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months
|
|---|---|---|---|
|
Overall Study
Lost to Follow-up
|
0
|
0
|
1
|
|
Overall Study
Withdrawal by Subject
|
1
|
0
|
0
|
Baseline Characteristics
A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
Baseline characteristics by cohort
| Measure |
60 Units/kg
n=5 Participants
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months
|
30 Units/kg
n=5 Participants
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months
|
Dose Adjusted
n=5 Participants
Taliglucerase alfa: Dose increased from 30 Units/kg to 45 or 60 Units/kg
|
Total
n=15 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
6.6 years
STANDARD_DEVIATION 3.1 • n=5 Participants
|
8.6 years
STANDARD_DEVIATION 3.8 • n=7 Participants
|
13.0 years
STANDARD_DEVIATION 4.1 • n=5 Participants
|
9.4 years
STANDARD_DEVIATION 4.4 • n=4 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
5 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
10 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
|
1 Participants
n=5 Participants
|
1 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
|
4 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
13 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
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Region of Enrollment
South Africa
|
2 participants
n=5 Participants
|
1 participants
n=7 Participants
|
0 participants
n=5 Participants
|
3 participants
n=4 Participants
|
|
Region of Enrollment
Israel
|
2 participants
n=5 Participants
|
1 participants
n=7 Participants
|
0 participants
n=5 Participants
|
3 participants
n=4 Participants
|
|
Region of Enrollment
Spain
|
0 participants
n=5 Participants
|
0 participants
n=7 Participants
|
2 participants
n=5 Participants
|
2 participants
n=4 Participants
|
|
Region of Enrollment
Serbia
|
0 participants
n=5 Participants
|
0 participants
n=7 Participants
|
2 participants
n=5 Participants
|
2 participants
n=4 Participants
|
|
Region of Enrollment
Paraguay
|
1 participants
n=5 Participants
|
3 participants
n=7 Participants
|
0 participants
n=5 Participants
|
4 participants
n=4 Participants
|
|
Region of Enrollment
Singapore
|
0 participants
n=5 Participants
|
0 participants
n=7 Participants
|
1 participants
n=5 Participants
|
1 participants
n=4 Participants
|
PRIMARY outcome
Timeframe: Baseline, months 9, 12 and 24Population: Two Switchover Patients completed 18 months treatment due to early closure of the study site and continued in a compassionate use program
Median and interquartile range. Baseline is the value obtained from the parent study: PB-06-005 for 30 and 60 Units/kg arms and PB-06-002 from Switchover arm.
Outcome measures
| Measure |
60 Units/kg
n=5 Participants
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months
|
30 Units/kg
n=5 Participants
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months
|
Switchover
n=5 Participants
Taliglucerase alfa: Maintained dose from PB-06-002 study
|
|---|---|---|---|
|
Hemoglobin
Baseline
|
10.6 g/dL
Interval 9.1 to 12.3
|
11.2 g/dL
Interval 9.1 to 12.3
|
13.5 g/dL
Interval 12.9 to 14.2
|
|
Hemoglobin
Month 9
|
11.9 g/dL
Interval 10.5 to 13.8
|
12.5 g/dL
Interval 10.1 to 13.7
|
13.9 g/dL
Interval 12.5 to 15.2
|
|
Hemoglobin
Month 12
|
12.2 g/dL
Interval 11.2 to 13.7
|
12.8 g/dL
Interval 11.2 to 14.2
|
13.7 g/dL
Interval 12.0 to 15.1
|
|
Hemoglobin
Month 24
|
12.8 g/dL
Interval 11.0 to 14.2
|
13.1 g/dL
Interval 11.5 to 14.5
|
14.2 g/dL
Interval 12.1 to 16.0
|
SECONDARY outcome
Timeframe: Baseline, months 9, 12 and 24Population: Chitotriosidase was not analyzed for the subjects in the Switchover group
Chitotriosidase. Baseline is the value obtained from the parent study: PB-06-005 for 30 and 60 Units/kg arms and PB-06-002 from Switchover arm.
Outcome measures
| Measure |
60 Units/kg
n=5 Participants
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months
|
30 Units/kg
n=4 Participants
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months
|
Switchover
Taliglucerase alfa: Maintained dose from PB-06-002 study
|
|---|---|---|---|
|
Chitotriosidase
Baseline
|
34968 nmol/mL*h
Standard Deviation 20928
|
30783 nmol/mL*h
Standard Deviation 17771
|
—
|
|
Chitotriosidase
Month 9
|
17416 nmol/mL*h
Standard Deviation 16821
|
13983 nmol/mL*h
Standard Deviation 10562
|
—
|
|
Chitotriosidase
Month 12
|
14677 nmol/mL*h
Standard Deviation 14418
|
13823 nmol/mL*h
Standard Deviation 11525
|
—
|
|
Chitotriosidase
Month 24
|
7449.5 nmol/mL*h
Standard Deviation 6966.5
|
10079 nmol/mL*h
Standard Deviation 9452.4
|
—
|
SECONDARY outcome
Timeframe: Baseline, months 12 and 24Population: Two Switchover Patients completed 18 months treatment due to early closure of the study site and continued in a compassionate use program
Spleen volume measured by MRI (or ultrasound). Baseline is the value obtained from the parent study: PB-06-005 for 30 and 60 Units/kg arms and PB-06-002 from Switchover arm.
Outcome measures
| Measure |
60 Units/kg
n=5 Participants
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months
|
30 Units/kg
n=5 Participants
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months
|
Switchover
n=5 Participants
Taliglucerase alfa: Maintained dose from PB-06-002 study
|
|---|---|---|---|
|
Spleen Volume
Baseline
|
1023.1 Milliliters
Standard Error 336.9
|
1244.7 Milliliters
Standard Error 317.6
|
313.0 Milliliters
Standard Error 69.8
|
|
Spleen Volume
12 Months
|
524.0 Milliliters
Standard Error 125.7
|
759.0 Milliliters
Standard Error 194.4
|
274.1 Milliliters
Standard Error 39.3
|
|
Spleen Volume
24 Months
|
395.8 Milliliters
Standard Error 76.8
|
719.0 Milliliters
Standard Error 161.2
|
249.5 Milliliters
Standard Error 69.8
|
|
Spleen Volume
33-36 Months
|
306.1 Milliliters
Standard Error 55.1
|
673.2 Milliliters
Standard Error 128.7
|
185.4 Milliliters
Standard Error 12.6
|
SECONDARY outcome
Timeframe: Baseline, months 9, 12, 24 and 33-36Population: Two Switchover Patients completed 18 months treatment due to early closure of the study site and continued in a compassionate use program
Platelet count. Baseline is the value obtained from the parent study: PB-06-005 for 30 and 60 Units/kg arms and PB-06-002 from Switchover arm.
Outcome measures
| Measure |
60 Units/kg
n=5 Participants
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months
|
30 Units/kg
n=5 Participants
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months
|
Switchover
n=5 Participants
Taliglucerase alfa: Maintained dose from PB-06-002 study
|
|---|---|---|---|
|
Platelet Count
Baseline
|
99600 Platelets/mm^3
Standard Error 19185
|
182000 Platelets/mm^3
Standard Error 27009
|
164587 Platelets/mm^3
Standard Error 17321
|
|
Platelet Count
Month 9
|
172600 Platelets/mm^3
Standard Error 38667
|
221400 Platelets/mm^3
Standard Error 30626
|
177400 Platelets/mm^3
Standard Error 16795
|
|
Platelet Count
Month 12
|
172200 Platelets/mm^3
Standard Error 39932
|
233800 Platelets/mm^3
Standard Error 32759
|
189000 Platelets/mm^3
Standard Error 7733.0
|
|
Platelet Count
Month 24
|
192800 Platelets/mm^3
Standard Error 26673
|
207600 Platelets/mm^3
Standard Error 18291
|
234800 Platelets/mm^3
Standard Error 14867
|
|
Platelet Count
Month 33-36
|
243750 Platelets/mm^3
Standard Error 18585
|
220200 Platelets/mm^3
Standard Error 29231
|
210000 Platelets/mm^3
Standard Error 10000
|
SECONDARY outcome
Timeframe: Baseline, months 12 and 24Population: Two Switchover Patients completed 18 months treatment due to early closure of the study site and continued in a compassionate use program
Liver volume by MRI or Ultrasound. Baseline is the value obtained from the parent study: PB-06-005 for 30 and 60 Units/kg arms and PB-06-002 from Switchover arm.
Outcome measures
| Measure |
60 Units/kg
n=5 Participants
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months
|
30 Units/kg
n=5 Participants
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months
|
Switchover
n=5 Participants
Taliglucerase alfa: Maintained dose from PB-06-002 study
|
|---|---|---|---|
|
Liver Volume
Baseline
|
991.7 Milliliters
Standard Error 134.7
|
1236.5 Milliliters
Standard Error 210.6
|
1345.6 Milliliters
Standard Error 193.1
|
|
Liver Volume
Month 12
|
849.1 Milliliters
Standard Error 121.6
|
1140.6 Milliliters
Standard Error 180.9
|
1460.7 Milliliters
Standard Error 227.8
|
|
Liver Volume
Month 24
|
865.5 Milliliters
Standard Error 82.4
|
1254.4 Milliliters
Standard Error 114.9
|
1480.7 Milliliters
Standard Error 253.3
|
|
Liver Volume
Month 33-36
|
890.6 Milliliters
Standard Error 118.2
|
1244.8 Milliliters
Standard Error 89.1
|
1274.2 Milliliters
Standard Error 189.7
|
Adverse Events
60 Units/kg
Serious events: 1 serious events
Other events: 5 other events
Deaths: 0 deaths
30 Units/kg
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Switchover
Serious events: 0 serious events
Other events: 5 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
60 Units/kg
n=5 participants at risk
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months
|
30 Units/kg
n=5 participants at risk
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months
|
Switchover
n=5 participants at risk
Taliglucerase alfa: Maintained dose from PB-06-002 study
|
|---|---|---|---|
|
Infections and infestations
Dengue Fever
|
20.0%
1/5 • Number of events 1
|
0.00%
0/5
|
0.00%
0/5
|
Other adverse events
| Measure |
60 Units/kg
n=5 participants at risk
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months
|
30 Units/kg
n=5 participants at risk
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months
|
Switchover
n=5 participants at risk
Taliglucerase alfa: Maintained dose from PB-06-002 study
|
|---|---|---|---|
|
Blood and lymphatic system disorders
LYMPHADENOPATHY
|
20.0%
1/5 • Number of events 1
|
40.0%
2/5 • Number of events 2
|
0.00%
0/5
|
|
Ear and labyrinth disorders
EAR PAIN
|
40.0%
2/5 • Number of events 2
|
0.00%
0/5
|
0.00%
0/5
|
|
Gastrointestinal disorders
ABDOMINAL PAIN
|
20.0%
1/5 • Number of events 1
|
40.0%
2/5 • Number of events 2
|
0.00%
0/5
|
|
Gastrointestinal disorders
DIARRHOEA
|
60.0%
3/5 • Number of events 3
|
0.00%
0/5
|
0.00%
0/5
|
|
Infections and infestations
DENGUE FEVER
|
0.00%
0/5
|
40.0%
2/5 • Number of events 2
|
0.00%
0/5
|
|
Infections and infestations
UPPER RESPIRATORY TRACT INFECTION
|
40.0%
2/5 • Number of events 2
|
20.0%
1/5 • Number of events 1
|
20.0%
1/5 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
PAIN IN EXTREMITY
|
40.0%
2/5 • Number of events 2
|
20.0%
1/5 • Number of events 1
|
0.00%
0/5
|
|
Infections and infestations
NASOPHARYNGITIS
|
20.0%
1/5 • Number of events 1
|
0.00%
0/5
|
40.0%
2/5 • Number of events 2
|
|
Nervous system disorders
HEADACHE
|
40.0%
2/5 • Number of events 2
|
20.0%
1/5 • Number of events 1
|
20.0%
1/5 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
COUGH
|
40.0%
2/5 • Number of events 2
|
20.0%
1/5 • Number of events 1
|
20.0%
1/5 • Number of events 1
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place