Trial Outcomes & Findings for Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase (NCT NCT00712348)
NCT ID: NCT00712348
Last Updated: 2018-10-04
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
31 participants
Primary outcome timeframe
Every 3 months from Baseline to Month 9
Results posted on
2018-10-04
Participant Flow
Participant milestones
| Measure |
Taliglucerase Alfa
Open label taliglucerase alfa treatment
Taliglucerase alfa: Intravenous infusion every 2 weeks
|
|---|---|
|
Overall Study
STARTED
|
31
|
|
Overall Study
COMPLETED
|
30
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
Taliglucerase Alfa
Open label taliglucerase alfa treatment
Taliglucerase alfa: Intravenous infusion every 2 weeks
|
|---|---|
|
Overall Study
Withdrawal by Subject
|
1
|
Baseline Characteristics
Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase
Baseline characteristics by cohort
| Measure |
Taliglucerase Alfa
n=31 Participants
Open label taliglucerase alfa treatment
Taliglucerase alfa: Intravenous infusion every 2 weeks
|
|---|---|
|
Age, Categorical
<=18 years
|
5 Participants
n=93 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
25 Participants
n=93 Participants
|
|
Age, Categorical
>=65 years
|
1 Participants
n=93 Participants
|
|
Age, Continuous
|
41.6 years
STANDARD_DEVIATION 17.4 • n=93 Participants
|
|
Sex: Female, Male
Female
|
14 Participants
n=93 Participants
|
|
Sex: Female, Male
Male
|
17 Participants
n=93 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
1 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
White
|
30 Participants
n=93 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
|
Region of Enrollment
United States
|
7 participants
n=93 Participants
|
|
Region of Enrollment
Canada
|
1 participants
n=93 Participants
|
|
Region of Enrollment
Spain
|
4 participants
n=93 Participants
|
|
Region of Enrollment
Australia
|
3 participants
n=93 Participants
|
|
Region of Enrollment
Israel
|
9 participants
n=93 Participants
|
|
Region of Enrollment
United Kingdom
|
2 participants
n=93 Participants
|
|
Region of Enrollment
Serbia
|
4 participants
n=93 Participants
|
|
Region of Enrollment
Singapore
|
1 participants
n=93 Participants
|
|
Religion
JEWISH - ASHKENAZI
|
14 participants
n=93 Participants
|
|
Religion
JEWISH - NONASHKENAZI
|
0 participants
n=93 Participants
|
|
Religion
NON JEWISH
|
15 participants
n=93 Participants
|
|
Religion
UNREPORTED
|
2 participants
n=93 Participants
|
PRIMARY outcome
Timeframe: Every 3 months from Baseline to Month 9Outcome measures
| Measure |
Taliglucerase Alfa
n=30 Participants
Open label taliglucerase alfa treatment
Taliglucerase alfa: Intravenous infusion every 2 weeks
|
|---|---|
|
Hemoglobin
Baseline
|
13.5 g/dL
Standard Deviation 1.4
|
|
Hemoglobin
Month 3
|
13.3 g/dL
Standard Deviation 1.5
|
|
Hemoglobin
Month 6
|
13.3 g/dL
Standard Deviation 1.6
|
|
Hemoglobin
Month 9
|
13.4 g/dL
Standard Deviation 1.5
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Every 3 months from Baseline to Month 9Outcome measures
| Measure |
Taliglucerase Alfa
n=30 Participants
Open label taliglucerase alfa treatment
Taliglucerase alfa: Intravenous infusion every 2 weeks
|
|---|---|
|
Platelet Count
Baseline
|
161137 platelets/mm^3
Standard Deviation 73387
|
|
Platelet Count
Month 3
|
150800 platelets/mm^3
Standard Deviation 66038
|
|
Platelet Count
Month 6
|
157586 platelets/mm^3
Standard Deviation 78357
|
|
Platelet Count
Month 9
|
161167 platelets/mm^3
Standard Deviation 80820
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline and 9 MonthsPopulation: 25 patients had spleen volume measured by MRI, 2 were MRI phobic and 3 were splenectomized
Spleen volume measured by MRI in mL
Outcome measures
| Measure |
Taliglucerase Alfa
n=25 Participants
Open label taliglucerase alfa treatment
Taliglucerase alfa: Intravenous infusion every 2 weeks
|
|---|---|
|
Spleen Volume
Baseline
|
720.5 mL
Standard Deviation 579.5
|
|
Spleen Volume
Month 9
|
654.7 mL
Standard Deviation 535.9
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline and 9 monthsPopulation: 2 patients were MRI phobic
Liver volume measured by MRI
Outcome measures
| Measure |
Taliglucerase Alfa
n=28 Participants
Open label taliglucerase alfa treatment
Taliglucerase alfa: Intravenous infusion every 2 weeks
|
|---|---|
|
Liver Volume
Baseline
|
1766 mL
Standard Deviation 474.5
|
|
Liver Volume
Month 9
|
1716 mL
Standard Deviation 455.6
|
Adverse Events
Taliglucerase Alfa
Serious events: 3 serious events
Other events: 29 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Taliglucerase Alfa
n=31 participants at risk
Open label taliglucerase alfa treatment
Taliglucerase alfa: Intravenous infusion every 2 weeks
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
EPISTAXIS
|
3.2%
1/31 • Number of events 1 • 9 months
|
|
Renal and urinary disorders
NEPHROLITHIASIS
|
3.2%
1/31 • Number of events 1 • 9 months
|
|
Reproductive system and breast disorders
PELVIC PROLAPSE
|
3.2%
1/31 • Number of events 1 • 9 months
|
Other adverse events
| Measure |
Taliglucerase Alfa
n=31 participants at risk
Open label taliglucerase alfa treatment
Taliglucerase alfa: Intravenous infusion every 2 weeks
|
|---|---|
|
Gastrointestinal disorders
DIARRHOEA
|
6.5%
2/31 • Number of events 2 • 9 months
|
|
General disorders
PAIN
|
6.5%
2/31 • Number of events 2 • 9 months
|
|
Infections and infestations
NASOPHARYNGITIS
|
12.9%
4/31 • Number of events 4 • 9 months
|
|
Infections and infestations
UPPER RESPIRATORY TRACT INFECTION
|
9.7%
3/31 • Number of events 3 • 9 months
|
|
Infections and infestations
URINARY TRACT INFECTION
|
9.7%
3/31 • Number of events 3 • 9 months
|
|
Injury, poisoning and procedural complications
INFUSION RELATED REACTION
|
12.9%
4/31 • Number of events 4 • 9 months
|
|
Musculoskeletal and connective tissue disorders
ARTHRALGIA
|
12.9%
4/31 • Number of events 4 • 9 months
|
|
Musculoskeletal and connective tissue disorders
PAIN IN EXTREMITY
|
9.7%
3/31 • Number of events 3 • 9 months
|
|
Nervous system disorders
HEADACHE
|
12.9%
4/31 • Number of events 4 • 9 months
|
|
Respiratory, thoracic and mediastinal disorders
COUGH
|
9.7%
3/31 • Number of events 3 • 9 months
|
|
Skin and subcutaneous tissue disorders
PRURITUS
|
6.5%
2/31 • Number of events 2 • 9 months
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place