Trial Outcomes & Findings for A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI (NCT NCT00299000)
NCT ID: NCT00299000
Last Updated: 2011-07-22
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE4
Target enrollment
4 participants
Primary outcome timeframe
52 weeks
Results posted on
2011-07-22
Participant Flow
Global study sites were hospitals. First Enrollment: 08MAY2006 Last Dose: 30APR2009
Participant milestones
| Measure |
Naglazyme, 1.0 mg/kg
weekly infusions for minimum of 52 weeks
|
Naglazyme, 2.0 mg/kg
weekly infusions for minimum of 52 weeks
|
|---|---|---|
|
Overall Study
STARTED
|
2
|
2
|
|
Overall Study
COMPLETED
|
2
|
2
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI
Baseline characteristics by cohort
| Measure |
Naglazyme, 1.0 mg/kg
n=2 Participants
weekly infusions for minimum of 52 weeks
|
Naglazyme, 2.0 mg/kg
n=2 Participants
weekly infusions for minimum of 52 weeks
|
Total
n=4 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
2 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
4 Participants
n=206 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Age Continuous
|
6.05 months
STANDARD_DEVIATION 3.89 • n=99 Participants
|
12.4 months
STANDARD_DEVIATION 0.42 • n=107 Participants
|
9.23 months
STANDARD_DEVIATION 4.31 • n=206 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
4 Participants
n=206 Participants
|
|
Race/Ethnicity, Customized
White, non-Hispanic
|
1 participants
n=99 Participants
|
1 participants
n=107 Participants
|
2 participants
n=206 Participants
|
|
Race/Ethnicity, Customized
Hispanic/Latino
|
1 participants
n=99 Participants
|
0 participants
n=107 Participants
|
1 participants
n=206 Participants
|
|
Race/Ethnicity, Customized
Other
|
0 participants
n=99 Participants
|
1 participants
n=107 Participants
|
1 participants
n=206 Participants
|
|
Region of Enrollment
United States
|
2 participants
n=99 Participants
|
0 participants
n=107 Participants
|
2 participants
n=206 Participants
|
|
Region of Enrollment
France
|
0 participants
n=99 Participants
|
1 participants
n=107 Participants
|
1 participants
n=206 Participants
|
|
Region of Enrollment
Portugal
|
0 participants
n=99 Participants
|
1 participants
n=107 Participants
|
1 participants
n=206 Participants
|
PRIMARY outcome
Timeframe: 52 weeksPopulation: Intention to treat.
Outcome measures
| Measure |
Naglazyme, 1.0 mg/kg
n=2 Participants
weekly infusions for minimum of 52 weeks
|
Naglazyme, 2.0 mg/kg
n=2 Participants
weekly infusions for minimum of 52 weeks
|
|---|---|---|
|
Change in Height
Height at baseline
|
67.3 centimeters
Standard Deviation 5.1
|
80.3 centimeters
Standard Deviation 1.8
|
|
Change in Height
Height at week 52
|
81.9 centimeters
Standard Deviation 1.8
|
91.3 centimeters
Standard Deviation 5.2
|
|
Change in Height
Change in height
|
14.6 centimeters
Standard Deviation 6.9
|
11.0 centimeters
Standard Deviation 7.0
|
PRIMARY outcome
Timeframe: 52 weeksOutcome measures
| Measure |
Naglazyme, 1.0 mg/kg
n=2 Participants
weekly infusions for minimum of 52 weeks
|
Naglazyme, 2.0 mg/kg
n=2 Participants
weekly infusions for minimum of 52 weeks
|
|---|---|---|
|
Change in Weight
weight at baseline
|
7.1 kilograms
Standard Deviation .3
|
10.2 kilograms
Standard Deviation .6
|
|
Change in Weight
weight at week 52
|
11.3 kilograms
Standard Deviation .3
|
13.3 kilograms
Standard Deviation 1.0
|
|
Change in Weight
change in weight
|
4.2 kilograms
Standard Deviation .6
|
3.1 kilograms
Standard Deviation 1.6
|
PRIMARY outcome
Timeframe: 52 weeksOutcome measures
| Measure |
Naglazyme, 1.0 mg/kg
n=2 Participants
weekly infusions for minimum of 52 weeks
|
Naglazyme, 2.0 mg/kg
n=2 Participants
weekly infusions for minimum of 52 weeks
|
|---|---|---|
|
Change in Haed Circumference
head circumference at baseline
|
43.5 centimeter
Standard Deviation 3.0
|
49.1 centimeter
Standard Deviation .5
|
|
Change in Haed Circumference
head circumference at 52 weeks
|
48.5 centimeter
Standard Deviation 1.0
|
51.8 centimeter
Standard Deviation .8
|
|
Change in Haed Circumference
Change in head circumference
|
5.0 centimeter
Standard Deviation 4.0
|
2.7 centimeter
Standard Deviation 1.3
|
SECONDARY outcome
Timeframe: minimum 52 weeks of dosingPopulation: Intention to treat.
Change in urinary GAG levels was calculated from baseline to week 52 of treatment.
Outcome measures
| Measure |
Naglazyme, 1.0 mg/kg
n=2 Participants
weekly infusions for minimum of 52 weeks
|
Naglazyme, 2.0 mg/kg
n=2 Participants
weekly infusions for minimum of 52 weeks
|
|---|---|---|
|
Change in Urinary Glycosaminoglycan Levels
Baseline
|
1041.87 ug/mg creatinine
Standard Deviation 86.32
|
698.95 ug/mg creatinine
Standard Deviation 142.34
|
|
Change in Urinary Glycosaminoglycan Levels
Week 52
|
261.15 ug/mg creatinine
Standard Deviation 54.38
|
178.10 ug/mg creatinine
Standard Deviation 42.43
|
|
Change in Urinary Glycosaminoglycan Levels
Change from Baseline to Week 52
|
-780.72 ug/mg creatinine
Standard Deviation 140.69
|
-520.85 ug/mg creatinine
Standard Deviation 99.91
|
Adverse Events
Naglazyme, 1.0 mg/kg
Serious events: 2 serious events
Other events: 2 other events
Deaths: 0 deaths
Naglazyme, 2.0 mg/kg
Serious events: 2 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Naglazyme, 1.0 mg/kg
n=2 participants at risk
weekly infusions for minimum of 52 weeks
|
Naglazyme, 2.0 mg/kg
n=2 participants at risk
weekly infusions for minimum of 52 weeks
|
|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
Adenoidal hypertrophy
|
0.00%
0/2 • All patients received weekly infusions of Nagalzyme for a minimum of 52 weeks. The range in time of exposure to Naglazyme for the duration of the study was 52.9 to 153.30 weeks.
|
50.0%
1/2 • Number of events 2 • All patients received weekly infusions of Nagalzyme for a minimum of 52 weeks. The range in time of exposure to Naglazyme for the duration of the study was 52.9 to 153.30 weeks.
|
|
Surgical and medical procedures
Cast application
|
0.00%
0/2 • All patients received weekly infusions of Nagalzyme for a minimum of 52 weeks. The range in time of exposure to Naglazyme for the duration of the study was 52.9 to 153.30 weeks.
|
50.0%
1/2 • Number of events 1 • All patients received weekly infusions of Nagalzyme for a minimum of 52 weeks. The range in time of exposure to Naglazyme for the duration of the study was 52.9 to 153.30 weeks.
|
|
Nervous system disorders
Febrile convulsion
|
0.00%
0/2 • All patients received weekly infusions of Nagalzyme for a minimum of 52 weeks. The range in time of exposure to Naglazyme for the duration of the study was 52.9 to 153.30 weeks.
|
50.0%
1/2 • Number of events 1 • All patients received weekly infusions of Nagalzyme for a minimum of 52 weeks. The range in time of exposure to Naglazyme for the duration of the study was 52.9 to 153.30 weeks.
|
|
Infections and infestations
Pneumonia
|
50.0%
1/2 • Number of events 1 • All patients received weekly infusions of Nagalzyme for a minimum of 52 weeks. The range in time of exposure to Naglazyme for the duration of the study was 52.9 to 153.30 weeks.
|
0.00%
0/2 • All patients received weekly infusions of Nagalzyme for a minimum of 52 weeks. The range in time of exposure to Naglazyme for the duration of the study was 52.9 to 153.30 weeks.
|
|
Vascular disorders
Poor venous access
|
50.0%
1/2 • Number of events 1 • All patients received weekly infusions of Nagalzyme for a minimum of 52 weeks. The range in time of exposure to Naglazyme for the duration of the study was 52.9 to 153.30 weeks.
|
0.00%
0/2 • All patients received weekly infusions of Nagalzyme for a minimum of 52 weeks. The range in time of exposure to Naglazyme for the duration of the study was 52.9 to 153.30 weeks.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory distress
|
50.0%
1/2 • Number of events 1 • All patients received weekly infusions of Nagalzyme for a minimum of 52 weeks. The range in time of exposure to Naglazyme for the duration of the study was 52.9 to 153.30 weeks.
|
0.00%
0/2 • All patients received weekly infusions of Nagalzyme for a minimum of 52 weeks. The range in time of exposure to Naglazyme for the duration of the study was 52.9 to 153.30 weeks.
|
|
Musculoskeletal and connective tissue disorders
Scoliosis
|
0.00%
0/2 • All patients received weekly infusions of Nagalzyme for a minimum of 52 weeks. The range in time of exposure to Naglazyme for the duration of the study was 52.9 to 153.30 weeks.
|
50.0%
1/2 • Number of events 3 • All patients received weekly infusions of Nagalzyme for a minimum of 52 weeks. The range in time of exposure to Naglazyme for the duration of the study was 52.9 to 153.30 weeks.
|
|
Infections and infestations
Tonsillitis
|
0.00%
0/2 • All patients received weekly infusions of Nagalzyme for a minimum of 52 weeks. The range in time of exposure to Naglazyme for the duration of the study was 52.9 to 153.30 weeks.
|
50.0%
1/2 • Number of events 1 • All patients received weekly infusions of Nagalzyme for a minimum of 52 weeks. The range in time of exposure to Naglazyme for the duration of the study was 52.9 to 153.30 weeks.
|
|
Gastrointestinal disorders
Umbilical hernia
|
0.00%
0/2 • All patients received weekly infusions of Nagalzyme for a minimum of 52 weeks. The range in time of exposure to Naglazyme for the duration of the study was 52.9 to 153.30 weeks.
|
50.0%
1/2 • Number of events 1 • All patients received weekly infusions of Nagalzyme for a minimum of 52 weeks. The range in time of exposure to Naglazyme for the duration of the study was 52.9 to 153.30 weeks.
|
Other adverse events
| Measure |
Naglazyme, 1.0 mg/kg
n=2 participants at risk
weekly infusions for minimum of 52 weeks
|
Naglazyme, 2.0 mg/kg
n=2 participants at risk
weekly infusions for minimum of 52 weeks
|
|---|---|---|
|
Gastrointestinal disorders
Diarrhea
|
100.0%
2/2 • Number of events 4 • All patients received weekly infusions of Nagalzyme for a minimum of 52 weeks. The range in time of exposure to Naglazyme for the duration of the study was 52.9 to 153.30 weeks.
|
100.0%
2/2 • Number of events 3 • All patients received weekly infusions of Nagalzyme for a minimum of 52 weeks. The range in time of exposure to Naglazyme for the duration of the study was 52.9 to 153.30 weeks.
|
|
Infections and infestations
Ear infection
|
100.0%
2/2 • Number of events 2 • All patients received weekly infusions of Nagalzyme for a minimum of 52 weeks. The range in time of exposure to Naglazyme for the duration of the study was 52.9 to 153.30 weeks.
|
100.0%
2/2 • Number of events 4 • All patients received weekly infusions of Nagalzyme for a minimum of 52 weeks. The range in time of exposure to Naglazyme for the duration of the study was 52.9 to 153.30 weeks.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
100.0%
2/2 • Number of events 15 • All patients received weekly infusions of Nagalzyme for a minimum of 52 weeks. The range in time of exposure to Naglazyme for the duration of the study was 52.9 to 153.30 weeks.
|
50.0%
1/2 • Number of events 1 • All patients received weekly infusions of Nagalzyme for a minimum of 52 weeks. The range in time of exposure to Naglazyme for the duration of the study was 52.9 to 153.30 weeks.
|
|
General disorders
Pyrexia
|
100.0%
2/2 • Number of events 8 • All patients received weekly infusions of Nagalzyme for a minimum of 52 weeks. The range in time of exposure to Naglazyme for the duration of the study was 52.9 to 153.30 weeks.
|
50.0%
1/2 • Number of events 31 • All patients received weekly infusions of Nagalzyme for a minimum of 52 weeks. The range in time of exposure to Naglazyme for the duration of the study was 52.9 to 153.30 weeks.
|
|
Infections and infestations
Rhinitis
|
50.0%
1/2 • Number of events 3 • All patients received weekly infusions of Nagalzyme for a minimum of 52 weeks. The range in time of exposure to Naglazyme for the duration of the study was 52.9 to 153.30 weeks.
|
100.0%
2/2 • Number of events 3 • All patients received weekly infusions of Nagalzyme for a minimum of 52 weeks. The range in time of exposure to Naglazyme for the duration of the study was 52.9 to 153.30 weeks.
|
|
Gastrointestinal disorders
Umbilical hernia
|
100.0%
2/2 • Number of events 3 • All patients received weekly infusions of Nagalzyme for a minimum of 52 weeks. The range in time of exposure to Naglazyme for the duration of the study was 52.9 to 153.30 weeks.
|
50.0%
1/2 • Number of events 1 • All patients received weekly infusions of Nagalzyme for a minimum of 52 weeks. The range in time of exposure to Naglazyme for the duration of the study was 52.9 to 153.30 weeks.
|
|
Infections and infestations
Upper respiratory tract infections
|
100.0%
2/2 • Number of events 9 • All patients received weekly infusions of Nagalzyme for a minimum of 52 weeks. The range in time of exposure to Naglazyme for the duration of the study was 52.9 to 153.30 weeks.
|
50.0%
1/2 • Number of events 3 • All patients received weekly infusions of Nagalzyme for a minimum of 52 weeks. The range in time of exposure to Naglazyme for the duration of the study was 52.9 to 153.30 weeks.
|
|
Gastrointestinal disorders
Vomiting
|
50.0%
1/2 • Number of events 2 • All patients received weekly infusions of Nagalzyme for a minimum of 52 weeks. The range in time of exposure to Naglazyme for the duration of the study was 52.9 to 153.30 weeks.
|
100.0%
2/2 • Number of events 3 • All patients received weekly infusions of Nagalzyme for a minimum of 52 weeks. The range in time of exposure to Naglazyme for the duration of the study was 52.9 to 153.30 weeks.
|
Additional Information
Medical Information Services
BioMarin Pharmaceutical Inc.
Phone: 800-983-4587
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee The first publication of the results shall be made by the Sponsor in a joint publication. If such a multicenter publication is not submitted within 12 months after conclusion of the study, the PI may publish the results from site individually, subject however to comply with the other terms of the agreement.
- Publication restrictions are in place
Restriction type: OTHER