Trial Outcomes & Findings for Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease (NCT NCT00258011)
NCT ID: NCT00258011
Last Updated: 2014-03-07
Results Overview
Overall Safety Summary of Adverse Events (AEs) during Treatment Safety assessment was based on the incidence of AE reports.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
3 participants
Primary outcome timeframe
Up to 73 Weeks
Results posted on
2014-03-07
Participant Flow
Participant milestones
| Measure |
Aldurazyme (Laronidase) Treatment
Patients received weekly infusions of Aldurazyme (laronidase) at an intravenous dose of 100 Units/kg (0.58 mg/kg) body weight (labeled dose) for up to 73 weeks.
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|---|---|
|
Overall Study
STARTED
|
3
|
|
Overall Study
COMPLETED
|
3
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease
Baseline characteristics by cohort
| Measure |
Aldurazyme (Laronidase) Treatment
n=3 Participants
Patients received weekly infusions of Aldurazyme (laronidase) at an intravenous dose of 100 Units/kg (0.58 mg/kg) body weight (labeled dose) for up to 73 weeks.
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|---|---|
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Age, Continuous
|
12.2 years
STANDARD_DEVIATION 12.57 • n=93 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=93 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=93 Participants
|
|
Race/Ethnicity, Customized
Asian (Japanese)
|
3 participants
n=93 Participants
|
PRIMARY outcome
Timeframe: Up to 73 WeeksPopulation: The study enrolled eligible MPS I patients prior to marketing authorization in Japan. The analysis was intention to treat.
Overall Safety Summary of Adverse Events (AEs) during Treatment Safety assessment was based on the incidence of AE reports.
Outcome measures
| Measure |
Aldurazyme (Laronidase) Treatment
n=3 Participants
Patients received weekly infusions of Aldurazyme (laronidase) at an intravenous dose of 100 Units/kg (0.58 mg/kg) body weight (labeled dose) for up to 73 weeks.
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|---|---|
|
Safety Evaluation
Having Adverse Events (AEs)
|
3 participants
Interval -81.1 to -60.6
|
|
Safety Evaluation
Having drug related AEs
|
0 participants
|
|
Safety Evaluation
Discontinuations due to AEs
|
0 participants
|
|
Safety Evaluation
Having Serious AEs
|
1 participants
|
|
Safety Evaluation
Having Severe AEs
|
1 participants
|
|
Safety Evaluation
Deaths
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0 participants
|
|
Safety Evaluation
Having infusion-associated reactions
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0 participants
|
SECONDARY outcome
Timeframe: Up to 73 WeeksPopulation: The study enrolled eligible MPS I patients prior to marketing authorization in Japan. The analysis was intention to treat.\> \* 1 patient final visit = Week 73; 1 patient final visit = Week 32; 1 patient final visit = Week 10
Percentage change in the concentration of GAG relative to creatinine in urine (ug GAG/mg creatinine) from baseline to last study visit. Greater decrease indicates greater response.
Outcome measures
| Measure |
Aldurazyme (Laronidase) Treatment
n=3 Participants
Patients received weekly infusions of Aldurazyme (laronidase) at an intravenous dose of 100 Units/kg (0.58 mg/kg) body weight (labeled dose) for up to 73 weeks.
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|---|---|
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Urinary Glycosaminoglycan (GAG) Excretion
Week 46 (1 patients analyzed)
|
-67.9 percent change in concentration of GAG
Standard Deviation 0
|
|
Urinary Glycosaminoglycan (GAG) Excretion
Baseline (3 patients analyzed)
|
0 percent change in concentration of GAG
Standard Deviation 0
|
|
Urinary Glycosaminoglycan (GAG) Excretion
Week 4 (3 patients analyzed)
|
-68.0 percent change in concentration of GAG
Standard Deviation 12.76
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|
Urinary Glycosaminoglycan (GAG) Excretion
Week 8 (3 patients analyzed)
|
-69.9 percent change in concentration of GAG
Standard Deviation 7.98
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|
Urinary Glycosaminoglycan (GAG) Excretion
Week 12 (2 patients analyzed)
|
-74.6 percent change in concentration of GAG
Standard Deviation 11.47
|
|
Urinary Glycosaminoglycan (GAG) Excretion
Week 16 (2 patients analyzed)
|
-71.0 percent change in concentration of GAG
Standard Deviation 9.40
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|
Urinary Glycosaminoglycan (GAG) Excretion
Week 20 (2 patients analyzed)
|
-60.6 percent change in concentration of GAG
Standard Deviation 12.85
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|
Urinary Glycosaminoglycan (GAG) Excretion
Week 26 (2 patients analyzed)
|
-71.5 percent change in concentration of GAG
Standard Deviation 5.84
|
|
Urinary Glycosaminoglycan (GAG) Excretion
Week 30 (2 patients analyzed)
|
-75.2 percent change in concentration of GAG
Standard Deviation 10.65
|
|
Urinary Glycosaminoglycan (GAG) Excretion
Week 34 (1 patients analyzed)
|
-77.7 percent change in concentration of GAG
Standard Deviation 0
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|
Urinary Glycosaminoglycan (GAG) Excretion
Week 38 (1 patients analyzed)
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-69.9 percent change in concentration of GAG
Standard Deviation 0
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Urinary Glycosaminoglycan (GAG) Excretion
Week 42 (1 patients analyzed)
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-78.0 percent change in concentration of GAG
Standard Deviation 0
|
|
Urinary Glycosaminoglycan (GAG) Excretion
Week 52 (1 patients analyzed)
|
-77.0 percent change in concentration of GAG
Standard Deviation 0
|
|
Urinary Glycosaminoglycan (GAG) Excretion
Week 64 (1 patients analyzed)
|
-81.1 percent change in concentration of GAG
Standard Deviation 0
|
|
Urinary Glycosaminoglycan (GAG) Excretion
Final Examination * (3 patients analyzed)
|
-69.7 percent change in concentration of GAG
Standard Deviation 20.23
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Adverse Events
Aldurazyme
Serious events: 1 serious events
Other events: 3 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Aldurazyme
n=3 participants at risk
Aldurazyme
|
|---|---|
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Infections and infestations
Bronchitis
|
33.3%
1/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Infections and infestations
Catheter site infection
|
33.3%
1/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Infections and infestations
Respiratory syncytial virus infection
|
33.3%
1/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Infections and infestations
Staphylococcal sepsis
|
33.3%
1/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Respiratory, thoracic and mediastinal disorders
Sleep apnoea syndrome
|
33.3%
1/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Respiratory, thoracic and mediastinal disorders
Tonsillar disorder
|
33.3%
1/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
Other adverse events
| Measure |
Aldurazyme
n=3 participants at risk
Aldurazyme
|
|---|---|
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Eye disorders
Conjunctivitis
|
33.3%
1/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Eye disorders
Ocular hyperaemia
|
33.3%
1/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Gastrointestinal disorders
Diarrhoea
|
33.3%
1/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
General disorders
Catheter related complication
|
33.3%
1/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
General disorders
Catheter site erythema
|
33.3%
1/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
General disorders
Catheter site pruritus
|
33.3%
1/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
General disorders
Pyrexia
|
33.3%
1/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Infections and infestations
Bronchitis
|
33.3%
1/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Infections and infestations
Catheter site infection
|
33.3%
1/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Infections and infestations
Gastroenteritis
|
33.3%
1/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Infections and infestations
Hordeolum
|
33.3%
1/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Infections and infestations
Influenza
|
33.3%
1/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Infections and infestations
Nasopharyngitis
|
100.0%
3/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Infections and infestations
Otitis media
|
66.7%
2/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Infections and infestations
Pharyngitis
|
33.3%
1/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Infections and infestations
Rhinitis
|
33.3%
1/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Infections and infestations
Varicella
|
33.3%
1/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Injury, poisoning and procedural complications
Arthropod bite
|
33.3%
1/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Injury, poisoning and procedural complications
Excoriation
|
33.3%
1/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Injury, poisoning and procedural complications
Head injury
|
33.3%
1/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Investigations
Oxygen saturation decreased
|
66.7%
2/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
33.3%
1/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
33.3%
1/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Musculoskeletal and connective tissue disorders
Monarthritis
|
33.3%
1/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Psychiatric disorders
Insomnia
|
33.3%
1/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
33.3%
1/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Respiratory, thoracic and mediastinal disorders
Haemoptysis
|
33.3%
1/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Respiratory, thoracic and mediastinal disorders
Pharyngeal erythema
|
33.3%
1/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Respiratory, thoracic and mediastinal disorders
Upper respiratory tract inflammation
|
33.3%
1/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Skin and subcutaneous tissue disorders
Dermatitis diaper
|
33.3%
1/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
33.3%
1/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Skin and subcutaneous tissue disorders
Eczema
|
33.3%
1/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Skin and subcutaneous tissue disorders
Heat rash
|
66.7%
2/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Skin and subcutaneous tissue disorders
Hyperkeratosis
|
33.3%
1/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Skin and subcutaneous tissue disorders
Rash
|
33.3%
1/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
33.3%
1/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Vascular disorders
Pallor
|
33.3%
1/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
|
Vascular disorders
Peripheral vascular disorder
|
33.3%
1/3
In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
|
Additional Information
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