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Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease

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ClinicalTrials.gov Identifier: NCT00258011
Recruitment Status : Completed
First Posted : November 24, 2005
Results First Posted : February 12, 2009
Last Update Posted : March 7, 2014
Sponsor:
Collaborator:
BioMarin/Genzyme LLC
Information provided by:
Sanofi

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Mucopolysaccharidosis I
Hurler Syndrome
Hurler-Scheie Syndrome
Scheie Syndrome
Intervention Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
Enrollment 3
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Aldurazyme (Laronidase) Treatment
Hide Arm/Group Description 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.
Period Title: Overall Study
Started 3
Completed 3
Not Completed 0
Arm/Group Title Aldurazyme (Laronidase) Treatment
Hide Arm/Group Description 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.
Overall Number of Baseline Participants 3
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 3 participants
12.2  (12.57)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants
Female
3
 100.0%
Male
0
   0.0%
Race/Ethnicity, Customized  
Measure Type: Number
Unit of measure:  Participants
Asian (Japanese) Number Analyzed 3 participants
3
1.Primary Outcome
Title Safety Evaluation
Hide Description Overall Safety Summary of Adverse Events (AEs) during Treatment Safety assessment was based on the incidence of AE reports.
Time Frame Up to 73 Weeks
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
The study enrolled eligible MPS I patients prior to marketing authorization in Japan. The analysis was intention to treat.
Arm/Group Title Aldurazyme (Laronidase) Treatment
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 3
Measure Type: Number
Unit of Measure: participants
Having Adverse Events (AEs) 3
Having drug related AEs 0
Discontinuations due to AEs 0
Having Serious AEs 1
Having Severe AEs 1
Deaths 0
Having infusion-associated reactions 0
2.Secondary Outcome
Title Urinary Glycosaminoglycan (GAG) Excretion
Hide Description 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.
Time Frame Up to 73 Weeks
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description

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

Arm/Group Title Aldurazyme (Laronidase) Treatment
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 3
Mean (Standard Deviation)
Unit of Measure: percent change in concentration of GAG
Baseline (3 patients analyzed) 0  (0)
Week 4 (3 patients analyzed) -68.0  (12.76)
Week 8 (3 patients analyzed) -69.9  (7.98)
Week 12 (2 patients analyzed) -74.6  (11.47)
Week 16 (2 patients analyzed) -71.0  (9.40)
Week 20 (2 patients analyzed) -60.6  (12.85)
Week 26 (2 patients analyzed) -71.5  (5.84)
Week 30 (2 patients analyzed) -75.2  (10.65)
Week 34 (1 patients analyzed) -77.7  (0)
Week 38 (1 patients analyzed) -69.9  (0)
Week 42 (1 patients analyzed) -78.0  (0)
Week 46 (1 patients analyzed) -67.9  (0)
Week 52 (1 patients analyzed) -77.0  (0)
Week 64 (1 patients analyzed) -81.1  (0)
Final Examination * (3 patients analyzed) -69.7  (20.23)
Time Frame [Not Specified]
Adverse Event Reporting Description 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.
 
Arm/Group Title Aldurazyme
Hide Arm/Group Description Aldurazyme
All-Cause Mortality
Aldurazyme
Affected / at Risk (%)
Total   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Aldurazyme
Affected / at Risk (%)
Total   1/3 (33.33%) 
Infections and infestations   
Bronchitis  1  1/3 (33.33%) 
Catheter site infection  1  1/3 (33.33%) 
Respiratory syncytial virus infection  1  1/3 (33.33%) 
Staphylococcal sepsis  1  1/3 (33.33%) 
Respiratory, thoracic and mediastinal disorders   
Sleep apnoea syndrome  1  1/3 (33.33%) 
Tonsillar disorder  1  1/3 (33.33%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MEDDRA 8.1
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Aldurazyme
Affected / at Risk (%)
Total   3/3 (100.00%) 
Eye disorders   
Conjunctivitis  1  1/3 (33.33%) 
Ocular hyperaemia  1  1/3 (33.33%) 
Gastrointestinal disorders   
Diarrhoea  1  1/3 (33.33%) 
General disorders   
Catheter related complication  1  1/3 (33.33%) 
Catheter site erythema  1  1/3 (33.33%) 
Catheter site pruritus  1  1/3 (33.33%) 
Pyrexia  1  1/3 (33.33%) 
Infections and infestations   
Bronchitis  1  1/3 (33.33%) 
Catheter site infection  1  1/3 (33.33%) 
Gastroenteritis  1  1/3 (33.33%) 
Hordeolum  1  1/3 (33.33%) 
Influenza  1  1/3 (33.33%) 
Nasopharyngitis  1  3/3 (100.00%) 
Otitis media  1  2/3 (66.67%) 
Pharyngitis  1  1/3 (33.33%) 
Rhinitis  1  1/3 (33.33%) 
Varicella  1  1/3 (33.33%) 
Injury, poisoning and procedural complications   
Arthropod bite  1  1/3 (33.33%) 
Excoriation  1  1/3 (33.33%) 
Head injury  1  1/3 (33.33%) 
Investigations   
Oxygen saturation decreased  1  2/3 (66.67%) 
Musculoskeletal and connective tissue disorders   
Arthralgia  1  1/3 (33.33%) 
Back pain  1  1/3 (33.33%) 
Monarthritis  1  1/3 (33.33%) 
Psychiatric disorders   
Insomnia  1  1/3 (33.33%) 
Respiratory, thoracic and mediastinal disorders   
Asthma  1  1/3 (33.33%) 
Haemoptysis  1  1/3 (33.33%) 
Pharyngeal erythema  1  1/3 (33.33%) 
Upper respiratory tract inflammation  1  1/3 (33.33%) 
Skin and subcutaneous tissue disorders   
Dermatitis diaper  1  1/3 (33.33%) 
Dry skin  1  1/3 (33.33%) 
Eczema  1  1/3 (33.33%) 
Heat rash  1  2/3 (66.67%) 
Hyperkeratosis  1  1/3 (33.33%) 
Rash  1  1/3 (33.33%) 
Urticaria  1  1/3 (33.33%) 
Vascular disorders   
Pallor  1  1/3 (33.33%) 
Peripheral vascular disorder  1  1/3 (33.33%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MEDDRA 8.1
Due to the rarity of the disease, this study is limited by its small sample size, lack of a control group and patients had variable treatment durations.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
In multi-site studies, PI can publish after Genzyme publishes or 18 months after study completion. PI gives Genzyme a draft 60 days before publication. Genzyme can ask that confidential information be removed, and can defer publication another 60 days upon notifying PI that it will file a patent application on inventions contained in the draft.
Results Point of Contact
Name/Title: Genzyme Medical Information
Organization: Genzyme Corporation
Phone: 800-745-4447
Responsible Party: Medical Monitor, Genzyme Corporation
ClinicalTrials.gov Identifier: NCT00258011     History of Changes
Other Study ID Numbers: ALID02205
First Submitted: November 22, 2005
First Posted: November 24, 2005
Results First Submitted: January 13, 2009
Results First Posted: February 12, 2009
Last Update Posted: March 7, 2014