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Trial record 1 of 1 for:    NCT00144781
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A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease

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ClinicalTrials.gov Identifier: NCT00144781
Recruitment Status : Completed
First Posted : September 5, 2005
Results First Posted : June 16, 2009
Last Update Posted : April 3, 2015
Sponsor:
Collaborator:
BioMarin/Genzyme LLC
Information provided by:
Sanofi

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Mucopolysaccharidosis I
Hurler's Syndrome
Hurler-Scheie Syndrome
Scheie Syndrome
Intervention Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
Enrollment 34
Recruitment Details  
Pre-assignment Details  
Arm/Group Title 0.58 mg/kg Aldurazyme Every Week 1.2 mg/kg Aldurazyme Every Week 1.2 mg/kg Aldurazyme Every 2 Weeks 1.8 mg/kg Aldurazyme Every 2 Weeks
Hide Arm/Group Description 0.58 mg Aldurazyme/kg of body weight (100 U/kg) administered every week (labeled dose). Final Visit is Week 27 for patients randomized to every week regimen. 1.2 mg Aldurazyme/kg of body weight (200 U/kg) administered every week. Final Visit is Week 27 for patients randomized to every week regimen. 1.2 mg Aldurazyme/kg of body weight (200 U/kg) administered every 2 weeks. Final Visit is Week 26 for patients randomized to every 2 week regimen. 1.8 mg Aldurazyme/kg of body weight (300 U/kg) administered every 2 weeks. Final Visit is Week 26 for patients randomized to every 2 week regimen.
Period Title: Overall Study
Started 8 9 8 9
Completed 8 7 8 9
Not Completed 0 2 0 0
Reason Not Completed
Adverse Event             0             1             0             0
Non-compliant             0             1             0             0
Arm/Group Title 0.58 mg/kg Aldurazyme Every Week 1.2 mg/kg Aldurazyme Every Week 1.2 mg/kg Aldurazyme Every 2 Weeks 1.8 mg/kg Aldurazyme Every 2 Weeks Total
Hide Arm/Group Description 0.58 mg Aldurazyme/kg of body weight (100 U/kg) administered every week (labeled dose). Final Visit is Week 27 for patients randomized to every week regimen. 1.2 mg Aldurazyme/kg of body weight (200 U/kg) administered every week. Final Visit is Week 27 for patients randomized to every week regimen. 1.2 mg Aldurazyme/kg of body weight (200 U/kg) administered every 2 weeks. Final Visit is Week 26 for patients randomized to every 2 week regimen. 1.8 mg Aldurazyme/kg of body weight (300 U/kg) administered every 2 weeks. Final Visit is Week 26 for patients randomized to every 2 week regimen. Total of all reporting groups
Overall Number of Baseline Participants 8 9 8 9 34
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 8 participants 9 participants 8 participants 9 participants 34 participants
8.5  (4.86) 8.0  (6.64) 9.1  (4.97) 9.3  (5.48) 8.7  (5.34)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants 9 participants 8 participants 9 participants 34 participants
Female
5
  62.5%
4
  44.4%
3
  37.5%
6
  66.7%
18
  52.9%
Male
3
  37.5%
5
  55.6%
5
  62.5%
3
  33.3%
16
  47.1%
Study Specific Race/Ethnicity  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 8 participants 9 participants 8 participants 9 participants 34 participants
Caucasian 4 6 5 6 21
Black 1 0 0 0 1
Hispanic 1 0 1 1 3
Asian 0 0 0 0 0
Other 2 3 2 2 9
1.Primary Outcome
Title Percent Change From Baseline to Week 26 in Urinary Glycosaminoglycan (GAG) Level
Hide Description Urinary GAG Level - Concentration of GAG relative to creatinine in urine. A greater decrease in GAG level indicates a greater response.
Time Frame Baseline to 26 Weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Based on the changes in urinary GAG levels observed in the Phase 3 double-blind study, a sample size of 8 patients per group would have sufficient power to detect a 29 percentage point difference between groups in the mean change in urinary GAG levels as being statistically significant. The analysis was intention to treat.
Arm/Group Title 0.58 mg/kg Aldurazyme Every Week 1.2 mg/kg Aldurazyme Every Week 1.2 mg/kg Aldurazyme Every 2 Weeks 1.8 mg/kg Aldurazyme Every 2 Weeks
Hide Arm/Group Description:
0.58 mg Aldurazyme/kg of body weight (100 U/kg) administered every week (labeled dose). Final Visit is Week 27 for patients randomized to every week regimen.
1.2 mg Aldurazyme/kg of body weight (200 U/kg) administered every week. Final Visit is Week 27 for patients randomized to every week regimen.
1.2 mg Aldurazyme/kg of body weight (200 U/kg) administered every 2 weeks. Final Visit is Week 26 for patients randomized to every 2 week regimen.
1.8 mg Aldurazyme/kg of body weight (300 U/kg) administered every 2 weeks. Final Visit is Week 26 for patients randomized to every 2 week regimen.
Overall Number of Participants Analyzed 8 7 8 9
Mean (95% Confidence Interval)
Unit of Measure: Percentage of Change in GAG Level
-57.57
(-73.28 to -41.87)
-66.63
(-80.42 to -52.83)
-66.15
(-72.88 to -59.43)
-62.66
(-67.66 to -57.66)
2.Secondary Outcome
Title Percent Change From Baseline to Week 26 in Liver Organ Volume
Hide Description A greater decrease in liver volume indicates a greater response.
Time Frame Baseline to 26 Weeks
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis was intention to treat.
Arm/Group Title 0.58 mg/kg Aldurazyme Every Week 1.2 mg/kg Aldurazyme Every Week 1.2 mg/kg Aldurazyme Every 2 Weeks 1.8 mg/kg Aldurazyme Every 2 Weeks
Hide Arm/Group Description:
0.58 mg Aldurazyme/kg of body weight (100 U/kg) administered every week (labeled dose). Final Visit is Week 27 for patients randomized to every week regimen.
1.2 mg Aldurazyme/kg of body weight (200 U/kg) administered every week. Final Visit is Week 27 for patients randomized to every week regimen.
1.2 mg Aldurazyme/kg of body weight (200 U/kg) administered every 2 weeks. Final Visit is Week 26 for patients randomized to every 2 week regimen.
1.8 mg Aldurazyme/kg of body weight (300 U/kg) administered every 2 weeks. Final Visit is Week 26 for patients randomized to every 2 week regimen.
Overall Number of Participants Analyzed 7 7 8 8
Mean (95% Confidence Interval)
Unit of Measure: Percentage of Change in Liver Volume
-26.27
(-33.52 to -19.10)
-30.67
(-38.19 to -23.15)
-30.74
(-45.99 to -15.48)
-32.14
(-40.58 to -23.70)
3.Secondary Outcome
Title Change From Baseline to Week 26 in Six Minute Walk Test (6MWT)
Hide Description Six Minute Walk Test: Distance walked (measured in Meters) in 6 minutes. A longer distance indicates a greater response.
Time Frame Baseline to 26 Weeks
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis was intention to treat.
Arm/Group Title 0.58 mg/kg Aldurazyme Every Week 1.2 mg/kg Aldurazyme Every Week 1.2 mg/kg Aldurazyme Every 2 Weeks 1.8 mg/kg Aldurazyme Every 2 Weeks
Hide Arm/Group Description:
0.58 mg Aldurazyme/kg of body weight (100 U/kg) administered every week (labeled dose). Final Visit is Week 27 for patients randomized to every week regimen.
1.2 mg Aldurazyme/kg of body weight (200 U/kg) administered every week. Final Visit is Week 27 for patients randomized to every week regimen.
1.2 mg Aldurazyme/kg of body weight (200 U/kg) administered every 2 weeks. Final Visit is Week 26 for patients randomized to every 2 week regimen.
1.8 mg Aldurazyme/kg of body weight (300 U/kg) administered every 2 weeks. Final Visit is Week 26 for patients randomized to every 2 week regimen.
Overall Number of Participants Analyzed 6 6 7 7
Mean (95% Confidence Interval)
Unit of Measure: meters
7.00
(-80.48 to 94.48)
-12.17
(-73.08 to 48.75)
25.43
(-44.39 to 95.25)
52.14
(-15.19 to 119.48)
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|Every Other|Week 1.2 mg/kg***Check Title*** Aldurazyme|Every Other|Week 1.8 mg/kg***Check Title*** Aldurazyme|Weekly|0.58 mg/kg***Check Title*** Aldurazyme|Weekly|1.2 mg/kg***Check Title***
Hide Arm/Group Description Aldurazyme|Every Other|Week 1.2 mg/kg***Check Description*** Aldurazyme|Every Other|Week 1.8 mg/kg***Check Description*** Aldurazyme|Weekly|0.58 mg/kg***Check Description*** Aldurazyme|Weekly|1.2 mg/kg***Check Description***
All-Cause Mortality
Aldurazyme|Every Other|Week 1.2 mg/kg***Check Title*** Aldurazyme|Every Other|Week 1.8 mg/kg***Check Title*** Aldurazyme|Weekly|0.58 mg/kg***Check Title*** Aldurazyme|Weekly|1.2 mg/kg***Check Title***
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/--   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Aldurazyme|Every Other|Week 1.2 mg/kg***Check Title*** Aldurazyme|Every Other|Week 1.8 mg/kg***Check Title*** Aldurazyme|Weekly|0.58 mg/kg***Check Title*** Aldurazyme|Weekly|1.2 mg/kg***Check Title***
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   2/8 (25.00%)   1/9 (11.11%)   1/8 (12.50%)   2/8 (25.00%) 
Gastrointestinal disorders         
Abdominal distension  1  0/8 (0.00%)  1/9 (11.11%)  0/8 (0.00%)  0/8 (0.00%) 
General disorders         
Abasia  1  1/8 (12.50%)  0/9 (0.00%)  0/8 (0.00%)  0/8 (0.00%) 
Infections and infestations         
Acute sinusitis  1  0/8 (0.00%)  0/9 (0.00%)  0/8 (0.00%)  1/8 (12.50%) 
Otitis media acute  1  0/8 (0.00%)  1/9 (11.11%)  0/8 (0.00%)  0/8 (0.00%) 
Pneumonia  1  1/8 (12.50%)  0/9 (0.00%)  0/8 (0.00%)  0/8 (0.00%) 
Injury, poisoning and procedural complications         
Femur fracture  1  0/8 (0.00%)  0/9 (0.00%)  1/8 (12.50%)  0/8 (0.00%) 
Respiratory, thoracic and mediastinal disorders         
Pulmonary oedema  1  1/8 (12.50%)  0/9 (0.00%)  0/8 (0.00%)  0/8 (0.00%) 
Respiratory failure  1  0/8 (0.00%)  0/9 (0.00%)  0/8 (0.00%)  1/8 (12.50%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 11.1
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Aldurazyme|Every Other|Week 1.2 mg/kg***Check Title*** Aldurazyme|Every Other|Week 1.8 mg/kg***Check Title*** Aldurazyme|Weekly|0.58 mg/kg***Check Title*** Aldurazyme|Weekly|1.2 mg/kg***Check Title***
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   8/8 (100.00%)   9/9 (100.00%)   8/8 (100.00%)   7/8 (87.50%) 
Blood and lymphatic system disorders         
Anaemia  1  0/8 (0.00%)  1/9 (11.11%)  0/8 (0.00%)  0/8 (0.00%) 
Eosinophilia  1  1/8 (12.50%)  0/9 (0.00%)  0/8 (0.00%)  0/8 (0.00%) 
White blood cell disorder  1  1/8 (12.50%)  0/9 (0.00%)  1/8 (12.50%)  0/8 (0.00%) 
Cardiac disorders         
Cyanosis  1  0/8 (0.00%)  1/9 (11.11%)  0/8 (0.00%)  1/8 (12.50%) 
Tachycardia  1  0/8 (0.00%)  0/9 (0.00%)  1/8 (12.50%)  0/8 (0.00%) 
Ear and labyrinth disorders         
Deafness  1  0/8 (0.00%)  1/9 (11.11%)  0/8 (0.00%)  0/8 (0.00%) 
Ear pain  1  0/8 (0.00%)  0/9 (0.00%)  1/8 (12.50%)  0/8 (0.00%) 
Otorrhoea  1  0/8 (0.00%)  1/9 (11.11%)  0/8 (0.00%)  0/8 (0.00%) 
Eye disorders         
Conjunctivitis  1  0/8 (0.00%)  1/9 (11.11%)  2/8 (25.00%)  0/8 (0.00%) 
Lacrimation increased  1  0/8 (0.00%)  0/9 (0.00%)  0/8 (0.00%)  1/8 (12.50%) 
Strabismus  1  0/8 (0.00%)  0/9 (0.00%)  0/8 (0.00%)  1/8 (12.50%) 
Vision blurred  1  0/8 (0.00%)  0/9 (0.00%)  0/8 (0.00%)  1/8 (12.50%) 
Visual acuity reduced  1  0/8 (0.00%)  1/9 (11.11%)  0/8 (0.00%)  0/8 (0.00%) 
Gastrointestinal disorders         
Abdominal distension  1  0/8 (0.00%)  2/9 (22.22%)  0/8 (0.00%)  0/8 (0.00%) 
Abdominal pain  1  1/8 (12.50%)  2/9 (22.22%)  4/8 (50.00%)  3/8 (37.50%) 
Abdominal pain upper  1  1/8 (12.50%)  0/9 (0.00%)  0/8 (0.00%)  2/8 (25.00%) 
Diarrhoea  1  4/8 (50.00%)  3/9 (33.33%)  6/8 (75.00%)  6/8 (75.00%) 
Flatulence  1  1/8 (12.50%)  0/9 (0.00%)  1/8 (12.50%)  1/8 (12.50%) 
Mouth ulceration  1  0/8 (0.00%)  0/9 (0.00%)  1/8 (12.50%)  0/8 (0.00%) 
Nausea  1  0/8 (0.00%)  1/9 (11.11%)  4/8 (50.00%)  4/8 (50.00%) 
Oral disorder  1  0/8 (0.00%)  0/9 (0.00%)  1/8 (12.50%)  0/8 (0.00%) 
Perianal erythema  1  2/8 (25.00%)  1/9 (11.11%)  0/8 (0.00%)  0/8 (0.00%) 
Stomach discomfort  1  0/8 (0.00%)  0/9 (0.00%)  0/8 (0.00%)  1/8 (12.50%) 
Teething  1  0/8 (0.00%)  1/9 (11.11%)  0/8 (0.00%)  0/8 (0.00%) 
Vomiting  1  2/8 (25.00%)  4/9 (44.44%)  3/8 (37.50%)  6/8 (75.00%) 
General disorders         
Abasia  1  0/8 (0.00%)  0/9 (0.00%)  1/8 (12.50%)  0/8 (0.00%) 
Application site dermatitis  1  0/8 (0.00%)  0/9 (0.00%)  0/8 (0.00%)  1/8 (12.50%) 
Catheter site pain  1  0/8 (0.00%)  0/9 (0.00%)  0/8 (0.00%)  1/8 (12.50%) 
Catheter site rash  1  0/8 (0.00%)  0/9 (0.00%)  0/8 (0.00%)  1/8 (12.50%) 
Catheter site related reaction  1  0/8 (0.00%)  1/9 (11.11%)  0/8 (0.00%)  1/8 (12.50%) 
Chest pain  1  0/8 (0.00%)  0/9 (0.00%)  2/8 (25.00%)  0/8 (0.00%) 
Chills  1  0/8 (0.00%)  0/9 (0.00%)  0/8 (0.00%)  1/8 (12.50%) 
Disease progression  1  1/8 (12.50%)  0/9 (0.00%)  0/8 (0.00%)  0/8 (0.00%) 
Fatigue  1  0/8 (0.00%)  0/9 (0.00%)  0/8 (0.00%)  1/8 (12.50%) 
Feeling hot  1  0/8 (0.00%)  0/9 (0.00%)  0/8 (0.00%)  1/8 (12.50%) 
Hernia obstructive  1  0/8 (0.00%)  1/9 (11.11%)  0/8 (0.00%)  0/8 (0.00%) 
Hernia pain  1  0/8 (0.00%)  0/9 (0.00%)  1/8 (12.50%)  1/8 (12.50%) 
Hyperthermia  1  0/8 (0.00%)  1/9 (11.11%)  0/8 (0.00%)  1/8 (12.50%) 
Influenza like illness  1  2/8 (25.00%)  0/9 (0.00%)  2/8 (25.00%)  3/8 (37.50%) 
Infusion site pruritus  1  0/8 (0.00%)  0/9 (0.00%)  0/8 (0.00%)  1/8 (12.50%) 
Infusion site rash  1  0/8 (0.00%)  0/9 (0.00%)  0/8 (0.00%)  1/8 (12.50%) 
Irritability  1  0/8 (0.00%)  1/9 (11.11%)  0/8 (0.00%)  0/8 (0.00%) 
Localised oedema  1  1/8 (12.50%)  0/9 (0.00%)  0/8 (0.00%)  0/8 (0.00%) 
Oedema  1  0/8 (0.00%)  0/9 (0.00%)  1/8 (12.50%)  0/8 (0.00%) 
Oedema peripheral  1  0/8 (0.00%)  0/9 (0.00%)  1/8 (12.50%)  0/8 (0.00%) 
Pain  1  1/8 (12.50%)  1/9 (11.11%)  2/8 (25.00%)  1/8 (12.50%) 
Pyrexia  1  5/8 (62.50%)  4/9 (44.44%)  6/8 (75.00%)  5/8 (62.50%) 
Vessel puncture site reaction  1  0/8 (0.00%)  0/9 (0.00%)  0/8 (0.00%)  1/8 (12.50%) 
Infections and infestations         
Acarodermatitis  1  0/8 (0.00%)  0/9 (0.00%)  1/8 (12.50%)  1/8 (12.50%) 
Bronchitis  1  1/8 (12.50%)  0/9 (0.00%)  1/8 (12.50%)  0/8 (0.00%) 
Cellulitis  1  0/8 (0.00%)  0/9 (0.00%)  0/8 (0.00%)  1/8 (12.50%) 
Conjunctivitis viral  1  0/8 (0.00%)  0/9 (0.00%)  2/8 (25.00%)  0/8 (0.00%) 
Dermatitis infected  1  0/8 (0.00%)  1/9 (11.11%)  0/8 (0.00%)  0/8 (0.00%) 
Ear infection  1  2/8 (25.00%)  1/9 (11.11%)  1/8 (12.50%)  0/8 (0.00%) 
Enterobiasis  1  1/8 (12.50%)  0/9 (0.00%)  0/8 (0.00%)  0/8 (0.00%) 
Flea infestation  1  0/8 (0.00%)  0/9 (0.00%)  1/8 (12.50%)  0/8 (0.00%) 
Genital candidiasis  1  1/8 (12.50%)  0/9 (0.00%)  1/8 (12.50%)  0/8 (0.00%) 
Herpes simplex  1  0/8 (0.00%)  0/9 (0.00%)  1/8 (12.50%)  0/8 (0.00%) 
Hordeolum  1  0/8 (0.00%)  0/9 (0.00%)  0/8 (0.00%)  1/8 (12.50%) 
Infection parasitic  1  0/8 (0.00%)  0/9 (0.00%)  1/8 (12.50%)  0/8 (0.00%) 
Influenza  1  1/8 (12.50%)  3/9 (33.33%)  2/8 (25.00%)  1/8 (12.50%) 
Lice infestation  1  1/8 (12.50%)  0/9 (0.00%)  0/8 (0.00%)  0/8 (0.00%) 
Localised infection  1  0/8 (0.00%)  0/9 (0.00%)  1/8 (12.50%)  0/8 (0.00%) 
Nasopharyngitis  1  3/8 (37.50%)  5/9 (55.56%)  2/8 (25.00%)  1/8 (12.50%) 
Oral herpes  1  1/8 (12.50%)  0/9 (0.00%)  0/8 (0.00%)  0/8 (0.00%) 
Otitis media  1  0/8 (0.00%)  0/9 (0.00%)  1/8 (12.50%)  1/8 (12.50%) 
Otitis media acute  1  1/8 (12.50%)  2/9 (22.22%)  0/8 (0.00%)  1/8 (12.50%) 
Paronychia  1  0/8 (0.00%)  1/9 (11.11%)  0/8 (0.00%)  0/8 (0.00%) 
Rhinitis  1  3/8 (37.50%)  2/9 (22.22%)  2/8 (25.00%)  3/8 (37.50%) 
Sinusitis  1  4/8 (50.00%)  2/9 (22.22%)  2/8 (25.00%)  3/8 (37.50%) 
Streptococcal impetigo  1  0/8 (0.00%)  0/9 (0.00%)  1/8 (12.50%)  0/8 (0.00%) 
Tonsillitis  1  0/8 (0.00%)  0/9 (0.00%)  1/8 (12.50%)  1/8 (12.50%) 
Upper respiratory tract infection  1  2/8 (25.00%)  1/9 (11.11%)  1/8 (12.50%)  3/8 (37.50%) 
Viral diarrhoea  1  1/8 (12.50%)  0/9 (0.00%)  0/8 (0.00%)  0/8 (0.00%) 
Injury, poisoning and procedural complications         
Arthropod bite  1  0/8 (0.00%)  1/9 (11.11%)  2/8 (25.00%)  1/8 (12.50%) 
Excoriation  1  1/8 (12.50%)  0/9 (0.00%)  0/8 (0.00%)  0/8 (0.00%) 
Limb injury  1  0/8 (0.00%)  1/9 (11.11%)  0/8 (0.00%)  0/8 (0.00%) 
Investigations         
Blood alkaline phosphatase increased  1  0/8 (0.00%)  0/9 (0.00%)  1/8 (12.50%)  0/8 (0.00%) 
Blood iron decreased  1  0/8 (0.00%)  0/9 (0.00%)  0/8 (0.00%)  1/8 (12.50%) 
Blood lactate dehydrogenase increased  1  0/8 (0.00%)  0/9 (0.00%)  0/8 (0.00%)  1/8 (12.50%) 
Blood pressure decreased  1  0/8 (0.00%)  0/9 (0.00%)  0/8 (0.00%)  1/8 (12.50%) 
Breath sounds abnormal  1  0/8 (0.00%)  0/9 (0.00%)  0/8 (0.00%)  1/8 (12.50%) 
Lymphocyte count increased  1  0/8 (0.00%)  1/9 (11.11%)  0/8 (0.00%)  1/8 (12.50%) 
Neutrophil count decreased  1  0/8 (0.00%)  0/9 (0.00%)  0/8 (0.00%)  1/8 (12.50%) 
Oxygen saturation decreased  1  0/8 (0.00%)  0/9 (0.00%)  0/8 (0.00%)  1/8 (12.50%) 
Platelet count decreased  1  3/8 (37.50%)  2/9 (22.22%)  0/8 (0.00%)  1/8 (12.50%) 
Platelet count increased  1  1/8 (12.50%)  0/9 (0.00%)  1/8 (12.50%)  0/8 (0.00%) 
Urine leukocyte esterase positive  1  0/8 (0.00%)  1/9 (11.11%)  0/8 (0.00%)  0/8 (0.00%) 
White blood cell count increased  1  0/8 (0.00%)  0/9 (0.00%)  0/8 (0.00%)  1/8 (12.50%) 
Metabolism and nutrition disorders         
Anorexia  1  1/8 (12.50%)  0/9 (0.00%)  0/8 (0.00%)  0/8 (0.00%) 
Decreased appetite  1  0/8 (0.00%)  2/9 (22.22%)  0/8 (0.00%)  0/8 (0.00%) 
Pica  1  1/8 (12.50%)  0/9 (0.00%)  0/8 (0.00%)  0/8 (0.00%) 
Polydipsia  1  0/8 (0.00%)  0/9 (0.00%)  1/8 (12.50%)  1/8 (12.50%) 
Weight gain poor  1  0/8 (0.00%)  0/9 (0.00%)  1/8 (12.50%)  0/8 (0.00%) 
Musculoskeletal and connective tissue disorders         
Arthralgia  1  0/8 (0.00%)  0/9 (0.00%)  2/8 (25.00%)  3/8 (37.50%) 
Back pain  1  0/8 (0.00%)  1/9 (11.11%)  0/8 (0.00%)  0/8 (0.00%) 
Groin pain  1  1/8 (12.50%)  0/9 (0.00%)  0/8 (0.00%)  0/8 (0.00%) 
Musculoskeletal stiffness  1  0/8 (0.00%)  0/9 (0.00%)  0/8 (0.00%)  1/8 (12.50%) 
Neck pain  1  1/8 (12.50%)  0/9 (0.00%)  1/8 (12.50%)  0/8 (0.00%) 
Pain in extremity  1  0/8 (0.00%)  0/9 (0.00%)  2/8 (25.00%)  1/8 (12.50%) 
Synovial cyst  1  0/8 (0.00%)  1/9 (11.11%)  0/8 (0.00%)  0/8 (0.00%) 
Nervous system disorders         
Convulsion  1  1/8 (12.50%)  0/9 (0.00%)  0/8 (0.00%)  0/8 (0.00%) 
Dizziness  1  0/8 (0.00%)  1/9 (11.11%)  0/8 (0.00%)  1/8 (12.50%) 
Headache  1  2/8 (25.00%)  0/9 (0.00%)  2/8 (25.00%)  4/8 (50.00%) 
Motor dysfunction  1  1/8 (12.50%)  0/9 (0.00%)  0/8 (0.00%)  0/8 (0.00%) 
Paraesthesia  1  0/8 (0.00%)  0/9 (0.00%)  1/8 (12.50%)  0/8 (0.00%) 
Tremor  1  0/8 (0.00%)  1/9 (11.11%)  0/8 (0.00%)  1/8 (12.50%) 
Psychiatric disorders         
Abnormal behaviour  1  1/8 (12.50%)  0/9 (0.00%)  1/8 (12.50%)  0/8 (0.00%) 
Depression  1  0/8 (0.00%)  1/9 (11.11%)  0/8 (0.00%)  0/8 (0.00%) 
Dysphoria  1  0/8 (0.00%)  0/9 (0.00%)  0/8 (0.00%)  1/8 (12.50%) 
Inappropriate affect  1  0/8 (0.00%)  1/9 (11.11%)  0/8 (0.00%)  0/8 (0.00%) 
Insomnia  1  2/8 (25.00%)  1/9 (11.11%)  0/8 (0.00%)  0/8 (0.00%) 
Renal and urinary disorders         
Leukocyturia  1  0/8 (0.00%)  1/9 (11.11%)  0/8 (0.00%)  0/8 (0.00%) 
Reproductive system and breast disorders         
Dysmenorrhoea  1  0/8 (0.00%)  1/9 (11.11%)  1/8 (12.50%)  0/8 (0.00%) 
Genital erythema  1  1/8 (12.50%)  0/9 (0.00%)  0/8 (0.00%)  0/8 (0.00%) 
Respiratory, thoracic and mediastinal disorders         
Adenoidal hypertrophy  1  0/8 (0.00%)  0/9 (0.00%)  0/8 (0.00%)  1/8 (12.50%) 
Allergic sinusitis  1  0/8 (0.00%)  1/9 (11.11%)  0/8 (0.00%)  0/8 (0.00%) 
Bronchospasm  1  1/8 (12.50%)  1/9 (11.11%)  1/8 (12.50%)  0/8 (0.00%) 
Cough  1  1/8 (12.50%)  2/9 (22.22%)  5/8 (62.50%)  5/8 (62.50%) 
Dysphonia  1  0/8 (0.00%)  1/9 (11.11%)  0/8 (0.00%)  1/8 (12.50%) 
Dyspnoea  1  0/8 (0.00%)  0/9 (0.00%)  0/8 (0.00%)  1/8 (12.50%) 
Nasal congestion  1  1/8 (12.50%)  3/9 (33.33%)  4/8 (50.00%)  2/8 (25.00%) 
Nocturnal dyspnoea  1  0/8 (0.00%)  0/9 (0.00%)  0/8 (0.00%)  1/8 (12.50%) 
Oropharyngeal pain  1  2/8 (25.00%)  0/9 (0.00%)  1/8 (12.50%)  4/8 (50.00%) 
Pharyngeal erythema  1  0/8 (0.00%)  1/9 (11.11%)  0/8 (0.00%)  0/8 (0.00%) 
Productive cough  1  0/8 (0.00%)  1/9 (11.11%)  0/8 (0.00%)  0/8 (0.00%) 
Pulmonary hypertension  1  1/8 (12.50%)  1/9 (11.11%)  0/8 (0.00%)  0/8 (0.00%) 
Respiratory tract congestion  1  0/8 (0.00%)  0/9 (0.00%)  0/8 (0.00%)  1/8 (12.50%) 
Rhinitis allergic  1  1/8 (12.50%)  0/9 (0.00%)  0/8 (0.00%)  1/8 (12.50%) 
Rhinorrhoea  1  2/8 (25.00%)  2/9 (22.22%)  3/8 (37.50%)  1/8 (12.50%) 
Sneezing  1  1/8 (12.50%)  1/9 (11.11%)  0/8 (0.00%)  0/8 (0.00%) 
Tachypnoea  1  0/8 (0.00%)  0/9 (0.00%)  1/8 (12.50%)  0/8 (0.00%) 
Wheezing  1  0/8 (0.00%)  0/9 (0.00%)  1/8 (12.50%)  0/8 (0.00%) 
Skin and subcutaneous tissue disorders         
Decubitus ulcer  1  0/8 (0.00%)  0/9 (0.00%)  1/8 (12.50%)  0/8 (0.00%) 
Dermatitis  1  2/8 (25.00%)  0/9 (0.00%)  1/8 (12.50%)  1/8 (12.50%) 
Dermatitis allergic  1  1/8 (12.50%)  0/9 (0.00%)  0/8 (0.00%)  0/8 (0.00%) 
Dermatitis atopic  1  0/8 (0.00%)  0/9 (0.00%)  1/8 (12.50%)  0/8 (0.00%) 
Dermatitis contact  1  0/8 (0.00%)  0/9 (0.00%)  0/8 (0.00%)  1/8 (12.50%) 
Dermatitis diaper  1  0/8 (0.00%)  0/9 (0.00%)  2/8 (25.00%)  1/8 (12.50%) 
Dry skin  1  1/8 (12.50%)  0/9 (0.00%)  0/8 (0.00%)  0/8 (0.00%) 
Eczema  1  1/8 (12.50%)  0/9 (0.00%)  1/8 (12.50%)  0/8 (0.00%) 
Erythema  1  0/8 (0.00%)  1/9 (11.11%)  0/8 (0.00%)  1/8 (12.50%) 
Heat rash  1  0/8 (0.00%)  0/9 (0.00%)  1/8 (12.50%)  0/8 (0.00%) 
Hyperhidrosis  1  0/8 (0.00%)  0/9 (0.00%)  0/8 (0.00%)  1/8 (12.50%) 
Milia  1  1/8 (12.50%)  0/9 (0.00%)  0/8 (0.00%)  0/8 (0.00%) 
Periorbital oedema  1  1/8 (12.50%)  0/9 (0.00%)  0/8 (0.00%)  0/8 (0.00%) 
Petechiae  1  1/8 (12.50%)  0/9 (0.00%)  0/8 (0.00%)  0/8 (0.00%) 
Pruritus  1  0/8 (0.00%)  0/9 (0.00%)  0/8 (0.00%)  2/8 (25.00%) 
Rash  1  1/8 (12.50%)  1/9 (11.11%)  0/8 (0.00%)  3/8 (37.50%) 
Rash papular  1  0/8 (0.00%)  0/9 (0.00%)  1/8 (12.50%)  0/8 (0.00%) 
Skin exfoliation  1  0/8 (0.00%)  0/9 (0.00%)  1/8 (12.50%)  0/8 (0.00%) 
Skin lesion  1  1/8 (12.50%)  1/9 (11.11%)  0/8 (0.00%)  0/8 (0.00%) 
Swelling face  1  0/8 (0.00%)  0/9 (0.00%)  1/8 (12.50%)  0/8 (0.00%) 
Urticaria  1  1/8 (12.50%)  2/9 (22.22%)  0/8 (0.00%)  1/8 (12.50%) 
Vascular disorders         
Hypertension  1  0/8 (0.00%)  1/9 (11.11%)  0/8 (0.00%)  0/8 (0.00%) 
Pallor  1  0/8 (0.00%)  0/9 (0.00%)  1/8 (12.50%)  1/8 (12.50%) 
Poor venous access  1  1/8 (12.50%)  0/9 (0.00%)  0/8 (0.00%)  0/8 (0.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 11.1
This study is limited by its small sample size and ability to detect small differences in urinary GAG levels between groups.
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: NCT00144781     History of Changes
Other Study ID Numbers: ALID-017-03
First Submitted: September 2, 2005
First Posted: September 5, 2005
Results First Submitted: March 27, 2009
Results First Posted: June 16, 2009
Last Update Posted: April 3, 2015