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Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) I

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00912925
Recruitment Status : Completed
First Posted : June 3, 2009
Results First Posted : August 19, 2009
Last Update Posted : April 7, 2015
Sponsor:
Collaborator:
BioMarin/Genzyme LLC
Information provided by:
Sanofi

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Conditions Mucopolysaccharidosis I
Hurlers Syndrome
Hurler-Scheie Syndrome
Interventions Biological: rhIDU (recombinant human-Alpha-L-Iduronidase)
Biological: Placebo
Enrollment 45
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Placebo Aldurazyme Treatment
Hide Arm/Group Description Patients in the placebo-control group were administered a solution of 100 millimolar (mM) sodium phosphate, 150 mM sodium chloride, and 0.001% polysorbate-80, adjusted to a pH of 5.8 administered intravenously over approximately 4 hours once weekly for 26 weeks. Patients in the active treatment group received Aldurazyme intravenously at a dose of 100 Units/kg (approximately 0.58 mg/kg = labeled dose) administered intravenously over approximately 4 hours once weekly for 26 weeks.
Period Title: Overall Study
Started 23 22
Completed 23 22
Not Completed 0 0
Arm/Group Title Placebo Aldurazyme Treatment Total
Hide Arm/Group Description Patients in the placebo-control group were administered a solution of 100 mM sodium phosphate, 150 mM sodium chloride, and 0.001% polysorbate-80, adjusted to a pH of 5.8 administered intravenously over approximately 4 hours once weekly for 26 weeks. Patients in the active treatment group received Aldurazyme intravenously at a dose of 100 Units/kg (approximately 0.58 mg/kg = labeled dose) administered intravenously over approximately 4 hours once weekly for 26 weeks. Total of all reporting groups
Overall Number of Baseline Participants 23 22 45
Hide Baseline Analysis Population Description
[Not Specified]
Age, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 23 participants 22 participants 45 participants
less than or equal to 12 years 10 12 22
13 to less than or equal to 18 years 8 3 11
19 to less than or equal to 65 years 5 7 12
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 23 participants 22 participants 45 participants
15.4  (7.63) 15.6  (8.63) 15.5  (8.04)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 23 participants 22 participants 45 participants
Female
12
  52.2%
11
  50.0%
23
  51.1%
Male
11
  47.8%
11
  50.0%
22
  48.9%
Race/Ethnicity, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 23 participants 22 participants 45 participants
Caucasian 21 16 37
Black 0 0 0
Hispanic 0 4 4
Asian 1 1 2
Other 1 1 2
1.Primary Outcome
Title Overall Change From Baseline to Week 26 in Percent Predicted Forced Vital Capacity (FVC)
Hide Description Percent Predicted Forced Vital Capacity (FVC): the maximal exhaled breathe volume following a maximal inhaled breath. Overall Change from Baseline to Week 26 in percent predicted FVC = (observed value)/(predicted value) * 100%). A higher value indicates a greater response.
Time Frame Baseline to Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Placebo Aldurazyme Treatment
Hide Arm/Group Description:
Patients in the placebo-control group were administered a solution of 100 mM sodium phosphate, 150 mM sodium chloride, and 0.001% polysorbate-80, adjusted to a pH of 5.8 administered intravenously over approximately 4 hours once weekly for 26 weeks.
Patients in the active treatment group received Aldurazyme intravenously at a dose of 100 Units/kg (approximately 0.58 mg/kg = labeled dose) administered intravenously over approximately 4 hours once weekly for 26 weeks.
Overall Number of Participants Analyzed 23 22
Mean (Standard Deviation)
Unit of Measure: Percent predicted FVC
Baseline 54.2  (16.00) 48.4  (14.85)
Week 26 51.5  (13.13) 50.2  (17.10)
Overall change from Baseline to Week 26 -2.7  (7.12) 1.8  (7.70)
2.Primary Outcome
Title Overall Change From Baseline to Week 26 in Six Minute Walk Test (6MWT)
Hide Description Six Minute Walk Test (6MWT): Distance walked (measured in meters) in 6 minutes. A longer distance indicates a greater response.
Time Frame Baseline to Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Placebo Aldurazyme Treatment
Hide Arm/Group Description:
Patients in the placebo-control group were administered a solution of 100 mM sodium phosphate, 150 mM sodium chloride, and 0.001% polysorbate-80, adjusted to a pH of 5.8 administered intravenously over approximately 4 hours once weekly for 26 weeks.
Patients in the active treatment group received Aldurazyme intravenously at a dose of 100 Units/kg (approximately 0.58 mg/kg = labeled dose) administered intravenously over approximately 4 hours once weekly for 26 weeks.
Overall Number of Participants Analyzed 23 22
Mean (Standard Deviation)
Unit of Measure: meters
Baseline 366.7  (113.68) 319.1  (131.41)
Week 26 348.3  (128.82) 338.8  (127.06)
Overall change from Baseline to Week 26 -18.4  (67.49) 19.7  (68.56)
3.Secondary Outcome
Title Overall Change From Baseline to Week 26 in Apnea/Hypopnea Index (AHI)
Hide Description Apnea/Hypopnea Index (AHI): Number of absent (apnea) and shallow (hypopnea) breaths per hour of sleep. Overall change from Baseline to Week 26 in AHI. A greater decrease in events indicates a greater response.
Time Frame Baseline to Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Placebo Aldurazyme Treatment
Hide Arm/Group Description:
Patients in the placebo-control group were administered a solution of 100 mM sodium phosphate, 150 mM sodium chloride, and 0.001% polysorbate-80, adjusted to a pH of 5.8 administered intravenously over approximately 4 hours once weekly for 26 weeks.
Patients in the active treatment group received Aldurazyme intravenously at a dose of 100 Units/kg (approximately 0.58 mg/kg = labeled dose) administered intravenously over approximately 4 hours once weekly for 26 weeks.
Overall Number of Participants Analyzed 19 20
Mean (Standard Deviation)
Unit of Measure: Events per hour
Baseline 14.7  (15.20) 20.5  (19.04)
Week 26 15.5  (14.93) 17.6  (16.75)
Overall change from Baseline to Week 26 0.4  (8.55) -2.9  (6.66)
4.Secondary Outcome
Title Overall Percent Change From Baseline to Week 26 in Liver Volume
Hide Description Liver Organ Volume: Volume of liver measured by Magnetic Resonance Imaging (MRI). Greater decrease in volume indicates a greater response.
Time Frame Baseline to Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Placebo Aldurazyme Treatment
Hide Arm/Group Description:
Patients in the placebo-control group were administered a solution of 100 mM sodium phosphate, 150 mM sodium chloride, and 0.001% polysorbate-80, adjusted to a pH of 5.8 administered intravenously over approximately 4 hours once weekly for 26 weeks.
Patients in the active treatment group received Aldurazyme intravenously at a dose of 100 Units/kg (approximately 0.58 mg/kg = labeled dose) administered intravenously over approximately 4 hours once weekly for 26 weeks.
Overall Number of Participants Analyzed 22 22
Mean (Standard Deviation)
Unit of Measure: Cubic centimeters
Baseline 1368.1  (314.88) 12212.2  (283.36)
Week 26 1366.5  (316.94) 979.8  (321.45)
Overall percent change from Baseline to Week 26 1.3  (19.22) -18.9  (19.44)
5.Secondary Outcome
Title Overall Change From Baseline to Week 26 in Child Health Assessment Questionnaire/Health Assessment Questionnaire (CHAQ/HAQ) Disability Index Score
Hide Description CHAQ/HAQ) = Patient questionnaire that measures the degree of disability on a scale of 0 (no disability) to 3 (maximal disability). A lower score indicates a greater response.
Time Frame Baseline to week 26
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Placebo Aldurazyme Treatment
Hide Arm/Group Description:
Patients in the placebo-control group were administered a solution of 100 mM sodium phosphate, 150 mM sodium chloride, and 0.001% polysorbate-80, adjusted to a pH of 5.8 administered intravenously over approximately 4 hours once weekly for 26 weeks.
Patients in the active treatment group received Aldurazyme intravenously at a dose of 100 Units/kg (approximately 0.58 mg/kg = labeled dose) administered intravenously over approximately 4 hours once weekly for 26 weeks.
Overall Number of Participants Analyzed 23 21
Mean (Standard Deviation)
Unit of Measure: Units on a scale
Baseline 1.9  (0.60) 2.0  (0.49)
Week 26 1.8  (0.71) 1.9  (0.58)
Overall change from Baseline to Week 26 -0.1  (0.43) -0.2  (0.39)
6.Secondary Outcome
Title Overall Change From Baseline to Week 26 in Active Joint Range of Motion (ROM)
Hide Description Active Joint Range of Motion (ROM): Shoulder Flexion Ability to maximally raise one's arm overhead without assistance. Shoulder range of motion (mean of left and right arms) measured in degrees (0-180) by goniometry. Greater degree of flexion indicates greater response.
Time Frame Baseline to Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Placebo Aldurazyme Treatment
Hide Arm/Group Description:
Patients in the placebo-control group were administered a solution of 100 mM sodium phosphate, 150 mM sodium chloride, and 0.001% polysorbate-80, adjusted to a pH of 5.8 administered intravenously over approximately 4 hours once weekly for 26 weeks.
Patients in the active treatment group received Aldurazyme intravenously at a dose of 100 Units/kg (approximately 0.58 mg/kg = labeled dose) administered intravenously over approximately 4 hours once weekly for 26 weeks.
Overall Number of Participants Analyzed 23 19
Mean (Standard Deviation)
Unit of Measure: Degrees
Baseline 89.8  (23.99) 96.1  (30.25)
Week 26 85.2  (32.66) 88.9  (37.69)
Overall change from baseline to week 26 -4.6  (27.18) -1.2  (29.78)
7.Secondary Outcome
Title Overall Percent Change From Baseline to Week 26 in Urinary Glycosaminoglycan (GAG) Levels
Hide Description Urinary Glycosaminoglycan (GAG) Levels: Concentration of GAG relative to creatinine in urine. A greater decrease in GAG level indicates a greater response.
Time Frame Baseline to Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Placebo Aldurazyme Treatment
Hide Arm/Group Description:
Patients in the placebo-control group were administered a solution of 100 mM sodium phosphate, 150 mM sodium chloride, and 0.001% polysorbate-80, adjusted to a pH of 5.8 administered intravenously over approximately 4 hours once weekly for 26 weeks.
Patients in the active treatment group received Aldurazyme intravenously at a dose of 100 Units/kg (approximately 0.58 mg/kg = labeled dose) administered intravenously over approximately 4 hours once weekly for 26 weeks.
Overall Number of Participants Analyzed 22 21
Mean (Standard Deviation)
Unit of Measure: ug/mg
Baseline 183.3  (72.05) 188.9  (60.93)
Week 26 250.2  (105.09) 81.3  (26.36)
Overall Percentage of Change in GAG Level 47.3  (59.34) -54.1  (19.49)
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 Placebo***Check Title*** Aldurazyme***Check Title***
Hide Arm/Group Description Placebo***Check Description*** Aldurazyme***Check Description***
All-Cause Mortality
Placebo***Check Title*** Aldurazyme***Check Title***
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Placebo***Check Title*** Aldurazyme***Check Title***
Affected / at Risk (%) Affected / at Risk (%)
Total   0/23 (0.00%)   3/22 (13.64%) 
Cardiac disorders     
Cardiac arrest  1  0/23 (0.00%)  1/22 (4.55%) 
Gastrointestinal disorders     
Constipation  1  0/23 (0.00%)  1/22 (4.55%) 
Infections and infestations     
Pneumonia  1  0/23 (0.00%)  1/22 (4.55%) 
Sepsis  1  0/23 (0.00%)  1/22 (4.55%) 
Injury, poisoning and procedural complications     
Ventriculoperitoneal shunt malfunction  1  0/23 (0.00%)  1/22 (4.55%) 
Renal and urinary disorders     
Renal failure acute  1  0/23 (0.00%)  1/22 (4.55%) 
Vascular disorders     
Aortic stenosis  1  0/23 (0.00%)  1/22 (4.55%) 
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%
Placebo***Check Title*** Aldurazyme***Check Title***
Affected / at Risk (%) Affected / at Risk (%)
Total   23/23 (100.00%)   21/22 (95.45%) 
Blood and lymphatic system disorders     
Anaemia  1  0/23 (0.00%)  1/22 (4.55%) 
Lymphadenopathy  1  1/23 (4.35%)  2/22 (9.09%) 
Lymphatic disorder  1  0/23 (0.00%)  1/22 (4.55%) 
Splenomegaly  1  1/23 (4.35%)  2/22 (9.09%) 
Thrombocytopenia  1  0/23 (0.00%)  2/22 (9.09%) 
Cardiac disorders     
Angina pectoris  1  0/23 (0.00%)  1/22 (4.55%) 
Arrhythmia  1  0/23 (0.00%)  1/22 (4.55%) 
Cardiac failure  1  0/23 (0.00%)  1/22 (4.55%) 
Tachycardia  1  0/23 (0.00%)  1/22 (4.55%) 
Ear and labyrinth disorders     
Ear pain  1  8/23 (34.78%)  1/22 (4.55%) 
Eye disorders     
Blepharitis  1  1/23 (4.35%)  0/22 (0.00%) 
Corneal opacity  1  0/23 (0.00%)  2/22 (9.09%) 
Eye discharge  1  1/23 (4.35%)  2/22 (9.09%) 
Eye pain  1  0/23 (0.00%)  1/22 (4.55%) 
Eye swelling  1  0/23 (0.00%)  1/22 (4.55%) 
Visual acuity reduced  1  1/23 (4.35%)  2/22 (9.09%) 
Gastrointestinal disorders     
Abdominal discomfort  1  0/23 (0.00%)  1/22 (4.55%) 
Abdominal distension  1  0/23 (0.00%)  1/22 (4.55%) 
Abdominal pain  1  2/23 (8.70%)  3/22 (13.64%) 
Abdominal pain upper  1  3/23 (13.04%)  0/22 (0.00%) 
Bowel movement irregularity  1  1/23 (4.35%)  0/22 (0.00%) 
Chapped lips  1  0/23 (0.00%)  1/22 (4.55%) 
Constipation  1  0/23 (0.00%)  1/22 (4.55%) 
Dental discomfort  1  1/23 (4.35%)  0/22 (0.00%) 
Diarrhoea  1  8/23 (34.78%)  7/22 (31.82%) 
Duodenogastric reflux  1  0/23 (0.00%)  1/22 (4.55%) 
Flatulence  1  1/23 (4.35%)  1/22 (4.55%) 
Hypoaesthesia oral  1  0/23 (0.00%)  1/22 (4.55%) 
Nausea  1  6/23 (26.09%)  3/22 (13.64%) 
Rectal haemorrhage  1  0/23 (0.00%)  1/22 (4.55%) 
Stomach discomfort  1  1/23 (4.35%)  1/22 (4.55%) 
Tooth discolouration  1  0/23 (0.00%)  1/22 (4.55%) 
Toothache  1  0/23 (0.00%)  1/22 (4.55%) 
Vomiting  1  9/23 (39.13%)  5/22 (22.73%) 
General disorders     
Application site rash  1  0/23 (0.00%)  1/22 (4.55%) 
Catheter site related reaction  1  1/23 (4.35%)  1/22 (4.55%) 
Chest discomfort  1  0/23 (0.00%)  2/22 (9.09%) 
Chest pain  1  1/23 (4.35%)  0/22 (0.00%) 
Chills  1  2/23 (8.70%)  1/22 (4.55%) 
Face oedema  1  0/23 (0.00%)  2/22 (9.09%) 
Facial pain  1  2/23 (8.70%)  0/22 (0.00%) 
Fatigue  1  4/23 (17.39%)  1/22 (4.55%) 
Feeling hot  1  1/23 (4.35%)  2/22 (9.09%) 
Gait disturbance  1  2/23 (8.70%)  0/22 (0.00%) 
Gravitational oedema  1  0/23 (0.00%)  2/22 (9.09%) 
Hernia pain  1  1/23 (4.35%)  0/22 (0.00%) 
Influenza like illness  1  3/23 (13.04%)  0/22 (0.00%) 
Infusion site swelling  1  1/23 (4.35%)  0/22 (0.00%) 
Injection site pain  1  0/23 (0.00%)  2/22 (9.09%) 
Injection site reaction  1  2/23 (8.70%)  4/22 (18.18%) 
Oedema peripheral  1  1/23 (4.35%)  0/22 (0.00%) 
Pain  1  0/23 (0.00%)  1/22 (4.55%) 
Pyrexia  1  13/23 (56.52%)  8/22 (36.36%) 
Hepatobiliary disorders     
Hepatomegaly  1  6/23 (26.09%)  4/22 (18.18%) 
Hyperbilirubinaemia  1  0/23 (0.00%)  2/22 (9.09%) 
Immune system disorders     
Corneal graft rejection  1  1/23 (4.35%)  0/22 (0.00%) 
Seasonal allergy  1  2/23 (8.70%)  0/22 (0.00%) 
Infections and infestations     
Abscess  1  0/23 (0.00%)  2/22 (9.09%) 
Catheter site infection  1  0/23 (0.00%)  1/22 (4.55%) 
Ear infection  1  4/23 (17.39%)  3/22 (13.64%) 
Fungal infection  1  0/23 (0.00%)  1/22 (4.55%) 
Influenza  1  1/23 (4.35%)  0/22 (0.00%) 
Laryngitis  1  1/23 (4.35%)  0/22 (0.00%) 
Lice infestation  1  0/23 (0.00%)  1/22 (4.55%) 
Localised infection  1  0/23 (0.00%)  1/22 (4.55%) 
Lower respiratory tract infection  1  1/23 (4.35%)  1/22 (4.55%) 
Nasopharyngitis  1  8/23 (34.78%)  4/22 (18.18%) 
Otitis externa  1  1/23 (4.35%)  0/22 (0.00%) 
Otitis media  1  3/23 (13.04%)  0/22 (0.00%) 
Otitis media chronic  1  0/23 (0.00%)  1/22 (4.55%) 
Perirectal abscess  1  0/23 (0.00%)  1/22 (4.55%) 
Rhinitis  1  0/23 (0.00%)  1/22 (4.55%) 
Skin infection  1  0/23 (0.00%)  1/22 (4.55%) 
Sputum purulent  1  0/23 (0.00%)  1/22 (4.55%) 
Tinea pedis  1  0/23 (0.00%)  1/22 (4.55%) 
Upper respiratory tract infection  1  4/23 (17.39%)  7/22 (31.82%) 
Urinary tract infection  1  3/23 (13.04%)  2/22 (9.09%) 
Viral infection  1  2/23 (8.70%)  2/22 (9.09%) 
Injury, poisoning and procedural complications     
Arthropod bite  1  1/23 (4.35%)  0/22 (0.00%) 
Contusion  1  1/23 (4.35%)  1/22 (4.55%) 
Fall  1  1/23 (4.35%)  1/22 (4.55%) 
Joint injury  1  1/23 (4.35%)  0/22 (0.00%) 
Procedural pain  1  0/23 (0.00%)  1/22 (4.55%) 
Thermal burn  1  1/23 (4.35%)  0/22 (0.00%) 
Wound  1  1/23 (4.35%)  0/22 (0.00%) 
Investigations     
Alanine aminotransferase increased  1  2/23 (8.70%)  1/22 (4.55%) 
Aspartate aminotransferase increased  1  2/23 (8.70%)  1/22 (4.55%) 
Bacteria urine  1  1/23 (4.35%)  0/22 (0.00%) 
Blood glucose increased  1  0/23 (0.00%)  1/22 (4.55%) 
Body temperature increased  1  2/23 (8.70%)  3/22 (13.64%) 
Brain scan abnormal  1  0/23 (0.00%)  1/22 (4.55%) 
Breath sounds abnormal  1  4/23 (17.39%)  2/22 (9.09%) 
Cardiac murmur  1  3/23 (13.04%)  2/22 (9.09%) 
Gallop rhythm present  1  0/23 (0.00%)  1/22 (4.55%) 
Intraocular pressure increased  1  1/23 (4.35%)  0/22 (0.00%) 
Liver function test abnormal  1  1/23 (4.35%)  0/22 (0.00%) 
Nitrite urine present  1  1/23 (4.35%)  0/22 (0.00%) 
Protein urine present  1  1/23 (4.35%)  0/22 (0.00%) 
Metabolism and nutrition disorders     
Dehydration  1  0/23 (0.00%)  1/22 (4.55%) 
Musculoskeletal and connective tissue disorders     
Arthralgia  1  5/23 (21.74%)  4/22 (18.18%) 
Arthropathy  1  1/23 (4.35%)  2/22 (9.09%) 
Back pain  1  5/23 (21.74%)  4/22 (18.18%) 
Foot deformity  1  1/23 (4.35%)  1/22 (4.55%) 
Joint crepitation  1  2/23 (8.70%)  1/22 (4.55%) 
Joint instability  1  4/23 (17.39%)  0/22 (0.00%) 
Joint stiffness  1  0/23 (0.00%)  1/22 (4.55%) 
Muscle spasms  1  2/23 (8.70%)  1/22 (4.55%) 
Muscle tightness  1  1/23 (4.35%)  0/22 (0.00%) 
Musculoskeletal chest pain  1  1/23 (4.35%)  0/22 (0.00%) 
Musculoskeletal discomfort  1  1/23 (4.35%)  0/22 (0.00%) 
Musculoskeletal pain  1  1/23 (4.35%)  1/22 (4.55%) 
Myalgia  1  2/23 (8.70%)  2/22 (9.09%) 
Neck pain  1  2/23 (8.70%)  3/22 (13.64%) 
Pain in extremity  1  5/23 (21.74%)  4/22 (18.18%) 
Soft tissue disorder  1  1/23 (4.35%)  0/22 (0.00%) 
Nervous system disorders     
Dizziness  1  5/23 (21.74%)  1/22 (4.55%) 
Dizziness postural  1  0/23 (0.00%)  1/22 (4.55%) 
Grand mal convulsion  1  0/23 (0.00%)  1/22 (4.55%) 
Headache  1  16/23 (69.57%)  11/22 (50.00%) 
Hyperaesthesia  1  1/23 (4.35%)  0/22 (0.00%) 
Hyperreflexia  1  0/23 (0.00%)  3/22 (13.64%) 
Hypoaesthesia  1  1/23 (4.35%)  0/22 (0.00%) 
Migraine  1  1/23 (4.35%)  1/22 (4.55%) 
Paraesthesia  1  1/23 (4.35%)  3/22 (13.64%) 
Sinus headache  1  1/23 (4.35%)  0/22 (0.00%) 
Somnolence  1  1/23 (4.35%)  0/22 (0.00%) 
Syncope  1  1/23 (4.35%)  0/22 (0.00%) 
Syncope vasovagal  1  1/23 (4.35%)  0/22 (0.00%) 
Psychiatric disorders     
Agitation  1  0/23 (0.00%)  1/22 (4.55%) 
Anxiety  1  2/23 (8.70%)  2/22 (9.09%) 
Insomnia  1  1/23 (4.35%)  1/22 (4.55%) 
Restlessness  1  0/23 (0.00%)  1/22 (4.55%) 
Reproductive system and breast disorders     
Dysmenorrhoea  1  2/23 (8.70%)  0/22 (0.00%) 
Menstrual discomfort  1  1/23 (4.35%)  0/22 (0.00%) 
Vaginal discharge  1  1/23 (4.35%)  0/22 (0.00%) 
Respiratory, thoracic and mediastinal disorders     
Choking  1  1/23 (4.35%)  0/22 (0.00%) 
Cough  1  6/23 (26.09%)  6/22 (27.27%) 
Dry throat  1  1/23 (4.35%)  0/22 (0.00%) 
Dysphonia  1  0/23 (0.00%)  1/22 (4.55%) 
Epistaxis  1  0/23 (0.00%)  1/22 (4.55%) 
Hyperventilation  1  1/23 (4.35%)  0/22 (0.00%) 
Hypoxia  1  0/23 (0.00%)  1/22 (4.55%) 
Nasal congestion  1  3/23 (13.04%)  2/22 (9.09%) 
Oropharyngeal pain  1  4/23 (17.39%)  1/22 (4.55%) 
Pulmonary congestion  1  1/23 (4.35%)  0/22 (0.00%) 
Respiratory failure  1  0/23 (0.00%)  1/22 (4.55%) 
Respiratory tract congestion  1  1/23 (4.35%)  1/22 (4.55%) 
Rhinorrhoea  1  0/23 (0.00%)  1/22 (4.55%) 
Rhonchi  1  0/23 (0.00%)  1/22 (4.55%) 
Sneezing  1  2/23 (8.70%)  1/22 (4.55%) 
Wheezing  1  2/23 (8.70%)  0/22 (0.00%) 
Skin and subcutaneous tissue disorders     
Acne  1  2/23 (8.70%)  1/22 (4.55%) 
Alopecia  1  1/23 (4.35%)  1/22 (4.55%) 
Decubitus ulcer  1  0/23 (0.00%)  1/22 (4.55%) 
Dermal cyst  1  0/23 (0.00%)  1/22 (4.55%) 
Dermatitis acneiform  1  0/23 (0.00%)  1/22 (4.55%) 
Dermatitis allergic  1  1/23 (4.35%)  0/22 (0.00%) 
Dry skin  1  1/23 (4.35%)  1/22 (4.55%) 
Erythema  1  1/23 (4.35%)  0/22 (0.00%) 
Heat rash  1  0/23 (0.00%)  1/22 (4.55%) 
Hyperhidrosis  1  1/23 (4.35%)  2/22 (9.09%) 
Ingrowing nail  1  1/23 (4.35%)  0/22 (0.00%) 
Pain of skin  1  1/23 (4.35%)  0/22 (0.00%) 
Petechiae  1  1/23 (4.35%)  1/22 (4.55%) 
Pruritus  1  1/23 (4.35%)  2/22 (9.09%) 
Psoriasis  1  1/23 (4.35%)  0/22 (0.00%) 
Rash  1  5/23 (21.74%)  8/22 (36.36%) 
Rash macular  1  0/23 (0.00%)  1/22 (4.55%) 
Skin disorder  1  1/23 (4.35%)  0/22 (0.00%) 
Skin swelling  1  1/23 (4.35%)  0/22 (0.00%) 
Surgical and medical procedures     
Ear tube removal  1  1/23 (4.35%)  0/22 (0.00%) 
Vascular disorders     
Flushing  1  4/23 (17.39%)  5/22 (22.73%) 
Hot flush  1  1/23 (4.35%)  0/22 (0.00%) 
Hypertension  1  1/23 (4.35%)  0/22 (0.00%) 
Hypotension  1  0/23 (0.00%)  2/22 (9.09%) 
Poor venous access  1  0/23 (0.00%)  3/22 (13.64%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 11.1
Rare disease with limited sample size, FVC calculations less reliable for patients whose height is below the 3rd% for general population.
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
Responsible Party: Medical Monitor, Genzyme Corporation
ClinicalTrials.gov Identifier: NCT00912925     History of Changes
Other Study ID Numbers: ALID-003-99
First Submitted: June 2, 2009
First Posted: June 3, 2009
Results First Submitted: July 2, 2009
Results First Posted: August 19, 2009
Last Update Posted: April 7, 2015