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| Name | Class |
|---|---|
| BioMarin/Genzyme LLC | INDUSTRY |
Not provided
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This study is being conducted to demonstrate the safety and clinical efficacy of Aldurazyme treatment in MPS I patients
Not provided
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Placebo | Placebo Comparator | 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. |
|
| Aldurazyme treatment | Active Comparator | 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| rhIDU (recombinant human-Alpha-L-Iduronidase) | Biological | Patients in the active treatment group received Aldurazyme intravenously at a dose of 100 units/kg (approximately 0.58mg/kg) administered intravenously over approximately 4 hours once weekly for 26 weeks. |
| Measure | Description | Time Frame |
|---|---|---|
| Overall Change From Baseline to Week 26 in Percent Predicted Forced Vital Capacity (FVC) | 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. | Baseline to Week 26 |
| Overall Change From Baseline to Week 26 in Six Minute Walk Test (6MWT) | Six Minute Walk Test (6MWT): Distance walked (measured in meters) in 6 minutes. A longer distance indicates a greater response. | Baseline to Week 26 |
| Measure | Description | Time Frame |
|---|---|---|
| Overall Change From Baseline to Week 26 in Apnea/Hypopnea Index (AHI) | 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. | Baseline to Week 26 |
| Overall Percent Change From Baseline to Week 26 in Liver Volume |
Not provided
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Medical Monitor | Genzyme, a Sanofi Company | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| New York | New York | United States | ||||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 33874971 | Derived | Tandon PK, Kakkis ED. The multi-domain responder index: a novel analysis tool to capture a broader assessment of clinical benefit in heterogeneous complex rare diseases. Orphanet J Rare Dis. 2021 Apr 19;16(1):183. doi: 10.1186/s13023-021-01805-5. |
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| ID | Title | Description |
|---|---|---|
| FG000 | Placebo | 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. |
| FG001 | Aldurazyme Treatment | 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. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Placebo | 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. |
| BG001 | Aldurazyme Treatment |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Customized | Number |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Overall Change From Baseline to Week 26 in Percent Predicted Forced Vital Capacity (FVC) | 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. | Posted | Mean | Standard Deviation | Percent predicted FVC | Baseline to Week 26 |
|
Not provided
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.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Placebo***Check Title*** | Placebo***Check Description*** |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cardiac arrest | Cardiac disorders | MedDRA 11.1 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 11.1 | Systematic Assessment |
Rare disease with limited sample size, FVC calculations less reliable for patients whose height is below the 3rd% for general population.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Genzyme Medical Information | Genzyme Corporation | medinfo@genzyme.com |
Not provided
| ID | Term |
|---|---|
| D008059 | Mucopolysaccharidosis I |
| ID | Term |
|---|---|
| D009083 | Mucopolysaccharidoses |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
Not provided
Not provided
Not provided
Not provided
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| Placebo | Biological | Patients in the Placebo-control group were administered a solution of 100mM sodium phosphate , 150mM sodium chloride, and 0.001% polysorbate-80, adjusted to a pH of 5.8 administered intravenously over a time period of approximately 4 hours once weekly for 26 weeks. |
|
Liver Organ Volume: Volume of liver measured by Magnetic Resonance Imaging (MRI). Greater decrease in volume indicates a greater response. |
| Baseline to Week 26 |
| Overall Change From Baseline to Week 26 in Child Health Assessment Questionnaire/Health Assessment Questionnaire (CHAQ/HAQ) Disability Index Score | 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. | Baseline to week 26 |
| Overall Change From Baseline to Week 26 in Active Joint Range of Motion (ROM) | 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. | Baseline to Week 26 |
| Overall Percent Change From Baseline to Week 26 in Urinary Glycosaminoglycan (GAG) Levels | Urinary Glycosaminoglycan (GAG) Levels: Concentration of GAG relative to creatinine in urine. A greater decrease in GAG level indicates a greater response. | Baseline to Week 26 |
| Chapel Hill |
| North Carolina |
| United States |
| Vancouver | British Columbia | Canada |
| Mainz | Germany |
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. |
| BG002 | Total | Total of all reporting groups |
| participants |
|
| Age, Continuous | Mean | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race/Ethnicity, Customized | Number | Participants |
|
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.
|
|
| Primary | Overall Change From Baseline to Week 26 in Six Minute Walk Test (6MWT) | Six Minute Walk Test (6MWT): Distance walked (measured in meters) in 6 minutes. A longer distance indicates a greater response. | Posted | Mean | Standard Deviation | meters | Baseline to Week 26 |
|
|
|
| Secondary | Overall Change From Baseline to Week 26 in Apnea/Hypopnea Index (AHI) | 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. | Posted | Mean | Standard Deviation | Events per hour | Baseline to Week 26 |
|
|
|
| Secondary | Overall Percent Change From Baseline to Week 26 in Liver Volume | Liver Organ Volume: Volume of liver measured by Magnetic Resonance Imaging (MRI). Greater decrease in volume indicates a greater response. | Posted | Mean | Standard Deviation | Cubic centimeters | Baseline to Week 26 |
|
|
|
| Secondary | Overall Change From Baseline to Week 26 in Child Health Assessment Questionnaire/Health Assessment Questionnaire (CHAQ/HAQ) Disability Index Score | 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. | Posted | Mean | Standard Deviation | Units on a scale | Baseline to week 26 |
|
|
|
| Secondary | Overall Change From Baseline to Week 26 in Active Joint Range of Motion (ROM) | 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. | Posted | Mean | Standard Deviation | Degrees | Baseline to Week 26 |
|
|
|
| Secondary | Overall Percent Change From Baseline to Week 26 in Urinary Glycosaminoglycan (GAG) Levels | Urinary Glycosaminoglycan (GAG) Levels: Concentration of GAG relative to creatinine in urine. A greater decrease in GAG level indicates a greater response. | Posted | Mean | Standard Deviation | ug/mg | Baseline to Week 26 |
|
|
|
| 0 |
| 23 |
| 23 |
| 23 |
| EG001 | Aldurazyme***Check Title*** | Aldurazyme***Check Description*** | 3 | 22 | 21 | 22 |
| Constipation | Gastrointestinal disorders | MedDRA 11.1 | Systematic Assessment |
|
| Pneumonia | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
|
| Sepsis | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
|
| Ventriculoperitoneal shunt malfunction | Injury, poisoning and procedural complications | MedDRA 11.1 | Systematic Assessment |
|
| Renal failure acute | Renal and urinary disorders | MedDRA 11.1 | Systematic Assessment |
|
| Aortic stenosis | Vascular disorders | MedDRA 11.1 | Systematic Assessment |
|
| Lymphadenopathy | Blood and lymphatic system disorders | MedDRA 11.1 | Systematic Assessment |
|
| Lymphatic disorder | Blood and lymphatic system disorders | MedDRA 11.1 | Systematic Assessment |
|
| Splenomegaly | Blood and lymphatic system disorders | MedDRA 11.1 | Systematic Assessment |
|
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA 11.1 | Systematic Assessment |
|
| Angina pectoris | Cardiac disorders | MedDRA 11.1 | Systematic Assessment |
|
| Arrhythmia | Cardiac disorders | MedDRA 11.1 | Systematic Assessment |
|
| Cardiac failure | Cardiac disorders | MedDRA 11.1 | Systematic Assessment |
|
| Tachycardia | Cardiac disorders | MedDRA 11.1 | Systematic Assessment |
|
| Ear pain | Ear and labyrinth disorders | MedDRA 11.1 | Systematic Assessment |
|
| Blepharitis | Eye disorders | MedDRA 11.1 | Systematic Assessment |
|
| Corneal opacity | Eye disorders | MedDRA 11.1 | Systematic Assessment |
|
| Eye discharge | Eye disorders | MedDRA 11.1 | Systematic Assessment |
|
| Eye pain | Eye disorders | MedDRA 11.1 | Systematic Assessment |
|
| Eye swelling | Eye disorders | MedDRA 11.1 | Systematic Assessment |
|
| Visual acuity reduced | Eye disorders | MedDRA 11.1 | Systematic Assessment |
|
| Abdominal discomfort | Gastrointestinal disorders | MedDRA 11.1 | Systematic Assessment |
|
| Abdominal distension | Gastrointestinal disorders | MedDRA 11.1 | Systematic Assessment |
|
| Abdominal pain | Gastrointestinal disorders | MedDRA 11.1 | Systematic Assessment |
|
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 11.1 | Systematic Assessment |
|
| Bowel movement irregularity | Gastrointestinal disorders | MedDRA 11.1 | Systematic Assessment |
|
| Chapped lips | Gastrointestinal disorders | MedDRA 11.1 | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | MedDRA 11.1 | Systematic Assessment |
|
| Dental discomfort | Gastrointestinal disorders | MedDRA 11.1 | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA 11.1 | Systematic Assessment |
|
| Duodenogastric reflux | Gastrointestinal disorders | MedDRA 11.1 | Systematic Assessment |
|
| Flatulence | Gastrointestinal disorders | MedDRA 11.1 | Systematic Assessment |
|
| Hypoaesthesia oral | Gastrointestinal disorders | MedDRA 11.1 | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA 11.1 | Systematic Assessment |
|
| Rectal haemorrhage | Gastrointestinal disorders | MedDRA 11.1 | Systematic Assessment |
|
| Stomach discomfort | Gastrointestinal disorders | MedDRA 11.1 | Systematic Assessment |
|
| Tooth discolouration | Gastrointestinal disorders | MedDRA 11.1 | Systematic Assessment |
|
| Toothache | Gastrointestinal disorders | MedDRA 11.1 | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA 11.1 | Systematic Assessment |
|
| Application site rash | General disorders | MedDRA 11.1 | Systematic Assessment |
|
| Catheter site related reaction | General disorders | MedDRA 11.1 | Systematic Assessment |
|
| Chest discomfort | General disorders | MedDRA 11.1 | Systematic Assessment |
|
| Chest pain | General disorders | MedDRA 11.1 | Systematic Assessment |
|
| Chills | General disorders | MedDRA 11.1 | Systematic Assessment |
|
| Face oedema | General disorders | MedDRA 11.1 | Systematic Assessment |
|
| Facial pain | General disorders | MedDRA 11.1 | Systematic Assessment |
|
| Fatigue | General disorders | MedDRA 11.1 | Systematic Assessment |
|
| Feeling hot | General disorders | MedDRA 11.1 | Systematic Assessment |
|
| Gait disturbance | General disorders | MedDRA 11.1 | Systematic Assessment |
|
| Gravitational oedema | General disorders | MedDRA 11.1 | Systematic Assessment |
|
| Hernia pain | General disorders | MedDRA 11.1 | Systematic Assessment |
|
| Influenza like illness | General disorders | MedDRA 11.1 | Systematic Assessment |
|
| Infusion site swelling | General disorders | MedDRA 11.1 | Systematic Assessment |
|
| Injection site pain | General disorders | MedDRA 11.1 | Systematic Assessment |
|
| Injection site reaction | General disorders | MedDRA 11.1 | Systematic Assessment |
|
| Oedema peripheral | General disorders | MedDRA 11.1 | Systematic Assessment |
|
| Pain | General disorders | MedDRA 11.1 | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA 11.1 | Systematic Assessment |
|
| Hepatomegaly | Hepatobiliary disorders | MedDRA 11.1 | Systematic Assessment |
|
| Hyperbilirubinaemia | Hepatobiliary disorders | MedDRA 11.1 | Systematic Assessment |
|
| Corneal graft rejection | Immune system disorders | MedDRA 11.1 | Systematic Assessment |
|
| Seasonal allergy | Immune system disorders | MedDRA 11.1 | Systematic Assessment |
|
| Abscess | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
|
| Catheter site infection | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
|
| Ear infection | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
|
| Fungal infection | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
|
| Influenza | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
|
| Laryngitis | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
|
| Lice infestation | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
|
| Localised infection | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
|
| Lower respiratory tract infection | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
|
| Otitis externa | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
|
| Otitis media | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
|
| Otitis media chronic | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
|
| Perirectal abscess | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
|
| Rhinitis | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
|
| Skin infection | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
|
| Sputum purulent | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
|
| Tinea pedis | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
|
| Urinary tract infection | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
|
| Viral infection | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
|
| Arthropod bite | Injury, poisoning and procedural complications | MedDRA 11.1 | Systematic Assessment |
|
| Contusion | Injury, poisoning and procedural complications | MedDRA 11.1 | Systematic Assessment |
|
| Fall | Injury, poisoning and procedural complications | MedDRA 11.1 | Systematic Assessment |
|
| Joint injury | Injury, poisoning and procedural complications | MedDRA 11.1 | Systematic Assessment |
|
| Procedural pain | Injury, poisoning and procedural complications | MedDRA 11.1 | Systematic Assessment |
|
| Thermal burn | Injury, poisoning and procedural complications | MedDRA 11.1 | Systematic Assessment |
|
| Wound | Injury, poisoning and procedural complications | MedDRA 11.1 | Systematic Assessment |
|
| Alanine aminotransferase increased | Investigations | MedDRA 11.1 | Systematic Assessment |
|
| Aspartate aminotransferase increased | Investigations | MedDRA 11.1 | Systematic Assessment |
|
| Bacteria urine | Investigations | MedDRA 11.1 | Systematic Assessment |
|
| Blood glucose increased | Investigations | MedDRA 11.1 | Systematic Assessment |
|
| Body temperature increased | Investigations | MedDRA 11.1 | Systematic Assessment |
|
| Brain scan abnormal | Investigations | MedDRA 11.1 | Systematic Assessment |
|
| Breath sounds abnormal | Investigations | MedDRA 11.1 | Systematic Assessment |
|
| Cardiac murmur | Investigations | MedDRA 11.1 | Systematic Assessment |
|
| Gallop rhythm present | Investigations | MedDRA 11.1 | Systematic Assessment |
|
| Intraocular pressure increased | Investigations | MedDRA 11.1 | Systematic Assessment |
|
| Liver function test abnormal | Investigations | MedDRA 11.1 | Systematic Assessment |
|
| Nitrite urine present | Investigations | MedDRA 11.1 | Systematic Assessment |
|
| Protein urine present | Investigations | MedDRA 11.1 | Systematic Assessment |
|
| Dehydration | Metabolism and nutrition disorders | MedDRA 11.1 | Systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 11.1 | Systematic Assessment |
|
| Arthropathy | Musculoskeletal and connective tissue disorders | MedDRA 11.1 | Systematic Assessment |
|
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 11.1 | Systematic Assessment |
|
| Foot deformity | Musculoskeletal and connective tissue disorders | MedDRA 11.1 | Systematic Assessment |
|
| Joint crepitation | Musculoskeletal and connective tissue disorders | MedDRA 11.1 | Systematic Assessment |
|
| Joint instability | Musculoskeletal and connective tissue disorders | MedDRA 11.1 | Systematic Assessment |
|
| Joint stiffness | Musculoskeletal and connective tissue disorders | MedDRA 11.1 | Systematic Assessment |
|
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA 11.1 | Systematic Assessment |
|
| Muscle tightness | Musculoskeletal and connective tissue disorders | MedDRA 11.1 | Systematic Assessment |
|
| Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | MedDRA 11.1 | Systematic Assessment |
|
| Musculoskeletal discomfort | Musculoskeletal and connective tissue disorders | MedDRA 11.1 | Systematic Assessment |
|
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA 11.1 | Systematic Assessment |
|
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 11.1 | Systematic Assessment |
|
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA 11.1 | Systematic Assessment |
|
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 11.1 | Systematic Assessment |
|
| Soft tissue disorder | Musculoskeletal and connective tissue disorders | MedDRA 11.1 | Systematic Assessment |
|
| Dizziness | Nervous system disorders | MedDRA 11.1 | Systematic Assessment |
|
| Dizziness postural | Nervous system disorders | MedDRA 11.1 | Systematic Assessment |
|
| Grand mal convulsion | Nervous system disorders | MedDRA 11.1 | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA 11.1 | Systematic Assessment |
|
| Hyperaesthesia | Nervous system disorders | MedDRA 11.1 | Systematic Assessment |
|
| Hyperreflexia | Nervous system disorders | MedDRA 11.1 | Systematic Assessment |
|
| Hypoaesthesia | Nervous system disorders | MedDRA 11.1 | Systematic Assessment |
|
| Migraine | Nervous system disorders | MedDRA 11.1 | Systematic Assessment |
|
| Paraesthesia | Nervous system disorders | MedDRA 11.1 | Systematic Assessment |
|
| Sinus headache | Nervous system disorders | MedDRA 11.1 | Systematic Assessment |
|
| Somnolence | Nervous system disorders | MedDRA 11.1 | Systematic Assessment |
|
| Syncope | Nervous system disorders | MedDRA 11.1 | Systematic Assessment |
|
| Syncope vasovagal | Nervous system disorders | MedDRA 11.1 | Systematic Assessment |
|
| Agitation | Psychiatric disorders | MedDRA 11.1 | Systematic Assessment |
|
| Anxiety | Psychiatric disorders | MedDRA 11.1 | Systematic Assessment |
|
| Insomnia | Psychiatric disorders | MedDRA 11.1 | Systematic Assessment |
|
| Restlessness | Psychiatric disorders | MedDRA 11.1 | Systematic Assessment |
|
| Dysmenorrhoea | Reproductive system and breast disorders | MedDRA 11.1 | Systematic Assessment |
|
| Menstrual discomfort | Reproductive system and breast disorders | MedDRA 11.1 | Systematic Assessment |
|
| Vaginal discharge | Reproductive system and breast disorders | MedDRA 11.1 | Systematic Assessment |
|
| Choking | Respiratory, thoracic and mediastinal disorders | MedDRA 11.1 | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 11.1 | Systematic Assessment |
|
| Dry throat | Respiratory, thoracic and mediastinal disorders | MedDRA 11.1 | Systematic Assessment |
|
| Dysphonia | Respiratory, thoracic and mediastinal disorders | MedDRA 11.1 | Systematic Assessment |
|
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 11.1 | Systematic Assessment |
|
| Hyperventilation | Respiratory, thoracic and mediastinal disorders | MedDRA 11.1 | Systematic Assessment |
|
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA 11.1 | Systematic Assessment |
|
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 11.1 | Systematic Assessment |
|
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 11.1 | Systematic Assessment |
|
| Pulmonary congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 11.1 | Systematic Assessment |
|
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 11.1 | Systematic Assessment |
|
| Respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 11.1 | Systematic Assessment |
|
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 11.1 | Systematic Assessment |
|
| Rhonchi | Respiratory, thoracic and mediastinal disorders | MedDRA 11.1 | Systematic Assessment |
|
| Sneezing | Respiratory, thoracic and mediastinal disorders | MedDRA 11.1 | Systematic Assessment |
|
| Wheezing | Respiratory, thoracic and mediastinal disorders | MedDRA 11.1 | Systematic Assessment |
|
| Acne | Skin and subcutaneous tissue disorders | MedDRA 11.1 | Systematic Assessment |
|
| Alopecia | Skin and subcutaneous tissue disorders | MedDRA 11.1 | Systematic Assessment |
|
| Decubitus ulcer | Skin and subcutaneous tissue disorders | MedDRA 11.1 | Systematic Assessment |
|
| Dermal cyst | Skin and subcutaneous tissue disorders | MedDRA 11.1 | Systematic Assessment |
|
| Dermatitis acneiform | Skin and subcutaneous tissue disorders | MedDRA 11.1 | Systematic Assessment |
|
| Dermatitis allergic | Skin and subcutaneous tissue disorders | MedDRA 11.1 | Systematic Assessment |
|
| Dry skin | Skin and subcutaneous tissue disorders | MedDRA 11.1 | Systematic Assessment |
|
| Erythema | Skin and subcutaneous tissue disorders | MedDRA 11.1 | Systematic Assessment |
|
| Heat rash | Skin and subcutaneous tissue disorders | MedDRA 11.1 | Systematic Assessment |
|
| Hyperhidrosis | Skin and subcutaneous tissue disorders | MedDRA 11.1 | Systematic Assessment |
|
| Ingrowing nail | Skin and subcutaneous tissue disorders | MedDRA 11.1 | Systematic Assessment |
|
| Pain of skin | Skin and subcutaneous tissue disorders | MedDRA 11.1 | Systematic Assessment |
|
| Petechiae | Skin and subcutaneous tissue disorders | MedDRA 11.1 | Systematic Assessment |
|
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 11.1 | Systematic Assessment |
|
| Psoriasis | Skin and subcutaneous tissue disorders | MedDRA 11.1 | Systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | MedDRA 11.1 | Systematic Assessment |
|
| Rash macular | Skin and subcutaneous tissue disorders | MedDRA 11.1 | Systematic Assessment |
|
| Skin disorder | Skin and subcutaneous tissue disorders | MedDRA 11.1 | Systematic Assessment |
|
| Skin swelling | Skin and subcutaneous tissue disorders | MedDRA 11.1 | Systematic Assessment |
|
| Ear tube removal | Surgical and medical procedures | MedDRA 11.1 | Systematic Assessment |
|
| Flushing | Vascular disorders | MedDRA 11.1 | Systematic Assessment |
|
| Hot flush | Vascular disorders | MedDRA 11.1 | Systematic Assessment |
|
| Hypertension | Vascular disorders | MedDRA 11.1 | Systematic Assessment |
|
| Hypotension | Vascular disorders | MedDRA 11.1 | Systematic Assessment |
|
| Poor venous access | Vascular disorders | MedDRA 11.1 | Systematic Assessment |
|
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.
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D016464 | Lysosomal Storage Diseases |
| D017520 | Mucinoses |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| Overall change from Baseline to Week 26 |
|
| Overall change from Baseline to Week 26 |
|
| Overall percent change from Baseline to Week 26 |
|
| Overall change from Baseline to Week 26 |
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| Overall change from baseline to week 26 |
|
| Overall Percentage of Change in GAG Level |
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