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| Name | Class |
|---|---|
| BioMarin/Genzyme LLC | INDUSTRY |
The main purpose of this study is to evaluate differences in the pharmacodynamic response of 4 Aldurazyme® (laronidase) dose regimens in patients with Mucopolysaccharidosis I (MPS I).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 1 | Active Comparator | 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. |
|
| 2 | Active Comparator | 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. |
|
| 3 | Active Comparator | 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. |
|
| 4 | Active Comparator | 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Aldurazyme (Recombinant Human Alpha-L-Iduronidase) | Biological | 0.58 mg/kg every week |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Percent Change From Baseline to Week 26 in Urinary Glycosaminoglycan (GAG) Level | Urinary GAG Level - Concentration of GAG relative to creatinine in urine. A greater decrease in GAG level indicates a greater response. | Baseline to 26 Weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Percent Change From Baseline to Week 26 in Liver Organ Volume | A greater decrease in liver volume indicates a greater response. | Baseline to 26 Weeks |
| Change From Baseline to Week 26 in Six Minute Walk Test (6MWT) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Medical Information | Genzyme, a Sanofi Company | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Hospital Infantil Joana de Gusmao | Florianópolis | Santa Catarina | CEP 88025-601 | Brazil | ||
| Universidade Federal de Minas Gerais |
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| ID | Title | Description |
|---|---|---|
| FG000 | 0.58 mg/kg Aldurazyme Every Week | 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. |
| FG001 | 1.2 mg/kg Aldurazyme Every Week |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Aldurazyme (Recombinant Human Alpha-L-Iduronidase) |
| Biological |
1.2 mg/kg every week |
|
| Aldurazyme (Recombinant Human Alpha-L-Iduronidase) | Biological | 1.2 mg/kg every other week |
|
| Aldurazyme (Recombinant Human Alpha-L-Iduronidase) | Biological | 1.8 mg/kg every other week |
|
Six Minute Walk Test: Distance walked (measured in Meters) in 6 minutes. A longer distance indicates a greater response.
| Baseline to 26 Weeks |
| Belo Horizonte |
| CEP 30130-100 |
| Brazil |
| Hospital de Clinical de Porto Alegre | Porto Alegre | CEP 90035-003 | Brazil |
| Universidade Federal de Sao Paulo | São Paulo | CEP 04023-062 | Brazil |
| Division of Clinical and Metabolic Genetics | Toronto | Ontario | M5G 1X8 | Canada |
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.
| FG002 | 1.2 mg/kg Aldurazyme Every 2 Weeks | 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. |
| FG003 | 1.8 mg/kg Aldurazyme Every 2 Weeks | 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. |
| COMPLETED |
|
| NOT COMPLETED |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | 0.58 mg/kg Aldurazyme Every Week | 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. |
| BG001 | 1.2 mg/kg Aldurazyme Every Week | 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. |
| BG002 | 1.2 mg/kg Aldurazyme Every 2 Weeks | 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. |
| BG003 | 1.8 mg/kg Aldurazyme Every 2 Weeks | 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. |
| BG004 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Study Specific Race/Ethnicity | Number | participants |
|
| 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 | Percent Change From Baseline to Week 26 in Urinary Glycosaminoglycan (GAG) Level | Urinary GAG Level - Concentration of GAG relative to creatinine in urine. A greater decrease in GAG level indicates a greater response. | 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. | Posted | Mean | 95% Confidence Interval | Percentage of Change in GAG Level | Baseline to 26 Weeks |
|
|
| ||||||||||||||||||||||||||||||||||
| Secondary | Percent Change From Baseline to Week 26 in Liver Organ Volume | A greater decrease in liver volume indicates a greater response. | The analysis was intention to treat. | Posted | Mean | 95% Confidence Interval | Percentage of Change in Liver Volume | Baseline to 26 Weeks |
| ||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline to Week 26 in Six Minute Walk Test (6MWT) | Six Minute Walk Test: Distance walked (measured in Meters) in 6 minutes. A longer distance indicates a greater response. | The analysis was intention to treat. | Posted | Mean | 95% Confidence Interval | meters | Baseline to 26 Weeks |
|
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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 | Aldurazyme|Every Other|Week 1.2 mg/kg***Check Title*** | Aldurazyme|Every Other|Week 1.2 mg/kg***Check Description*** | 2 | 8 | 8 | 8 | ||
| EG001 | Aldurazyme|Every Other|Week 1.8 mg/kg***Check Title*** | Aldurazyme|Every Other|Week 1.8 mg/kg***Check Description*** | 1 | 9 | 9 | 9 | ||
| EG002 | Aldurazyme|Weekly|0.58 mg/kg***Check Title*** | Aldurazyme|Weekly|0.58 mg/kg***Check Description*** | 1 | 8 | 8 | 8 | ||
| EG003 | Aldurazyme|Weekly|1.2 mg/kg***Check Title*** | Aldurazyme|Weekly|1.2 mg/kg***Check Description*** | 2 | 8 | 7 | 8 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal distension | Gastrointestinal disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Abasia | General disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Acute sinusitis | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
| |
| Otitis media acute | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
| |
| Femur fracture | Injury, poisoning and procedural complications | MedDRA 11.1 | Systematic Assessment |
| |
| Pulmonary oedema | Respiratory, thoracic and mediastinal disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Respiratory failure | Respiratory, thoracic and mediastinal 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 |
| |
| Eosinophilia | Blood and lymphatic system disorders | MedDRA 11.1 | Systematic Assessment |
| |
| White blood cell disorder | Blood and lymphatic system disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Cyanosis | Cardiac disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Deafness | Ear and labyrinth disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Ear pain | Ear and labyrinth disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Otorrhoea | Ear and labyrinth disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Conjunctivitis | Eye disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Lacrimation increased | Eye disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Strabismus | Eye disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Vision blurred | Eye disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Visual acuity reduced | Eye 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 |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Flatulence | Gastrointestinal disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Mouth ulceration | Gastrointestinal disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Oral disorder | Gastrointestinal disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Perianal erythema | Gastrointestinal disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Stomach discomfort | Gastrointestinal disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Teething | Gastrointestinal disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Abasia | General disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Application site dermatitis | General disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Catheter site pain | General disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Catheter site rash | General disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Catheter site related reaction | General disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Chest pain | General disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Chills | General disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Disease progression | General disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Feeling hot | General disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Hernia obstructive | General disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Hernia pain | General disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Hyperthermia | General disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Infusion site pruritus | General disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Infusion site rash | General disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Irritability | General disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Localised oedema | General disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Oedema | 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 |
| |
| Vessel puncture site reaction | General disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Acarodermatitis | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
| |
| Conjunctivitis viral | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
| |
| Dermatitis infected | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
| |
| Enterobiasis | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
| |
| Flea infestation | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
| |
| Genital candidiasis | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
| |
| Herpes simplex | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
| |
| Hordeolum | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
| |
| Infection parasitic | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
| |
| Influenza | 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 |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
| |
| Oral herpes | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
| |
| Otitis media | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
| |
| Otitis media acute | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
| |
| Paronychia | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
| |
| Streptococcal impetigo | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
| |
| Tonsillitis | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
| |
| Viral diarrhoea | Infections and infestations | MedDRA 11.1 | Systematic Assessment |
| |
| Arthropod bite | Injury, poisoning and procedural complications | MedDRA 11.1 | Systematic Assessment |
| |
| Excoriation | Injury, poisoning and procedural complications | MedDRA 11.1 | Systematic Assessment |
| |
| Limb injury | Injury, poisoning and procedural complications | MedDRA 11.1 | Systematic Assessment |
| |
| Blood alkaline phosphatase increased | Investigations | MedDRA 11.1 | Systematic Assessment |
| |
| Blood iron decreased | Investigations | MedDRA 11.1 | Systematic Assessment |
| |
| Blood lactate dehydrogenase increased | Investigations | MedDRA 11.1 | Systematic Assessment |
| |
| Blood pressure decreased | Investigations | MedDRA 11.1 | Systematic Assessment |
| |
| Breath sounds abnormal | Investigations | MedDRA 11.1 | Systematic Assessment |
| |
| Lymphocyte count increased | Investigations | MedDRA 11.1 | Systematic Assessment |
| |
| Neutrophil count decreased | Investigations | MedDRA 11.1 | Systematic Assessment |
| |
| Oxygen saturation decreased | Investigations | MedDRA 11.1 | Systematic Assessment |
| |
| Platelet count decreased | Investigations | MedDRA 11.1 | Systematic Assessment |
| |
| Platelet count increased | Investigations | MedDRA 11.1 | Systematic Assessment |
| |
| Urine leukocyte esterase positive | Investigations | MedDRA 11.1 | Systematic Assessment |
| |
| White blood cell count increased | Investigations | MedDRA 11.1 | Systematic Assessment |
| |
| Anorexia | Metabolism and nutrition disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Pica | Metabolism and nutrition disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Polydipsia | Metabolism and nutrition disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Weight gain poor | Metabolism and nutrition disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Groin pain | Musculoskeletal and connective tissue disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Musculoskeletal stiffness | 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 |
| |
| Synovial cyst | Musculoskeletal and connective tissue disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Convulsion | Nervous system disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Motor dysfunction | Nervous system disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Paraesthesia | Nervous system disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Tremor | Nervous system disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Abnormal behaviour | Psychiatric disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Dysphoria | Psychiatric disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Inappropriate affect | Psychiatric disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Leukocyturia | Renal and urinary disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Dysmenorrhoea | Reproductive system and breast disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Genital erythema | Reproductive system and breast disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Adenoidal hypertrophy | Respiratory, thoracic and mediastinal disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Allergic sinusitis | Respiratory, thoracic and mediastinal disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Bronchospasm | Respiratory, thoracic and mediastinal disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Dysphonia | Respiratory, thoracic and mediastinal disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Nocturnal dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Pharyngeal erythema | Respiratory, thoracic and mediastinal disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Pulmonary hypertension | Respiratory, thoracic and mediastinal disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Rhinitis allergic | Respiratory, thoracic and mediastinal disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Sneezing | Respiratory, thoracic and mediastinal disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Tachypnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Wheezing | Respiratory, thoracic and mediastinal disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Decubitus ulcer | Skin and subcutaneous tissue disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Dermatitis | Skin and subcutaneous tissue disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Dermatitis allergic | Skin and subcutaneous tissue disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Dermatitis atopic | Skin and subcutaneous tissue disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Dermatitis contact | Skin and subcutaneous tissue disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Dermatitis diaper | Skin and subcutaneous tissue disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Dry skin | Skin and subcutaneous tissue disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Eczema | 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 |
| |
| Milia | Skin and subcutaneous tissue disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Periorbital oedema | 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 |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Rash papular | Skin and subcutaneous tissue disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Skin exfoliation | Skin and subcutaneous tissue disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Skin lesion | Skin and subcutaneous tissue disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Swelling face | Skin and subcutaneous tissue disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Pallor | Vascular disorders | MedDRA 11.1 | Systematic Assessment |
| |
| Poor venous access | Vascular disorders | MedDRA 11.1 | Systematic Assessment |
|
This study is limited by its small sample size and ability to detect small differences in urinary GAG levels between groups.
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.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Genzyme Medical Information | Genzyme Corporation | 800-745-4447 |
| ID | Term |
|---|---|
| D008059 | Mucopolysaccharidosis I |
| ID | Term |
|---|---|
| D009083 | Mucopolysaccharidoses |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| 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 |
Not provided
Not provided
| ID | Term |
|---|---|
| D007068 | Iduronidase |
| ID | Term |
|---|---|
| D006026 | Glycoside Hydrolases |
| D006867 | Hydrolases |
| D004798 | Enzymes |
| D045762 | Enzymes and Coenzymes |
Not provided
Not provided
| Male |
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| Black |
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| Hispanic |
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| Asian |
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| Other |
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