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The purpose of this study is to evaluate the long-term efficacy and safety of rhASB treatment in patients with Mucopolysaccharidosis VI.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| rhASB/rhASB | Other | N-acetylgalactosamine 4-sulfatase |
|
| Placebo/rhASB | Other |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| N-acetylgalactosamine 4-sulfatase | Drug |
|
| |
| Placebo/rhASB |
| Measure | Description | Time Frame |
|---|---|---|
| 12-Minute Walk Test | Mean change in meters walked in 12 minutes. Mean change is the mean difference between the 12-Minute Walk Test at 96 weeks and that measured before first ever treatment with rhASB. For the rhASB/rhASB group, mean change is calculated for Week 96 - Baseline. For the placebo/rhASB group, mean change is calculated for Week 96 - Week 24. | Baseline of ASB-03-05 through week 96 of ASB-03-06 |
| Measure | Description | Time Frame |
|---|---|---|
| 3-Minute Stair Climb | Mean change in number of stairs climbed per minute in 3 minutes. Mean change is the mean difference between the 3-Minute Stair Climb at 96 weeks and that measured before first ever treatment with rhASB. Mean change is calculated for Week 96 - Baseline for the rhASB/rhASB group and for Week 96 - Week 24 for the placebo/rhASB group. | Baseline ASB-03-05 through week 96 of ASB-03-06. |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| BioMarin Pharmaceutical Inc. | Novato | California | 94949 | 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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Global study sites were hospitals and medical clinics.
First Enrollment: 05FEB04
Last Dose: 28SEP06
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| ID | Title | Description |
|---|---|---|
| FG000 | rhASB/rhASB | Patients received rhASB in the double-blind study(ASB-03-05, NCT00067470)and continued to receive rhASB in the extension study(ASB-03-06). |
| FG001 | Placebo/rhASB | Patients received placebo in the double-blind study (ASB-03-05, NCT00067470)then received rhASB in the extension study (ASB-03-06). |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | rhASB/rhASB | Patients received rhASB in the double-blind study(ASB-03-05, NCT00067470)and continued to receive rhASB in the extension study(ASB-03-06). |
| BG001 | Placebo/rhASB | Patients received placebo in the double-blind study (ASB-03-05, NCT00067470)then received rhASB in the extension study (ASB-03-06). |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age Continuous | Demographic and baseline characteristics were recorded at baseline in the double-blind study, ASB-03-05. |
| 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 | 12-Minute Walk Test | Mean change in meters walked in 12 minutes. Mean change is the mean difference between the 12-Minute Walk Test at 96 weeks and that measured before first ever treatment with rhASB. For the rhASB/rhASB group, mean change is calculated for Week 96 - Baseline. For the placebo/rhASB group, mean change is calculated for Week 96 - Week 24. | The efficacy analysis included all subjects who continued in the extension study (ASB-03-06) except the 1 subject from the rhASB/rhASB group and 1 subject from the placebo/rhASB group who missed the Week 96 measurement. | Posted | Mean | Standard Deviation | meters | Baseline of ASB-03-05 through week 96 of ASB-03-06 |
|
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | All Participants | rhASB/rhASB and placebo/rhASB groups were combined for analysis. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Body increased temperature | Investigations | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Ear infection | Infections and infestations | MedDRA (6.1) | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Information Services | BioMarin Pharmaceutical Inc. | 8009834587 | medinfo@bmrn.com |
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| ID | Term |
|---|---|
| D009087 | Mucopolysaccharidosis VI |
| ID | Term |
|---|---|
| D009083 | Mucopolysaccharidoses |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
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| ID | Term |
|---|---|
| D020051 | N-Acetylgalactosamine-4-Sulfatase |
| ID | Term |
|---|---|
| D001192 | Arylsulfatases |
| D013429 | Sulfatases |
| D004950 | Esterases |
| D006867 | Hydrolases |
| D004798 |
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| Drug |
|
| Change in Urinary Glycosaminoglycans (GAG) Level | Mean change in urinary GAG level for the first 72 weeks of rhASB treatment. For the rhASB/rhASB group, mean change was calculated for Week 72 -Baseline. For the placebo/rhASB, mean change was calculated for Week 96 - Week 24. | 72 weeks |
| BG002 | Total | Total of all reporting groups |
| Mean |
| Standard Deviation |
| years |
|
| Sex: Female, Male | Demographic and baseline characteristics were recorded at baseline in the double-blind study, ASB-03-05. | Count of Participants | Participants |
|
| Race/Ethnicity, Customized | Demographic and baseline characteristics were recorded at baseline in the double-blind study, ASB-03-05. | Number | participants |
|
| Region of Enrollment | Demographic and baseline characteristics were recorded at baseline in the double-blind study, ASB-03-05. | Number | participants |
|
| OG001 | Placebo/rhASB | Patients received placebo in the double-blind study (ASB-03-05, NCT00067470)then received rhASB in the extension study (ASB-03-06). |
|
|
| Secondary | 3-Minute Stair Climb | Mean change in number of stairs climbed per minute in 3 minutes. Mean change is the mean difference between the 3-Minute Stair Climb at 96 weeks and that measured before first ever treatment with rhASB. Mean change is calculated for Week 96 - Baseline for the rhASB/rhASB group and for Week 96 - Week 24 for the placebo/rhASB group. | The efficacy analysis included all subjects who continued in the extension study (ASB-03-06) except 1 subject from the placebo/rhASB group who missed the Week 96 measurement. | Posted | Mean | Standard Deviation | stairs/min | Baseline ASB-03-05 through week 96 of ASB-03-06. |
|
|
|
| Secondary | Change in Urinary Glycosaminoglycans (GAG) Level | Mean change in urinary GAG level for the first 72 weeks of rhASB treatment. For the rhASB/rhASB group, mean change was calculated for Week 72 -Baseline. For the placebo/rhASB, mean change was calculated for Week 96 - Week 24. | rhASB/rhASB and placebo/rhASB groups were combined for the analysis, representing 72 weeks of rhASB treatment in each group. | Posted | Mean | Standard Deviation | microgram/mg creatinine | 72 weeks |
|
|
|
| 14 |
| 38 |
| 38 |
| 38 |
| Broncopneumopathy | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Cardiac failure | Cardiac disorders | Systematic Assessment |
|
| Cervical cord compression | Nervous system disorders | Systematic Assessment |
|
| Cervical myelopathy | Nervous system disorders | Systematic Assessment |
|
| Convulsion | Nervous system disorders | Systematic Assessment |
|
| Corneal Abscess | Infections and infestations | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Cyst | General disorders | Systematic Assessment |
|
| Dizziness | Nervous system disorders | Systematic Assessment |
|
| Haemorrhage | Vascular disorders | Systematic Assessment |
|
| Hip Dysplasia | Congenital, familial and genetic disorders | Systematic Assessment |
|
| Hip surgery | Surgical and medical procedures | Systematic Assessment |
|
| Inguinal hernia | Gastrointestinal disorders | Systematic Assessment |
|
| Inguinal hernia obstructive | Gastrointestinal disorders | Systematic Assessment |
|
| Intracranial pressure increased | Nervous system disorders | Systematic Assessment |
|
| Intraocular pressure increased | Investigations | Systematic Assessment |
|
| Mastoiditis | Infections and infestations | Systematic Assessment |
|
| Mitral valve stenosis | Cardiac disorders | Systematic Assessment |
|
| Osteotomy | Surgical and medical procedures | Systematic Assessment |
|
| Pneumonia | Infections and infestations | Systematic Assessment |
|
| Pneumonitis | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Respiratory distress | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Spinal cord compression | Nervous system disorders | Systematic Assessment |
|
| Staphylococcal sepsis | Infections and infestations | Systematic Assessment |
|
| Tension headache | Nervous system disorders | Systematic Assessment |
|
| Tracheal stenosis | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Vertigo | Ear and labyrinth disorders | Systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA (6.1) | Systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA (6.1) | Systematic Assessment |
|
| Gastroenteritis | Infections and infestations | MedDRA (6.1) | Systematic Assessment |
|
| Bronchitis | Infections and infestations | MedDRA (6.1) | Systematic Assessment |
|
| Otitis externa | Infections and infestations | MedDRA (6.1) | Systematic Assessment |
|
| Influenza | Infections and infestations | MedDRA (6.1) | Systematic Assessment |
|
| Otitis media acute | Infections and infestations | MedDRA (6.1) | Systematic Assessment |
|
| Sinusitis | Infections and infestations | MedDRA (6.1) | Systematic Assessment |
|
| Hordeolum | Infections and infestations | MedDRA (6.1) | Systematic Assessment |
|
| Pharyngitis | Infections and infestations | MedDRA (6.1) | Systematic Assessment |
|
| Laryngitis | Infections and infestations | MedDRA (6.1) | Systematic Assessment |
|
| Pneumonia | Infections and infestations | MedDRA (6.1) | Systematic Assessment |
|
| Dental Caries | Infections and infestations | MedDRA (6.1) | Systematic Assessment |
|
| Respiratory tract infection | Infections and infestations | MedDRA (6.1) | Systematic Assessment |
|
| Tinea pedis | Infections and infestations | MedDRA (6.1) | Systematic Assessment |
|
| Otitis media | Infections and infestations | MedDRA (6.1) | Systematic Assessment |
|
| Varicella | Infections and infestations | MedDRA (6.1) | Systematic Assessment |
|
| Bronchopneumonia | Infections and infestations | MedDRA (6.1) | Systematic Assessment |
|
| Folliculitis | Infections and infestations | MedDRA (6.1) | Systematic Assessment |
|
| Fungal infection | Infections and infestations | MedDRA (6.1) | Systematic Assessment |
|
| Furuncle | Infections and infestations | MedDRA (6.1) | Systematic Assessment |
|
| Lower respiratory tract infection | Infections and infestations | MedDRA (6.1) | Systematic Assessment |
|
| Subcutaneous abscess | Infections and infestations | MedDRA (6.1) | Systematic Assessment |
|
| Tonsillitis | Infections and infestations | MedDRA (6.1) | Systematic Assessment |
|
| Tooth abscess | Infections and infestations | MedDRA (6.1) | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (6.1) | Systematic Assessment |
|
| Pharyngolaryngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (6.1) | Systematic Assessment |
|
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA (6.1) | Systematic Assessment |
|
| Bronchospasm | Respiratory, thoracic and mediastinal disorders | MedDRA (6.1) | Systematic Assessment |
|
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (6.1) | Systematic Assessment |
|
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (6.1) | Systematic Assessment |
|
| Pulmonary hypertention | Respiratory, thoracic and mediastinal disorders | MedDRA (6.1) | Systematic Assessment |
|
| Rhinitis | Respiratory, thoracic and mediastinal disorders | MedDRA (6.1) | Systematic Assessment |
|
| Wheezing | Respiratory, thoracic and mediastinal disorders | MedDRA (6.1) | Systematic Assessment |
|
| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA (6.1) | Systematic Assessment |
|
| Restrictive pulmonary disease | Respiratory, thoracic and mediastinal disorders | MedDRA (6.1) | Systematic Assessment |
|
| Apnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (6.1) | Systematic Assessment |
|
| Chest pain | Respiratory, thoracic and mediastinal disorders | MedDRA (6.1) | Systematic Assessment |
|
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA (6.1) | Systematic Assessment |
|
| Increased bronchial secretion | Respiratory, thoracic and mediastinal disorders | MedDRA (6.1) | Systematic Assessment |
|
| Oropharyngeal swelling | Respiratory, thoracic and mediastinal disorders | MedDRA (6.1) | Systematic Assessment |
|
| Tachypnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (6.1) | Systematic Assessment |
|
| Upper respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (6.1) | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA (6.1) | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA (6.1) | Systematic Assessment |
|
| Abdominal pain | Gastrointestinal disorders | MedDRA (6.1) | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA (6.1) | Systematic Assessment |
|
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (6.1) | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | MedDRA (6.1) | Systematic Assessment |
|
| Toothache | Gastrointestinal disorders | MedDRA (6.1) | Systematic Assessment |
|
| Dyspepsia | Gastrointestinal disorders | MedDRA (6.1) | Systematic Assessment |
|
| Abdominal distension | Gastrointestinal disorders | MedDRA (6.1) | Systematic Assessment |
|
| Inguinal hernia | Gastrointestinal disorders | MedDRA (6.1) | Systematic Assessment |
|
| Mouth ulceration | Gastrointestinal disorders | MedDRA (6.1) | Systematic Assessment |
|
| Stomatitis | Gastrointestinal disorders | MedDRA (6.1) | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA (6.1) | Systematic Assessment |
|
| Dizziness | Nervous system disorders | MedDRA (6.1) | Systematic Assessment |
|
| Paraesthesia | Nervous system disorders | MedDRA (6.1) | Systematic Assessment |
|
| Hyperreflexia | Nervous system disorders | MedDRA (6.1) | Systematic Assessment |
|
| Hypoaesthenia | Nervous system disorders | MedDRA (6.1) | Systematic Assessment |
|
| Tremor | Nervous system disorders | MedDRA (6.1) | Systematic Assessment |
|
| Cervical myelopathy | Nervous system disorders | MedDRA (6.1) | Systematic Assessment |
|
| Migrane | Nervous system disorders | MedDRA (6.1) | Systematic Assessment |
|
| Somnolence | Nervous system disorders | MedDRA (6.1) | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA (6.1) | Systematic Assessment |
|
| Fatigue | General disorders | MedDRA (6.1) | Systematic Assessment |
|
| Influenza like illness | General disorders | MedDRA (6.1) | Systematic Assessment |
|
| Chest pain | General disorders | MedDRA (6.1) | Systematic Assessment |
|
| Pain | General disorders | MedDRA (6.1) | Systematic Assessment |
|
| Hernia pain | General disorders | MedDRA (6.1) | Systematic Assessment |
|
| Infusion site pain | General disorders | MedDRA (6.1) | Systematic Assessment |
|
| Oedema peripheral | General disorders | MedDRA (6.1) | Systematic Assessment |
|
| Rigors | General disorders | MedDRA (6.1) | Systematic Assessment |
|
| Athralgia | Musculoskeletal and connective tissue disorders | MedDRA (6.1) | Systematic Assessment |
|
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (6.1) | Systematic Assessment |
|
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (6.1) | Systematic Assessment |
|
| Bone pain | Musculoskeletal and connective tissue disorders | MedDRA (6.1) | Systematic Assessment |
|
| Joint stiffness | Musculoskeletal and connective tissue disorders | MedDRA (6.1) | Systematic Assessment |
|
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA (6.1) | Systematic Assessment |
|
| Hip deformity | Musculoskeletal and connective tissue disorders | MedDRA (6.1) | Systematic Assessment |
|
| Groin pain | Musculoskeletal and connective tissue disorders | MedDRA (6.1) | Systematic Assessment |
|
| Intervertibral disc disorder | Musculoskeletal and connective tissue disorders | MedDRA (6.1) | Systematic Assessment |
|
| Joint contracture | Musculoskeletal and connective tissue disorders | MedDRA (6.1) | Systematic Assessment |
|
| Kyphoscoliosis | Musculoskeletal and connective tissue disorders | MedDRA (6.1) | Systematic Assessment |
|
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA (6.1) | Systematic Assessment |
|
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA (6.1) | Systematic Assessment |
|
| Rash papular | Skin and subcutaneous tissue disorders | MedDRA (6.1) | Systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | MedDRA (6.1) | Systematic Assessment |
|
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA (6.1) | Systematic Assessment |
|
| Erythema | Skin and subcutaneous tissue disorders | MedDRA (6.1) | Systematic Assessment |
|
| Prurigo | Skin and subcutaneous tissue disorders | MedDRA (6.1) | Systematic Assessment |
|
| Acne | Skin and subcutaneous tissue disorders | MedDRA (6.1) | Systematic Assessment |
|
| Eczema | Skin and subcutaneous tissue disorders | MedDRA (6.1) | Systematic Assessment |
|
| Exanthem | Skin and subcutaneous tissue disorders | MedDRA (6.1) | Systematic Assessment |
|
| Face oedema | Skin and subcutaneous tissue disorders | MedDRA (6.1) | Systematic Assessment |
|
| Pityriasis rosea | Skin and subcutaneous tissue disorders | MedDRA (6.1) | Systematic Assessment |
|
| Rash erythematous | Skin and subcutaneous tissue disorders | MedDRA (6.1) | Systematic Assessment |
|
| Conjunctivitis | Eye disorders | MedDRA (6.1) | Systematic Assessment |
|
| Eye irritation | Eye disorders | MedDRA (6.1) | Systematic Assessment |
|
| Eye pain | Eye disorders | MedDRA (6.1) | Systematic Assessment |
|
| Eye redness | Eye disorders | MedDRA (6.1) | Systematic Assessment |
|
| Visual acuity reduced | Eye disorders | MedDRA (6.1) | Systematic Assessment |
|
| Eyelid oedema | Eye disorders | MedDRA (6.1) | Systematic Assessment |
|
| Glaucoma | Eye disorders | MedDRA (6.1) | Systematic Assessment |
|
| Keratoconjunctivitis sicca | Eye disorders | MedDRA (6.1) | Systematic Assessment |
|
| Ear pain | Ear and labyrinth disorders | MedDRA (6.1) | Systematic Assessment |
|
| Hypoacusis | Ear and labyrinth disorders | MedDRA (6.1) | Systematic Assessment |
|
| Otorrhoea | Ear and labyrinth disorders | MedDRA (6.1) | Systematic Assessment |
|
| Cerumen impaction | Ear and labyrinth disorders | MedDRA (6.1) | Systematic Assessment |
|
| Post procedural pain | Injury, poisoning and procedural complications | MedDRA (6.1) | Systematic Assessment |
|
| Head injury | Injury, poisoning and procedural complications | MedDRA (6.1) | Systematic Assessment |
|
| Procedural site reaction | Injury, poisoning and procedural complications | MedDRA (6.1) | Systematic Assessment |
|
| Excoriation | Injury, poisoning and procedural complications | MedDRA (6.1) | Systematic Assessment |
|
| Fall | Injury, poisoning and procedural complications | MedDRA (6.1) | Systematic Assessment |
|
| Joint injury | Injury, poisoning and procedural complications | MedDRA (6.1) | Systematic Assessment |
|
| Hypotension | Vascular disorders | MedDRA (6.1) | Systematic Assessment |
|
| Poor venous access | Vascular disorders | MedDRA (6.1) | Systematic Assessment |
|
| Haematoma | Vascular disorders | MedDRA (6.1) | Systematic Assessment |
|
| Hyperaemia | Vascular disorders | MedDRA (6.1) | Systematic Assessment |
|
| Hypertension | Vascular disorders | MedDRA (6.1) | Systematic Assessment |
|
| Cardiac valve disease | Cardiac disorders | MedDRA (6.1) | Systematic Assessment |
|
| Atrial hypertrophy | Cardiac disorders | MedDRA (6.1) | Systematic Assessment |
|
| Cardiac failure | Cardiac disorders | MedDRA (6.1) | Systematic Assessment |
|
| Cyanosis | Cardiac disorders | MedDRA (6.1) | Systematic Assessment |
|
| Dilatation atrial | Cardiac disorders | MedDRA (6.1) | Systematic Assessment |
|
| Neutrophil count increased | Investigations | MedDRA (6.1) | Systematic Assessment |
|
| QRS axis abnormal | Investigations | MedDRA (6.1) | Systematic Assessment |
|
| Blood alkaline phosphatase increased | Investigations | MedDRA (6.1) | Systematic Assessment |
|
| Cardiac murmur | Investigations | MedDRA (6.1) | Systematic Assessment |
|
| Oxygen saturation decreased | Investigations | MedDRA (6.1) | Systematic Assessment |
|
| Anaemia | Blood and lymphatic system disorders | MedDRA (6.1) | Systematic Assessment |
|
| Leukopenia | Blood and lymphatic system disorders | MedDRA (6.1) | Systematic Assessment |
|
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA (6.1) | Systematic Assessment |
|
| Iron deficiency | Metabolism and nutrition disorders | MedDRA (6.1) | Systematic Assessment |
|
| Abnormal behavior | Psychiatric disorders | MedDRA (6.1) | Systematic Assessment |
|
| Anxiety | Psychiatric disorders | MedDRA (6.1) | Systematic Assessment |
|
| Dysuria | Renal and urinary disorders | MedDRA (6.1) | Systematic Assessment |
|
| Hepatomegaly | Hepatobiliary disorders | MedDRA (6.1) | Systematic Assessment |
|
| Skin Papilloma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (6.1) | Systematic Assessment |
|
| Tooth extraction | Surgical and medical procedures | MedDRA (6.1) | Systematic Assessment |
|
The first publication of the results shall be made by Sponsor in a joint publication. If such a multicenter publication is not submitted within 12 months after conclusion of the study, the PI may publish the results from their site individually, subject however to comply with the other terms of the agreement.
| 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 |
| Enzymes |
| D045762 | Enzymes and Coenzymes |
| D002811 | Chondroitinsulfatases |
| D002810 | Chondroitinases and Chondroitin Lyases |