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| ID | Type | Description | Link |
|---|---|---|---|
| 2011-001805-28 | EudraCT Number |
Not provided
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The study was terminated because BioMarin decided to end the overall development program based on competing corporate priorities.
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This is a Phase 2 open-label, multiple dose study of BMN 701 administered by IV infusion every 2 weeks (qow) to patients with late-onset Pompe disease.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| BMN 701 20mg/kg | Experimental | BMN 701 20mg/kg IV every other week |
|
| BMN 701 10mg/kg | Experimental | BMN 701 10mg/kg IV every other week |
|
| BMN 701 5mg/kg | Experimental | BMN 701 5mg/kg IV every other week |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| BMN 701 | Biological | GILT-tagged recombinant human GAA |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With a Positive Anti-BMN 701 Antibody | Status of Anti-BMN 701 antibody is corresponding to the test results of blood samples. | Baseline, Week 144 |
| Number of Participants With a Positive Anti-BMN 701 Antibody Response | Status of Anti-IGF-I antibody is corresponding to the test results of blood samples | Baseline, Week 144 |
| Number of Participants With a Positive Anti-BMN 701 Antibody Response | Status of Anti-IGF-II antibody is corresponding to the test results of blood samples | Baseline, Week 144 |
| Measure | Description | Time Frame |
|---|---|---|
| Percent Predicted Maximal Inspiratory Pressure (MIP) | Pulmonary Function Test: Percent Predicted Maximal Inspiratory Pressure | Baseline, Week 144 |
| Percent Predicted Maximum Expiratory Pressure (MEP) |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
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| Name | Affiliation | Role |
|---|---|---|
| Medical Monitor | BioMarin Pharmaceutical | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Univ of California San Diego School of Medicine | San Diego | California | 92103 | United States | ||
| University of Florida College of Medicine |
Not provided
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| ID | Title | Description |
|---|---|---|
| FG000 | BMN 701 5 mg/kg | IV infusion at dose levels of 5 mg/kg |
| FG001 | BMN 701 10 mg/kg | IV infusion at dose levels of 10 mg/kg |
| FG002 | BMN 701 20 mg/kg | IV infusion at dose levels of 20 mg/kg |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | BMN 701 5 mg/kg | IV infusion at dose levels of 5 mg/kg |
| BG001 | BMN 701 10 mg/kg | IV infusion at dose levels of 10 mg/kg |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| 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 | Number of Participants With a Positive Anti-BMN 701 Antibody | Status of Anti-BMN 701 antibody is corresponding to the test results of blood samples. | Full Analysis Set. Please note the overall number of participants reflects the number of patients in that arm, while the outcome measurement number of participants reflects the number of patients for which data is available and analyzed. | Posted | Count of Participants | Participants | Baseline, Week 144 |
|
284.9 weeks (maximum exposure) +4 weeks follow up
Not provided
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 | BMN 701 5 mg/kg | BMN 701 5 mg/kg | 0 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cardiac failure acute | Cardiac disorders | MedDRA 19.0 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 19.0 | Systematic Assessment |
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Debra Lounsbury, Principal Scientist, Clinical Sciences | BioMarin Pharmaceutical Inc. | 415-506-6348 | DLounsbury@bmrn.com |
Not provided
| ID | Term |
|---|---|
| D006009 | Glycogen Storage Disease Type II |
| ID | Term |
|---|---|
| D020140 | Lysosomal Storage Diseases, Nervous System |
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
| D001927 | Brain Diseases |
Not provided
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Pulmonary Function Test: Percent Predicted Maximum Expiratory Pressure
| Baseline, Week 144 |
| 6 Minutes Walk Test (Meters) | Distance walked within 6 minutes | Baseline, Week 144 |
| Maximum Voluntary Ventilation (MVV) | Pulmonary function test: Maximum Voluntary Ventilation (MVV) | Baseline, Week 144 |
| Percent Predicted Upright Forced Vital Capacity (FVC) | Pulmonary function test: Percent Predicted Upright Forced Vital Capacity | Baseline, Week 144 |
| Change From Baseline in Urine Tetrasaccharide Concentration at Week 144 | Change from Baseline in Urine Tetrasaccharide Concentration at Week 144 | Baseline, Week 144 |
| Plasma IGF-I Concentration | Plasma IGF-I concentration from lab | Baseline, Week 144 |
| Plasma IGF-II Concentration | Plasma IGF-II concentration from lab | Baseline, Week 144 |
| Insulin-like Growth Factor Binding Protein 3 (IGFBP3) | insulin-like growth factor binding protein 3 from lab | Baseline, Week 144 |
| Gainesville |
| Florida |
| 32610 |
| United States |
| Tampa General Hospital | Tampa | Florida | 33606 | United States |
| University of Kansas Medical Center | Kansas City | Kansas | 66160 | United States |
| Royal Brisbane and Women's Hospital | Herston | Queensland | 4029 | Australia |
| Royal Adelaide Hospital | North Adelaide | South Australia | 5006 | Australia |
| Hôpital Pitié-Salpêtrière | Paris | 75013 | France |
| Villa Metabolica, ZKJM MC University Mainz | Mainz | 55131 | Germany |
| Auckland City and Starship Children's Hospital | Auckland | 1142 | New Zealand |
| University Hospitals Birmingham NHS Foundation Trust | Birmingham | B15 2TH | United Kingdom |
| Royal Free Hospital | London | NW3 2QG | United Kingdom |
| Salford Royal NHS Foundation Trust | Salford | M5 5AP | United Kingdom |
| Other - study termination |
|
| BG002 | BMN 701 20 mg/kg | IV infusion at dose levels of 20 mg/kg |
| BG003 | Total | Total of all reporting groups |
| years |
|
| Age, Customized | Count of Participants | Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| Region of Enrollment | Count of Participants | Participants |
|
| OG002 | BMN 701 20 mg/kg | IV infusion at dose levels of 20 mg/kg |
|
|
| Primary | Number of Participants With a Positive Anti-BMN 701 Antibody Response | Status of Anti-IGF-I antibody is corresponding to the test results of blood samples | Full Analysis Set. | Posted | Count of Participants | Participants | Baseline, Week 144 |
|
|
|
| Primary | Number of Participants With a Positive Anti-BMN 701 Antibody Response | Status of Anti-IGF-II antibody is corresponding to the test results of blood samples | Full Analysis Set. | Posted | Count of Participants | Participants | Baseline, Week 144 |
|
|
|
| Secondary | Percent Predicted Maximal Inspiratory Pressure (MIP) | Pulmonary Function Test: Percent Predicted Maximal Inspiratory Pressure | Full Analysis Set. No data collected for outcomes with 0 participants analyzed. | Posted | Mean | Standard Deviation | Percent of Predicted | Baseline, Week 144 |
|
|
|
| Secondary | Percent Predicted Maximum Expiratory Pressure (MEP) | Pulmonary Function Test: Percent Predicted Maximum Expiratory Pressure | Full Analysis Set. No data collected for outcomes with 0 participants analyzed. | Posted | Mean | Standard Deviation | Percent of Predicted | Baseline, Week 144 |
|
|
|
| Secondary | 6 Minutes Walk Test (Meters) | Distance walked within 6 minutes | Full Analysis Set. | Posted | Mean | Standard Deviation | meter | Baseline, Week 144 |
|
|
|
| Secondary | Maximum Voluntary Ventilation (MVV) | Pulmonary function test: Maximum Voluntary Ventilation (MVV) | Full Analysis Set. No data collected for outcomes with 0 participants analyzed. | Posted | Mean | Standard Deviation | L/min | Baseline, Week 144 |
|
|
|
| Secondary | Percent Predicted Upright Forced Vital Capacity (FVC) | Pulmonary function test: Percent Predicted Upright Forced Vital Capacity | Full Analysis Set. | Posted | Mean | Standard Deviation | Percent Predicted | Baseline, Week 144 |
|
|
|
| Secondary | Percent Predicted Upright Forced Vital Capacity (FVC) | Pulmonary function test: Percent Predicted Upright Forced Vital capacity | Full Analysis Set. | Posted | Mean | Standard Deviation | Percent Predicted | Baseline, Week 144 |
|
|
|
| Secondary | Change From Baseline in Urine Tetrasaccharide Concentration at Week 144 | Change from Baseline in Urine Tetrasaccharide Concentration at Week 144 | Full Analysis Set. No data collected for outcomes with 0 participants analyzed. | Posted | Mean | Standard Deviation | mmol/mol | Baseline, Week 144 |
|
|
|
| Secondary | Plasma IGF-I Concentration | Plasma IGF-I concentration from lab | Full Analysis Set. No data collected for outcomes with 0 participants analyzed. Please note: the overall number of participants reflects the number of patients in that arm, while the outcome measurement number of participants reflects the number of patients for which data is available and analyzed. | Posted | Mean | Standard Deviation | nmol/L | Baseline, Week 144 |
|
|
|
| Secondary | Plasma IGF-II Concentration | Plasma IGF-II concentration from lab | Full Analysis Set. No data collected for outcomes with 0 participants analyzed. Please note: the overall number of participants reflects the number of patients in that arm, while the outcome measurement number of participants reflects the number of patients for which data is available and analyzed. | Posted | Mean | Standard Deviation | nmol/L | Baseline, Week 144 |
|
|
|
| Secondary | Insulin-like Growth Factor Binding Protein 3 (IGFBP3) | insulin-like growth factor binding protein 3 from lab | Full Analysis Set. No data collected for outcomes with 0 participants analyzed. Please note: the overall number of participants reflects the number of patients in that arm, while the outcome measurement number of participants reflects the number of patients for which data is available and analyzed. | Posted | Mean | Standard Deviation | nmol/L | Baseline, Week 144 |
|
|
|
| 3 |
| 2 |
| 3 |
| 3 |
| 3 |
| EG001 | BMN 701 10 mg/kg | BMN 701 10 mg/kg | 0 | 3 | 1 | 3 | 3 | 3 |
| EG002 | BMN 701 20 mg/kg | BMN 701 20 mg/kg | 0 | 15 | 7 | 15 | 15 | 15 |
| EG003 | Total | Total | 0 | 21 | 10 | 21 | 21 | 21 |
| Myocardial infarction | Cardiac disorders | MedDRA 19.0 | Systematic Assessment |
|
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
|
| Fatigue | General disorders | MedDRA 19.0 | Systematic Assessment |
|
| Anaphylactoid reaction | Immune system disorders | MedDRA 19.0 | Systematic Assessment |
|
| Pneumonia | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
|
| Fall | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
|
| Tendon rupture | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
|
| Hypoglycaemia | Metabolism and nutrition disorders | MedDRA 19.0 | Systematic Assessment |
|
| Cerebrovascular accident | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
|
| Presyncope | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
|
| Prerenal failure | Renal and urinary disorders | MedDRA 19.0 | Systematic Assessment |
|
| Chronic respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
|
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
|
| Stridor | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
|
| Iron deficiency anaemia | Blood and lymphatic system disorders | MedDRA 19.0 | Systematic Assessment |
|
| Bradycardia | Cardiac disorders | MedDRA 19.0 | Systematic Assessment |
|
| Cardiac failure | Cardiac disorders | MedDRA 19.0 | Systematic Assessment |
|
| Diastolic dysfunction | Cardiac disorders | MedDRA 19.0 | Systematic Assessment |
|
| Left ventricular hypertrophy | Cardiac disorders | MedDRA 19.0 | Systematic Assessment |
|
| Sinus tachycardia | Cardiac disorders | MedDRA 19.0 | Systematic Assessment |
|
| Tachycardia | Cardiac disorders | MedDRA 19.0 | Systematic Assessment |
|
| Ear discomfort | Ear and labyrinth disorders | MedDRA 19.0 | Systematic Assessment |
|
| Tinnitus | Ear and labyrinth disorders | MedDRA 19.0 | Systematic Assessment |
|
| Vertigo positional | Ear and labyrinth disorders | MedDRA 19.0 | Systematic Assessment |
|
| Androgen deficiency | Endocrine disorders | MedDRA 19.0 | Systematic Assessment |
|
| Blindness transient | Eye disorders | MedDRA 19.0 | Systematic Assessment |
|
| Ocular hyperaemia | Eye disorders | MedDRA 19.0 | Systematic Assessment |
|
| Visual impairment | Eye disorders | MedDRA 19.0 | Systematic Assessment |
|
| Abdominal discomfort | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
|
| Abdominal distension | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
|
| Abdominal pain | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
|
| Abdominal pain lower | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
|
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
|
| Abdominal tenderness | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
|
| Dental caries | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
|
| Dyspepsia | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
|
| Flatulence | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
|
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
|
| Irritable bowel syndrome | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
|
| Painful defaecation | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
|
| Rectal haemorrhage | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
|
| Salivary hypersecretion | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
|
| Tooth loss | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
|
| Toothache | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
|
| Application site erythema | General disorders | MedDRA 19.0 | Systematic Assessment |
|
| Asthenia | General disorders | MedDRA 19.0 | Systematic Assessment |
|
| Catheter site erythema | General disorders | MedDRA 19.0 | Systematic Assessment |
|
| Catheter site inflammation | General disorders | MedDRA 19.0 | Systematic Assessment |
|
| Catheter site pain | General disorders | MedDRA 19.0 | Systematic Assessment |
|
| Chest discomfort | General disorders | MedDRA 19.0 | Systematic Assessment |
|
| Chest pain | General disorders | MedDRA 19.0 | Systematic Assessment |
|
| Chills | General disorders | MedDRA 19.0 | Systematic Assessment |
|
| Discomfort | General disorders | MedDRA 19.0 | Systematic Assessment |
|
| Extravasation | General disorders | MedDRA 19.0 | Systematic Assessment |
|
| Fatigue | General disorders | MedDRA 19.0 | Systematic Assessment |
|
| Feeling abnormal | General disorders | MedDRA 19.0 | Systematic Assessment |
|
| Feeling cold | General disorders | MedDRA 19.0 | Systematic Assessment |
|
| Feeling hot | General disorders | MedDRA 19.0 | Systematic Assessment |
|
| Feeling of body temperature change | General disorders | MedDRA 19.0 | Systematic Assessment |
|
| Gait disturbance | General disorders | MedDRA 19.0 | Systematic Assessment |
|
| Influenza like illness | General disorders | MedDRA 19.0 | Systematic Assessment |
|
| Infusion site extravasation | General disorders | MedDRA 19.0 | Systematic Assessment |
|
| Infusion site pain | General disorders | MedDRA 19.0 | Systematic Assessment |
|
| Infusion site swelling | General disorders | MedDRA 19.0 | Systematic Assessment |
|
| Malaise | General disorders | MedDRA 19.0 | Systematic Assessment |
|
| Non-cardiac chest pain | General disorders | MedDRA 19.0 | Systematic Assessment |
|
| Oedema peripheral | General disorders | MedDRA 19.0 | Systematic Assessment |
|
| Pain | General disorders | MedDRA 19.0 | Systematic Assessment |
|
| Peripheral swelling | General disorders | MedDRA 19.0 | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA 19.0 | Systematic Assessment |
|
| Temperature intolerance | General disorders | MedDRA 19.0 | Systematic Assessment |
|
| Tenderness | General disorders | MedDRA 19.0 | Systematic Assessment |
|
| Vessel puncture site bruise | General disorders | MedDRA 19.0 | Systematic Assessment |
|
| Cholelithiasis | Hepatobiliary disorders | MedDRA 19.0 | Systematic Assessment |
|
| Seasonal allergy | Immune system disorders | MedDRA 19.0 | Systematic Assessment |
|
| Blister infected | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
|
| Bronchitis | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
|
| Ear infection | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
|
| Fungal infection | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
|
| Gastroenteritis viral | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
|
| Herpes simplex | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
|
| Influenza | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
|
| Lower respiratory tract infection | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
|
| Lower respiratory tract infection viral | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
|
| Rhinitis | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
|
| Sinusitis | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
|
| Urinary tract infection | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
|
| Viral infection | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
|
| Viral upper respiratory tract infection | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
|
| Contusion | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
|
| Epicondylitis | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
|
| Eye contusion | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
|
| Fall | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
|
| Head injury | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
|
| Joint injury | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
|
| Laceration | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
|
| Muscle contusion | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
|
| Muscle strain | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
|
| Post procedural contusion | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
|
| Post procedural haemorrhage | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
|
| Procedural pain | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
|
| Skin abrasion | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
|
| Skin wound | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
|
| Tendon injury | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
|
| Thermal burn | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
|
| Tooth fracture | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
|
| Wound | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
|
| Ammonia increased | Investigations | MedDRA 19.0 | Systematic Assessment |
|
| Bacterial test positive | Investigations | MedDRA 19.0 | Systematic Assessment |
|
| Blood calcium decreased | Investigations | MedDRA 19.0 | Systematic Assessment |
|
| Blood glucose decreased | Investigations | MedDRA 19.0 | Systematic Assessment |
|
| Blood immunoglobulin E increased | Investigations | MedDRA 19.0 | Systematic Assessment |
|
| Cardiac murmur | Investigations | MedDRA 19.0 | Systematic Assessment |
|
| Complement factor increased | Investigations | MedDRA 19.0 | Systematic Assessment |
|
| Lipase increased | Investigations | MedDRA 19.0 | Systematic Assessment |
|
| Pulmonary physical examination abnormal | Investigations | MedDRA 19.0 | Systematic Assessment |
|
| Troponin increased | Investigations | MedDRA 19.0 | Systematic Assessment |
|
| Tryptase increased | Investigations | MedDRA 19.0 | Systematic Assessment |
|
| Dehydration | Metabolism and nutrition disorders | MedDRA 19.0 | Systematic Assessment |
|
| Glucose tolerance impaired | Metabolism and nutrition disorders | MedDRA 19.0 | Systematic Assessment |
|
| Hyperglycaemia | Metabolism and nutrition disorders | MedDRA 19.0 | Systematic Assessment |
|
| Hyperkalaemia | Metabolism and nutrition disorders | MedDRA 19.0 | Systematic Assessment |
|
| Hypoglycaemia | Metabolism and nutrition disorders | MedDRA 19.0 | Systematic Assessment |
|
| Iron deficiency | Metabolism and nutrition disorders | MedDRA 19.0 | Systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
|
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
|
| Bursitis | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
|
| Flank pain | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
|
| Groin pain | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
|
| Intervertebral disc protrusion | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
|
| Joint swelling | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
|
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
|
| Muscle twitching | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
|
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
|
| Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
|
| Musculoskeletal discomfort | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
|
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
|
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
|
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
|
| Osteopenia | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
|
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
|
| Spinal pain | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
|
| Tendonitis | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Systematic Assessment |
|
| Skin papilloma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 19.0 | Systematic Assessment |
|
| Balance disorder | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
|
| Burning sensation | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
|
| Dizziness | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
|
| Hypoaesthesia | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
|
| Lethargy | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
|
| Migraine | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
|
| Paraesthesia | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
|
| Presyncope | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
|
| Restless legs syndrome | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
|
| Sciatica | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
|
| Sinus headache | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
|
| Tremor | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
|
| Tunnel vision | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
|
| Device occlusion | Product Issues | MedDRA 19.0 | Systematic Assessment |
|
| Anxiety | Psychiatric disorders | MedDRA 19.0 | Systematic Assessment |
|
| Confusional state | Psychiatric disorders | MedDRA 19.0 | Systematic Assessment |
|
| Insomnia | Psychiatric disorders | MedDRA 19.0 | Systematic Assessment |
|
| Mood swings | Psychiatric disorders | MedDRA 19.0 | Systematic Assessment |
|
| Nervousness | Psychiatric disorders | MedDRA 19.0 | Systematic Assessment |
|
| Restlessness | Psychiatric disorders | MedDRA 19.0 | Systematic Assessment |
|
| Sleep terror | Psychiatric disorders | MedDRA 19.0 | Systematic Assessment |
|
| Haematuria | Renal and urinary disorders | MedDRA 19.0 | Systematic Assessment |
|
| Renal impairment | Renal and urinary disorders | MedDRA 19.0 | Systematic Assessment |
|
| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
|
| Chronic respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
|
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
|
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
|
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
|
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
|
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
|
| Paranasal sinus hypersecretion | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
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| Pleuritic pain | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
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| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
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| Pulmonary congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
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| Rales | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
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| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
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| Tachypnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
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| Wheezing | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
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| Angioedema | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
|
| Cold sweat | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
|
| Decubitus ulcer | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
|
| Dermal cyst | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
|
| Erythema | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
|
| Hyperhidrosis | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
|
| Night sweats | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
|
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
|
| Rash macular | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
|
| Rash papular | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
|
| Rash pruritic | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
|
| Rosacea | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
|
| Skin lesion | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
|
| Skin mass | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
|
| Skin ulcer | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
|
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA 19.0 | Systematic Assessment |
|
| Flushing | Vascular disorders | MedDRA 19.0 | Systematic Assessment |
|
| Haematoma | Vascular disorders | MedDRA 19.0 | Systematic Assessment |
|
| Hot flush | Vascular disorders | MedDRA 19.0 | Systematic Assessment |
|
| Hypertension | Vascular disorders | MedDRA 19.0 | Systematic Assessment |
|
| Hypotension | Vascular disorders | MedDRA 19.0 | Systematic Assessment |
|
| Pallor | Vascular disorders | MedDRA 19.0 | Systematic Assessment |
|
Not provided
Not provided
Not provided
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D006008 | Glycogen Storage Disease |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D016464 | Lysosomal Storage Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| Week 144 |
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| Week 144 |
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| Change from Baseline to Week 144 |
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| Change from Baseline to Week 144 |
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| Change from Baseline to Week 144 |
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| Change from Baseline to Week 144 |
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| Change from Baseline to Week 144 |
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| Week 144 |
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| Week 144 |
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| Week 144 |
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