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The purpose of this study is to determine whether the administration of iduronate-2-sulfatase enzyme in a weekly or every other week therapy frequency is safe and efficacious in patients with MPS II.
MPS II is a rare, X-linked, lysosomal storage disorder caused by a deficiency in the enzyme iduronate-2-sulfatase. Because of this deficiency, glycosaminoglycans (GAG) accumulate in multiple tissues and organs, resulting in progressive cellular and organ system dysfunction. The purpose of this study is to determine if one year of therapy with iduronate-2-sulfatase enzyme replacement therapy, at a dose of 0.5mg/kg, weekly or every other week, is safe, and results in clinically meaningful improvement in multiple organ function, compared with a placebo group. Upon completion of the study, patients will be eligible to enroll in an open-label maintenance study.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Idursulfase weekly (0.5 mg/kg) | Experimental |
| |
| Idursulfase every other week (0.5 mg/kg) | Experimental |
| |
| Placebo | Placebo Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Iduronate-2-sulfatase enzyme replacement therapy | Biological | Patients will receive weekly infusions of idursulfase at a dose of 0.5 mg/kg. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Ranked Adjusted 2-Component Composite Variable Score Based on Change From Baseline to Week 53 | The 2-component composite variable consists of the sum of the ranked changes from baseline to Week 53 for percent predicted Forced Vital Capacity (FVC) and 6-Minute Walking Test (6MWT) total distance walked. For the 2 treatment groups being compared, ranking occurred within the comparison treatment groups combined (idursulfase weekly and placebo treatment groups). These comparison groups were pooled and ranked for each component separately. Within each component (% predicted FVC, 6MWT), the change from baseline was then ranked. The lowest change value was assigned a rank of 1, the next lowest a rank of 2, etc. The composite score for each participant was the sum of the 2 ranked scores corresponding to the 2 individual components (% predicted FVC and 6MWT) for each participant. Thus, the greater the composite score (greater the sum of the ranks of the changes from baseline, where the lowest change was ranked as 1), the greater the improvement. | Baseline, Week 53 |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Mean Global Joint Range of Motion (JROM) Score at Week 53 | Change was calculated at Week 53 from baseline. Global JROM (% of normal range of motion) is the average of 11 ratios multiplied by 100. Ratios are Left/Right means of passive range of motion in Shoulder (Flexion/Extension, Abduction, Internal/External Rotation), Elbow (Flexion/Extension), Wrist (Flexion/Extension), Index Finger (Flexion/Extension [Combined Metacarpophalangeal joint (MCP), Proximal interphalangeal joint (PIP), Distal interphalangeal joint (DIP) motion]), Hip (Flexion/Extension, Abduction, Internal/External Rotation), Knee (Flexion/Extension), and Ankle (Dorsiflexion) divided by the normal range (American Academy of Orthopedic Surgeons and American Medical Association). |
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Inclusion Criteria:
To be eligible to participate in this study, patients must meet the following inclusion criteria prior to enrollment:
The diagnosis of MPS II will be determined by the investigator based upon both clinical and biochemical criteria.
All patients must have at least one of the following Clinical Criteria considered by the investigator to be MPS II-related:
In addition, patients must have the following Biochemical Criteria:
Must be male, 5 to 25 years of age.
Forced vital capacity of <80% of predicted obtained at the baseline evaluation of this study.
Must be able to adequately perform the testing required in this study, including reproducible pulmonary function testing by spirometry, as judged by the investigator.
Patient, patient's parent(s), or legally authorized guardian must have voluntarily signed an Institutional Review Board (IRB)/Independent Ethics Committee (IEC)-approved informed consent form after all relevant aspects of the study have been explained and discussed with the patient.
Exclusion Criteria:
Patients meeting any of the following criteria are not eligible for participation in this study:
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| Name | Affiliation | Role |
|---|---|---|
| Study Director | Takeda | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital Oakland | Oakland | California | 94609 | United States | ||
| St. Louis Children's Hospital, Washington University |
| 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. | |
| 23837440 | Derived | Raluy-Callado M, Chen WH, Whiteman DA, Fang J, Wiklund I. The impact of Hunter syndrome (mucopolysaccharidosis type II) on health-related quality of life. Orphanet J Rare Dis. 2013 Jul 10;8:101. doi: 10.1186/1750-1172-8-101. |
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| ID | Title | Description |
|---|---|---|
| FG000 | Idursulfase Weekly (0.5 mg/kg) | Idursulfase 0.5 milligram per kilogram (mg/kg) administered once-weekly by intravenous infusion for one year (52 infusions). |
| FG001 | Idursulfase Every Other Week (EOW) (0.5 mg/kg) |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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|
| iduronate-2-sulfatase enzyme replacement therapy | Biological | Patients will receive every other week infusions of idursulfase at a dose of 0.5 mg/kg. |
|
|
| Placebo | Biological | Patients will receive weekly infusions of placebo. |
|
| Baseline, Week 53 |
| Mean Combined Liver and Spleen Volume at Baseline | Liver and Spleen volume was determined by Magnetic Resonance Imaging (MRI). | Baseline |
| Percent Change From Baseline in Mean Combined Liver and Spleen Volume at Week 53 | Liver and Spleen volume was determined by Magnetic Resonance Imaging (MRI). Change was calculated at Week 53 from baseline. | Baseline, Week 53 |
| Change From Baseline in Mean Normalized Urine Glycosaminoglycan (GAG) Levels at Week 53 | Mean normalized urine GAG was analyzed using urine testing. Change was calculated at Week 53 from baseline. The urine GAG levels were normalized to urine creatinine and were reported as microgram GAG per milligram creatinine (mcg GAG/mg creatinine). | Baseline, Week 53 |
| Mean Cardiac Left Ventricular Mass Index (LVMI) at Baseline | Cardiac LVMI was determined by echocardiography. LVMI is the left ventricular mass (LVM, in grams [g]) indexed to body surface area (BSA), in square meter [m^2]. LVMI (in gram per square meter [g/m^2]) = LVM divided by BSA. | Baseline |
| Percent Change From Baseline in Mean Cardiac Left Ventricular Mass Index (LVMI) at Week 53 | Cardiac LVMI was determined by echocardiography. Change was calculated at Week 53 from baseline. LVMI is the LVM, in grams indexed to BSA, in square meter [m^2]. LVMI in g/m^2 = LVM divided by BSA. | Baseline, Week 53 |
| St Louis |
| Missouri |
| 63110 |
| United States |
| University of North Carolina at Chapel Hill | Chapel Hill | North Carolina | 27599 | United States |
| Texas Children's Hospital, Baylor College of Medicine | Houston | Texas | 77030 | United States |
| Hospital de Clinicas de Porto Alegre | Porto Alegre | Brazil |
| Children's Hospital, Johannes-Gutenburg Universitaet Mainz | Mainz | Germany |
| Addenbrooke's Hospital | Cambridge | England | CB2 2QQ | United Kingdom |
| Great Ormond Street Hospital for Sick Children | London | England | WC1N3JH | United Kingdom |
| Royal Manchester Children's Hospital | Manchester | England | M27 4HA | United Kingdom |
Idursulfase 0.5 mg/kg administered every other week by intravenous infusion for one year (26 infusions) along with placebo matching to idursulfase every other week (alternating with idursulfase) by intravenous infusion for one year (26 infusions).
| FG002 | Placebo | Placebo matching to idursulfase administered once-weekly by intravenous infusion for one year (52 infusions). |
| COMPLETED |
|
| NOT COMPLETED |
|
|
Intent-to-treat (ITT) population included all randomized participants.
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| ID | Title | Description |
|---|---|---|
| BG000 | Idursulfase Weekly (0.5 mg/kg) | Idursulfase 0.5 mg/kg administered once-weekly by intravenous infusion for one year (52 infusions). |
| BG001 | Idursulfase EOW (0.5 mg/kg) | Idursulfase 0.5 mg/kg administered every other week by intravenous infusion for one year (26 infusions) along with placebo matching to idursulfase every other week (alternating with idursulfase) by intravenous infusion for one year (26 infusions). |
| BG002 | Placebo | Placebo matching to idursulfase administered once-weekly by intravenous infusion for one year (52 infusions). |
| BG003 | 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 | Age at randomization. | Mean | Standard Deviation | years |
| ||||||||||||||
| Age, Customized | Age at randomization. The participant who was less than (<) 5 years old was considered for randomization to be in 5 to 11 years category. Participants who were greater than (>) 25 years old were considered for randomization to be in 19 to 25 years category. | Count of Participants | Participants |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Region of Enrollment | Count of Participants | 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 | |||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Secondary | Change From Baseline in Mean Global Joint Range of Motion (JROM) Score at Week 53 | Change was calculated at Week 53 from baseline. Global JROM (% of normal range of motion) is the average of 11 ratios multiplied by 100. Ratios are Left/Right means of passive range of motion in Shoulder (Flexion/Extension, Abduction, Internal/External Rotation), Elbow (Flexion/Extension), Wrist (Flexion/Extension), Index Finger (Flexion/Extension [Combined Metacarpophalangeal joint (MCP), Proximal interphalangeal joint (PIP), Distal interphalangeal joint (DIP) motion]), Hip (Flexion/Extension, Abduction, Internal/External Rotation), Knee (Flexion/Extension), and Ankle (Dorsiflexion) divided by the normal range (American Academy of Orthopedic Surgeons and American Medical Association). | ITT population. Here, number of participants analyzed = participants who were evaluable for this measure. | Posted | Mean | Standard Error | percentage of normal range of motion | Baseline, Week 53 |
|
|
| ||||||||||||||||||||||||||||||||
| Secondary | Mean Combined Liver and Spleen Volume at Baseline | Liver and Spleen volume was determined by Magnetic Resonance Imaging (MRI). | ITT population. Here, number of participants analyzed = participants who were evaluable for this measure. | Posted | Mean | Standard Error | milliliter (mL) | Baseline |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Percent Change From Baseline in Mean Combined Liver and Spleen Volume at Week 53 | Liver and Spleen volume was determined by Magnetic Resonance Imaging (MRI). Change was calculated at Week 53 from baseline. | ITT population. Here, number of participants analyzed = participants who were evaluable for this measure. | Posted | Mean | Standard Error | percent change | Baseline, Week 53 |
| ||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Mean Normalized Urine Glycosaminoglycan (GAG) Levels at Week 53 | Mean normalized urine GAG was analyzed using urine testing. Change was calculated at Week 53 from baseline. The urine GAG levels were normalized to urine creatinine and were reported as microgram GAG per milligram creatinine (mcg GAG/mg creatinine). | ITT population. | Posted | Mean | Standard Error | mcg GAG/mg creatinine | Baseline, Week 53 |
| ||||||||||||||||||||||||||||||||||
| Primary | Ranked Adjusted 2-Component Composite Variable Score Based on Change From Baseline to Week 53 | The 2-component composite variable consists of the sum of the ranked changes from baseline to Week 53 for percent predicted Forced Vital Capacity (FVC) and 6-Minute Walking Test (6MWT) total distance walked. For the 2 treatment groups being compared, ranking occurred within the comparison treatment groups combined (idursulfase weekly and placebo treatment groups). These comparison groups were pooled and ranked for each component separately. Within each component (% predicted FVC, 6MWT), the change from baseline was then ranked. The lowest change value was assigned a rank of 1, the next lowest a rank of 2, etc. The composite score for each participant was the sum of the 2 ranked scores corresponding to the 2 individual components (% predicted FVC and 6MWT) for each participant. Thus, the greater the composite score (greater the sum of the ranks of the changes from baseline, where the lowest change was ranked as 1), the greater the improvement. | Intent-to-treat (ITT) population, Only participants receiving "Idursulfase Weekly" or "Placebo" were to be analyzed for this outcome. | Posted | Mean | Standard Error | sum of the ranked scores | Baseline, Week 53 |
| ||||||||||||||||||||||||||||||||||
| Secondary | Mean Cardiac Left Ventricular Mass Index (LVMI) at Baseline | Cardiac LVMI was determined by echocardiography. LVMI is the left ventricular mass (LVM, in grams [g]) indexed to body surface area (BSA), in square meter [m^2]. LVMI (in gram per square meter [g/m^2]) = LVM divided by BSA. | ITT population. Here, number of participants analyzed = participants who were evaluable for this measure. | Posted | Mean | Standard Error | gram per square meter (g/m^2) | Baseline |
| ||||||||||||||||||||||||||||||||||
| Secondary | Percent Change From Baseline in Mean Cardiac Left Ventricular Mass Index (LVMI) at Week 53 | Cardiac LVMI was determined by echocardiography. Change was calculated at Week 53 from baseline. LVMI is the LVM, in grams indexed to BSA, in square meter [m^2]. LVMI in g/m^2 = LVM divided by BSA. | ITT population. Here, number of participants analyzed = participants who were evaluable for this measure. | Posted | Mean | Standard Error | percent change | Baseline, Week 53 |
|
52 weeks
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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 | Idursulfase Weekly (0.5 mg/kg) | Idursulfase 0.5 mg/kg administered once-weekly by intravenous infusion for one year (52 infusions). | 9 | 32 | 32 | 32 | ||
| EG001 | Idursulfase EOW (0.5 mg/kg) | Idursulfase 0.5 mg/kg administered every other week by intravenous infusion for one year (26 infusions) along with placebo matching to idursulfase every other week (alternating with idursulfase) by intravenous infusion for one year (26 infusions). | 8 | 32 | 32 | 32 | ||
| EG002 | Placebo | Placebo matching to idursulfase administered once-weekly by intravenous infusion for one year (52 infusions). | 9 | 32 | 32 | 32 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Aortic valve incompetence | Cardiac disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Arrhythmia not otherwise specified (nos) | Cardiac disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Atrial tachycardia | Cardiac disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Cyanosis nos | Cardiac disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Heart valve insufficiency | Cardiac disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Ear disorder nos | Ear and labyrinth disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Hearing impaired | Ear and labyrinth disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Otorrhoea | Ear and labyrinth disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Appendicitis | Gastrointestinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Pancreatitis acute | Gastrointestinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Umbilical hernia nos | Gastrointestinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Hernia nos | General disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Bronchopneumonia nos | Infections and infestations | MedDRA (5.1) | Systematic Assessment |
| |
| Localised infection | Infections and infestations | MedDRA (5.1) | Systematic Assessment |
| |
| Otitis media chronic nos | Infections and infestations | MedDRA (5.1) | Systematic Assessment |
| |
| Otitis media serous nos | Infections and infestations | MedDRA (5.1) | Systematic Assessment |
| |
| Pilonidal sinus infected | Infections and infestations | MedDRA (5.1) | Systematic Assessment |
| |
| Pneumonia streptococcal | Infections and infestations | MedDRA (5.1) | Systematic Assessment |
| |
| Tooth caries nos | Infections and infestations | MedDRA (5.1) | Systematic Assessment |
| |
| Wound infection due to staphylococcus aureus | Infections and infestations | MedDRA (5.1) | Systematic Assessment |
| |
| Anaesthesia intubation complication | Injury, poisoning and procedural complications | MedDRA (5.1) | Systematic Assessment |
| |
| Medical device complication | Injury, poisoning and procedural complications | MedDRA (5.1) | Systematic Assessment |
| |
| Blood carbon dioxide increased | Investigations | MedDRA (5.1) | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Carpal tunnel syndrome | Nervous system disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Adjustment disorder with depressed mood | Psychiatric disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Phobia | Psychiatric disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Adenoidal hypertrophy | Respiratory, thoracic and mediastinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Bronchospasm nos | Respiratory, thoracic and mediastinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Tonsillar hypertrophy | Respiratory, thoracic and mediastinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Rash nos | Skin and subcutaneous tissue disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Orthostatic hypotension | Vascular disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Poor venous access | Vascular disorders | MedDRA (5.1) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Lymphadenopathy | Blood and lymphatic system disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Atrial hypertrophy | Cardiac disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Tachycardia nos | Cardiac disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Ventricular hypertrophy | Cardiac disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Cerumen impaction | Ear and labyrinth disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Deafness nos | Ear and labyrinth disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Deafness unilateral | Ear and labyrinth disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Ear disorder nos | Ear and labyrinth disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Ear haemorrhage | Ear and labyrinth disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Ear pain | Ear and labyrinth disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Eustachian tube dysfunction | Ear and labyrinth disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Hypoacusis | Ear and labyrinth disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Otorrhoea | Ear and labyrinth disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Sensation of block in ear | Ear and labyrinth disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Tympanic membrane perforation | Ear and labyrinth disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Conjunctivitis | Eye disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Conjunctivitis allergic | Eye disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Eye pain | Eye disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Hypermetropia | Eye disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Lacrimation increased | Eye disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Myopia | Eye disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Vision blurred | Eye disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Abdominal discomfort | Gastrointestinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Abdominal pain nos | Gastrointestinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Diarrhoea nos | Gastrointestinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Flatulence | Gastrointestinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Gastroenteritis nos | Gastrointestinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Inguinal hernia nos | Gastrointestinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Loose stools | Gastrointestinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Swollen tongue | Gastrointestinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Toothache | Gastrointestinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Umbilical hernia nos | Gastrointestinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Vomiting nos | Gastrointestinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Catheter site pain | General disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Chest pain | General disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Fall | General disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Gait abnormal | General disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Hernia pain | General disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Inflammation localised | General disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Infusion site pain | General disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Infusion site swelling | General disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Injection site bruising | General disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Injection site extravasation | General disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Injection site haemorrhage | General disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Lethargy | General disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Malaise | General disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Pain nos | General disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Rigors | General disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Sensation of foreign body nos | General disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Ear infection nos | Infections and infestations | MedDRA (5.1) | Systematic Assessment |
| |
| Furuncle | Infections and infestations | MedDRA (5.1) | Systematic Assessment |
| |
| Herpes simplex | Infections and infestations | MedDRA (5.1) | Systematic Assessment |
| |
| Hordeolum | Infections and infestations | MedDRA (5.1) | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA (5.1) | Systematic Assessment |
| |
| Lice infestation | Infections and infestations | MedDRA (5.1) | Systematic Assessment |
| |
| Localised infection | Infections and infestations | MedDRA (5.1) | Systematic Assessment |
| |
| Lower respiratory tract infection nos | Infections and infestations | MedDRA (5.1) | Systematic Assessment |
| |
| Otitis media nos | Infections and infestations | MedDRA (5.1) | Systematic Assessment |
| |
| Otitis media serous nos | Infections and infestations | MedDRA (5.1) | Systematic Assessment |
| |
| Respiratory tract infection nos | Infections and infestations | MedDRA (5.1) | Systematic Assessment |
| |
| Sinusitis nos | Infections and infestations | MedDRA (5.1) | Systematic Assessment |
| |
| Tonsillitis | Infections and infestations | MedDRA (5.1) | Systematic Assessment |
| |
| Tracheobronchitis | Infections and infestations | MedDRA (5.1) | Systematic Assessment |
| |
| Upper respiratory tract infection nos | Infections and infestations | MedDRA (5.1) | Systematic Assessment |
| |
| Urinary tract infection nos | Infections and infestations | MedDRA (5.1) | Systematic Assessment |
| |
| Varicella | Infections and infestations | MedDRA (5.1) | Systematic Assessment |
| |
| Viral infection nos | Infections and infestations | MedDRA (5.1) | Systematic Assessment |
| |
| Abrasion nos | Injury, poisoning and procedural complications | MedDRA (5.1) | Systematic Assessment |
| |
| Arthropod bite | Injury, poisoning and procedural complications | MedDRA (5.1) | Systematic Assessment |
| |
| Head injury | Injury, poisoning and procedural complications | MedDRA (5.1) | Systematic Assessment |
| |
| Muscle strain | Injury, poisoning and procedural complications | MedDRA (5.1) | Systematic Assessment |
| |
| Post procedural pain | Injury, poisoning and procedural complications | MedDRA (5.1) | Systematic Assessment |
| |
| Skin laceration | Injury, poisoning and procedural complications | MedDRA (5.1) | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA (5.1) | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA (5.1) | Systematic Assessment |
| |
| Blood alkaline phosphatase nos increased | Investigations | MedDRA (5.1) | Systematic Assessment |
| |
| Blood lactate dehydrogenase increased | Investigations | MedDRA (5.1) | Systematic Assessment |
| |
| Blood triglycerides increased | Investigations | MedDRA (5.1) | Systematic Assessment |
| |
| Electrocardiogram abnormal nos | Investigations | MedDRA (5.1) | Systematic Assessment |
| |
| Gamma-Glutamyltransferase increased | Investigations | MedDRA (5.1) | Systematic Assessment |
| |
| Haematocrit decreased | Investigations | MedDRA (5.1) | Systematic Assessment |
| |
| Haemoglobin decreased | Investigations | MedDRA (5.1) | Systematic Assessment |
| |
| Appetite decreased nos | Metabolism and nutrition disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Bone pain | Musculoskeletal and connective tissue disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Chest wall pain | Musculoskeletal and connective tissue disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Groin pain | Musculoskeletal and connective tissue disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Muscle cramp | Musculoskeletal and connective tissue disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Pain in foot | Musculoskeletal and connective tissue disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Pain in limb | Musculoskeletal and connective tissue disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Peripheral swelling | Musculoskeletal and connective tissue disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Carpal tunnel syndrome | Nervous system disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Hyperreflexia | Nervous system disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Hypoaesthesia | Nervous system disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Insomnia | Nervous system disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Migraine nos | Nervous system disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Paraesthesia | Nervous system disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Post-Traumatic headache | Nervous system disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Somnolence | Nervous system disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Tremor | Nervous system disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Abnormal behaviour nos | Psychiatric disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Testicular pain | Reproductive system and breast disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Asthma nos | Respiratory, thoracic and mediastinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Bronchitis nos | Respiratory, thoracic and mediastinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Bronchospasm nos | Respiratory, thoracic and mediastinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Dyspnoea nos | Respiratory, thoracic and mediastinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Nasal passage irritation | Respiratory, thoracic and mediastinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Nasopharyngitis | Respiratory, thoracic and mediastinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Obstructive airways disorder nos | Respiratory, thoracic and mediastinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Pharyngitis | Respiratory, thoracic and mediastinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Rhinitis allergic nos | Respiratory, thoracic and mediastinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Rhinitis nos | Respiratory, thoracic and mediastinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Rhonchi | Respiratory, thoracic and mediastinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Sleep apnoea syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Sneezing | Respiratory, thoracic and mediastinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Tachypnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Upper respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Wheezing | Respiratory, thoracic and mediastinal disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Acne nos | Skin and subcutaneous tissue disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Contusion | Skin and subcutaneous tissue disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Dyshidrosis | Skin and subcutaneous tissue disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Eczema | Skin and subcutaneous tissue disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Erythema | Skin and subcutaneous tissue disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Rash macular | Skin and subcutaneous tissue disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Rash nos | Skin and subcutaneous tissue disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Rash pruritic | Skin and subcutaneous tissue disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Sweating increased | Skin and subcutaneous tissue disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Urticaria nos | Skin and subcutaneous tissue disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Flushing | Vascular disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Hypertension nos | Vascular disorders | MedDRA (5.1) | Systematic Assessment |
| |
| Hypotension nos | Vascular disorders | MedDRA (5.1) | Systematic Assessment |
|
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Shire | +1 866 842 5335 | ClinicalTransparency@shire.com |
| ID | Term |
|---|---|
| D016532 | Mucopolysaccharidosis II |
| D013398 | Sudden Infant Death |
| ID | Term |
|---|---|
| D038901 | X-Linked Intellectual Disability |
| D008607 | Intellectual Disability |
| D019954 | Neurobehavioral Manifestations |
| D009461 | Neurologic Manifestations |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D009083 | Mucopolysaccharidoses |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D016464 | Lysosomal Storage Diseases |
| D017520 | Mucinoses |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D003645 | Death, Sudden |
| D003643 | Death |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D066088 | Infant Death |
Not provided
Not provided
| ID | Term |
|---|---|
| C517982 | idursulfase |
Not provided
Not provided
Not provided
| 12 to 18 years |
|
| 19 to 25 years |
|
| greater than equal to (>=) 26 years |
|
| Male |
|
| South America |
|
| Europe |
|
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