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MPS IIIA, also known as Sanfilippo A, is an inherited lysosomal storage disease (LSD). MPS IIIA is caused by a deficiency in sulfamidase, one of the enzymes involved in the lysosomal degradation of the glycosaminoglycan (GAG) heparan sulfate (HS). The natural course of MPS IIIA is characterized by devastating neurodegeneration with initially mild somatic involvement. The aims of the present study is to assess the dose related safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of SOBI003, a chemically modified recombinant human (rh) Sulfamidase developed as an enzyme replacement therapy (ERT).
This is an open-label, non-controlled, parallel, sequential ascending multiple-dose, multicenter study to assess the dose related safety, tolerability, PK and PD of SOBI003 in pediatric MPS IIIA patients. Patients between 1 and 6 years of age who have not received previous treatment for MPS IIIA with an ERT, gene- or stem cell therapy will be eligible to participate in the study. The study is planned to consist of 3 dose cohorts, each comprising 3 patients. Treatment initiations will be staggered within each cohort in order to be able to observe, interpret and treat possible adverse reactions. SOBI003 is administered as weekly i.v. infusions over a period of 24 weeks. Upon completion of the 24-week treatment period with satisfactory tolerability, the patient is offered to receive continued SOBI003 treatment by participation in an extension study.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Dose group 1 | Experimental | SOBI003 dose 3 mg/kg once weekly for 24 weeks |
|
| Dose group 2 | Experimental | SOBI003 dose 10 mg/kg once weekly for 24 weeks |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| SOBI003 | Drug | Weekly i.v.infusion |
|
| Measure | Description | Time Frame |
|---|---|---|
| Safety as Measured by Adverse Events Frequencies (by Type and Severity) | Number of adverse events, by type and severity, from start of infusion up to 24 weeks | From start of first infusion up to Week 24 |
| Measure | Description | Time Frame |
|---|---|---|
| The Observed Serum Concentration Immediately Before the Start of Infusion of SOBI003 | The observed serum concentration immediately before the start of infusion of SOBI003 (CPre-dose). | Weeks 1, 2, 3, 4, 8, 12, and 24 |
| The Observed Serum Concentration at the End of Infusion of SOBI003 |
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Inclusion Criteria:
Informed consent obtained from the patient's legally authorized representative(s)
Patients with MPS IIIA, as confirmed by both:
Chronological age of ≥12 and ≤72 months (i.e., 1 to 6 years) at the time of the first SOBI003 infusion and a developmental age ≥12 months at screening as assessed by the Vineland Adaptive Behavior Scales, Second Edition (VABS-II)
Medically stable patient who is expected to be able to comply with study procedures
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Paul Harmatz, MD | Childrens's Hospital and Research Center Oakland | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Childrens's Hospital and Research Center | Oakland | California | 94609 | United States | ||
| University of North Carolina Hospitals |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 35835061 | Derived | Harmatz P, Muenzer J, Ezgu F, Dalen P, Huledal G, Lindqvist D, Gelius SS, Wiken M, Onnestam K, Broijersen A. Chemically modified recombinant human sulfamidase (SOBI003) in mucopolysaccharidosis IIIA patients: Results from an open, non-controlled, multicenter study. Mol Genet Metab. 2022 Aug;136(4):249-259. doi: 10.1016/j.ymgme.2022.06.008. Epub 2022 Jun 28. |
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| ID | Title | Description |
|---|---|---|
| FG000 | Dose Group 1 | SOBI003 dose 3 mg/kg once weekly for 24 weeks SOBI003: Weekly i.v.infusion |
| FG001 | Dose Group 2 | SOBI003 dose 10 mg/kg once weekly for 24 weeks SOBI003: Weekly i.v.infusion |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Dose Group 1 | SOBI003 dose 3 mg/kg once weekly for 24 weeks SOBI003: Weekly i.v.infusion |
| BG001 | Dose Group 2 | SOBI003 dose 10 mg/kg once weekly for 24 weeks SOBI003: Weekly i.v.infusion |
| 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 day of first infusion (months) |
| 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 | Safety as Measured by Adverse Events Frequencies (by Type and Severity) | Number of adverse events, by type and severity, from start of infusion up to 24 weeks | Posted | Number | number of events | From start of first infusion up to Week 24 |
|
The period for recording adverse events, including Serious Adverse Events (SAEs), began upon receiving the first dose of SOBI003 and ended at completion of the week 24 visit. In addition SAEs was reported from the time for signing the informed consent form until 28 days past the last dose of SOBI003.
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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 | Dose Group 1 | SOBI003 dose 3 mg/kg once weekly for 24 weeks SOBI003: Weekly i.v.infusion |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pyrexia | General disorders | MedDRA 20.1 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Iron deficiency anaemia | Blood and lymphatic system disorders | MedDRA 20.1 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Anders Bröijersén, Medical Director | Swedish Orphan Biovitrum AB | +46 8 697 20 00 | anders.broijersen@sobi.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jul 6, 2018 | Feb 22, 2021 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Mar 24, 2020 | Feb 22, 2021 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D009084 | Mucopolysaccharidosis III |
| ID | Term |
|---|---|
| D009083 | Mucopolysaccharidoses |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
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The study was designed to include three arms, up to 20 mg/kg. After a company decision to end the development of the compound it was decided to not start a third cohort, but if stated safe the dose could increase up to 20 mg/kg in cohort 1 and 2.
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The observed serum concentration at the end of infusion of SOBI003 (CEnd of inf) |
| Weeks 1, 2, 3, 4, 8, 12, and 24 |
| The Time of the End of the Infusion of SOBI003 | The time of the end of infusion of SOBI003 (tEnd of inf) | Weeks 1, 2, 3, 4, 8, 12, and 24 |
| The Maximum Observed Serum Concentration of SOBI003 | The Maximum Observed Serum Concentration of SOBI003 (Cmax) | 0, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96 hours post-dose on Weeks 1, 4, 12, and 24 |
| The Time at Which the Maximum Serum Concentration of SOBI003 is Observed | The time after start of infusion at which the maximum serum concentration is observed (tmax) | Weeks 1, 4, 12, and 24 |
| The Minimum Observed Serum Concentration of SOBI003 | The minimum observed serum concentration of SOBI003 (CTrough) | Weeks 1, 4, 12, and 24 |
| Clearance | Clearance (CL) of SOBI003 | Weeks 1, 4, 12, and 24 |
| Area Under the Serum Concentration-time Curve From Time 0 to 168 Hours | Area under the serum concentration-time curve from time 0 to 168 hours (AUC 0-168h) | 0,1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 120, 144, 168 hours post-dose on Weeks 1, 4,12, and 24 |
| The Half-life | The half-life of SOBI003 in serum (T1/2) | Weeks 1, 4, 12, and 24 |
| SOBI003 Concentration in Cerebrospinal Fluid | SOBI003 concentration in cerebrospinal fluid | Weeks 12 and 24 |
| Number of Patients Having Anti-drug Antibodies in Serum | Number of patients in each dose group having anti-drug antibodies in serum | Weeks 2,4,8,12 and 24 |
| Patients Having Anti-drug Antibodies in Cerebrospinal Fluid | Percent of patients having anti-drug antibodies in cerebrospinal fluid | Weeks 12 and 24 |
| Change From Baseline in Heparan Sulfate Levels in Cerebrospinal Fluid | Change from baseline, in percent, of Heparan Sulfate levels in cerebrospinal fluid | Baseline, weeks 12, and 24 |
| Change From Baseline in Heparan Sulfate Levels in Serum | Change from baseline in Heparan sulfate levels in serum | Weeks 2, 3, 4, 8, 12 and 24 |
| Change From Baseline in Heparan Sulfate Levels in Urine | Change from baseline in Heparan sulfate levels in urine | Weeks 2, 3, 4, 8, 12 and 24 |
| Change From Baseline in Neurocognitive Development Quotient | Quotient between age equivalent score and age, 0 - 100%, where high values are desirable. The age equivalent score represent the age of the typical and normal individual who would achieve the same result as the one who was tested. The age equivalent scores are assessed by the Bayley Scales of Infant and Toddler Development®, third edition cognitive subtest or the Kaufman Assessment Battery for Children, Second edition. The Bayley Scales of Infant and Toddler Development-Third Edition is an individually administered test designed to assess developmental functioning of infants and toddlers. The Bayley-III assesses development in five areas: cognitive, language, motor, social-emotional, and adaptive behavior. The Kaufman Assessment Battery for Children (K-ABC) is a clinical instrument for assessing cognitive development. | Week 24 |
| Change From Baseline in Age-equivalence Score | The age equivalent score represent the age in months of the typical and normal individual who would achieve the same result as the one who was tested. The age equivalent scores are assessed by the Bayley Scales of Infant and Toddler Development®, third edition cognitive subtest or the Kaufman Assessment Battery for Children, Second edition. The Bayley Scales of Infant and Toddler Development-Third Edition is an individually administered test designed to assess developmental functioning of infants and toddlers. The Bayley-III assesses development in five areas: cognitive, language, motor, social-emotional, and adaptive behavior. The Kaufman Assessment Battery for Children (K-ABC) is a clinical instrument for assessing cognitive development. | Week 24 |
| Change From Baseline in Age-equivalence Score as Assessed by VABS-II | The age equivalent score represent the age in months of the typical and normal individual who would achieve the same result as the one who was tested. The age equivalent scores are assessed by Vinelandâ„¢ Adaptive Behavior Scales, Expanded Interview Form, Second edition (VABS-II). The Vineland is designed to measure adaptive behavior of individuals from birth to age 90. The Vineland-II contains 5 domains each with 2-3 subdomains. The main domains are: Communication, Daily Living Skills, Socialization, Motor Skills, and Maladaptive Behavior. | Week 24 |
| Change From Baseline in Gray Matter Volume | Grey matter contains most of the brain's neuronal cell bodies. The grey matter includes regions of the brain involved in muscle control, and sensory perception such as seeing and hearing, memory, emotions, speech, decision making, and self-control. The gray matter volume will be measured by volumetric magnetic resonance imaging (MRI). | Week 24 |
| Change From Baseline in Pediatric Quality of Life Inventory (PedsQLâ„¢) Total Score | Pediatric Quality of Life Inventory (PedsQLâ„¢) is a modular approach to measuring health-related quality of life (HRQOL) in healthy children and adolescents and those with acute and chronic health conditions. Lower scores indicate better functioning. Min score = 0, and max score = 144. | Week 24 |
| Change From Baseline in PedsQLâ„¢ Family Impact Module Total Score | Pediatric Quality of Life Inventory (PedsQLâ„¢) is a modular approach to measuring health-related quality of life in healthy children and adolescents and those with acute and chronic health conditions. The measure includes a scale, from where the categorical score "4", "3", "2", "1", and "0" was reversed and linearly transformed to a 0-100 scale to 4=0, 3=25, 2=50, 1=75 and 0=100, where 100 = minimum and 0 = maximum. The Total Score is the sum of all 36 items in the test divided by the number of items answered. Higher scores indicate better functioning. | Week 24 |
| Chapel Hill |
| North Carolina |
| 27599 |
| United States |
| University Medical Center Hamburg-Eppendorf | Hamburg | Germany |
| Gazi University Hospital | Ankara | Turkey (Türkiye) |
| BG002 | Total | Total of all reporting groups |
| Mean |
| Standard Deviation |
| months |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Units |
|---|
| Counts |
|---|
| Participants |
|
|
| Secondary | The Observed Serum Concentration Immediately Before the Start of Infusion of SOBI003 | The observed serum concentration immediately before the start of infusion of SOBI003 (CPre-dose). | The table report number of available pharmacokinetic (PK) samples | Posted | Median | Full Range | ng/mL | Weeks 1, 2, 3, 4, 8, 12, and 24 |
|
|
|
| Secondary | The Observed Serum Concentration at the End of Infusion of SOBI003 | The observed serum concentration at the end of infusion of SOBI003 (CEnd of inf) | Number of analysed are available samples | Posted | Mean | Full Range | ng/mL | Weeks 1, 2, 3, 4, 8, 12, and 24 |
|
|
|
| Secondary | The Time of the End of the Infusion of SOBI003 | The time of the end of infusion of SOBI003 (tEnd of inf) | Number of analysed are available samples | Posted | Median | Full Range | Hours | Weeks 1, 2, 3, 4, 8, 12, and 24 |
|
|
|
| Secondary | The Maximum Observed Serum Concentration of SOBI003 | The Maximum Observed Serum Concentration of SOBI003 (Cmax) | Samples was taken centrally and/or peripherally. | Posted | Median | Full Range | ng/mL | 0, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96 hours post-dose on Weeks 1, 4, 12, and 24 |
|
|
|
| Secondary | The Time at Which the Maximum Serum Concentration of SOBI003 is Observed | The time after start of infusion at which the maximum serum concentration is observed (tmax) | Posted | Median | Full Range | Hours | Weeks 1, 4, 12, and 24 |
|
|
|
| Secondary | The Minimum Observed Serum Concentration of SOBI003 | The minimum observed serum concentration of SOBI003 (CTrough) | Posted | Median | Full Range | ng/mL | Weeks 1, 4, 12, and 24 |
|
|
|
| Secondary | Clearance | Clearance (CL) of SOBI003 | Posted | Median | Full Range | (mL/h)/kg | Weeks 1, 4, 12, and 24 |
|
|
|
| Secondary | Area Under the Serum Concentration-time Curve From Time 0 to 168 Hours | Area under the serum concentration-time curve from time 0 to 168 hours (AUC 0-168h) | Number of analysed are available samples | Posted | Median | Full Range | h*ng/mL | 0,1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 120, 144, 168 hours post-dose on Weeks 1, 4,12, and 24 |
|
|
|
| Secondary | The Half-life | The half-life of SOBI003 in serum (T1/2) | Posted | Median | Full Range | Hours | Weeks 1, 4, 12, and 24 |
|
|
|
| Secondary | SOBI003 Concentration in Cerebrospinal Fluid | SOBI003 concentration in cerebrospinal fluid | Number of analysed are available samples | Posted | Median | Full Range | mg/L | Weeks 12 and 24 |
|
|
|
| Secondary | Number of Patients Having Anti-drug Antibodies in Serum | Number of patients in each dose group having anti-drug antibodies in serum | Posted | Count of Participants | Participants | Weeks 2,4,8,12 and 24 |
|
|
|
| Secondary | Patients Having Anti-drug Antibodies in Cerebrospinal Fluid | Percent of patients having anti-drug antibodies in cerebrospinal fluid | Posted | Number | percent of participants in dose group | Weeks 12 and 24 |
|
|
|
| Secondary | Change From Baseline in Heparan Sulfate Levels in Cerebrospinal Fluid | Change from baseline, in percent, of Heparan Sulfate levels in cerebrospinal fluid | Posted | Median | Full Range | percent change | Baseline, weeks 12, and 24 |
|
|
|
| Secondary | Change From Baseline in Heparan Sulfate Levels in Serum | Change from baseline in Heparan sulfate levels in serum | Posted | Median | Full Range | mg/L | Weeks 2, 3, 4, 8, 12 and 24 |
|
|
|
| Secondary | Change From Baseline in Heparan Sulfate Levels in Urine | Change from baseline in Heparan sulfate levels in urine | Posted | Median | Full Range | g/mol | Weeks 2, 3, 4, 8, 12 and 24 |
|
|
|
| Secondary | Change From Baseline in Neurocognitive Development Quotient | Quotient between age equivalent score and age, 0 - 100%, where high values are desirable. The age equivalent score represent the age of the typical and normal individual who would achieve the same result as the one who was tested. The age equivalent scores are assessed by the Bayley Scales of Infant and Toddler Development®, third edition cognitive subtest or the Kaufman Assessment Battery for Children, Second edition. The Bayley Scales of Infant and Toddler Development-Third Edition is an individually administered test designed to assess developmental functioning of infants and toddlers. The Bayley-III assesses development in five areas: cognitive, language, motor, social-emotional, and adaptive behavior. The Kaufman Assessment Battery for Children (K-ABC) is a clinical instrument for assessing cognitive development. | Posted | Median | Full Range | unitless | Week 24 |
|
|
|
| Secondary | Change From Baseline in Age-equivalence Score | The age equivalent score represent the age in months of the typical and normal individual who would achieve the same result as the one who was tested. The age equivalent scores are assessed by the Bayley Scales of Infant and Toddler Development®, third edition cognitive subtest or the Kaufman Assessment Battery for Children, Second edition. The Bayley Scales of Infant and Toddler Development-Third Edition is an individually administered test designed to assess developmental functioning of infants and toddlers. The Bayley-III assesses development in five areas: cognitive, language, motor, social-emotional, and adaptive behavior. The Kaufman Assessment Battery for Children (K-ABC) is a clinical instrument for assessing cognitive development. | Posted | Median | Full Range | Months | Week 24 |
|
|
|
| Secondary | Change From Baseline in Age-equivalence Score as Assessed by VABS-II | The age equivalent score represent the age in months of the typical and normal individual who would achieve the same result as the one who was tested. The age equivalent scores are assessed by Vinelandâ„¢ Adaptive Behavior Scales, Expanded Interview Form, Second edition (VABS-II). The Vineland is designed to measure adaptive behavior of individuals from birth to age 90. The Vineland-II contains 5 domains each with 2-3 subdomains. The main domains are: Communication, Daily Living Skills, Socialization, Motor Skills, and Maladaptive Behavior. | Posted | Median | Full Range | Months | Week 24 |
|
|
|
| Secondary | Change From Baseline in Gray Matter Volume | Grey matter contains most of the brain's neuronal cell bodies. The grey matter includes regions of the brain involved in muscle control, and sensory perception such as seeing and hearing, memory, emotions, speech, decision making, and self-control. The gray matter volume will be measured by volumetric magnetic resonance imaging (MRI). | Posted | Median | Full Range | mL | Week 24 |
|
|
|
| Secondary | Change From Baseline in Pediatric Quality of Life Inventory (PedsQLâ„¢) Total Score | Pediatric Quality of Life Inventory (PedsQLâ„¢) is a modular approach to measuring health-related quality of life (HRQOL) in healthy children and adolescents and those with acute and chronic health conditions. Lower scores indicate better functioning. Min score = 0, and max score = 144. | Posted | Mean | Standard Deviation | Units on a scale | Week 24 |
|
|
|
| Secondary | Change From Baseline in PedsQLâ„¢ Family Impact Module Total Score | Pediatric Quality of Life Inventory (PedsQLâ„¢) is a modular approach to measuring health-related quality of life in healthy children and adolescents and those with acute and chronic health conditions. The measure includes a scale, from where the categorical score "4", "3", "2", "1", and "0" was reversed and linearly transformed to a 0-100 scale to 4=0, 3=25, 2=50, 1=75 and 0=100, where 100 = minimum and 0 = maximum. The Total Score is the sum of all 36 items in the test divided by the number of items answered. Higher scores indicate better functioning. | Posted | Mean | Standard Deviation | Units on a scale | Week 24 |
|
|
|
| 0 |
| 3 |
| 1 |
| 3 |
| 3 |
| 3 |
| EG001 | Dose Group 2 | SOBI003 dose 10 mg/kg once weekly for 24 weeks SOBI003: Weekly i.v.infusion | 0 | 3 | 1 | 3 | 3 | 3 |
| Device related infestations | Infections and infestations | MedDRA 20.1 | Systematic Assessment |
|
| Endocarditis | Infections and infestations | MedDRA 20.1 | Systematic Assessment |
|
| Pneumonia | Infections and infestations | MedDRA 20.1 | Systematic Assessment |
|
| Tachycardia | Cardiac disorders | MedDRA 20.1 | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA 20.1 | Systematic Assessment |
|
| Medical device site haemorrhage | General disorders | MedDRA 20.1 | Systematic Assessment |
|
| Conjunctivitis | Infections and infestations | MedDRA 20.1 | Systematic Assessment |
|
| Gastroenteritis | Infections and infestations | MedDRA 20.1 | Systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA 20.1 | Systematic Assessment |
|
| Viral infection | Infections and infestations | MedDRA 20.1 | Systematic Assessment |
|
| Vascular access site occlusion | Injury, poisoning and procedural complications | MedDRA 20.1 | Systematic Assessment |
|
| Tremor | Nervous system disorders | MedDRA 20.1 | Systematic Assessment |
|
| Device damage | Product Issues | MedDRA 20.1 | Systematic Assessment |
|
| Device malfunction | Product Issues | MedDRA 20.1 | Systematic Assessment |
|
| Anxiety | Psychiatric disorders | MedDRA 20.1 | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 20.1 | Systematic Assessment |
|
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA 20.1 | Systematic Assessment |
|
| Hyperaemia | Vascular disorders | MedDRA 20.1 | Systematic Assessment |
|
| Aortic valve thickening | Cardiac disorders | MedDRA 20.1 | Systematic Assessment |
|
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 20.1 | Systematic Assessment |
|
| Abnormal faeces | Gastrointestinal disorders | MedDRA 20.1 | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA 20.1 | Systematic Assessment |
|
| Faeces discoloured | Gastrointestinal disorders | MedDRA 20.1 | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA 20.1 | Systematic Assessment |
|
| Application site irritation | General disorders | MedDRA 20.1 | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA 20.1 | Systematic Assessment |
|
| Otitis media acute | Infections and infestations | MedDRA 20.1 | Systematic Assessment |
|
| Arthropod bite | Injury, poisoning and procedural complications | MedDRA 20.1 | Systematic Assessment |
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| Contusion | Injury, poisoning and procedural complications | MedDRA 20.1 | Systematic Assessment |
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| Fall | Injury, poisoning and procedural complications | MedDRA 20.1 | Systematic Assessment |
|
| Lip injury | Injury, poisoning and procedural complications | MedDRA 20.1 | Systematic Assessment |
|
| Skin abrasion | Injury, poisoning and procedural complications | MedDRA 20.1 | Systematic Assessment |
|
| Vascular access complication | Injury, poisoning and procedural complications | MedDRA 20.1 | Systematic Assessment |
|
| Activated partial thromboplastin time prolonged | Investigations | MedDRA 20.1 | Systematic Assessment |
|
| Blood fibrinogen increased | Investigations | MedDRA 20.1 | Systematic Assessment |
|
| Blood pressure diastolic increased | Investigations | MedDRA 20.1 | Systematic Assessment |
|
| Blood pressure systolic increased | Investigations | MedDRA 20.1 | Systematic Assessment |
|
| C-reactive protein increased | Investigations | MedDRA 20.1 | Systematic Assessment |
|
| CSF glucose decreased | Investigations | MedDRA 20.1 | Systematic Assessment |
|
| CSF protein increased | Investigations | MedDRA 20.1 | Systematic Assessment |
|
| Eosinophil count increased | Investigations | MedDRA 20.1 | Systematic Assessment |
|
| Haemoglobin increased | Investigations | MedDRA 20.1 | Systematic Assessment |
|
| Monocyte count decreased | Investigations | MedDRA 20.1 | Systematic Assessment |
|
| Neutrophil count increased | Investigations | MedDRA 20.1 | Systematic Assessment |
|
| Oxygen saturation decreased | Investigations | MedDRA 20.1 | Systematic Assessment |
|
| Platelet count decreased | Investigations | MedDRA 20.1 | Systematic Assessment |
|
| Red blood cell count increased | Investigations | MedDRA 20.1 | Systematic Assessment |
|
| White blood cell count increased | Investigations | MedDRA 20.1 | Systematic Assessment |
|
| Clonus | Nervous system disorders | MedDRA 20.1 | Systematic Assessment |
|
| Device dislocation | Product Issues | MedDRA 20.1 | Systematic Assessment |
|
| Restlessness | Psychiatric disorders | MedDRA 20.1 | Systematic Assessment |
|
| Dermatitis diaper | Skin and subcutaneous tissue disorders | MedDRA 20.1 | Systematic Assessment |
|
| Erythema | Skin and subcutaneous tissue disorders | MedDRA 20.1 | Systematic Assessment |
|
| Hyperhidrosis | Skin and subcutaneous tissue disorders | MedDRA 20.1 | Systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | MedDRA 20.1 | Systematic Assessment |
|
| Rash erythematous | Skin and subcutaneous tissue disorders | MedDRA 20.1 | Systematic Assessment |
|
| Vascular device user | Social circumstances | MedDRA 20.1 | Systematic Assessment |
|
Not provided
Not provided
| 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 |
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