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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 aim of the present study is to assess the safety, tolerability and efficacy of long-term SOBI003 treatment. SOBI003 is a chemically modified recombinant human (rh) Sulfamidase developed as an enzyme replacement therapy (ERT).
This is an open, single-arm, multicenter extension study to assess the safety, tolerability and efficacy of long-term SOBI003 treatment in pediatric MPS IIIA patients. The study is an extension of the First in Human (FIH) SOBI003-001 study, allowing continuous treatment of SOBI003 for up to 2 years. Study patients who complete Week 24 of the FIH study (SOBI003-001) will be invited to continue to Week 25 in the extension study.
When entering the extension study, these patients will receive the highest dose that has been declared safe in the ongoing FIH study (SOBI003-001). Upon completion of the FIH study, an analysis aimed at selecting the dose for forthcoming studies will take place. Once the dose has been selected, this dose will be applied to all patients enrolled in the extension study. The total duration of the extension study for an individual patient is 80 weeks, resulting in a total of 104 weeks (2 years) of SOBI003 treatment.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| SOBI003 | Experimental | SOBI003 solution, 20 mg/mL, is mixed with NaCl 0.9% infusion solution prior to administration. For a bodyweight < 25 kg, the total infusion volume is 100 mL. For a bodyweight ≥ 25 kg, the total infusion volume is 250 mL. SOBI003 is administered as i.v. infusions given once weekly for a duration of 80 weeks (from Week 25 until Week 104 following the first 24 weeks of SOBI003 administration in the FIH study (SOBI003-001) study. The SOBI003 dose will be adjusted to the highest dose that has been declared safe by the safety review committee on the FIH study.Hence, dose adjustments may occur a couple of times on the extension study until the final decided dose has been determined. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| SOBI003 | Drug | weekly i.v. infusion |
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| Measure | Description | Time Frame |
|---|---|---|
| Safety as Measured by Adverse Events Frequencies (by Type and Severity) | Number of adverse events, by type and severity, from week 25 up to week 104 | From infusion week 25 up to week 104 |
| Measure | Description | Time Frame |
|---|---|---|
| The Observed SOBI003 Serum Concentration Immediately Before the Start of Infusion of SOBI003 | The observed SOBI003 serum concentration immediately before the start of infusion of SOBI003 (CPre-dose) Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Paul Harmatz, MD | Children´s Hospital and Research Center, Oakland | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children´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 initial dose 3 mg/kg, once weekly from infusion week 25 to week 104 SOBI003: weekly i.v. infusion |
| FG001 | Dose Group 2 | SOBI003 initial dose 10 mg/kg, once weekly from infusion week 25 to week 104 SOBI003: weekly i.v. infusion |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Dose Group 1 | SOBI003 initial dose 3 mg/kg, once weekly from infusion week 25 to week 104 SOBI003: weekly i.v. infusion |
| BG001 | Dose Group 2 | SOBI003 initial dose 10 mg/kg, once weekly from infusion week 25 to week 104 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 | 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 | Safety as Measured by Adverse Events Frequencies (by Type and Severity) | Number of adverse events, by type and severity, from week 25 up to week 104 | Posted | Number | events | From infusion week 25 up to week 104 |
|
The period for recording adverse events, including Serious Adverse Events (SAEs), began Day 1 of week 25, when first dose in study SOBI003-002 was given, and ended at completion of week 104 visit. In addition SAEs was reported from the time of 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 initial dose 3 mg/kg, once weekly from infusion week 25 to week 104 SOBI003: weekly i.v. infusion |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pyrexia | General disorders | MedDRA 23.0 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Iron deficiency anaemia | Blood and lymphatic system disorders | MedDRA 23.0 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Global Communication | Sobi | +4686972000 | info@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 | Jun 29, 2018 | Dec 7, 2021 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Sep 13, 2018 | Dec 7, 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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| Weeks 38, 52, 78 and 104 |
| The Observed SOBI003 Serum Concentration at the End of Infusion of SOBI003 | The observed SOBI003 serum concentration at the end of infusion of SOBI003 (CEnd of inf) Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture. | Weeks 38, 52, 78 and 104 |
| The Time of the End of the Infusion of SOBI003 | The time of the end of infusion of SOBI003 (tEnd of inf) Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture. | Weeks 38, 52, 78 and 104 |
| The Maximum Observed Serum Concentration of SOBI003 | The maximum observed serum concentration of SOBI003 (Cmax) Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture. | Weeks 38, 52, 78 and 104 |
| 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) Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture. | Weeks 38, 52, 78 and 104 |
| The Minimum Observed Serum Concentration of SOBI003 | The minimum observed serum concentration of SOBI003 (CTrough) Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture. | Weeks 38, 52, 78 and 104 |
| Clearance | Clearance (CL) of SOBI003 Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture. | Weeks 38, 52, 78 and 104 |
| Area Under the SOBI003 Serum Concentration-time Curve From Time 0 to168 Hours | Area under the SOBI003 serum concentration-time curve from time 0 to 168 hours (AUC 0-168h) Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture. | 0,1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 120, 144, 168 hours post-dose on Weeks 38, 52, 78 and 104 |
| The Half-life | The half-life of SOBI003 in serum (T1/2) Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture. | Weeks 38, 52, 78 and 104 |
| SOBI003 Concentration in Cerebrospinal Fluid | Concentration of SOBI003 in cerebrospinal fluid | Weeks 52 and 104 |
| Number of Patients Having Anti-drug Antibodies in Serum | Number of patients in each dose group having anti-drug antibodies in serum | Weeks 38, 52, 78 and 104 |
| Number of Patients Having Anti-drug Antibodies in Cerebrospinal Fluid | Number of patients in each dose group having anti-drug antibodies cerebrospinal fluid | Weeks 52 and 104 |
| Change From Baseline in Heparan Sulfate Concentration in Cerebrospinal Fluid | Change from baseline, in percent, of Heparan Sulfate levels in cerebrospinal fluid | Weeks 52 and 104 |
| Change From Baseline in Heparan Sulfate Levels in Serum | Change from baseline in Heparan sulfate, in mg/L, levels in serum | Weeks 38, 52, 78 and 104 |
| Change From Baseline in Heparan Sulfate Levels in Urine | Change from baseline in Heparan sulfate levels, in g/mol, in urine | Weeks 38, 52, 78 and 104 |
| 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. These results are truly "unitless"/"dimensionless" because they represents quotients of values from the same scale, which means that the units in the denominator and | Weeks 52 and 104 |
| 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 52 and 104 |
| Age-equivalence Score as Assessed Either by the BSID-III, Cognitive Subtest, or the KABC-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 Vineland™ 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 52 and 104 |
| Change From Baseline in Age-equivalence Score as Assessed Either by the BSID-III, Cognitive Subtest, or the KABC-II. | The age equivalent score represents 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 either by the Bayley Scales of Infant and Toddler Development®, third edition, (BSID-III) cognitive subtest or the Kaufman Assessment Battery for Children, Second edition (KABC-II) depending on chronological age of the subject. Quotient between age equivalent score and age, 0 - 100%, where high values are desirable. The BSID-III is an individually administered test designed to assess developmental functioning of infants and toddlers. The BSID-III assesses development in five areas: cognitive, language, motor, social-emotional, and adaptive behavior. The KABC-II is a clinical instrument for assessing cognitive development. The unit and measurement is the same in both scales (BSID-III and KABC-II): Age-equivalent score. | Week 52 and 104 |
| 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 Vineland™ 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 52 and 104 |
| 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 Vineland™ 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 52 and 104 |
| 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) at weeks 52 and 104. | Week 52 and 104 |
| Pediatric Quality of Life Inventory (PedsQL™) Total Score | Pediatric Quality of Life Inventory (PedsQL™) is a modular approach to measuring health-related quality of life 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 52 and 104 |
| Change From Baseline in Pediatric Quality of Life Inventory (PedsQL™) Total Score | Pediatric Quality of Life Inventory (PedsQL™) 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. Higher scores indicate better functioning. Min score = 0, and max score = 144. | Week 52 and 104 |
| PedsQL™ Family Impact Module Total Score | Pediatric Quality of Life Inventory (PedsQL™) 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 Total Score is the sum of all 36 items in the test divided by the number of items answered. Higher scores indicate better functioning. Min score = 0, and max score = 144. | Week 52 and 104 |
| Change From Baseline in PedsQL™ Family Impact Module Total Score | Pediatric Quality of Life Inventory (PedsQL™) 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 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 52 and 104 |
| Chapel Hill |
| North Carolina |
| 27599 |
| United States |
| Gazi University Hospital | Ankara | Turkey (Türkiye) |
| BG002 | Total | Total of all reporting groups |
| months |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Units | Counts |
|---|
| Participants |
|
|
| Secondary | The Observed SOBI003 Serum Concentration Immediately Before the Start of Infusion of SOBI003 | The observed SOBI003 serum concentration immediately before the start of infusion of SOBI003 (CPre-dose) Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture. | The table report number of available pharmacokinetic (PK) samples | Posted | Mean | Standard Deviation | ng/mL | Weeks 38, 52, 78 and 104 |
|
|
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| Secondary | The Observed SOBI003 Serum Concentration at the End of Infusion of SOBI003 | The observed SOBI003 serum concentration at the end of infusion of SOBI003 (CEnd of inf) Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture. | The table report number of available pharmacokinetic (PK) samples | Posted | Mean | Standard Deviation | ng/mL | Weeks 38, 52, 78 and 104 |
|
|
|
| Secondary | The Time of the End of the Infusion of SOBI003 | The time of the end of infusion of SOBI003 (tEnd of inf) Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture. | Number of analysed are available samples | Posted | Median | Full Range | Hours | Weeks 38, 52, 78 and 104 |
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| Secondary | The Maximum Observed Serum Concentration of SOBI003 | The maximum observed serum concentration of SOBI003 (Cmax) Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture. | Number of analysed are available samples | Posted | Median | Full Range | ng/mL | Weeks 38, 52, 78 and 104 |
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|
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| 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) Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture. | Number of analysed are available samples | Posted | Median | Full Range | Hours | Weeks 38, 52, 78 and 104 |
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| Secondary | The Minimum Observed Serum Concentration of SOBI003 | The minimum observed serum concentration of SOBI003 (CTrough) Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture. | Number of analysed are available samples | Posted | Median | Full Range | ng/mL | Weeks 38, 52, 78 and 104 |
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| Secondary | Clearance | Clearance (CL) of SOBI003 Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture. | Number of analysed are available samples | Posted | Median | Full Range | mL/min | Weeks 38, 52, 78 and 104 |
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| Secondary | Area Under the SOBI003 Serum Concentration-time Curve From Time 0 to168 Hours | Area under the SOBI003 serum concentration-time curve from time 0 to 168 hours (AUC 0-168h) Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture. | 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 38, 52, 78 and 104 |
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| Secondary | The Half-life | The half-life of SOBI003 in serum (T1/2) Blood samples for serum PK analysis were collected either by centrally (i.e., using a catheter in the central venous port) or peripheral (i.e., by venipuncture). Central venous sampling is more convenient than repeated venipuncture. | Number of analysed are available samples | Posted | Weeks 38, 52, 78 and 104 |
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| Secondary | SOBI003 Concentration in Cerebrospinal Fluid | Concentration of SOBI003 in cerebrospinal fluid | Number analysed are available samples | Posted | Median | Full Range | ng/mL | Weeks 52 and 104 |
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| 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 38, 52, 78 and 104 |
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| Secondary | Number of Patients Having Anti-drug Antibodies in Cerebrospinal Fluid | Number of patients in each dose group having anti-drug antibodies cerebrospinal fluid | Posted | Count of Participants | Participants | Weeks 52 and 104 |
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| Secondary | Change From Baseline in Heparan Sulfate Concentration in Cerebrospinal Fluid | Change from baseline, in percent, of Heparan Sulfate levels in cerebrospinal fluid | Posted | Median | Full Range | mg/L | Weeks 52 and 104 |
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| Secondary | Change From Baseline in Heparan Sulfate Levels in Serum | Change from baseline in Heparan sulfate, in mg/L, levels in serum | Posted | Median | Full Range | mg/L | Weeks 38, 52, 78 and 104 |
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| Secondary | Change From Baseline in Heparan Sulfate Levels in Urine | Change from baseline in Heparan sulfate levels, in g/mol, in urine | Number of analysed are available samples | Posted | Median | Full Range | g/mol | Weeks 38, 52, 78 and 104 |
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| 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. These results are truly "unitless"/"dimensionless" because they represents quotients of values from the same scale, which means that the units in the denominator and | Number of analysed are number of assessments | Posted | Median | Full Range | Unitless | Weeks 52 and 104 |
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| 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. | Number of analyses are available assessments | Posted | Median | Full Range | Months | Week 52 and 104 |
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| Secondary | Age-equivalence Score as Assessed Either by the BSID-III, Cognitive Subtest, or the KABC-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 Vineland™ 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. | Number of analyses are available assessments | Posted | Median | Full Range | Months | Week 52 and 104 |
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| Secondary | Change From Baseline in Age-equivalence Score as Assessed Either by the BSID-III, Cognitive Subtest, or the KABC-II. | The age equivalent score represents 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 either by the Bayley Scales of Infant and Toddler Development®, third edition, (BSID-III) cognitive subtest or the Kaufman Assessment Battery for Children, Second edition (KABC-II) depending on chronological age of the subject. Quotient between age equivalent score and age, 0 - 100%, where high values are desirable. The BSID-III is an individually administered test designed to assess developmental functioning of infants and toddlers. The BSID-III assesses development in five areas: cognitive, language, motor, social-emotional, and adaptive behavior. The KABC-II is a clinical instrument for assessing cognitive development. The unit and measurement is the same in both scales (BSID-III and KABC-II): Age-equivalent score. | Number of analysed are available assessments | Posted | Median | Full Range | Months | Week 52 and 104 |
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| Secondary | 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 Vineland™ 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 52 and 104 |
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| 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 Vineland™ 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 52 and 104 |
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| 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) at weeks 52 and 104. | Number analysed is available assessments | Posted | Median | Full Range | mL | Week 52 and 104 |
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| Secondary | Pediatric Quality of Life Inventory (PedsQL™) Total Score | Pediatric Quality of Life Inventory (PedsQL™) is a modular approach to measuring health-related quality of life 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 52 and 104 |
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| Secondary | Change From Baseline in Pediatric Quality of Life Inventory (PedsQL™) Total Score | Pediatric Quality of Life Inventory (PedsQL™) 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. Higher scores indicate better functioning. Min score = 0, and max score = 144. | Posted | Mean | Standard Deviation | Units on a scale | Week 52 and 104 |
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| Secondary | PedsQL™ Family Impact Module Total Score | Pediatric Quality of Life Inventory (PedsQL™) 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 Total Score is the sum of all 36 items in the test divided by the number of items answered. Higher scores indicate better functioning. Min score = 0, and max score = 144. | Posted | Mean | Standard Deviation | Units on a scale | Week 52 and 104 |
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| Secondary | Change From Baseline in PedsQL™ Family Impact Module Total Score | Pediatric Quality of Life Inventory (PedsQL™) 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 Total Score is the sum of all 36 items in the test divided by the number of items answered. Higher scores indicate better functioning. | Number analysed is available assessments | Posted | Mean | Standard Deviation | Units on a scale | Week 52 and 104 |
|
|
|
| 0 |
| 3 |
| 2 |
| 3 |
| 3 |
| 3 |
| EG001 | Dose Group 2 | SOBI003 initial dose 10 mg/kg, once weekly from infusion week 25 to week 104 SOBI003: weekly i.v. infusion | 0 | 3 | 2 | 3 | 3 | 3 |
| Upper respiratory tract infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
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| Device related infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
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| Endocarditis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
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| Infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
|
| Influenza | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
|
| Pneumonia | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
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| Staphylococcal infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
|
| Incision site haemorrhage | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
|
| Aortic valve thickening | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
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| Cyanosis | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
|
| Tachycardia | Cardiac disorders | MedDRA 23.0 | Systematic Assessment |
|
| Auricular swelling | Ear and labyrinth disorders | MedDRA 23.0 | Systematic Assessment |
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| Otorrhoea | Ear and labyrinth disorders | MedDRA 23.0 | Systematic Assessment |
|
| Eye swelling | Eye disorders | MedDRA 23.0 | Systematic Assessment |
|
| Abdominal pain | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
|
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
|
| Abnormal faeces | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
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| Aphthous ulcer | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
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| Constipation | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
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| Dental caries | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
|
| Faeces discoloured | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
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| Haematochezia | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
|
| Lip disorder | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
|
| Oral contusion | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
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| Retching | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
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| Application site erythema | General disorders | MedDRA 23.0 | Systematic Assessment |
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| Catheter site extravasation | General disorders | MedDRA 23.0 | Systematic Assessment |
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| Catheter site inflammation | General disorders | MedDRA 23.0 | Systematic Assessment |
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| Catheter site injury | General disorders | MedDRA 23.0 | Systematic Assessment |
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| Complication associated with device | General disorders | MedDRA 23.0 | Systematic Assessment |
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| Fatigue | General disorders | MedDRA 23.0 | Systematic Assessment |
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| Infusion site bruising | General disorders | MedDRA 23.0 | Systematic Assessment |
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| Infusion site irritation | General disorders | MedDRA 23.0 | Systematic Assessment |
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| Medical device site haemorrhage | General disorders | MedDRA 23.0 | Systematic Assessment |
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| Oedema | General disorders | MedDRA 23.0 | Systematic Assessment |
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| Pain | General disorders | MedDRA 23.0 | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA 23.0 | Systematic Assessment |
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| Drug hypersensitivity | Immune system disorders | MedDRA 23.0 | Systematic Assessment |
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| Hypersensitivity | Immune system disorders | MedDRA 23.0 | Systematic Assessment |
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| Seasonal allergy | Immune system disorders | MedDRA 23.0 | Systematic Assessment |
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| Abscess | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
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| Cellulitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
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| Conjunctivitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
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| Corona virus infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
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| Device related infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
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| Ear infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
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| Endocarditis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
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| Furuncle | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
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| Gastroenteritis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
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| Infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
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| Influenza | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
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| Nasopharyngitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
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| Otitis media acute | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
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| Pharyngitis streptococcal | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
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| Pneumonia | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
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| Staphylococcal infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
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| Streptococcal infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
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| Tonsillitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
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| Upper respiratory tract infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
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| Viral infection | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
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| Arthropod bite | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
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| Contusion | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
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| Fall | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
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| Foreign body in gastrointestinal tract | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
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| Human bite | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
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| Incision site haemorrhage | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
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| Lip injury | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
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| Scratch | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
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| Skin abrasion | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
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| Soft tissue injury | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
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| Vascular access complication | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
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| Vascular access site occlusion | Injury, poisoning and procedural complications | MedDRA 23.0 | Systematic Assessment |
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| Activated partial thromboplastin time prolonged | Investigations | MedDRA 23.0 | Systematic Assessment |
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| Blood fibrinogen increased | Investigations | MedDRA 23.0 | Systematic Assessment |
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| Blood pressure diastolic increased | Investigations | MedDRA 23.0 | Systematic Assessment |
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| Blood pressure systolic increased | Investigations | MedDRA 23.0 | Systematic Assessment |
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| C-reactive protein increased | Investigations | MedDRA 23.0 | Systematic Assessment |
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| CSF glucose decreased | Investigations | MedDRA 23.0 | Systematic Assessment |
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| CSF protein increased | Investigations | MedDRA 23.0 | Systematic Assessment |
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| Cardiac murmur | Investigations | MedDRA 23.0 | Systematic Assessment |
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| Eosinophil count increased | Investigations | MedDRA 23.0 | Systematic Assessment |
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| Haemoglobin increased | Investigations | MedDRA 23.0 | Systematic Assessment |
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| Heart rate increased | Investigations | MedDRA 23.0 | Systematic Assessment |
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| Monocyte count decreased | Investigations | MedDRA 23.0 | Systematic Assessment |
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| Neutrophil count increased | Investigations | MedDRA 23.0 | Systematic Assessment |
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| Oxygen saturation decreased | Investigations | MedDRA 23.0 | Systematic Assessment |
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| Platelet count decreased | Investigations | MedDRA 23.0 | Systematic Assessment |
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| Red blood cell count increased | Investigations | MedDRA 23.0 | Systematic Assessment |
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| Vitamin D decreased | Investigations | MedDRA 23.0 | Systematic Assessment |
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| Weight decreased | Investigations | MedDRA 23.0 | Systematic Assessment |
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| White blood cell count increased | Investigations | MedDRA 23.0 | Systematic Assessment |
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| Muscle tightness | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
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| Skin papilloma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 23.0 | Systematic Assessment |
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| Ataxia | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
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| Clonus | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
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| Drooling | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
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| Tremor | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
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| Device damage | Product Issues | MedDRA 23.0 | Systematic Assessment |
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| Device dislocation | Product Issues | MedDRA 23.0 | Systematic Assessment |
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| Device malfunction | Product Issues | MedDRA 23.0 | Systematic Assessment |
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| Abnormal behaviour | Psychiatric disorders | MedDRA 23.0 | Systematic Assessment |
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| Anxiety | Psychiatric disorders | MedDRA 23.0 | Systematic Assessment |
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| Irritability | Psychiatric disorders | MedDRA 23.0 | Systematic Assessment |
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| Restlessness | Psychiatric disorders | MedDRA 23.0 | Systematic Assessment |
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| Sleep disorder | Psychiatric disorders | MedDRA 23.0 | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
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| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
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| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
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| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
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| Sneezing | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
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| Tachypnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
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| Wheezing | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
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| Dermatitis | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
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| Dermatitis allergic | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
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| Dermatitis contact | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
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| Dermatitis diaper | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
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| Dry skin | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
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| Erythema | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
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| Generalised erythema | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
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| Hyperhidrosis | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
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| Hyperkeratosis | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
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| Livedo reticularis | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
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| Rash | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
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| Rash erythematous | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
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| Rash maculo-papular | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
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| Skin discolouration | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
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| Skin fissures | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
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| Skin irritation | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
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| Swelling face | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
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| Urticaria | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
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| Flushing | Vascular disorders | MedDRA 23.0 | Systematic Assessment |
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| Hyperaemia | Vascular disorders | MedDRA 23.0 | Systematic Assessment |
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| Hypertension | Vascular disorders | MedDRA 23.0 | Systematic Assessment |
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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 |
| Week 52 - central serum |
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| Week 78 - central serum |
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| Week 104 - central serum |
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| Week 38 - peripheral serum |
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| Week 52 - peripheral serum |
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| Week 78 - peripheral serum |
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| Week 104 - peripheral serum |
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| Week 38 - peripheral serum |
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| Week 52 - central serum |
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| Week 52 - peripheral serum |
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| Week 78 - central serum |
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| Week 78 - peripheral serum |
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| Week 104 - central serum |
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| Week 104 - peripheral serum |
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| Week 52 - central serum |
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| Week 78 - central serum |
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| Week 104 - central serum |
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|
| Week 38 - peripheral serum |
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| Week 52- peripheral serum |
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| Week 78- peripheral serum |
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| Week 104- peripheral serum |
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| Week 52 - central serum |
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| Week 78 - central serum |
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| Week 104 - central serum |
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| Week 38 - peripheral serum |
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| Week 52 - peripheral serum |
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| Week 78 - peripheral serum |
|
|
| Week 104 - peripheral serum |
|
|
| Week 52 - central serum |
|
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| Week 78 - central serum |
|
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| Week 104 - central serum |
|
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| Week 38 - peripheral serum |
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| Week 52 - peripheral serum |
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| Week 78 - peripheral serum |
|
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| Week 104 - peripheral serum |
|
|
| Week 52 - central serum |
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| Week 78 - central serum |
|
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| Week 104 - central serum |
|
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| Week 38 - peripheral serum |
|
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| Week 52 - peripheral serum |
|
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| Week 78 - peripheral serum |
|
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| Week 104 - peripheral serum |
|
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| Week 78 - central serum |
|
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| Week 104 - central serum |
|
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| Week 38 - peripheral serum |
|
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| Week 52 - peripheral serum |
|
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| Week 78 - peripheral serum |
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| Week 104 - peripheral serum |
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| Week 78 - central serum |
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| Week 104 - central serum |
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| Week 38 - peripheral serum |
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| Week 52 - peripheral serum |
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| Week 78 - peripheral serum |
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| Week 104 - peripheral serum |
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| Week 104 |
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| Week 78 |
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| Week 104 |
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| Week 78 |
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| Week 104 |
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| Week 52 |
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| Week 78 |
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| Week 104 |
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| Week 104 |
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| Week 104 |
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| Week 104 |
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| Week 104 |
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| Week 104 |
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| Week 104 |
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