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| ID | Type | Description | Link |
|---|---|---|---|
| 2014-004143-13 | EudraCT Number |
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This extension study will allow participants that completed Study HGT-HIT-094 to continue receiving Elaprase treatment in conjunction with idursulfase IT or to continue receiving Elaprase treatment and begin concurrent IT treatment for those that did not receive idursulfase IT treatment in Study HGT-HIT-094.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Idursulfase-IT | Experimental | Participants received 10 milligrams (mg) of idursulfase-IT intrathecally via intrathecal drug delivery device (IDDD) or lumbar puncture (LP) once every 28 days along with standard-of-care therapy with Elaprase, for 470 weeks (for participants who began intrathecal [IT] treatment in this study) and 480 weeks (for participants who began IT treatment in HGT-HIT-094 and continued to receive IT treatment in this study). Participants who were younger than 3 years of age received an adjusted dose of 7.5 mg (>8 months to 30 months of age) or 10 mg (>30 months to 3years of age) of idursulfase-IT. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Idursulfase-IT | Drug | Participants received 10 mg of idursulfase-IT intrathecally via IDDD or LP once every 28 days. Participants who were younger than 3 years of age received an adjusted dose of 7.5 mg (>8 months to 30 months of age) and 10 mg (>30 months to 3 years of age). |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Adverse Events (AEs) | An AE is any noxious, pathologic, or unintended change in anatomical, physiologic, or metabolic function as indicated by physical signs, symptoms, or laboratory changes occurring in any phase of a clinical study, whether or not considered investigational product-related. | Up to 9 years |
| Number of Participants With Clinically Significant Changes in Vital Signs | Participants were assessed for clinically significant changes in vital signs like injection (IT) vital signs and regular vital signs (temperature, pulse, blood pressure [systolic and diastolic], oxygen saturation, and respiration rate). | Up to 9 years |
| Number of Participants With Clinically Significant Changes in Laboratory Parameters | Participants were assessed for clinically significant changes in laboratory parameters such as chemistry, hematology, urinalysis and cerebrospinal fluid (CSF) values. | Up to 9 years |
| Number of Participants With Clinically Significant Changes in 12-lead Electrocardiogram (ECG) Findings | Participants were assessed for clinically significant changes in 12-lead ECG findings (such as heart rate, PR interval, QRS interval, QT interval, and the corrected QT interval). | Up to 9 years |
| Maximum Observed Serum Concentration (Cmax) of Idursulfase | Idursulfase concentrations in serum were determined using a validated Enzyme -Linked Immunosorbent Assay (ELISA) method. Concentration for Cmax is presented in this endpoint. | Predose and at 30 min, 60 min, 120 min, 4 hours, 6 hours, 8 hours, 12 hours, 24 hours, 30 hours and 36 hours post-dose at Week 100 in relation to HGT-HIT-094 (Week 48 of this study) |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Differential Ability Scales, Second Edition (DAS-II) Standard Cluster Scores at Month 67 | DAS-II was used to assess all participants of age 2 years, 6 months or older. DAS-II comprises 2 overlapping batteries. Early Years battery (EYB) was designed for children ages 2 years, 6 months through 6 years, 11 months. The School Age Battery (SAB) was designed for children ages 7 years, 0 months through 17 years, 11months. These batteries are fully co-normed for ages 5 years, 0 months, through 8 years, 11 months. The cluster areas include general conceptual ability (GCA), verbal, nonverbal, spatial, and special nonverbal composite (SNC). The cluster area score represents a score (mean=100 and standard deviation=15) on which higher scores indicate higher level of cognitive ability. |
| Measure | Description | Time Frame |
|---|---|---|
| Maximum Observed Concentration (Cmax) of Idursulfase in Cerebrospinal Fluid (CSF) | Data was not collected for this outcome measure due to Sponsor decision. | Predose each week up to 9 years |
| Change From Baseline in Age Equivalent Scores of the Bayley Scales of Infant Development, Third Edition (BSID-III) Domains |
Inclusion Criteria:
Exclusion Criteria:
The participant has experienced, in the opinion of the investigator, a safety or medical issue that contraindicates treatment with idursulfase-IT, including, but not limited to, uncontrolled seizure disorder, bleeding disorder, and clinically relevant hypertension.
The participant has a known hypersensitivity to any of the components of idursulfase-IT.
The participant has clinically relevant intracranial hypertension.
The participant is enrolled in another clinical study, other than HGT-HIT-094 (NCT02055118), that involves clinical investigations or use of any investigational product (drug or [intrathecal/spinal] device) within 30 days prior to study enrollment or at any time during the study.
The participant has any known or suspected hypersensitivity to anesthesia or is thought to be at an unacceptably high risk for anesthesia due to compromised airways or other conditions.
The participant has a condition that is contraindicated as described in the SOPH-A-PORT® Mini S, Implantable Access Port, Spinal, Mini Unattached, with Guidewire (SOPH-A-PORT Mini S) intrathecal drug delivery device (IDDD) Instructions for Use, including:
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Takeda | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital and Research Center at Oakland | Oakland | California | 94609 | United States | ||
| Ann & Robert H Lurie Children's Hospital of Chicago |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37915038 | Derived | Yee KS, Chirila C, Davenport E, Mladsi D, Barnett C, Kronenberger WG. A post hoc analysis of Projected Retained Ability Scores (PRAS) for the longitudinal assessment of cognitive functioning in patients with neuronopathic mucopolysaccharidosis II receiving intrathecal idursulfase-IT. Orphanet J Rare Dis. 2023 Nov 2;18(1):343. doi: 10.1186/s13023-023-02957-2. | |
| 35961250 |
| Label | URL |
|---|---|
| To obtain more information on the study, click here/on this link | View source |
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Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
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IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
Participants who completed end of study (EOS) Visit Week 52 assessments of Study HGT-HIT-094 (NCT02055118) and who met the eligibility criteria were enrolled in this extension study. Some of the enrolled participants who were treated with idursulfase-IT in Study HGT-HIT-094 were analyzed as per the reference timepoints of HGT-HIT-094 study for some evaluations. For others, the reference timepoints after enrollment to the extension study were considered.
Participants took part in the study at various investigative sites in Australia, Canada, France, Mexico, Spain, the United Kingdom, and the United States of America (USA) from 14 April 2015 to 18 April 2024.
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| ID | Title | Description |
|---|---|---|
| FG000 | Idursulfase-IT | Participants received 10 milligrams (mg) of idursulfase-IT intrathecally via intrathecal drug delivery device (IDDD) or lumbar puncture (LP) once every 28 days along with standard-of-care therapy with Elaprase, for 470 weeks (for participants who began intrathecal [IT] treatment in this study) and 480 weeks (for participants who began IT treatment in HGT-HIT-094 and continued to receive IT treatment in this study). Participants who were younger than 3 years of age received an adjusted dose of 7.5 mg (>8 months to 30 months of age) or 10 mg (>30 months to 3 years of age) of idursulfase-IT. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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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 | Oct 9, 2018 | Apr 16, 2025 |
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| Elaprase | Drug | Participants received intravenous (IV) Elaprase infusions at a minimum of 48 hours after IT administration of idursulfase-IT. |
|
| Percent Change From Baseline in the Concentration of Glycosaminoglycan (GAG) in CSF at Month 67 | The percent change in concentration of GAG in CSF was assessed. GAGs are long sugar chains that are like building blocks for the body's tissues, especially connective tissues like cartilage, skin, and tendons, and play a crucial role in cell signaling and interactions. | For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 67 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 55 of this study. |
| Percent Change From Baseline in the Concentration of GAG in Urine at Month 67 | The percent change in concentration of GAG in urine was assessed. GAGs are long sugar chains that are like building blocks for the body's tissues, especially connective tissues like cartilage, skin, and tendons, and play a crucial role in cell signaling and interactions. | For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 67 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 55 of this study. |
| Number of Participants Who Reported Positive for Anti-idursulfase Antibodies in CSF | Up to 9 years |
| Number of Participants Who Reported Positive for Anti-idursulfase Antibodies in Serum | Up to 9 years |
| For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 67 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 55 of this study. |
| Change From Baseline in Standard Scores of the Vineland Adaptive Behavior Scales, Second Edition (VABS-II) Domains at Month 67 | The VABS-II test measures adaptive behaviors, including the ability to cope with environmental changes, to learn new everyday skills, and to demonstrate independence. This test measures the following 4 key domains: communication, daily living skills, socialization, motor skills, and the adaptive behavior composite [ABC] (a composite of the other 4 domains). The standard scores for four key domains were mean=100 and standard deviation=15, where higher scores indicate a higher level of cognitive ability. Positive change from baseline indicates improvement in adaptive functioning. The ABC score (a composite of the other 4 domains) ranges from 20 to 160 on which higher scores indicate a higher level of adaptive functioning. A positive change from baseline value indicates improvement in adaptive functioning. | For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 67 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 55 of this study. |
| Change From Baseline in Standard Composite Scores of the VABS-II Domains at Month 67 | The VABS-II test measures adaptive behaviors, including the ability to cope with environmental changes, to learn new everyday skills, and to demonstrate independence. This test measures the following 4 key domains: communication, daily living skills, socialization, motor skills, and the ABC (a composite of the other 4 domains). The ABC score ranges from 20 to 160 on which higher scores indicate a higher level of adaptive functioning. A positive change value indicates improvement in adaptive functioning. | For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 67 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 55 of this study. |
| Change From Baseline in Age Equivalents Score of the Differential Ability Scales, Second Edition (DAS-II) at Month 61 | DAS-II comprises 2 overlapping batteries; EYB= 2 years,6 months through 6 years,11 months and SAB=7 years,0 months through 17 years,11 months. Core subtests include Verbal Comprehension, Picture Similarities, Naming Vocabulary, Pattern Construction, Matrices and Copying for DAS-II Early Years and Recall of Designs, Word Definitions, Pattern Construction, Matrices, Verbal Similarities, and Sequential and Quantitative Reasoning for DAS-II School Years. Standard subtests scores (mean=50 and standard deviation of 10) for each subtest were converted to age equivalent scores (AES). Higher AES indicates greater cognitive ability. Negative change from baseline indicates worsening. | For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 61 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 49 of this study. |
| Change From Baseline in Developmental Quotients (DQ) of the DAS-II at Month 61 | DAS-II was used to assess all participants of age 2 years,6 months or older.DAS-II comprises 2 overlapping batteries.EYB was designed for children ages 2 years,6 months through 6 years,11 months.SAB was designed for children ages 7 years,0 months through 17 years,11 months.Core subtests include Verbal Comprehension,Picture Similarities,Naming Vocabulary,Pattern Construction,Matrices, & Copying for DAS-II Early Years&Recall of Designs,Word Definitions, Pattern Construction,Matrices,Verbal Similarities, & Sequential and Quantitative Reasoning for the DAS-II School Years. The DQ was computed as a ratio and expressed as a percentage using the AES divided by the age at testing ([AES/chronological age] × 100; range, 0-100). The higher score indicates greater cognitive ability. The subtest score represent a score (mean = 50 and standard deviation of 10) on which higher scores indicate a higher level of cognitive ability. A positive change value indicates improvement in cognitive ability. | For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 61 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 49 of this study. |
| Change From Baseline in T-scores of the Core Subtests DAS-II at Month 61 | DAS-II was used to assess all participants of age 2 years, 6 months or older. DAS-II comprises 2 overlapping batteries. EYB was designed for children ages 2 years, 6 months through 6 years, 11 months. SAB was designed for children ages 7 years, 0 months through 17 years, 11months. The core subtests include Verbal Comprehension, Picture Similarities, Naming Vocabulary, Pattern Construction, Matrices, and Copying for the DAS-II Early Years and Recall of Designs, Word Definitions, Pattern Construction, Matrices, Verbal Similarities, and Sequential and Quantitative Reasoning for the DAS-II School Years. Core subtests score represent a score (mean = 50 and standard deviation of 10) on which higher scores indicate a higher level of cognitive ability. | For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 61 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 49 of this study. |
| Change From Baseline in Age Equivalents Score of the VABS-II Sub Domains at Month 67 | The VABS-II test measures adaptive behaviors, including the ability to cope with environmental changes, to learn new everyday skills, and to demonstrate independence. This test measures the following subdomains of 5 key domains: Communication (Receptive, Expressive, Written), Daily Living Skills(Personal, Domestic, Community), Socialization (Interpersonal Relationships, Play and Leisure Time, Coping Skills), Motor Skills (Gross, Fine). Standard subdomain scores (mean=100 and standard deviation of 15) for each subdomain were converted to AES. Higher AES indicates greater cognitive ability. Negative change from baseline indicates worsening. | For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 67 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 55 of this study. |
| Change From Baseline in DQ of the VABS-II Sub Domains at Month 67 | The VABS-II test measures adaptive behaviors, including the ability to cope with environmental changes, to learn new everyday skills, and to demonstrate independence. This test measures the following subdomains of 5 key domains: Communication (Receptive, Expressive, Written), Daily Living Skills (Personal, Domestic, Community), Socialization (Interpersonal Relationships, Play and Leisure Time, Coping Skills), Motor Skills (Gross, Fine). The DQ was computed as a ratio and expressed as a percentage using the age-equivalent score divided by the age at testing ([age-equivalent score/chronological age] × 100; range, 0-100). Higher scores indicate better cognitive ability. A positive change from baseline value indicates improvement in cognition. | For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 67 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 55 of this study. |
| Change From Baseline in v-Scores of the VABS-II Sub Domains at Month 67 | The VABS-II test measures adaptive behaviors, including the ability to cope with environmental changes, to learn new everyday skills, and to demonstrate independence. This test measures the following subdomains of 5 key domains: Communication (Receptive, Expressive, Written), Daily Living Skills (Personal, Domestic, Community), Socialization (Interpersonal Relationships, Play and Leisure Time, Coping Skills), Motor Skills (Gross, Fine). The V-scale scores represent a score (mean = 15 and standard deviation of 3; range: 1-24) on which higher scores indicate a higher level of adaptive functioning. | For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 67 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 55 of this study. |
| Change From Baseline in v-Scale Scores of the VABS-II Maladaptive Behavior Index and Its Subscales at Month 67 | The VABS-II test measures adaptive behaviors, including the ability to cope with environmental changes, to learn new everyday skills, and to demonstrate independence. The maladaptive behavior index is a composite of the internalizing, externalizing, and other types of undesirable behavior that may interfere with the individual's adaptive functioning. The v-Scale scores represent a score (mean = 15 and standard deviation of 3; range: 1-24) on which higher scores indicate a higher level of adaptive functioning. | For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 67 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 55 of this study. |
| Number of Participants With Observed Maladaptive Levels of the VABS-II Maladaptive Behavior Index and Its Subscales at Month 61 | The VABS-II test measures adaptive behaviors, including the ability to cope with environmental changes, to learn new everyday skills, and to demonstrate independence. The maladaptive behavior index is a composite of the internalizing, externalizing, and other types of undesirable behavior that may interfere with the individual's adaptive functioning. Only categories having non-zero values are reported. | For participants who began IT treatment in HGT-HIT-094: At Month 61 in relation to HGT-HIT-094; For participants who began IT treatment in this study: At Month 49 of this study. |
| Change From Baseline in Brain Structure Volume as Measured by Magnetic Resonance Imaging (MRI) | Brain structure volume was assessed from brain total intracranial volume, brain total tissue volume, brain total white matter, brain total gray matter, and total CSF volume as measured by MRI. | For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 120 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 108 of this study. |
This pre-specified optional outcome measure was not assessed. |
| For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 67 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 55 of this study. |
| Change From Baseline in Development Quotient (DQ) of the Bayley Scales of Infant Development, Third Edition (BSID-III) Domains | This pre-specified optional outcome measure was not assessed. | For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 67 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 55 of this study. |
| Chicago |
| Illinois |
| 60611 |
| United States |
| University of North Carolina | Chapel Hill | North Carolina | 27514 | United States |
| Women's and Children's Hospital | Adelaide | 5006 | Australia |
| The Hospital for Sick Children | Toronto | Ontario | M5G 1X8 | Canada |
| Hôpital Femme Mère Enfants | Bron | 69677 | France |
| Instituto Nacional de Pediatría | Coyoacán | Mexico City | 04530 | Mexico |
| Hospital Infantil Universitario Niño Jesus | Madrid | 28009 | Spain |
| Royal Manchester Children's Hospital | Manchester | M13 9WL | United Kingdom |
| Muenzer J, Burton BK, Harmatz P, Gutierrez-Solana LG, Ruiz-Garcia M, Jones SA, Guffon N, Inbar-Feigenberg M, Bratkovic D, Hale M, Wu Y, Yee KS, Whiteman DAH, Alexanderian D; SHP609-302 study group. Long-term open-label extension study of the safety and efficacy of intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II. Mol Genet Metab. 2022 Sep-Oct;137(1-2):92-103. doi: 10.1016/j.ymgme.2022.07.016. Epub 2022 Aug 2. |
| COMPLETED |
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| NOT COMPLETED |
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The Safety Population included all the participants in Study SHP609-302 who underwent IDDD implantation or received at least 1 dose of study drug (full or partial).
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| ID | Title | Description |
|---|---|---|
| BG000 | Idursulfase-IT | Participants received 10 mg of idursulfase-IT intrathecally via IDDD or LP once every 28 days along with standard-of-care therapy with Elaprase, for 470 weeks (for participants who began IT treatment in this study) and 480 weeks (for participants who began IT treatment in HGT-HIT-094 and continued to receive IT treatment in this study). Participants who were younger than 3 years of age received an adjusted dose of 7.5 mg (>8 months to 30 months of age) or 10 mg (>30 months to 3 years of age) of idursulfase-IT. |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Participants With Adverse Events (AEs) | An AE is any noxious, pathologic, or unintended change in anatomical, physiologic, or metabolic function as indicated by physical signs, symptoms, or laboratory changes occurring in any phase of a clinical study, whether or not considered investigational product-related. | The Safety Population included all the participants in Study SHP609-302 who underwent IDDD implantation or received at least 1 dose of study drug (full or partial). | Posted | Count of Participants | Participants | Up to 9 years |
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| Primary | Number of Participants With Clinically Significant Changes in Vital Signs | Participants were assessed for clinically significant changes in vital signs like injection (IT) vital signs and regular vital signs (temperature, pulse, blood pressure [systolic and diastolic], oxygen saturation, and respiration rate). | The Safety Population included all the participants in Study SHP609-302 who underwent IDDD implantation or received at least 1 dose of study drug (full or partial). | Posted | Count of Participants | Participants | Up to 9 years |
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| Primary | Number of Participants With Clinically Significant Changes in Laboratory Parameters | Participants were assessed for clinically significant changes in laboratory parameters such as chemistry, hematology, urinalysis and cerebrospinal fluid (CSF) values. | The Safety Population included all the participants in Study SHP609-302 who underwent IDDD implantation or received at least 1 dose of study drug (full or partial). | Posted | Count of Participants | Participants | Up to 9 years |
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| Primary | Number of Participants With Clinically Significant Changes in 12-lead Electrocardiogram (ECG) Findings | Participants were assessed for clinically significant changes in 12-lead ECG findings (such as heart rate, PR interval, QRS interval, QT interval, and the corrected QT interval). | The Safety Population included all the participants in Study SHP609-302 who underwent IDDD implantation or received at least 1 dose of study drug (full or partial). | Posted | Count of Participants | Participants | Up to 9 years |
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| Primary | Maximum Observed Serum Concentration (Cmax) of Idursulfase | Idursulfase concentrations in serum were determined using a validated Enzyme -Linked Immunosorbent Assay (ELISA) method. Concentration for Cmax is presented in this endpoint. | The Pharmacokinetic Population included all the participants in Study SHP609-302 who received study drug and participated in the scheduled pharmacokinetic studies, and for whom at least 1 post-dose pharmacokinetic blood sample was collected. Overall number of participants analyzed is the number of participants with data available for analyses. Participants were divided into 3 groups for this outcome measure based on the time they received IT treatment in relation to prior study HGT-HIT-094. | Posted | Mean | Standard Deviation | nanograms per milliliter (ng/mL) | Predose and at 30 min, 60 min, 120 min, 4 hours, 6 hours, 8 hours, 12 hours, 24 hours, 30 hours and 36 hours post-dose at Week 100 in relation to HGT-HIT-094 (Week 48 of this study) |
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| Other Pre-specified | Maximum Observed Concentration (Cmax) of Idursulfase in Cerebrospinal Fluid (CSF) | Data was not collected for this outcome measure due to Sponsor decision. | Not Posted | Predose each week up to 9 years | Participants | |||||||||||||||||||||||||||||||
| Primary | Percent Change From Baseline in the Concentration of Glycosaminoglycan (GAG) in CSF at Month 67 | The percent change in concentration of GAG in CSF was assessed. GAGs are long sugar chains that are like building blocks for the body's tissues, especially connective tissues like cartilage, skin, and tendons, and play a crucial role in cell signaling and interactions. | The Safety Population included all the participants in Study SHP609-302 who underwent IDDD implantation or received at least 1 dose of study drug (full or partial). Overall number of participants analyzed is the number of participants with data available for analyses. | Posted | Mean | Standard Deviation | percent change | For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 67 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 55 of this study. |
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| Primary | Percent Change From Baseline in the Concentration of GAG in Urine at Month 67 | The percent change in concentration of GAG in urine was assessed. GAGs are long sugar chains that are like building blocks for the body's tissues, especially connective tissues like cartilage, skin, and tendons, and play a crucial role in cell signaling and interactions. | The Safety Population included all the participants in Study SHP609-302 who underwent IDDD implantation or received at least 1 dose of study drug (full or partial). Overall number of participants analyzed is the number of participants with data available for analyses. | Posted | Mean | Standard Deviation | percent change | For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 67 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 55 of this study. |
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| Primary | Number of Participants Who Reported Positive for Anti-idursulfase Antibodies in CSF | The Safety Population included all the participants in Study SHP609-302 who underwent IDDD implantation or received at least 1 dose of study drug (full or partial). | Posted | Count of Participants | Participants | Up to 9 years |
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| Primary | Number of Participants Who Reported Positive for Anti-idursulfase Antibodies in Serum | The Safety Population included all the participants in Study SHP609-302 who underwent IDDD implantation or received at least 1 dose of study drug (full or partial). | Posted | Count of Participants | Participants | Up to 9 years |
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| Secondary | Change From Baseline in Differential Ability Scales, Second Edition (DAS-II) Standard Cluster Scores at Month 67 | DAS-II was used to assess all participants of age 2 years, 6 months or older. DAS-II comprises 2 overlapping batteries. Early Years battery (EYB) was designed for children ages 2 years, 6 months through 6 years, 11 months. The School Age Battery (SAB) was designed for children ages 7 years, 0 months through 17 years, 11months. These batteries are fully co-normed for ages 5 years, 0 months, through 8 years, 11 months. The cluster areas include general conceptual ability (GCA), verbal, nonverbal, spatial, and special nonverbal composite (SNC). The cluster area score represents a score (mean=100 and standard deviation=15) on which higher scores indicate higher level of cognitive ability. | The Safety Population included all the participants in Study SHP609-302 who underwent IDDD implantation or received at least 1 dose of study drug (full or partial). Overall number of participants analyzed is the number of participants with data available for analyses. Number analyzed is the number of participants available for analyses for the specified category. | Posted | Mean | Standard Deviation | score on a scale | For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 67 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 55 of this study. |
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| Other Pre-specified | Change From Baseline in Age Equivalent Scores of the Bayley Scales of Infant Development, Third Edition (BSID-III) Domains | This pre-specified optional outcome measure was not assessed. | Not Posted | For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 67 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 55 of this study. | Participants | |||||||||||||||||||||||||||||||
| Other Pre-specified | Change From Baseline in Development Quotient (DQ) of the Bayley Scales of Infant Development, Third Edition (BSID-III) Domains | This pre-specified optional outcome measure was not assessed. | Not Posted | For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 67 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 55 of this study. | Participants | |||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Standard Scores of the Vineland Adaptive Behavior Scales, Second Edition (VABS-II) Domains at Month 67 | The VABS-II test measures adaptive behaviors, including the ability to cope with environmental changes, to learn new everyday skills, and to demonstrate independence. This test measures the following 4 key domains: communication, daily living skills, socialization, motor skills, and the adaptive behavior composite [ABC] (a composite of the other 4 domains). The standard scores for four key domains were mean=100 and standard deviation=15, where higher scores indicate a higher level of cognitive ability. Positive change from baseline indicates improvement in adaptive functioning. The ABC score (a composite of the other 4 domains) ranges from 20 to 160 on which higher scores indicate a higher level of adaptive functioning. A positive change from baseline value indicates improvement in adaptive functioning. | The Safety Population included all the participants in Study SHP609-302 who underwent IDDD implantation or received at least 1 dose of study drug (full or partial). Overall number of participants analyzed is the number of participants with data available for analyses. | Posted | Mean | Standard Deviation | score on a scale | For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 67 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 55 of this study. |
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| Secondary | Change From Baseline in Standard Composite Scores of the VABS-II Domains at Month 67 | The VABS-II test measures adaptive behaviors, including the ability to cope with environmental changes, to learn new everyday skills, and to demonstrate independence. This test measures the following 4 key domains: communication, daily living skills, socialization, motor skills, and the ABC (a composite of the other 4 domains). The ABC score ranges from 20 to 160 on which higher scores indicate a higher level of adaptive functioning. A positive change value indicates improvement in adaptive functioning. | The Safety Population included all the participants in Study SHP609-302 who underwent IDDD implantation or received at least 1 dose of study drug (full or partial). Overall number of participants analyzed is the number of participants with data available for analyses. Number analyzed is the number of participants with data available for analyses for the specified category. | Posted | Mean | Standard Deviation | score on a scale | For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 67 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 55 of this study. |
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| Secondary | Change From Baseline in Age Equivalents Score of the Differential Ability Scales, Second Edition (DAS-II) at Month 61 | DAS-II comprises 2 overlapping batteries; EYB= 2 years,6 months through 6 years,11 months and SAB=7 years,0 months through 17 years,11 months. Core subtests include Verbal Comprehension, Picture Similarities, Naming Vocabulary, Pattern Construction, Matrices and Copying for DAS-II Early Years and Recall of Designs, Word Definitions, Pattern Construction, Matrices, Verbal Similarities, and Sequential and Quantitative Reasoning for DAS-II School Years. Standard subtests scores (mean=50 and standard deviation of 10) for each subtest were converted to age equivalent scores (AES). Higher AES indicates greater cognitive ability. Negative change from baseline indicates worsening. | The Safety Population included all the participants in Study SHP609-302 who underwent IDDD implantation or received at least 1 dose of study drug (full or partial). Overall number of participants analyzed is the number of participants with data available for analyses. Number analyzed is the number of participants with data available for analyses for the specified category. | Posted | Mean | Standard Deviation | score on a scale | For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 61 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 49 of this study. |
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| Secondary | Change From Baseline in Developmental Quotients (DQ) of the DAS-II at Month 61 | DAS-II was used to assess all participants of age 2 years,6 months or older.DAS-II comprises 2 overlapping batteries.EYB was designed for children ages 2 years,6 months through 6 years,11 months.SAB was designed for children ages 7 years,0 months through 17 years,11 months.Core subtests include Verbal Comprehension,Picture Similarities,Naming Vocabulary,Pattern Construction,Matrices, & Copying for DAS-II Early Years&Recall of Designs,Word Definitions, Pattern Construction,Matrices,Verbal Similarities, & Sequential and Quantitative Reasoning for the DAS-II School Years. The DQ was computed as a ratio and expressed as a percentage using the AES divided by the age at testing ([AES/chronological age] × 100; range, 0-100). The higher score indicates greater cognitive ability. The subtest score represent a score (mean = 50 and standard deviation of 10) on which higher scores indicate a higher level of cognitive ability. A positive change value indicates improvement in cognitive ability. | The Safety Population included all the participants in Study SHP609-302 who underwent IDDD implantation or received at least 1 dose of study drug (full or partial). Overall number of participants analyzed is the number of participants with data available for analyses. Number analyzed is the number of participants with data available for analyses for the specified category. | Posted | Mean | Standard Deviation | score on a scale | For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 61 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 49 of this study. |
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| Secondary | Change From Baseline in T-scores of the Core Subtests DAS-II at Month 61 | DAS-II was used to assess all participants of age 2 years, 6 months or older. DAS-II comprises 2 overlapping batteries. EYB was designed for children ages 2 years, 6 months through 6 years, 11 months. SAB was designed for children ages 7 years, 0 months through 17 years, 11months. The core subtests include Verbal Comprehension, Picture Similarities, Naming Vocabulary, Pattern Construction, Matrices, and Copying for the DAS-II Early Years and Recall of Designs, Word Definitions, Pattern Construction, Matrices, Verbal Similarities, and Sequential and Quantitative Reasoning for the DAS-II School Years. Core subtests score represent a score (mean = 50 and standard deviation of 10) on which higher scores indicate a higher level of cognitive ability. | The Safety Population included all the participants in Study SHP609-302 who underwent IDDD implantation or received at least 1 dose of study drug (full or partial). Overall number of participants analyzed is the number of participants with data available for analyses. Number analyzed is the number of participants with data available for analyses for the specified category | Posted | Mean | Standard Deviation | score on a scale | For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 61 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 49 of this study. |
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| Secondary | Change From Baseline in Age Equivalents Score of the VABS-II Sub Domains at Month 67 | The VABS-II test measures adaptive behaviors, including the ability to cope with environmental changes, to learn new everyday skills, and to demonstrate independence. This test measures the following subdomains of 5 key domains: Communication (Receptive, Expressive, Written), Daily Living Skills(Personal, Domestic, Community), Socialization (Interpersonal Relationships, Play and Leisure Time, Coping Skills), Motor Skills (Gross, Fine). Standard subdomain scores (mean=100 and standard deviation of 15) for each subdomain were converted to AES. Higher AES indicates greater cognitive ability. Negative change from baseline indicates worsening. | The Safety Population included all the participants in Study SHP609-302 who underwent IDDD implantation or received at least 1 dose of study drug (full or partial). Overall number of participants analyzed is the number of participants with data available for analyses. | Posted | Mean | Standard Deviation | score on a scale | For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 67 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 55 of this study. |
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| Secondary | Change From Baseline in DQ of the VABS-II Sub Domains at Month 67 | The VABS-II test measures adaptive behaviors, including the ability to cope with environmental changes, to learn new everyday skills, and to demonstrate independence. This test measures the following subdomains of 5 key domains: Communication (Receptive, Expressive, Written), Daily Living Skills (Personal, Domestic, Community), Socialization (Interpersonal Relationships, Play and Leisure Time, Coping Skills), Motor Skills (Gross, Fine). The DQ was computed as a ratio and expressed as a percentage using the age-equivalent score divided by the age at testing ([age-equivalent score/chronological age] × 100; range, 0-100). Higher scores indicate better cognitive ability. A positive change from baseline value indicates improvement in cognition. | The Safety Population included all the participants in Study SHP609-302 who underwent IDDD implantation or received at least 1 dose of study drug (full or partial). Overall number of participants analyzed is the number of participants with data available for analyses. | Posted | Mean | Standard Deviation | score on a scale | For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 67 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 55 of this study. |
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| Secondary | Change From Baseline in v-Scores of the VABS-II Sub Domains at Month 67 | The VABS-II test measures adaptive behaviors, including the ability to cope with environmental changes, to learn new everyday skills, and to demonstrate independence. This test measures the following subdomains of 5 key domains: Communication (Receptive, Expressive, Written), Daily Living Skills (Personal, Domestic, Community), Socialization (Interpersonal Relationships, Play and Leisure Time, Coping Skills), Motor Skills (Gross, Fine). The V-scale scores represent a score (mean = 15 and standard deviation of 3; range: 1-24) on which higher scores indicate a higher level of adaptive functioning. | The Safety Population included all the participants in Study SHP609-302 who underwent IDDD implantation or received at least 1 dose of study drug (full or partial). Overall number of participants analyzed is the number of participants with data available for analyses. | Posted | Mean | Standard Deviation | score on a scale | For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 67 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 55 of this study. |
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| Secondary | Change From Baseline in v-Scale Scores of the VABS-II Maladaptive Behavior Index and Its Subscales at Month 67 | The VABS-II test measures adaptive behaviors, including the ability to cope with environmental changes, to learn new everyday skills, and to demonstrate independence. The maladaptive behavior index is a composite of the internalizing, externalizing, and other types of undesirable behavior that may interfere with the individual's adaptive functioning. The v-Scale scores represent a score (mean = 15 and standard deviation of 3; range: 1-24) on which higher scores indicate a higher level of adaptive functioning. | The Safety Population included all the participants in Study SHP609-302 who underwent IDDD implantation or received at least 1 dose of study drug (full or partial). Overall number of participants analyzed is the number of participants with data available for analyses. | Posted | Mean | Standard Deviation | score on a scale | For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 67 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 55 of this study. |
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| Secondary | Number of Participants With Observed Maladaptive Levels of the VABS-II Maladaptive Behavior Index and Its Subscales at Month 61 | The VABS-II test measures adaptive behaviors, including the ability to cope with environmental changes, to learn new everyday skills, and to demonstrate independence. The maladaptive behavior index is a composite of the internalizing, externalizing, and other types of undesirable behavior that may interfere with the individual's adaptive functioning. Only categories having non-zero values are reported. | The Safety Population included all the participants in Study SHP609-302 who underwent IDDD implantation or received at least 1 dose of study drug (full or partial). Overall number of participants analyzed is the number of participants with data available for analyses. | Posted | Count of Participants | Participants | For participants who began IT treatment in HGT-HIT-094: At Month 61 in relation to HGT-HIT-094; For participants who began IT treatment in this study: At Month 49 of this study. |
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| Secondary | Change From Baseline in Brain Structure Volume as Measured by Magnetic Resonance Imaging (MRI) | Brain structure volume was assessed from brain total intracranial volume, brain total tissue volume, brain total white matter, brain total gray matter, and total CSF volume as measured by MRI. | The Safety Population included all the participants in Study SHP609-302 who underwent IDDD implantation or received at least 1 dose of study drug (full or partial). Overall number of participants analyzed is the number of participants with data available for analyses. | Posted | Mean | Standard Deviation | cubic centimeters (cm^3) | For participants who began IT treatment in HGT-HIT-094: From Baseline of HGT-HIT-094 up to Month 120 in relation to HGT-HIT-094; For participants who began IT treatment in this study: From Baseline of this study up to Month 108 of this study. |
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Up to 9 years
The Safety Population included all the participants in Study SHP609-302 who underwent IDDD implantation or received at least 1 dose of study drug (full or partial).
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Idursulfase-IT | Participants received 10 mg of idursulfase-IT intrathecally via IDDD or LP once every 28 days along with standard-of-care therapy with Elaprase, for 470 weeks (for participants who began IT treatment in this study) and 480 weeks (for participants who began IT treatment in HGT-HIT-094 and continued to receive IT treatment in this study). Participants who were younger than 3 years of age received an adjusted dose of 7.5 mg (>8 months to 30 months of age) or 10 mg (>30 months to 3 years of age) of idursulfase-IT. | 0 | 56 | 48 | 56 | 56 | 56 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain | Gastrointestinal disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 16.1 | Systematic Assessment |
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| Adenoidal hypertrophy | Respiratory, thoracic and mediastinal disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Albumin CSF increased | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| Anaphylactic reaction | Immune system disorders | MedDRA 16.1 | Systematic Assessment |
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| Aortic valve stenosis | Cardiac disorders | MedDRA 16.1 | Systematic Assessment |
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| Arthritis reactive | Musculoskeletal and connective tissue disorders | MedDRA 16.1 | Systematic Assessment |
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| Asthenia | General disorders | MedDRA 16.1 | Systematic Assessment |
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| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA 16.1 | Systematic Assessment |
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| Bacteraemia | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
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| Bronchospasm | Respiratory, thoracic and mediastinal disorders | MedDRA 16.1 | Systematic Assessment |
| |
| CSF cell count increased | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| CSF glucose decreased | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| CSF lymphocyte count increased | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| CSF protein increased | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| CSF white blood cell count | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| Campylobacter infection | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Carpal tunnel syndrome | Nervous system disorders | MedDRA 16.1 | Systematic Assessment |
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| Catheter site cellulitis | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
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| Central nervous system infection | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
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| Cerebrospinal fluid leakage | Nervous system disorders | MedDRA 16.1 | Systematic Assessment |
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| Cholelithiasis | Hepatobiliary disorders | MedDRA 16.1 | Systematic Assessment |
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| Clostridium difficile colitis | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
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| Constipation | Gastrointestinal disorders | MedDRA 16.1 | Systematic Assessment |
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| Convulsion | Nervous system disorders | MedDRA 16.1 | Systematic Assessment |
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| Corona virus infection | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
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| Deafness | Ear and labyrinth disorders | MedDRA 16.1 | Systematic Assessment |
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| Dental caries | Gastrointestinal disorders | MedDRA 16.1 | Systematic Assessment |
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| Device breakage | General disorders | MedDRA 16.1 | Systematic Assessment |
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| Device deployment issue | General disorders | MedDRA 16.1 | Systematic Assessment |
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| Device dislocation | General disorders | MedDRA 16.1 | Systematic Assessment |
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| Device extrusion | General disorders | MedDRA 16.1 | Systematic Assessment |
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| Device failure | General disorders | MedDRA 16.1 | Systematic Assessment |
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| Device kink | General disorders | MedDRA 16.1 | Systematic Assessment |
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| Device malfunction | General disorders | MedDRA 16.1 | Systematic Assessment |
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| Device misuse | General disorders | MedDRA 16.1 | Systematic Assessment |
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| Device related infection | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
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| Drug reaction with eosinophilia and systemic symptoms | Skin and subcutaneous tissue disorders | MedDRA 16.1 | Systematic Assessment |
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| Endocarditis | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
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| Epilepsy | Nervous system disorders | MedDRA 16.1 | Systematic Assessment |
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| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 16.1 | Systematic Assessment |
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| Fatigue | General disorders | MedDRA 16.1 | Systematic Assessment |
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| Foreign body aspiration | Injury, poisoning and procedural complications | MedDRA 16.1 | Systematic Assessment |
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| Gastroenteritis viral | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
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| Haematochezia | Gastrointestinal disorders | MedDRA 16.1 | Systematic Assessment |
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| Hypersensitivity | Immune system disorders | MedDRA 16.1 | Systematic Assessment |
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| Hyperthermia | General disorders | MedDRA 16.1 | Systematic Assessment |
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| Hypotension | Vascular disorders | MedDRA 16.1 | Systematic Assessment |
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| Hypothermia | General disorders | MedDRA 16.1 | Systematic Assessment |
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| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Implant site abscess | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
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| Implant site effusion | General disorders | MedDRA 16.1 | Systematic Assessment |
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| Implant site infection | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
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| Inflammation | General disorders | MedDRA 16.1 | Systematic Assessment |
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| Influenza | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Injection site swelling | General disorders | MedDRA 16.1 | Systematic Assessment |
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| Insomnia | Psychiatric disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Medical device site reaction | General disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Meningitis bacterial | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Meningitis streptococcal | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Nocardiosis | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Otitis media | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Parainfluenzae virus infection | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Pleocytosis | Nervous system disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Procedural nausea | Injury, poisoning and procedural complications | MedDRA 16.1 | Systematic Assessment |
| |
| Procedural pain | Injury, poisoning and procedural complications | MedDRA 16.1 | Systematic Assessment |
| |
| Procedural vomiting | Injury, poisoning and procedural complications | MedDRA 16.1 | Systematic Assessment |
| |
| Pseudomeningocele | Injury, poisoning and procedural complications | MedDRA 16.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Respiratory distress | Respiratory, thoracic and mediastinal disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Respiratory syncytial virus infection | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Sleep disorder | Psychiatric disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Spondylolisthesis | Musculoskeletal and connective tissue disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Staphylococcal bacteraemia | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Staphylococcal infection | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Staphylococcus test positive | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| Staring | Psychiatric disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Stenotrophomonas infection | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Streptococcal sepsis | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Testicular torsion | Reproductive system and breast disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Tonsillitis | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Toothache | Gastrointestinal disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Vascular complication associated with device | General disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Wound dehiscence | Injury, poisoning and procedural complications | MedDRA 16.1 | Systematic Assessment |
| |
| Wound infection | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain | Gastrointestinal disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Abnormal behaviour | Psychiatric disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Achilles tendon discomfort | Musculoskeletal and connective tissue disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Adenoidal hypertrophy | Respiratory, thoracic and mediastinal disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Adenovirus infection | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Aggression | Psychiatric disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Agitation | Psychiatric disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| Albumin CSF increased | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| Anaemia | Blood and lymphatic system disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Arthropod bite | Injury, poisoning and procedural complications | MedDRA 16.1 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Astigmatism | Eye disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Attention deficit/hyperactivity disorder | Psychiatric disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Blister | Skin and subcutaneous tissue disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Blood bicarbonate decreased | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| Blood calcium decreased | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| Blood pressure diastolic decreased | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| Blood pressure diastolic increased | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| Blood pressure increased | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| Blood pressure systolic decreased | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| Blood pressure systolic increased | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| Blood thyroid stimulating hormone increased | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| Blood triglycerides increased | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| Blood uric acid increased | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| Body temperature decreased | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Bronchospasm | Respiratory, thoracic and mediastinal disorders | MedDRA 16.1 | Systematic Assessment |
| |
| CSF cell count increased | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| CSF glucose decreased | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| CSF neutrophil count increased | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| CSF protein increased | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| CSF test abnormal | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| CSF white blood cell count increased | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| Carbon dioxide decreased | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| Cardiac murmur | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| Carpal tunnel syndrome | Nervous system disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Catheter site effusion | General disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Catheter site erythema | General disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Catheter site extravasation | General disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Catheter site pain | General disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Catheter site swelling | General disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Cerebrospinal fluid retention | Nervous system disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Cerumen impaction | Ear and labyrinth disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Choking | Respiratory, thoracic and mediastinal disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Conjunctivitis | Eye disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 16.1 | Systematic Assessment |
| |
| Corona virus infection | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Coronavirus test positive | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Croup infectious | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Deafness | Ear and labyrinth disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Dental caries | Gastrointestinal disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Dermatitis allergic | Skin and subcutaneous tissue disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Dermatitis contact | Skin and subcutaneous tissue disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Dermatitis diaper | Skin and subcutaneous tissue disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Developmental hip dysplasia | Congenital, familial and genetic disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Device breakage | General disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Device malfunction | General disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Device occlusion | General disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Diastolic hypertension | Vascular disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Diastolic hypotension | Vascular disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Drug hypersensitivity | Immune system disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Dry skin | Skin and subcutaneous tissue disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Dysphemia | Psychiatric disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Ear pain | Ear and labyrinth disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Eczema | Skin and subcutaneous tissue disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Electrocardiogram QT prolonged | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| Enuresis | Renal and urinary disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Eosinophil count increased | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| Eosinophil percentage increased | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| Eosinophilia | Blood and lymphatic system disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Erythema | Skin and subcutaneous tissue disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Excoriation | Injury, poisoning and procedural complications | MedDRA 16.1 | Systematic Assessment |
| |
| Eye swelling | Eye disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Face oedema | General disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 16.1 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Febrile convulsion | Nervous system disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Flushing | Vascular disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Foot deformity | Musculoskeletal and connective tissue disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Foramen magnum stenosis | Congenital, familial and genetic disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Frequent bowel movements | Gastrointestinal disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Fungal skin infection | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Gait disturbance | General disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Haematochezia | Gastrointestinal disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Haematocrit decreased | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| Haemoglobin decreased | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| Hand-foot-and-mouth disease | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Head injury | Injury, poisoning and procedural complications | MedDRA 16.1 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Heart rate decreased | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| Heart rate increased | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| Hepatomegaly | Hepatobiliary disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Hypersensitivity | Immune system disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Implant site effusion | General disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Implant site swelling | General disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Incision site oedema | Injury, poisoning and procedural complications | MedDRA 16.1 | Systematic Assessment |
| |
| Incision site pain | Injury, poisoning and procedural complications | MedDRA 16.1 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Infusion site swelling | General disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Intervertebral disc protrusion | Musculoskeletal and connective tissue disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Iron deficiency | Metabolism and nutrition disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Irritability | General disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Joint injury | Injury, poisoning and procedural complications | MedDRA 16.1 | Systematic Assessment |
| |
| Laceration | Injury, poisoning and procedural complications | MedDRA 16.1 | Systematic Assessment |
| |
| Laryngitis | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Lethargy | Nervous system disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Ligament sprain | Injury, poisoning and procedural complications | MedDRA 16.1 | Systematic Assessment |
| |
| Lower respiratory tract infection | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Medical device complication | General disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Medical device pain | General disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Metapneumovirus infection | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Middle ear effusion | Ear and labyrinth disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Motion sickness | Ear and labyrinth disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Nerve compression | Nervous system disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Neutrophil count decreased | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| Neutrophil count increased | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| Nuclear magnetic resonance imaging brain abnormal | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| Ocular hyperaemia | Eye disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Oral herpes | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Otitis externa | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Otitis media | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Otitis media acute | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Otorrhoea | Ear and labyrinth disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Oxygen saturation decreased | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| Pain | General disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Pharyngitis streptococcal | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Platelet count decreased | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Post procedural haemorrhage | Injury, poisoning and procedural complications | MedDRA 16.1 | Systematic Assessment |
| |
| Procedural headache | Injury, poisoning and procedural complications | MedDRA 16.1 | Systematic Assessment |
| |
| Procedural hypertension | Injury, poisoning and procedural complications | MedDRA 16.1 | Systematic Assessment |
| |
| Procedural hypotension | Injury, poisoning and procedural complications | MedDRA 16.1 | Systematic Assessment |
| |
| Procedural nausea | Injury, poisoning and procedural complications | MedDRA 16.1 | Systematic Assessment |
| |
| Procedural pain | Injury, poisoning and procedural complications | MedDRA 16.1 | Systematic Assessment |
| |
| Procedural vomiting | Injury, poisoning and procedural complications | MedDRA 16.1 | Systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Proteinuria | Renal and urinary disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Pseudomeningocele | Injury, poisoning and procedural complications | MedDRA 16.1 | Systematic Assessment |
| |
| Psychomotor hyperactivity | Nervous system disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Pulmonary oedema | Respiratory, thoracic and mediastinal disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Rash erythematous | Skin and subcutaneous tissue disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Rash generalised | Skin and subcutaneous tissue disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Rash macular | Skin and subcutaneous tissue disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Rash maculo-papular | Skin and subcutaneous tissue disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Red blood cell count decreased | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| Red blood cells CSF positive | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| Red man syndrome | Skin and subcutaneous tissue disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Respiratory distress | Respiratory, thoracic and mediastinal disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Respiratory syncytial virus infection | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Retching | Gastrointestinal disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Scab | Skin and subcutaneous tissue disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Scoliosis | Musculoskeletal and connective tissue disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Scratch | Injury, poisoning and procedural complications | MedDRA 16.1 | Systematic Assessment |
| |
| Seasonal allergy | Immune system disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Sinus bradycardia | Cardiac disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Sinus tachycardia | Cardiac disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Sleep apnoea syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Sleep disorder | Psychiatric disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Spinal disorder | Musculoskeletal and connective tissue disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Spondylolisthesis | Musculoskeletal and connective tissue disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Staphylococcal infection | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Staring | Psychiatric disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Swelling face | Skin and subcutaneous tissue disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Systolic hypertension | Vascular disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Thermal burn | Injury, poisoning and procedural complications | MedDRA 16.1 | Systematic Assessment |
| |
| Tinea pedis | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Toe walking | Musculoskeletal and connective tissue disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Tooth abscess | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Tooth fracture | Injury, poisoning and procedural complications | MedDRA 16.1 | Systematic Assessment |
| |
| Tooth infection | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Toothache | Gastrointestinal disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Tympanic membrane perforation | Ear and labyrinth disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Umbilical hernia | Gastrointestinal disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Upper respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Upper-airway cough syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Vascular complication associated with device | General disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA 16.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 16.1 | Systematic Assessment |
| |
| Wheezing | Respiratory, thoracic and mediastinal disorders | MedDRA 16.1 | Systematic Assessment |
| |
| White blood cell count decreased | Investigations | MedDRA 16.1 | Systematic Assessment |
| |
| White blood cell count increased | Investigations | MedDRA 16.1 | Systematic Assessment |
|
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Takeda | +1-877-825-3327 | TrialDisclosures@takeda.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Feb 21, 2023 | Apr 16, 2025 | SAP_001.pdf |
| ID | Term |
|---|---|
| D016532 | Mucopolysaccharidosis II |
| 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 |
Not provided
Not provided
| ID | Term |
|---|---|
| C517982 | idursulfase |
Not provided
Not provided
Not provided
| Unknown or Not Reported |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
|
|
|
| OG002 |
| Idursulfase-IT 10 mg, Former Substudy |
The Former Substudy group included participants who received IT treatment in HGT-HIT-094 substudy. |
|
|
| Units |
|---|
| Counts |
|---|
| Participants |
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| Units |
|---|
| Counts |
|---|
| Participants |
|
|
|
|
|
|
|
|
|
|
|
|
Participants received 10 mg of idursulfase-IT intrathecally via IDDD or LP once every 28 days along with standard-of-care therapy with Elaprase, for 470 weeks (for participants who began IT treatment in this study) and 480 weeks (for participants who began IT treatment in HGT-HIT-094 and continued to receive IT treatment in this study). Participants who were younger than 3 years of age received an adjusted dose of 7.5 mg (>8 months to 30 months of age) or 10 mg (>30 months to 3 years of age) of idursulfase-IT. |
|
|
|
|
|
|
|
|
|
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
| Participants |
|
|