A Phase 2/3 interventional study of Idursulfase-IT and Elaprase in Hunter Syndrome, sponsored by Shire. Completed at 9 sites in 7 countries. Open to male participants aged Up to 18 Years. Per ClinicalTrials.gov, last updated 2025-06-19.
Sponsored by Shire · Phase 2/3, Interventional, and Treatment
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.
71 studies on the registry are indexed under Mucopolysaccharidosis II; 8 are open to participants now.
This study's enrollment of 56 is above the median of 20 across 43 interventional studies indexed under Mucopolysaccharidosis II.
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Exclusion Criteria:
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:
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.
Drug: Idursulfase-IT · Drug: Elaprase
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).
Also known as: HGT-2310
Participants received intravenous (IV) Elaprase infusions at a minimum of 48 hours after IT administration of idursulfase-IT.
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.
Time frame: 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).
Time frame: 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.
Time frame: 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).
Time frame: 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.
Time frame: 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)
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.
Time frame: 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.
Time frame: 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
Time frame: Up to 9 years
Number of Participants Who Reported Positive for Anti-idursulfase Antibodies in Serum
Time frame: Up to 9 years
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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Maximum Observed Concentration (Cmax) of Idursulfase in Cerebrospinal Fluid (CSF)
Data was not collected for this outcome measure due to Sponsor decision.
Time frame: 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
This pre-specified optional outcome measure was not assessed.
Time frame: 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.
Time frame: 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 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.
| Milestone | Idursulfase-IT |
|---|---|
| Started | 56 |
| Completed | 23 |
| Not completed | 33 |
| Withdrew: Withdrawal by subject | 9 |
| Withdrew: Participation terminated by investigator | 1 |
| Withdrew: Site terminated by sponsor | 21 |
| Withdrew: Technical problems | 1 |
| Withdrew: Reason not specified | 1 |
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.
| Participants | Idursulfase-IT |
|---|---|
| Number of Participants With Adverse Events (AEs) | 56 |
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).
| Participants | Idursulfase-IT |
|---|---|
| Number of Participants With Clinically Significant Changes in Vital Signs | 0 |
Participants were assessed for clinically significant changes in laboratory parameters such as chemistry, hematology, urinalysis and cerebrospinal fluid (CSF) values.
| Participants | Idursulfase-IT |
|---|---|
| Number of Participants With Clinically Significant Changes in Laboratory Parameters | 0 |
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).
| Participants | Idursulfase-IT |
|---|---|
| Number of Participants With Clinically Significant Changes in 12-lead Electrocardiogram (ECG) Findings | 0 |
Idursulfase concentrations in serum were determined using a validated Enzyme -Linked Immunosorbent Assay (ELISA) method. Concentration for Cmax is presented in this endpoint.
| nanograms per milliliter (ng/mL) | Idursulfase-IT 10 mg, Delayed IT | Idursulfase-IT 10 mg, Early IT | Idursulfase-IT 10 mg, Former Substudy |
|---|---|---|---|
| Maximum Observed Serum Concentration (Cmax) of Idursulfase | 128.19 ± 169.427 | 104.89 ± 66.879 | 120.23 ± 57.308 |
Data was not collected for this outcome measure due to Sponsor decision.
Results for this outcome have not been posted.
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.
| percent change | Idursulfase-IT |
|---|---|
| Percent Change From Baseline in the Concentration of Glycosaminoglycan (GAG) in CSF at Month 67 | -71.15 ± 17.842 |
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.
| percent change | Idursulfase-IT |
|---|---|
| Percent Change From Baseline in the Concentration of GAG in Urine at Month 67 | -32.44 ± 29.366 |
| Participants | Idursulfase-IT |
|---|---|
| Number of Participants Who Reported Positive for Anti-idursulfase Antibodies in CSF | 25 |
| Participants | Idursulfase-IT |
|---|---|
| Number of Participants Who Reported Positive for Anti-idursulfase Antibodies in Serum | 46 |
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.
| score on a scale | Idursulfase-IT |
|---|---|
| GCA | -6.0 ± 8.49 |
| Verbal | -23.7 ± 21.59 |
| Nonverbal | -20.5 ± 7.78 |
| Spatial | 0.3 ± 18.88 |
| SNC | -5.5 ± 6.36 |
This pre-specified optional outcome measure was not assessed.
Results for this outcome have not been posted.
This pre-specified optional outcome measure was not assessed.
Results for this outcome have not been posted.
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.
| score on a scale | Idursulfase-IT |
|---|---|
| Communication | -13.5 ± 4.95 |
| Daily Living Skills | -9.5 ± 0.71 |
| Socialization | -14.5 ± 9.19 |
| Motor Skills | 11.5 ± 7.78 |
| ABC | -13.5 ± 2.12 |
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.
| score on a scale | Idursulfase-IT |
|---|---|
| Baseline Age <6 Years | -13.5 ± 2.12 |
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.
| score on a scale | Idursulfase-IT |
|---|---|
| EYB: Verbal Comprehension | 1.50 ± 1.768 |
| EYB: Picture Similarities | 2.63 ± 2.652 |
| EYB: Naming Vocabulary | 3.13 ± 1.237 |
| EYB: Pattern Construction | 1.38 ± 1.237 |
| EYB: Matrices | 3.38 ± 1.237 |
| EYB: Copying | 1.13 ± 1.591 |
| SAB: Recall of Designs | 0.00 ± NA |
| SAB: Word Definitions | -0.25 ± NA |
| SAB: Pattern Construction | 0.00 ± NA |
| SAB: Matrices | 2.50 ± NA |
| SAB: Verbal Similarities | 0.00 ± NA |
| SAB: Sequential & Quantitative Reasoning | 0.75 ± NA |
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.
| score on a scale | Idursulfase-IT |
|---|---|
| EYB: Verbal Comprehension | -27.45 ± 13.223 |
| EYB: Picture Similarities | -7.75 ± 23.971 |
| EYB: Naming Vocabulary | -10.95 ± 4.596 |
| EYB: Pattern Construction | -22.25 ± 7.425 |
| EYB: Matrices | -9.20 ± 12.021 |
| EYB: Copying | -34.95 ± 15.627 |
| SAB: Recall of Designs | -25.90 ± NA |
| SAB: Word Definitions | -29.30 ± NA |
| SAB: Pattern Construction | -28.50 ± NA |
| SAB: Matrices | 2.20 ± NA |
| SAB: Verbal Similarities | -25.90 ± NA |
| SAB: Sequential & Quantitative Reasoning | -19.80 ± NA |
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.
| score on a scale | Idursulfase-IT |
|---|---|
| EYB: Verbal Comprehension | -14.5 ± 9.19 |
| EYB: Picture Similarities | -6.5 ± 10.61 |
| EYB: Naming Vocabulary | -8.5 ± 4.95 |
| EYB: Pattern Construction | -10.0 ± 9.90 |
| EYB: Matrices | 2.5 ± 2.12 |
| EYB: Copying | -14.0 ± 26.87 |
| SAB: Recall of Designs | -4.0 ± NA |
| SAB: Word Definitions | -28.0 ± NA |
| SAB: Pattern Construction | -13.0 ± NA |
| SAB: Matrices | -3.0 ± NA |
| SAB: Verbal Similarities | -24.0 ± NA |
| SAB: Sequential & Quantitative Reasoning | -2.0 ± NA |
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.
| score on a scale | Idursulfase-IT |
|---|---|
| Communication: Receptive | 2.13 ± 0.412 |
| Communication: Expressive | 2.54 ± 1.591 |
| Communication: Written | 1.58 ± 1.296 |
| Daily Living Skills: Personal | 4.50 ± 1.296 |
| Daily Living Skills: Domestic | 3.42 ± 0.825 |
| Daily Living Skills: Community | 1.83 ± 0.943 |
| Socialization: Interpersonal Relationships | 1.92 ± 4.832 |
| Socialization: Play and Leisure Time | 2.25 ± 1.768 |
| Socialization: Coping Skills | 0.75 ± 1.296 |
| Motor Skills: Gross | 9.75 ± 8.721 |
| Motor Skills: Fine | 2.33 ± 0.118 |
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.
| score on a scale | Idursulfase-IT |
|---|---|
| Communication: Receptive | -7.30 ± 19.375 |
| Communication: Expressive | -6.65 ± 23.547 |
| Communication: Written | -40.15 ± 10.677 |
| Daily Living Skills: Personal | 9.50 ± 13.011 |
| Daily Living Skills: Domestic | -22.60 ± 0.424 |
| Daily Living Skills: Community | -27.15 ± 16.900 |
| Socialization: Interpersonal Relationships | -26.00 ± 44.265 |
| Socialization: Play and Leisure Time | -18.00 ± 13.435 |
| Socialization: Coping Skills | -44.65 ± 31.183 |
| Motor Skills: Gross | 47.35 ± 66.539 |
| Motor Skills: Fine | -19.90 ± 6.788 |
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.
| score on a scale | Idursulfase-IT |
|---|---|
| Communication: Receptive | -0.5 ± 2.12 |
| Communication: Expressive | -1.0 ± 2.83 |
| Communication: Written | -8.0 ± 2.83 |
| Daily Living Skills: Personal | 1.0 ± 1.41 |
| Daily Living Skills: Domestic | -2.0 ± 1.41 |
| Daily Living Skills: Community | -5.0 ± 1.41 |
| Socialization: Interpersonal Relationships | -3.0 ± 5.66 |
| Socialization: Play and Leisure Time | -2.0 ± 1.41 |
| Socialization: Coping Skills | -3.5 ± 2.12 |
| Motor Skills: Gross | 2.5 ± 2.12 |
| Motor Skills: Fine | 1.5 ± 0.71 |
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.
| score on a scale | Idursulfase-IT |
|---|---|
| Maladaptive Behavior Index | -3.0 ± NA |
| Internalizing | -4.0 ± NA |
| Externalizing | -1.0 ± NA |
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.
| Participants | Idursulfase-IT |
|---|---|
| Maladaptive Behavior Index; Average | 7 |
| Maladaptive Behavior Index; Elevated | 1 |
| Internalizing; Average | 6 |
| Internalizing; Elevated | 2 |
| Externalizing; Average | 2 |
| Externalizing; Elevated | 6 |
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.
| cubic centimeters (cm^3) | Idursulfase-IT |
|---|---|
| Brain Total Intracranial Volume | -19.813 ± 70.994 |
| Brain Total Tissue Volume | -38.573 ± 134.021 |
| Brain Total White Matter Volume | 98.180 ± 64.194 |
| Brain Total Gray Matter Volume | -136.750 ± 79.547 |
| Total CSF Volume | 58.480 ± 52.382 |
Collected over Up to 9 years. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Idursulfase-IT | 0/56 (0%) | 48/56 (85.7%) | 56/56 (100%) |
| Event | Idursulfase-IT |
|---|---|
| Device failureGeneral disorders | 22/56 |
| Cerebrospinal fluid leakageNervous system disorders | 12/56 |
| PyrexiaGeneral disorders | 10/56 |
| Vascular complication associated with deviceGeneral disorders | 7/56 |
| BacteraemiaInfections and infestations | 4/56 |
| Device breakageGeneral disorders | 4/56 |
| Device kinkGeneral disorders | 4/56 |
| Device related infectionInfections and infestations | 4/56 |
| Central nervous system infectionInfections and infestations | 3/56 |
| Device dislocationGeneral disorders | 3/56 |
| Event | Idursulfase-IT |
|---|---|
| PyrexiaGeneral disorders | 43/56 |
| CoughRespiratory, thoracic and mediastinal disorders | 40/56 |
| VomitingGastrointestinal disorders | 40/56 |
| NasopharyngitisInfections and infestations | 38/56 |
| Upper respiratory tract infectionInfections and infestations | 37/56 |
| Procedural painInjury, poisoning and procedural complications | 33/56 |
| Ear infectionInfections and infestations | 32/56 |
| DiarrhoeaGastrointestinal disorders | 30/56 |
| HeadacheNervous system disorders | 27/56 |
| RhinorrhoeaRespiratory, thoracic and mediastinal disorders | 25/56 |
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).
| Age, Continuous(years) | Idursulfase-IT |
|---|---|
| Mean | 4.89 ± 2.117 |
| Sex: Female, Male(Participants) | Idursulfase-IT |
|---|---|
| Female | 0 |
| Male | 56 |
| Ethnicity (NIH/OMB)(Participants) | Idursulfase-IT |
|---|---|
| Hispanic or Latino | 14 |
| Not Hispanic or Latino | 38 |
| Unknown or Not Reported | 4 |
| Race (NIH/OMB)(Participants) | Idursulfase-IT |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 4 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 1 |
| White | 41 |
| More than one race | 0 |
| Unknown or Not Reported | 10 |
Documents are hosted by the registry — open the source record to download them.
Plan to share: Yes — 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.
Supporting information: Study protocol, Sap, Icf, Csr
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