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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT02055118
Registration number
NCT02055118
Ethics application status
Date submitted
17/01/2014
Date registered
4/02/2014
Titles & IDs
Public title
Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment
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Scientific title
A Controlled, Randomized, Two-arm, Open-label, Assessor-blinded, Multicenter Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment
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Secondary ID [1]
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2013-002885-38
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Secondary ID [2]
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HGT-HIT-094
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Universal Trial Number (UTN)
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Trial acronym
AIM-IT
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Hunter Syndrome
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Condition category
Condition code
Other
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Research that is not of generic health relevance and not applicable to specific health categories listed above
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Mental Health
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Other mental health disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Other - idursulfase-IT
Other interventions - No IT treatment
Experimental: idursulfase-IT - 10 mg administered via IT using IDDD (intrathecal drug delivery device) once a month for 52 weeks.
Other: Nontreatment control - Patients will receive weekly standard of care treatment with IV Elaprase only.
Treatment: Other: idursulfase-IT
10mg
Other interventions: No IT treatment
Standard of Care
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Intervention code [1]
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Treatment: Other
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Intervention code [2]
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Other interventions
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Change From Baseline in the Differential Ability Scales, Second Edition (DAS-II) General Conceptual Ability (GCA) Standard Score at Week 52
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Assessment method [1]
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The DAS-II was used to assess cognitive development in all randomized participants. The GCA standard score of the DAS-II was used to obtain a general measure of cognitive ability. The GCA score represent a score (mean = 100 and standard deviation of 15) on which higher scores indicate a higher level of cognitive ability. The score ranges from 30 to 170. A positive change value indicates improvement in cognitive ability.
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Timepoint [1]
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Baseline, Week 52
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Secondary outcome [1]
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Change From Baseline in the Vineland Adaptive Behavior Scales, Second Edition (VABS-II) Adaptive Behavior Composite (ABC) Score at Week 52
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Assessment method [1]
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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 (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.
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Timepoint [1]
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Baseline, Week 52
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Secondary outcome [2]
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Change From Baseline in the Differential Ability Scales, Second Edition (DAS-II) General Conceptual Ability (GCA) Standard Score at Weeks 16, 28 and 40
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Assessment method [2]
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The DAS-II was used to assess cognitive development in all randomized participants. The GCA standard score of the DAS-II was used to obtain a general measure of cognitive ability. The GCA score represent a score (mean = 100 and standard deviation of 15) on which higher scores indicate a higher level of cognitive ability. The score ranges from 30 to 170. A positive change value indicates improvement in cognitive ability.
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Timepoint [2]
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Baseline, Week 16, Week 28 and Week 40
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Secondary outcome [3]
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Change From Baseline in the Vineland Adaptive Behavior Scales, Second Edition (VABS-II) Adaptive Behavior Composite (ABC) Score at Week 16, 28 and 40
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Assessment method [3]
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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 (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.
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Timepoint [3]
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Baseline, Week 16, Week 28 and Week 40
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Secondary outcome [4]
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Change From Baseline in Differential Ability Scales, Second Edition (DAS-II) Cluster Standard Scores at Weeks 16, 28, 40 and 52
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Assessment method [4]
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The DAS-II was used to assess cognitive development in all randomized participants. The cluster scores represent a score (mean = 100 and standard deviation of 15) on which higher scores indicate a higher level of cognitive ability in each cluster: verbal (score range: 31-169), nonverbal (score range: 31-166) and spatial (score range: 32-170).
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Timepoint [4]
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Baseline, Week 16, Week 28, Week 40 and Week 52
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Secondary outcome [5]
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Change From Baseline in Vineland Adaptive Behavior Scales, Second Edition (VABS-II) Standard Scores of Other Domains at Weeks 16, 28, 40, 52
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Assessment method [5]
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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 (a composite of the other 4 domains). The standard scores represent a score (mean = 100 and standard deviation of 15) on which higher scores indicate a higher level of cognitive ability. A positive change value indicates improvement in adaptive functioning. Communication, daily living skills, socialization and motor skills domains were reported here. The range for individual standard scores is 20-160.
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Timepoint [5]
0
0
Baseline, Week 16, Week 28, Week 40 and Week 52
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Secondary outcome [6]
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Change From Baseline of Age Equivalent Score for Early Years of Core Subtests of the Differential Ability Scales, Second Edition (DAS-II) at Weeks 16, 28, 40 and 52
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Assessment method [6]
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The DAS-II was used to assess cognitive development in all randomized participants. The early years battery is designed for children ages 2 years 6 months through 6 years 11 months. Standardized scores were converted to age equivalent scores to measure ability, skill, and knowledge expressed as the age at which most individuals reach the same level. The mean age equivalent score is obtained by averaging out the age-equivalent scores. 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.
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Timepoint [6]
0
0
Baseline, Week 16, Week 28, Week 40 and Week 52
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Secondary outcome [7]
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Change From Baseline of Age Equivalents for School Age of Core Subtests of the Differential Ability Scales, Second Edition (DAS-II) at Weeks 16, 28, 40 and 52
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Assessment method [7]
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The DAS-II was used to assess cognitive development in all randomized participants. The school age battery is designed for children ages 7 years 0 months through 17 years 11 months. Standardized scores were converted to age equivalent scores to measure ability, skill, and knowledge expressed as the age at which most individuals reach the same level. The mean age equivalent score is obtained by averaging out the age-equivalent scores. The higher score indicates greater cognitive ability. The subtest score represents 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. In the below table, SQR stands for "Sequential \& Quantitative Reasoning".
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Timepoint [7]
0
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Baseline, Week 16, Week 28, Week 40 and Week 52
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Secondary outcome [8]
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Change From Baseline of Development Quotients for Early Years of Core Subtests of the Differential Ability Scales, Second Edition (DAS-II) at Weeks 16, 28, 40 and 52
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Assessment method [8]
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The DAS-II was used to assess cognitive development in all randomized participants. The early years battery is designed for children ages 2 years 6 months through 6 years 11 months. The DQ is a means to express a neurodevelopmental/cognitive delay. 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). 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.
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Timepoint [8]
0
0
Baseline, Week 16, Week 28, Week 40 and Week 52
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Secondary outcome [9]
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Change From Baseline of Development Quotients for School Age of Core Subtests of the Differential Ability Scales, Second Edition (DAS-II) at Weeks 16, 28, 40 and 52
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Assessment method [9]
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0
The DAS-II was used to assess cognitive development in all randomized participants. The school age battery is designed for children ages 7 years 0 months through 17 years 11 months. The DQ is a means to express a neurodevelopmental/cognitive delay. 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). 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. In the below table, SQR stands for "Sequential \& Quantitative Reasoning".
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Timepoint [9]
0
0
Baseline, Week 16, Week 28, Week 40 and Week 52
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Secondary outcome [10]
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Change From Baseline of T-scores for Early Years of Core Subtests of the Differential Ability Scale, Second Edition (DAS-II) at Weeks 16, 28, 40 and 52
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Assessment method [10]
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0
The DAS-II was used to assess cognitive development in all randomized participants. The early years battery is designed for children ages 2 years 6 months through 6 years 11 months. 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.
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Timepoint [10]
0
0
Baseline, Week 16, Week 28, Week 40 and Week 52
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Secondary outcome [11]
0
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Change From Baseline of T-scores for School Age of Core Subtests of the Differential Ability Scale, Second Edition (DAS-II) at Weeks 16, 28, 40 and 52
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Assessment method [11]
0
0
The DAS-II was used to assess cognitive development in all randomized participants. The school age battery is designed for children ages 7 years 0 months through 17 years 11 months. 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. In the below table, SQR stands for "Sequential \& Quantitative Reasoning".
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Timepoint [11]
0
0
Baseline, Week 16, Week 28, Week 40 and Week 52
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Secondary outcome [12]
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Change From Baseline of Age Equivalents Scores of Sub-domains of the Vineland Adaptive Behavior Scales, Second Edition (VABS-II) at Weeks 16, 28, 40 and 52
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Assessment method [12]
0
0
The VABS-II test measures adaptive behaviors, including the ability to cope with environmental changes, to learn new everyday skills, and to demonstrate independence. Standardized scores (range 40-160) were converted to age equivalent scores to measure ability, skill, and knowledge expressed as the age at which most individuals reach the same level. The mean age equivalent score is obtained by averaging out the age-equivalent scores for the all the sub-domains except for Gross and Fine motor skills (range: 0, unbound). A positive value indicates improvement in health and cognition.
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Timepoint [12]
0
0
Baseline, Week 16, Week 28, Week 40 and Week 52
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Secondary outcome [13]
0
0
Change From Baseline of Development Quotients of Sub-domains of the Vineland Adaptive Behavior Scales, Second Edition (VABS-II) at Weeks 16, 28, 40 and 52
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Assessment method [13]
0
0
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 DQ is a means to express a neurodevelopmental/cognitive delay. 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). The overall DQ score is calculated from the mean age-equivalent score obtained by averaging out the age equivalent scores for the all the sub-domains except for Gross and Fine motor skills. This test measures the following 4 key domains: communication, daily living skills, socialization, motor skills, and the adaptive behavior composite (a composite of the other 4 domains). A positive value indicates improvement in health and cognition.
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Timepoint [13]
0
0
Baseline, Week 16, Week 28, Week 40 and Week 52
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Secondary outcome [14]
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Change From Baseline of V-Scale Scores of Sub-domains of the Vineland Adaptive Behavior Scales, Second Edition (VABS-II) at Weeks 16, 28, 40 and 52
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Assessment method [14]
0
0
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 (a composite of the other 4 domains). 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. A positive change value indicates improvement in adaptive functioning.
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Timepoint [14]
0
0
Baseline, Week 16, Week 28, Week 40 and Week 52
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Secondary outcome [15]
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Change From Baseline of V-Scale Scores of Maladaptive Behavior Index and Its Sub-scales at Weeks 16, 28, 40 and 52
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Assessment method [15]
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The VABS-II test measures adaptive behaviors, including the ability to cope with environmental changes, to learn new everyday skills, and to demonstrate independence. 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 greater maladaptive behaviors. A positive change value indicates increase of maladaptive behavior.
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Timepoint [15]
0
0
Baseline, Week 16, Week 28, Week 40 and Week 52
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Secondary outcome [16]
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Observed Maladaptive Levels of Maladaptive Behavior Index and Its Sub-scales of Vineland Adaptive Behavior Scales, Second Edition (VABS-II)
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Assessment method [16]
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The VABS-II test measures adaptive behaviors, including the ability to cope with environmental changes, to learn new everyday skills, and to demonstrate independence. Maladaptive behavior index (MBI) 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 greater maladaptive behaviors. The v-scale score ranges for MBI, externalizing and internalizing scores are defined as clinically significant: 21-24, elevated: 18-20, average: 1-17.
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Timepoint [16]
0
0
Baseline, Week 16, Week 28, Week 40 and Week 52
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Secondary outcome [17]
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Maximum Observed Drug Concentration (Cmax) of Idursulfase After IT Administration
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Assessment method [17]
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The Cmax of idursulfase after IT administration was reported.
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Timepoint [17]
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Pre-dose, 30, 60, 120 minutes, 4, 6, 8, 12, 24, 30 and 36 hour (h) post-dose on Weeks 4, 24, and 48
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Secondary outcome [18]
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Time to Reach Maximum Drug Concentration (Tmax) of Idursulfase After IT Administration
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Assessment method [18]
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The tmax of idursulfase after IT administration was reported.
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Timepoint [18]
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Pre-dose, 30, 60, 120 minutes, 4, 6, 8, 12, 24, 30 and 36 hour (h) post-dose on Weeks 4, 24, and 48
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Secondary outcome [19]
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Area Under the Concentration Versus Time Curve From Zero From the Time of Dosing to the Last Measurable Concentration (AUC0-t) of Idursulfase After IT Administration
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Assessment method [19]
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The AUC0-t of idursulfase after IT administration was reported.
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Timepoint [19]
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Pre-dose, 30, 60, 120 minutes, 4, 6, 8, 12, 24, 30 and 36 hour (h) post-dose on Weeks 4, 24, and 48
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Secondary outcome [20]
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Terminal Half-life (t1/2) of Idursulfase After IT Administration
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Assessment method [20]
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The t1/2 of idursulfase after IT administration was reported.
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Timepoint [20]
0
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Pre-dose, 30, 60, 120 minutes, 4, 6, 8, 12, 24, 30 and 36 hour (h) post-dose on Weeks 4, 24, and 48
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Secondary outcome [21]
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Total Body Clearance for Extravascular Administration Divided by the Fraction of Dose Absorbed (CL/F) of Idursulfase After IT Administration
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Assessment method [21]
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The CL/F of idursulfase after IT administration was reported.
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Timepoint [21]
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Pre-dose, 30, 60, 120 minutes, 4, 6, 8, 12, 24, 30 and 36 hour (h) post-dose on Weeks 4, 24, and 48
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Secondary outcome [22]
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Change From Baseline in the Concentration of Glycosaminoglycans (GAG) in Cerebrospinal Fluid (CSF) at Week 52
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Assessment method [22]
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Change from baseline in the concentration of GAG in CSF was reported.
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Timepoint [22]
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Baseline, Week 52
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Secondary outcome [23]
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Concentration of Idursulfase in Cerebrospinal Fluid (CSF)
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Assessment method [23]
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CSF samples were collected via the IDDD or lumbar puncture prior to the injection of Idursulfase-IT.
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Timepoint [23]
0
0
Pre-dose on Week 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44 and 48
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Secondary outcome [24]
0
0
Participant Response to Quality of Life EuroQol-5D (EQ-5D) Questionnaire at Week 52
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Assessment method [24]
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The EQ-5D provides a descriptive profile and index value for health status. The questionnaire measures 5 dimensions of health status: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. For each dimension, there are 5 levels of response: no problems, slight problems, moderate problems, severe problems, and unable to do/extreme problems.
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Timepoint [24]
0
0
Week 52
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Secondary outcome [25]
0
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Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Intrathecal Drug Delivery Device (IDDD)-Related Adverse Events
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Assessment method [25]
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An adverse event (AE) was 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. Treatment-emergent AEs for the no IT treatment group were defined as all AEs occurring on or after the date of randomization and at or before the end of the study (EOS) visit. Treatment-emergent AEs for the IT treatment group were defined as all AEs occurring on or after the date of the first IDDD implant surgery or Treatment-Emergentfirst dose of the investigational product (whichever was earlier) and at or before the EOS visit (+30 days) or 2 weeks after the removal of the last IDDD (whichever was later).
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Timepoint [25]
0
0
From start of study treatment up to Week 53
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Secondary outcome [26]
0
0
Composite Scores of Bayley Scales of Infant Development (BSID-III) Scale in Substudy Population
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Assessment method [26]
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0
Participants who were younger than 3 years were assessed using the BSID-III. The BSID--III is a series of measurements to assess the motor (fine and gross), language (receptive and expressive), and cognitive development of infants and toddlers and consists of a series of developmental play tasks. The composite score for the cognitive scale, language scale, and motor scale are normed and have a mean=100, SD=15 and range of 40-160. Higher values denote stronger skills and abilities in the domain, indicating better outcomes. Participant wise data at evaluable timepoints was reported for this outcome.
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Timepoint [26]
0
0
Baseline up to Week 52
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Secondary outcome [27]
0
0
Percentile Scores of Bayley Scales of Infant Development (BSID-III) Scale in Substudy Population
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Assessment method [27]
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0
Participants who were younger than 3 years were assessed using the BSID-III. The BSID--III is a series of measurements to assess the motor (fine and gross), language (receptive and expressive), and cognitive development of infants and toddlers and consists of a series of developmental play tasks. Percentile scores range from 1 to 99 with 50 as the mean and median. Higher percentile means higher the rank of the child relative to the normed population. Participant wise data at evaluable timepoints was reported for this outcome.
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Timepoint [27]
0
0
Baseline up to Week 52
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Secondary outcome [28]
0
0
Age Equivalent Scores of Bayley Scales of Infant Development (BSID-III) Scale in Substudy Population
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Assessment method [28]
0
0
Participants who were younger than 3 years were assessed using the BSID-III. The BSID--III is a series of measurements to assess the motor (fine and gross), language (receptive and expressive), and cognitive development of infants and toddlers and consists of a series of developmental play tasks. Standardized scores (range 40-160) were converted to age- equivalent scores to measure ability, skill, and knowledge expressed as the age at which most individuals reach the same level (age norm; range: 0, unbound). Higher values present better outcomes. Participant wise data at evaluable timepoints was reported for this outcome.
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Timepoint [28]
0
0
Baseline up to Week 52
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Secondary outcome [29]
0
0
Chronological Age of Bayley Scales of Infant Development (BSID-III) Scale in Substudy Population
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Assessment method [29]
0
0
Participants who were younger than 3 years were assessed using the BSID-III. The BSID--III is a series of measurements to assess the motor (fine and gross), language (receptive and expressive), and cognitive development of infants and toddlers and consists of a series of developmental play tasks. Chronological age of the participants when assessed by the BSID-III scale was reported. Range 16.59 - 45.21 months. Participant wise data at evaluable timepoints was reported for this outcome.
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Timepoint [29]
0
0
Baseline up to Week 52
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Secondary outcome [30]
0
0
Development Quotient (DQ) of Bayley Scales of Infant Development (BSID-III) Scale in Substudy Population
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Assessment method [30]
0
0
Participants who were younger than 3 years were assessed using the BSID-III. The BSID--III is a series of measurements to assess the motor (fine and gross), language (receptive and expressive), and cognitive development of infants and toddlers and consists of a series of developmental play tasks. The development quotient (DQ) is a means to express a neurodevelopmental/cognitive delay which will be 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 values present better outcomes. Participant wise data at evaluable timepoints was reported for this outcome.
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Timepoint [30]
0
0
Baseline up to Week 52
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Secondary outcome [31]
0
0
Raw Scores of Bayley Scales of Infant Development (BSID-III) Scale in Substudy Population
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Assessment method [31]
0
0
Participants who were younger than 3 years were assessed using the BSID-III. The BSID--III is a series of measurements to assess the motor (fine and gross), language (receptive and expressive), and cognitive development of infants and toddlers and consists of a series of developmental play tasks. Raw scores are converted to scaled scores that are based on normed populations. Raw score ranges: Cognitive scale 0-91, Receptive communication 0-49, Expressive communication 0-48, Fine motor 0-66 and Gross motor 0-72. Higher values indicate better outcomes. Participant wise data at evaluable timepoints was reported for this outcome.
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Timepoint [31]
0
0
Baseline up to Week 52
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Eligibility
Key inclusion criteria
Inclusion Criteria Inclusion Criteria for the Pivotal Study
Patients must meet all of the following criteria to be considered eligible for randomization in the pivotal study:
1. The patient is male and is =3 and <18 years of age at the time of informed consent.
(Patients who are younger than 3 years of age may be enrolled in a separate substudy provided that they meet other inclusion criteria, provided below.)
2. The patient must have a documented diagnosis of MPS II.
3. The patient has evidence at Screening of Hunter syndrome-related cognitive impairment defined as follows:
A patient who is =3 and <13 years of age must have one of the following criteria (3a OR 3b):
1. A GCA score =55 and =85 OR
2. If the patient has a GCA score at Screening >85, there must be evidence of a decrease in GCA score of =10 points over 12 months from a previously documented test result in observational study HGT-HIT-090.
A patient who is =13 and <18 years of age must have both of the following criteria (3c AND 3d):
3. A GCA score of =55 and =85. AND
4. There must be evidence of a decrease in GCA score of =10 points over 12 months from a previously documented
4. The patient has received and tolerated a minimum of 4 months of therapy with Elaprase during the period immediately prior to Screening.
5. The patient must have sufficient auditory capacity, with a hearing aid(s), if needed, in the Investigator's judgment to complete the required protocol testing and must be compliant with wearing the hearing aid(s), if needed, on scheduled testing days.
6. The patient's parent(s) or legally authorized guardian(s) must have voluntarily signed an Institutional Review Board/Independent Ethics Committee approved informed consent form after all relevant aspects of the study have been explained and discussed. Consent of the patient's parent(s) or legally authorized guardian(s) and the patient's assent, if applicable, must be obtained prior to the start of any study procedures.
Inclusion Criteria for the Substudy
Patients must meet all of the following criteria to be considered eligible for enrollment in the separate substudy:
1. The patient is male and is <3 years of age at the time of informed consent.
2. The patient must have a documented diagnosis of MPS II.
3. The patient has evidence at Screening of Hunter syndrome-related cognitive impairment
4. The patient has received and tolerated a minimum of 4 months of therapy with Elaprase during the period immediately prior to Screening.
5. The patient must have sufficient auditory capacity, with a hearing aid(s), if needed, in the Investigator's judgment to complete the required protocol testing and must be compliant with wearing the hearing aid(s), if needed, on scheduled testing days.
6. The patient's parent(s) or legally authorized guardian(s) must have voluntarily signed an Institutional Review Board/Independent Ethics Committee approved informed consent form after all relevant aspects of the study have been explained and discussed. Consent of the patient's parent(s) or legally authorized guardian(s) must be obtained prior to the start of any study procedures.
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Minimum age
No limit
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Maximum age
18
Years
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Sex
Males
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Can healthy volunteers participate?
No
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Key exclusion criteria
Exclusion Criteria
Patients who meet any of the following criteria are not eligible to be randomized into the pivotal study or enrolled in the separate substudy:
1. The patient has clinically significant non-Hunter syndrome-related CNS involvement (such as Fragile-X syndrome) which is judged by the Investigator to be likely to interfere with the accurate administration and interpretation of protocol assessments.
2. The patient has a large chromosomal deletion or complex rearrangement that includes a deletion of the FMR1 and/or FMR2 genes.
3. The patient has a significant medical or psychiatric comorbidity(ies) that might affect study data or confound the integrity of study results.
4. The patient has contra-indications for performance of lumbar puncture such as musculoskeletal/spinal abnormalities or risk of abnormal bleeding.
5. The patient has a history of complications from previous lumbar punctures or technical challenges in conducting lumbar punctures such that the potential risks would exceed possible benefits for the patient.
6. The patient has an opening CSF pressure upon lumbar puncture that exceeds 30.0 cm H2O.
7. The patient has experienced infusion-related anaphylactoid event(s) or has evidence of consistent severe adverse events related to treatment with Elaprase which, in the Investigator's opinion, may pose an unnecessary risk to the patient.
8. The patient has received a cord blood or bone marrow transplant at any time or has received blood product transfusions within 90 days prior to Screening.
9. The patient has a history of poorly controlled seizure disorder.
10. The patient is unable to comply with the protocol (eg, has significant hearing or vision impairment, a clinically relevant medical condition making implementation of the protocol difficult, unstable social situation, known clinically significant psychiatric/behavioral instability, is unable to return for safety evaluations, or is otherwise unlikely to complete the study), as determined by the Investigator.
11. The patient is enrolled in another clinical study that involves clinical investigation 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.
12. The patient 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.
13. The patient has a condition that is contraindicated as described in the SOPH-A-PORT Mini S IDDD Instructions for Use (IFU), including but not limited to the presence of a CSF shunt device in the patient.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
24/03/2014
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
28/09/2017
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Sample size
Target
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Accrual to date
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Final
58
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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Women's and Children's Hospital, 72 King William Road - North Adelaide
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Recruitment postcode(s) [1]
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5006 - North Adelaide
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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California
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United States of America
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Illinois
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United States of America
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North Carolina
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Canada
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Ontario
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France
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Bron
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Mexico
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Ciudad De México
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Spain
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Madrid
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United Kingdom
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Manchester
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Shire
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Ethics approval
Ethics application status
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Summary
Brief summary
Study HGT-HIT-094 is a multicenter study designed to determine the effect on clinical parameters of neurodevelopmental status of monthly IT administration of idursulfase-IT 10 mg for 12 months in pediatric patients with Hunter syndrome and cognitive impairment who have previously received and tolerated a minimum of 4 months of therapy with Elaprase.
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Trial website
https://clinicaltrials.gov/study/NCT02055118
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Trial related presentations / publications
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; HGT-HIT-094 Study Group. Intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II: Results from a phase 2/3 randomized study. Mol Genet Metab. 2022 Sep-Oct;137(1-2):127-139. doi: 10.1016/j.ymgme.2022.07.017. Epub 2022 Aug 2. Erratum In: Mol Genet Metab. 2023 Nov;140(3):107645. doi: 10.1016/j.ymgme.2023.107645. 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. Erratum In: Mol Genet Metab. 2023 Nov;140(3):107646. doi: 10.1016/j.ymgme.2023.107646.
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Public notes
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Contacts
Principal investigator
Name
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Study Director
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Address
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Takeda
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Contact person for public queries
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Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
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What data in particular will be shared?
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 document/s available: Study protocol, Statistical analysis plan (SAP), Informed consent form (ICF), Clinical study report (CSR)
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When will data be available (start and end dates)?
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Available to whom?
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.
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Available for what types of analyses?
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How or where can data be obtained?
IPD available at link: https://vivli.org/ourmember/takeda/
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What supporting documents are/will be available?
No Supporting Document Provided
Type
Other Details
Attachment
Study protocol
Study Protocol: original
https://cdn.clinicaltrials.gov/large-docs/18/NCT02055118/Prot_000.pdf
Study protocol
Study Protocol: Amendment 1
https://cdn.clinicaltrials.gov/large-docs/18/NCT02055118/Prot_001.pdf
Study protocol
Study Protocol: Amendment 2
https://cdn.clinicaltrials.gov/large-docs/18/NCT02055118/Prot_002.pdf
Study protocol
Study Protocol: Amendment 3
https://cdn.clinicaltrials.gov/large-docs/18/NCT02055118/Prot_003.pdf
Study protocol
Study Protocol: Amendment 4
https://cdn.clinicaltrials.gov/large-docs/18/NCT02055118/Prot_004.pdf
Study protocol
Study Protocol: Amendment 5
https://cdn.clinicaltrials.gov/large-docs/18/NCT02055118/Prot_005.pdf
Statistical analysis plan
https://cdn.clinicaltrials.gov/large-docs/18/NCT02055118/SAP_006.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT02055118