A Phase 4 interventional study of Elaprase for intravenous (IV) infusion in Hunter Syndrome, sponsored by Shire. Completed at 8 sites in 8 countries. Open to male participants aged Up to 5 Years. Per ClinicalTrials.gov, last updated 2026-04-02.
Sponsored by Shire (part of Takeda) · Phase 4, Interventional, and Treatment
This long-term study will provide Elaprase treatment to children enrolled in this study and will utilize data from both enrolled patients and Hunter Outcome Survey (HOS) patient registry data to conduct the primary growth analysis to assess changes in height and weight in patients with Mucopolysaccharidosis II (Hunter syndrome) MPS II.
77 studies on the registry are indexed under Mucopolysaccharidosis II; 8 are open to participants now.
This study's enrollment of 21 is close to the median of 22 across 48 interventional studies indexed under Mucopolysaccharidosis II.
Browse Mucopolysaccharidosis II studies →Shire is the lead sponsor of 351 studies on the registry; 2 are open to participants now.
Of its 48 completed or terminated interventional studies of FDA-regulated products, 48 (100%) have results posted.
First submitted before Shire became part of Takeda.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Group 1: Prospective Patient Group
The patient must have a documented diagnosis of MPS II. Of the 3 criteria below, the combinations (3a AND 3b) or (3a AND 3c) will be accepted as diagnostic of MPS II:
Group 2: Retrospective Data Inclusion Criteria:
Retrospective Patient Group patients will be enrolled in HOS and not Study SHP-ELA-401; however, their growth data may be included in the analysis for Study SHP-ELA-401 if the following data inclusion criteria are met.
Exclusion Criteria:
Group 1: Prospective Patient Group
Group 2: Retrospective Data Exclusion Criteria:
HOS patients that meet the following criteria are not eligible to be included into the Study SHP-ELA-401 Primary Growth Analysis:
1. Patient was treated with growth hormone or other medications or interventions intended to promote growth in the time period covered by the analysis.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants received IV infusions of Elaprase, once-weekly at a dose of 0.5 mg/kg. Thereafter, each enrolled participant continued receiving one infusion of Elaprase every week. They were be followed for a minimum of 5 years after initiation of Elaprase treatment, or until they reached their 10th birthday, whichever was longer.
Drug: Elaprase for intravenous (IV) infusion
Participants in the HOS patient registry, who were treated, were combined with the Prospective Set in the Primary Growth Analysis for this study using their height and weight data.
Participants in the HOS patient registry, we were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.
Patients enrolled in this study will receive once-weekly IV infusions of Elaprase at a dose of 0.5 mg/kg and will be followed for a minimum of 5 years after initiation of Elaprase treatment, or until they reach their 10th birthday, whichever is longer.Height and weight data from HOS will be utilized in the Primary Growth Analysis for this study.
Also known as: Idursulfase
Height Overall
The effect of treatment on growth evaluated in terms of height. As pre-specified in the statistical analysis plan (SAP), descriptive analysis for this outcome measure was planned for the Combined Set and HOS Untreated Set arms and the assessment for Primary Growth Analysis was considered for the Combined Set in comparison to the HOS Untreated Set. The Combined Set included treated participants enrolled in this study (prospective participants) as well as treated participants from the HOS registry.
Time frame: Prospective participants: From Baseline through End-of-Study (approximately 9.75 years); HOS participants followed up to a maximum of 9.5 years where participant data were accessed from the registry between the period of 08/01/07 to 02/06/22
Weight Overall
The effect of treatment on growth was evaluated, in terms of weight. As pre-specified in the SAP, descriptive analysis for this outcome measure was planned for the Combined Set and HOS Untreated Set arms and the assessment for Primary Growth Analysis was considered for the Combined Set in comparison to the HOS Untreated Set. The Combined Set included treated participants enrolled in this study (prospective participants) as well as treated participants from the HOS registry.
Time frame: Prospective participants: From Baseline through End-of-Study (approximately 9.75 years); HOS participants followed up to a maximum of 9.5 years where participant data were accessed from the registry between the period of 08/01/07 to 02/06/22
Change From Baseline in Height Measured by Z-score
Z-score(standard score) for height was calculated as number of standard deviations(SD) by which mean height of each arm was above/below the mean height of the reference population.Z-scores were calculated based on World Health Organization Drug Dictionary(WHO-DD) growth charts(for age less or equal to\[≤\] 24 months) \& Centers for Disease Control \& Prevention(CDC) growth charts(for age more than\[\>\]24 months) normal height-for-age data.Normal growth Z-score range: -1 to +1.Z-score:0 represents the reference mean \& 50th percentile.Z-score of more than or equal to(≥) +2 indicates above normal range \& taller than average \& Z-score ≤ -2 indicates shorter stature than average \& may indicate growth issues.Score is calculated as Z=(Actual value for participant in study-Mean value of healthy population)/(SD of healthy population).As per SAP,descriptive analysis was planned for Combined \& HOS Untreated Set arms, with Primary Growth Analysis assessed for Combined Set compared to HOS Untreated Set.
Time frame: Prospective participants: From Baseline through End-of-Study (approximately 9.75 years); HOS participants followed up to a maximum of 9.5 years where participant data were accessed from the registry between the period of 08/01/07 to 02/06/22
Change From Baseline in Weight Measured by Z-score
Z-score (standard score) for weight was calculated as the number of SDs by which the mean weight of each arm was above or below the mean weight of the reference population. Z-scores were calculated based on WHO-DD growth charts (for age ≤ 24 months) \& CDC growth charts (for age \> 24 months) normal weight-for-age data. The normal range for growth Z score is defined as -1 to +1. Z-score: 0 represents reference population mean and the 50th percentile. Positive Z-score of ≥ +2 indicates above average weight (overweight). Negative Z-score of ≤ -2 indicates below average weight (underweight). The score is calculated as Z=(Actual value for participant in study-Mean value of healthy population)/(SD of healthy population). As per SAP, descriptive analysis was planned for Combined \& HOS Untreated Set arms, with Primary Growth Analysis assessed for Combined Set compared to HOS Untreated Set.
Time frame: Prospective participants: From Baseline through End-of-Study (approximately 9.75 years); HOS participants followed up to a maximum of 9.5 years where participant data were accessed from the registry between the period of 08/01/07 to 02/06/22
Number of Participants With Clinical Significant Abnormal Neurological Examination
A full physical examination will be performed with a thorough review of body systems. Physical examinations will include a review of the patient's general appearance, neurological examination, as well as evaluation of the body systems. Any abnormal change in findings will be recorded as an adverse event (AE).
Time frame: From Screening to End-of-Study (approximately 9.75 years)
Number of Participants With Treatment-emergent Adverse Events (TEAE)
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 product-related. This includes an exacerbation of a pre-existing condition. A TEAE is defined as an AE with an onset that occurs after receiving study drug.
Time frame: From screening to End-of-Study (approximately 9.75 years)
Number of Participants With Clinically Significant Abnormal Urinalysis Values
Reported here is the number of participants with clinically significant abnormal values in urinalysis tests. Only tests with at least 1 participant with clinically significant abnormal values are reported. Participant was clinically significant abnormal if there was at least one abnormal and clinically significant post-baseline value.
Time frame: From screening to End-of-Study (approximately 9.75 years)
Number of Participants With Clinically Significant Abnormal Serum Chemistry Values
Reported here is the number of participants with clinically significant abnormal values in serum chemistry tests. Only tests with at least 1 participant with clinically significant abnormal values are reported. Participant was clinically significant abnormal if there was at least one abnormal and clinically significant post-baseline value.
Time frame: From screening to End-of-Study (approximately 9.75 years)
Number of Participants With Clinically Significant Abnormal Hematology Values
Reported here is the number of participants with clinically significant abnormal values in haematological tests. Only tests with at least 1 participant with clinically significant abnormal values are reported. Participant was clinically significant abnormal if there was at least one abnormal and clinically significant post-baseline value.
Time frame: From screening to End-of-Study (approximately 9.75 years)
Observed Value of Height Velocity From Baseline to End of Study
Height velocity was calculated as the difference in height, divided by the difference in age between consecutive study visits.
Time frame: Prospective participants: From Baseline till End-of-Study Treatment (approximately 9.75 years); HOS participants followed up to a maximum of 9.5 years where participant data were accessed from the registry between the period of 08/01/07 to 02/06/22
Observed Value of Weight Velocity From Baseline to End of Study
Weight velocity was be calculated as the difference in weight, divided by the difference in age between consecutive study visits.
Time frame: Prospective participants: From Baseline till End-of- Study Treatment (Approximately 9.75 years); HOS participants followed up to a maximum of 9.5 years where participant data were accessed from the registry between the period of 08/01/07 to 02/06/22
Percent Change From Baseline for Urinary Glycosaminoglycans (uGAG) Levels Normalized to Urine Creatinine
Urinary GAG levels were normalized to urine creatinine (normalized uGAG) and reported as mg uGAG/ millimoles (mmol) creatinine.
Time frame: Baseline to End-of-Study (Approximately 9.75 years)
Normalized uGAG Divided by Upper Llimit of Normal for Age (uGAG/ULN) Every 12 Months
Normalized uGAG was divided by the upper limit of normal for age (uGAG/ULN), where the ULN for uGAG was obtained from Mayo Clinic.
Time frame: Baseline to End-of-Study (Approximately 9.75 years)
Liver Volume
Liver volume was assessed using abdominal ultrasonography.
Time frame: Baseline up to 24 Months
Spleen Volume
Spleen volume was assessed using abdominal ultrasonography.
Time frame: Baseline up to 24 Months
Joint Mobility, as Measured by Joint Range of Motion (JROM) Scores, Including Upper-Limb and Lower-Limb Joint Scores
Global JROM (% normal range of motion \[ROM\]) is average of 11 ratios multiplied by 100. Ratios are Left/Right means of passive ROM in Shoulder (Flexion \[flex\]/Extension \[ext\], Abduction, Internal/External Rotation), Elbow (flex/ext), Wrist (flex/ext), Index Finger (flex/ext \[Combined Metacarpophalangeal joint, Proximal interphalangeal joint, Distal interphalangeal joint motion\]), Hip (flex/ext, Abduction, Internal/External Rotation), Knee(flex/ext) \& Ankle (Dorsiflexion) divided by normal range (reference: American Academy of Orthopedic Surgeons and American Medical Association). For reported values of upper limb (UL) \& lower limb (LL) scores, UL score is average of 3 joint scores in UL (shoulder-elbow-wrist) \& LL score is average of 3 joint scores in LL (hip-knee-ankle). Joint motion (in degrees) was averaged across both sides, divided by normal value \& multiplied by 100 to yield a percent score. Score \>100% occurs when measured joint motion exceeds normal reference values.
Time frame: From Baseline to End-of-Study (approximately 9.75 years)
Distance Walked, as Measured by Six Minute Walk Test (6MWT)
The 6MWT was conducted according to the American Thoracic Society guidelines for the 6MWT in participants who were able to walk. The distance achieved in meters was recorded.
Time frame: From Baseline to End-of-Study (approximately 9.75 years)
Quality of Life, as Measured by Hunter-Syndrome Functional Outcome in Clinical Understanding Scale (HS-FOCUS)
The participant's QoL was assessed using the HS-FOCUS (shortened version) questionnaire. The HS-FOCUS (shortened version) questionnaire has 5 function domains (walking/standing, grip/reach, schooling/work, activities, and breathing). The scale of the 5 function domains ranges from 0 to 3, with a 3-score denoting highest disability: 0: with no difficulty;1: with some difficulty; 2: with much difficulty; 3: unable to do; Missing: Does not apply. The response option "Does not apply" is treated as "missing" with no score, the same as if the item had not been completed in the questionnaire. Higher scores indicate worse functional outcomes/greater disability.
Time frame: Baseline to End-of-Study (Approximately 9.75 years)
Impact of Illness on Ability to Function in Daily Life, as Measured by Childhood Health Assessment Questionnaire (CHAQ Parent Report)
Impact on ability to function in daily life was measured by the CHAQ (Parent Report). The CHAQ includes 30 items measured on a scale of 0 to 3: 0=without any difficulty; 1=with some difficulty; 2=with much difficulty; 3=Unable to do; Missing: Does not apply. It evaluates functional abilities across 8 domains (dressing, hygiene, arising, eating, walking, reach, grip and activities). The result is referred as Disability Index. The highest scoring item in each category determines the score for that category with higher scores indicating worse functioning/higher disability. The Discomfort Index and Health Status Index are measured on separate 15 cm scales. The distance from the left end of the scale to the respondent's mark is measured and multiplied by 0.2 to calculate the score (range 0-3). Discomfort and Health Status Index scores were rescaled to 0-100 scales. Higher scores indicate greater discomfort/worse health status.
Time frame: Baseline to End-of-Study (approximately 9.75 years)
Adaptive Behavior, as Measured by the Vineland Adaptive Behavior Scales (VABS II)
Adaptive behavior was assessed using parent/caregiver report on the VABS-II, a standardized norm-referenced tool to evaluate adaptive behavior for ages 0-90.VABS-II has 1 composite score(Adaptive Behavior Composite \[ABC\]), reflecting overall adaptive ability.ABC comprises 4 domain scores in participants \<7years old(Communication,Daily Living Skills,Socialization,\& Motor Skills)\& 3 domain scores in participants ≥7years of age(Communication,Daily Living Skills,\& Socialization).ABC standard score is derived from domain standard scores per VABS-II manual(not a simple sum/average of the reported standard scores).Domain scores are standard scores derived from combination of 11 subdomain scores according to VABS-II scoring rules/manual.Scale for ABC \& domain standard scores ranges between 20 \& 160.ABC \& domain scores have a normative mean=100,with SD=15 \& subdomain scores are normed with a mean=15 \& SD=3.Higher scores indicate better,while lower scores indicate worse adaptive functioning.
Time frame: Baseline to End-of-Study (approximately 9.75 years)
Anti-Idursulfase Antibodies (ADA) in Serum
Blood samples were collected and analyzed for determination of anti-idursulfase antibodies every 6 months in SHP-ELA-401. Analysis of anti-idursulfase antibodies including neutralizing antibodies (NAb) was conducted using validated 3-tier immunoassay methods (screening, confirmatory, and titer).
Time frame: From Baseline to End-of-Study (Approximately 9.75 years)
Participants took part in the study at various investigative sites globally from 28 October 2015 to 29 July 2025.
| Milestone | Prospective Set | Hunter Outcome Survey (HOS) Treated Set | HOS Untreated Set |
|---|---|---|---|
| Started | 21 | 19 | 26 |
| Completed | 17 | 19 | 26 |
| Not completed | 4 | 0 | 0 |
| Withdrew: Death | 1 | 0 | 0 |
| Withdrew: Non-compliance with study drug | 2 | 0 | 0 |
| Withdrew: Reason not specified | 1 | 0 | 0 |
The effect of treatment on growth evaluated in terms of height. As pre-specified in the statistical analysis plan (SAP), descriptive analysis for this outcome measure was planned for the Combined Set and HOS Untreated Set arms and the assessment for Primary Growth Analysis was considered for the Combined Set in comparison to the HOS Untreated Set. The Combined Set included treated participants enrolled in this study (prospective participants) as well as treated participants from the HOS registry.
| centimeter (cm) | Combined Set | HOS Untreated Set |
|---|---|---|
| Height Overall | 113.759 ± 1.211 | 108.143 ± 1.7668 |
The effect of treatment on growth was evaluated, in terms of weight. As pre-specified in the SAP, descriptive analysis for this outcome measure was planned for the Combined Set and HOS Untreated Set arms and the assessment for Primary Growth Analysis was considered for the Combined Set in comparison to the HOS Untreated Set. The Combined Set included treated participants enrolled in this study (prospective participants) as well as treated participants from the HOS registry.
| kilograms (kg) | Combined Set | HOS Untreated Set |
|---|---|---|
| Weight Overall | 25.164 ± 0.8382 | 26.11 ± 1.1647 |
Z-score(standard score) for height was calculated as number of standard deviations(SD) by which mean height of each arm was above/below the mean height of the reference population.Z-scores were calculated based on World Health Organization Drug Dictionary(WHO-DD) growth charts(for age less or equal to\[≤\] 24 months) \& Centers for Disease Control \& Prevention(CDC) growth charts(for age more than\[\>\]24 months) normal height-for-age data.Normal growth Z-score range: -1 to +1.Z-score:0 represents the reference mean \& 50th percentile.Z-score of more than or equal to(≥) +2 indicates above normal range \& taller than average \& Z-score ≤ -2 indicates shorter stature than average \& may indicate growth issues.Score is calculated as Z=(Actual value for participant in study-Mean value of healthy population)/(SD of healthy population).As per SAP,descriptive analysis was planned for Combined \& HOS Untreated Set arms, with Primary Growth Analysis assessed for Combined Set compared to HOS Untreated Set.
| Z-score | Combined Set | HOS Untreated Set |
|---|---|---|
| Change From Baseline in Height Measured by Z-score | -1.135 ± 1.3480 | -4.228 ± 1.4616 |
Z-score (standard score) for weight was calculated as the number of SDs by which the mean weight of each arm was above or below the mean weight of the reference population. Z-scores were calculated based on WHO-DD growth charts (for age ≤ 24 months) \& CDC growth charts (for age \> 24 months) normal weight-for-age data. The normal range for growth Z score is defined as -1 to +1. Z-score: 0 represents reference population mean and the 50th percentile. Positive Z-score of ≥ +2 indicates above average weight (overweight). Negative Z-score of ≤ -2 indicates below average weight (underweight). The score is calculated as Z=(Actual value for participant in study-Mean value of healthy population)/(SD of healthy population). As per SAP, descriptive analysis was planned for Combined \& HOS Untreated Set arms, with Primary Growth Analysis assessed for Combined Set compared to HOS Untreated Set.
| Z-score | Combined Set | HOS Untreated Set |
|---|---|---|
| Change From Baseline in Weight Measured by Z-score | -1.161 ± 1.3259 | -2.337 ± 1.9014 |
A full physical examination will be performed with a thorough review of body systems. Physical examinations will include a review of the patient's general appearance, neurological examination, as well as evaluation of the body systems. Any abnormal change in findings will be recorded as an adverse event (AE).
| Participants | Prospective Set |
|---|---|
| Number of Participants With Clinical Significant Abnormal Neurological Examination | 0 |
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 product-related. This includes an exacerbation of a pre-existing condition. A TEAE is defined as an AE with an onset that occurs after receiving study drug.
| Participants | Prospective Set |
|---|---|
| Number of Participants With Treatment-emergent Adverse Events (TEAE) | 21 |
Reported here is the number of participants with clinically significant abnormal values in urinalysis tests. Only tests with at least 1 participant with clinically significant abnormal values are reported. Participant was clinically significant abnormal if there was at least one abnormal and clinically significant post-baseline value.
| Participants | Prospective Set |
|---|---|
| Bacteria (/High-power field (HPF)) | 5 |
| Erythrocytes (/HPF) | 1 |
| Glucose (milligrams per deciliter (mg/dl)) | 1 |
| Leukocyte Esterase | 1 |
| Mucous Threads | 1 |
| Nitrite | 4 |
| Occult Blood | 1 |
| Protein (mg/dL) | 3 |
| Specimen Appearance | 6 |
| Squamous Epithelial Cells (/HPF) | 2 |
| Squamous Transitional Epithelial Cells (/HPF) | 1 |
| Triple Phosphate Crystals (/HPF) | 2 |
| Yeast Cells | 1 |
| pH (pH) | 3 |
Reported here is the number of participants with clinically significant abnormal values in serum chemistry tests. Only tests with at least 1 participant with clinically significant abnormal values are reported. Participant was clinically significant abnormal if there was at least one abnormal and clinically significant post-baseline value.
| Participants | Prospective Set |
|---|---|
| Alanine Aminotransferase (units per liter (U/L)) | 1 |
| Aspartate Aminotransferase (U/L) | 1 |
| Gamma Glutamyl Transferase (U/L) | 1 |
Reported here is the number of participants with clinically significant abnormal values in haematological tests. Only tests with at least 1 participant with clinically significant abnormal values are reported. Participant was clinically significant abnormal if there was at least one abnormal and clinically significant post-baseline value.
| Participants | Prospective Set |
|---|---|
| Ery. Mean Corpuscular Hemoglobin (picogram (pg)) | 1 |
| Ery. Mean Corpuscular Volume (femtoliter (fL)) | 1 |
| Hemoglobin (gram per litre (g/L)) | 1 |
Height velocity was calculated as the difference in height, divided by the difference in age between consecutive study visits.
| centimeters (cm)/year | Combined Set | Prospective Set | HOS Untreated Set |
|---|---|---|---|
| Overall Height Velocity: BL to < 6 Years of Age | 6.556 ± 2.6803 | 5.401 ± 2.5815 | 6.557 ± 2.5902 |
| Overall Height Velocity: 6 Years of Age to EOS | 3.571 ± 1.8064 | 2.699 ± 1.7610 | 1.761 ± 1.2178 |
Weight velocity was be calculated as the difference in weight, divided by the difference in age between consecutive study visits.
| kilograms (kg)/year | Combined Set | Prospective Set | HOS Untreated Set |
|---|---|---|---|
| Overall Velocity: Baseline to < 6 Years of Age | 2.475 ± 1.4550 | 1.772 ± 1.2029 | 3.670 ± 2.0005 |
| Overall Velocity: 6 Years of Age to EOS | 2.305 ± 1.7134 | 1.573 ± 1.3198 | 1.445 ± 0.8324 |
Urinary GAG levels were normalized to urine creatinine (normalized uGAG) and reported as mg uGAG/ millimoles (mmol) creatinine.
| percent change | Prospective Set |
|---|---|
| Percent Change From Baseline for Urinary Glycosaminoglycans (uGAG) Levels Normalized to Urine Creatinine | -70.446 ± 17.8741 |
Normalized uGAG was divided by the upper limit of normal for age (uGAG/ULN), where the ULN for uGAG was obtained from Mayo Clinic.
| Normalized uGAG/ULN | Prospective Set |
|---|---|
| Normalized uGAG/ULN: Baseline | 5.052 ± 1.0376 |
| Normalized uGAG/ULN: 12 Months | 2.315 ± 0.7655 |
| Normalized uGAG/ULN: 24 Months | 2.096 ± 0.7657 |
| Normalized uGAG/ULN: 36 Months | 2.182 ± 0.9339 |
| Normalized uGAG/ULN: 48 Months | 2.063 ± 0.6853 |
| Normalized uGAG/ULN: 60 Months | 2.197 ± 0.8870 |
| Normalized uGAG/ULN: 72 Months | 2.190 ± 1.0945 |
| Normalized uGAG/ULN: 84 Months | 0.830 ± NA |
| Normalized uGAG/ULN: End of Study | 2.055 ± 1.1866 |
Liver volume was assessed using abdominal ultrasonography.
| milliliters (mL) | Prospective Set |
|---|---|
| Liver Volume: Baseline | 105.855 ± 16.8925 |
| Liver Volume: 24 Months | 87.811 ± 7.0147 |
Spleen volume was assessed using abdominal ultrasonography.
| mL | Prospective Set |
|---|---|
| Spleen Volume: Baseline | 104.593 ± 29.9658 |
| Spleen Volume: 24 Months | 98.427 ± 47.8022 |
Global JROM (% normal range of motion \[ROM\]) is average of 11 ratios multiplied by 100. Ratios are Left/Right means of passive ROM in Shoulder (Flexion \[flex\]/Extension \[ext\], Abduction, Internal/External Rotation), Elbow (flex/ext), Wrist (flex/ext), Index Finger (flex/ext \[Combined Metacarpophalangeal joint, Proximal interphalangeal joint, Distal interphalangeal joint motion\]), Hip (flex/ext, Abduction, Internal/External Rotation), Knee(flex/ext) \& Ankle (Dorsiflexion) divided by normal range (reference: American Academy of Orthopedic Surgeons and American Medical Association). For reported values of upper limb (UL) \& lower limb (LL) scores, UL score is average of 3 joint scores in UL (shoulder-elbow-wrist) \& LL score is average of 3 joint scores in LL (hip-knee-ankle). Joint motion (in degrees) was averaged across both sides, divided by normal value \& multiplied by 100 to yield a percent score. Score \>100% occurs when measured joint motion exceeds normal reference values.
| percent score | Prospective Set |
|---|---|
| Lower Limb | 94.50 ± 31.634 |
| Upper Limb | 104.24 ± 21.471 |
The 6MWT was conducted according to the American Thoracic Society guidelines for the 6MWT in participants who were able to walk. The distance achieved in meters was recorded.
| meters | Prospective Set |
|---|---|
| Distance Walked, as Measured by Six Minute Walk Test (6MWT) | 224.2 ± 178.42 |
The participant's QoL was assessed using the HS-FOCUS (shortened version) questionnaire. The HS-FOCUS (shortened version) questionnaire has 5 function domains (walking/standing, grip/reach, schooling/work, activities, and breathing). The scale of the 5 function domains ranges from 0 to 3, with a 3-score denoting highest disability: 0: with no difficulty;1: with some difficulty; 2: with much difficulty; 3: unable to do; Missing: Does not apply. The response option "Does not apply" is treated as "missing" with no score, the same as if the item had not been completed in the questionnaire. Higher scores indicate worse functional outcomes/greater disability.
| score on a scale | Prospective Set |
|---|---|
| End of Study: Walking/Standing | 1.19 ± 1.042 |
| End of Study: Grip/Reach | 1.83 ± 0.986 |
| End of Study: School/Work | 1.97 ± 1.166 |
| End of Study: Activities | 1.68 ± 1.238 |
| End of Study: Breathing | 1.00 ± 0.929 |
Impact on ability to function in daily life was measured by the CHAQ (Parent Report). The CHAQ includes 30 items measured on a scale of 0 to 3: 0=without any difficulty; 1=with some difficulty; 2=with much difficulty; 3=Unable to do; Missing: Does not apply. It evaluates functional abilities across 8 domains (dressing, hygiene, arising, eating, walking, reach, grip and activities). The result is referred as Disability Index. The highest scoring item in each category determines the score for that category with higher scores indicating worse functioning/higher disability. The Discomfort Index and Health Status Index are measured on separate 15 cm scales. The distance from the left end of the scale to the respondent's mark is measured and multiplied by 0.2 to calculate the score (range 0-3). Discomfort and Health Status Index scores were rescaled to 0-100 scales. Higher scores indicate greater discomfort/worse health status.
| score on a scale | Prospective Set |
|---|---|
| Disability Index | 2.143 ± 0.7334 |
| Discomfort Index | 16.4 ± 25.57 |
| Health Status Index | 51.9 ± 33.12 |
Adaptive behavior was assessed using parent/caregiver report on the VABS-II, a standardized norm-referenced tool to evaluate adaptive behavior for ages 0-90.VABS-II has 1 composite score(Adaptive Behavior Composite \[ABC\]), reflecting overall adaptive ability.ABC comprises 4 domain scores in participants \<7years old(Communication,Daily Living Skills,Socialization,\& Motor Skills)\& 3 domain scores in participants ≥7years of age(Communication,Daily Living Skills,\& Socialization).ABC standard score is derived from domain standard scores per VABS-II manual(not a simple sum/average of the reported standard scores).Domain scores are standard scores derived from combination of 11 subdomain scores according to VABS-II scoring rules/manual.Scale for ABC \& domain standard scores ranges between 20 \& 160.ABC \& domain scores have a normative mean=100,with SD=15 \& subdomain scores are normed with a mean=15 \& SD=3.Higher scores indicate better,while lower scores indicate worse adaptive functioning.
| score on a scale | Prospective Set |
|---|---|
| EOS:Adaptive Behavior Composite Standard Score | 48.1 ± 19.07 |
| EOS:Communication Domain Standard Score | 44.4 ± 15.19 |
| EOS:Daily Living Domain Standard Score | 51.6 ± 22.53 |
| EOS:Motor Skills Domain Standard Score | 28.5 ± 13.22 |
| EOS: Social Domain Standard Score | 53.6 ± 19.88 |
Blood samples were collected and analyzed for determination of anti-idursulfase antibodies every 6 months in SHP-ELA-401. Analysis of anti-idursulfase antibodies including neutralizing antibodies (NAb) was conducted using validated 3-tier immunoassay methods (screening, confirmatory, and titer).
| Participants | Prospective Set |
|---|---|
| ADA Positive (ADA+) | 21 |
| NAb Positive (NAb+) | 15 |
Collected over From baseline until the end of study (up to approximately 9.75 years). Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Prospective Set | 1/21 (4.8%) | 11/21 (52.4%) | 21/21 (100%) |
| Event | Prospective Set |
|---|---|
| PneumoniaInfections and infestations | 6/21 |
| BronchitisInfections and infestations | 3/21 |
| COVID-19Infections and infestations | 2/21 |
| BradycardiaCardiac disorders | 1/21 |
| ChillsGeneral disorders | 1/21 |
| Dengue feverInfections and infestations | 1/21 |
| DyspnoeaRespiratory, thoracic and mediastinal disorders | 1/21 |
| EczemaSkin and subcutaneous tissue disorders | 1/21 |
| HydrocephalusNervous system disorders | 1/21 |
| HypernatraemiaMetabolism and nutrition disorders | 1/21 |
| Event | Prospective Set |
|---|---|
| NasopharyngitisInfections and infestations | 14/21 |
| DiarrhoeaGastrointestinal disorders | 13/21 |
| Upper respiratory tract infectionInfections and infestations | 13/21 |
| BronchitisInfections and infestations | 11/21 |
| PyrexiaGeneral disorders | 11/21 |
| COVID-19Infections and infestations | 7/21 |
| RhinitisInfections and infestations | 7/21 |
| Viral infectionInfections and infestations | 7/21 |
| CoughRespiratory, thoracic and mediastinal disorders | 6/21 |
| PharyngitisInfections and infestations | 6/21 |
Efficacy Set: all prospective participants, who had a baseline and at least 1 post-baseline efficacy assessment. HOS Treated Set included all participants enrolled in HOS registry that met criteria. HOS Untreated Set included untreated participants from HOS registry that met criteria.
| Age, Customized(Participants) | Prospective Set | HOS Treated Set | HOS Untreated Set | Total |
|---|---|---|---|---|
| Less than (<)2 Years | 2 | 2 | 15 | 19 |
| Greater than equal to (>=) 2 Years | 19 | 17 | 11 | 47 |
| Sex: Female, Male(Participants) | Prospective Set | HOS Treated Set | HOS Untreated Set | Total |
|---|---|---|---|---|
| Female | 0 | 0 | 0 | 0 |
| Male | 21 | 19 | 26 | 66 |
| Ethnicity (NIH/OMB)(Participants) | Prospective Set | HOS Treated Set | HOS Untreated Set | Total |
|---|---|---|---|---|
| Hispanic or Latino | 2 | 0 | 0 | 2 |
| Not Hispanic or Latino | 9 | 0 | 0 | 9 |
| Unknown or Not Reported | 10 | 19 | 26 | 55 |
| Race (NIH/OMB)(Participants) | Prospective Set | HOS Treated Set | HOS Untreated Set | Total |
|---|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 | 0 |
| Asian | 15 | 1 | 1 | 17 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 |
| Black or African American | 0 | 1 | 2 | 3 |
| White | 4 | 14 | 23 | 41 |
| More than one race | 0 | 0 | 0 | 0 |
| Unknown or Not Reported | 2 | 3 | 0 | 5 |
PDFs hosted by the registry, ClinicalTrials.gov.
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, Statistical analysis plan, Informed consent form, Clinical study report
Access criteria: 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.
Data sharing details ↗ · vivli.org
Ran from Oct 2015 to primary completion in Jul 2025, 117 months.
Results were first posted in Apr 2026, 8 months after primary completion.
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Studies of the drug tested here: Idursulfase