Hunter SyndromePhase 4NCT02455622
Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age
A Phase 4 study testing Elaprase for intravenous (IV) infusion in people with Hunter Syndrome, run by Shire at 8 sites in 8 countries.
Trial summary
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… Read more about this study
- Who is it for?
- Ages Up to 5 years, men only
- Conditions the study is for
- Hunter Syndrome
- What is being tested?
- Elaprase for intravenous (IV) infusion
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 8 sitesin 8 countries
- Study dates
- 28 Oct 2015to 29 Jul 2025
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 5 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Group 1: Prospective Patient Group
- The patient is male.
- The patient is Elaprase-naïve at study entry.
- 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:
- The patient has a deficiency in I2S enzyme activity of ≤10% of the lower limit of the normal range as measured in plasma, fibroblasts, or leukocytes (based on the reference laboratory's normal range). AND
- The patient has a documented mutation in the I2S gene. OR
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- The patient has a normal enzyme activity level of one other sulfatase as measured in plasma, fibroblasts, or leukocytes (based on the normal range of measuring laboratory).
- The patient will be <6 years of age at the start of Elaprase treatment.
- The patient, patient's parent(s) or legally authorized guardian(s) must have voluntarily signed an Institutional Review Board (IRB)/Independent Ethics Committee (IEC) 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, as relevant, must be obtained.
- 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.
- The patient is male.
- The patient is enrolled in HOS.
- The patient was <6 years of age at the start of Elaprase treatment.
- The patient received Elaprase weekly treatment for at least 5 years.
- The patient had a height assessment and a weight assessment documented within 3 months before or after Elaprase treatment start.
- The patient has had annual height and weight assessments from start of Elaprase through age 10 years.
- The patient, patient's parent(s), or legally authorized guardian(s) agree(s) to data collection.
- The patient, patient's parent(s), or legally authorized guardian(s) must have signed an IRB/IEC-approved informed consent form after all relevant aspects of the HOS study have been explained and discussed. Consent of the patient's parent(s) or legally authorized guardian(s) and the patient's assent, as relevant, must be obtained.
Who cannot join
- Group 1: Prospective Patient Group
- The patient has received treatment with any investigational drug or device within the 30 days prior to study entry.
- The patient has received or is receiving treatment with idursulfase-IT.
- The patient has received growth hormones, a cord blood infusion, or a bone marrow transplant at any time.
- The patient has received blood product transfusions within 90 days prior to Screening.
- The patient is unable to comply with the protocol as determined by the Investigator.
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- 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.
Trial locations and contacts
8 sites in 8 countries.
8 sites in 8 countries
United States1 site
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Dominican Republic1 site
Hospital Infantil Dr Robert Reid Cabral
Santo Domingo, 10101
Germany1 site
Universitätsmedizin der Johannes Gutenberg-Universität Mainz
Mainz, 55131
Serbia1 site
Mother and Child Health Care Institute of Serbia Dr Vukan Cupic
Belgrade, 11000
About this study
Official title: A Long-Term, Open-Label, Multicenter, Phase IV Study to Assess Longitudinal Changes on Height and Weight in Patients With MPS II Who Are Receiving Elaprase and Started Treatment With Elaprase at <6 Years of Age
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.
- Conditions
- Hunter Syndrome
- Being tested
- Elaprase for intravenous (IV) infusion
- Medical subject headings
- Mucopolysaccharidosis II
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Elaprase for intravenous (IV) infusionDrug
Also called Idursulfase
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.
| Study group | Role | What they receive |
|---|---|---|
| Prospective Set | Experimental | 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. Receives: Drug: Elaprase for intravenous (IV) infusion |
| Hunter Outcome Survey (HOS) Treated | No intervention | 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. |
| HOS Untreated | No intervention | 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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).
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: From screening to End-of-Study (approximately 9.75 years)
Secondary outcomes (12)
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Baseline to End-of-Study (Approximately 9.75 years)
Liver Volume
Liver volume was assessed using abdominal ultrasonography.
Measured: Baseline up to 24 Months
Spleen Volume
Spleen volume was assessed using abdominal ultrasonography.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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).
Measured: From Baseline to End-of-Study (Approximately 9.75 years)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 May 2015 (estimated)
- Study start28 Oct 2015
- Primary completion29 Jul 2025
- Study completion29 Jul 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Hunter SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 21 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Takeda Development Center Americas, Inc.
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Shire
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02455622
- Sponsor's protocol ID
- SHP-ELA-401
- Other IDs
- 2014-004804-31, 2014
- Status last verified
- Mar 2026
- First submitted
- 20 May 2015
- Last updated
- 2 Apr 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- 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
Study at a glance
From the registry record, last updated by the sponsor on 2 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Long-Term, Open-Label, Multicenter, Phase IV Study to Assess Longitudinal Changes on Height and Weight in Patients With MPS II Who Are Receiving Elaprase and Started Treatment With Elaprase at <6 Years of Age
- ClinicalTrials.gov ID
- NCT02455622
- Acronym / sponsor ID
- SHP-ELA-401
- Conditions
- Hunter Syndrome
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 21 participants (actual)
- Lead sponsor
- ShireIndustry (pharma, biotech, devices)
- Collaborators
- Takeda Development Center Americas, Inc.
- Study start
- 28 Oct 2015 (actual)
- Primary completion
- 29 Jul 2025 (actual)
- Study completion
- 29 Jul 2025 (actual)
- First posted
- 28 May 2015 (estimated)
- Results
- Posted 2 Apr 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Prospective Set | Experimental | Drug: Elaprase for intravenous (IV) infusionParticipants 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. |
| Hunter Outcome Survey (HOS) Treated | No intervention | –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. |
| HOS Untreated | No intervention | –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. |
Who may qualify
- Ages
- Up to 5 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (19 listed)
- Group 1: Prospective Patient Group
- 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.
- The patient is male.
- The patient is enrolled in HOS.
- The patient was <6 years of age at the start of Elaprase treatment.
Key exclusion criteria (9 listed)
- Group 1: Prospective Patient Group
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Elaprase for intravenous (IV) infusionAlso: IdursulfasePatients 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. | Drug | Prospective Set |
Primary outcome
Height Overall
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
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.
Weight Overall
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
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.
Change From Baseline in Height Measured by Z-score
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
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.
Change From Baseline in Weight Measured by Z-score
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
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.
Number of Participants With Clinical Significant Abnormal Neurological Examination
Time frame: From Screening to End-of-Study (approximately 9.75 years)
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).
Number of Participants With Treatment-emergent Adverse Events (TEAE)
Time frame: From screening to End-of-Study (approximately 9.75 years)
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.
Number of Participants With Clinically Significant Abnormal Urinalysis Values
Time frame: From screening to End-of-Study (approximately 9.75 years)
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.
Number of Participants With Clinically Significant Abnormal Serum Chemistry Values
Time frame: From screening to End-of-Study (approximately 9.75 years)
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.
Number of Participants With Clinically Significant Abnormal Hematology Values
Time frame: From screening to End-of-Study (approximately 9.75 years)
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.
12 secondary outcomes
Observed Value of Height Velocity From Baseline to End of Study
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
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
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
Time frame: Baseline to End-of-Study (Approximately 9.75 years)
Liver Volume
Time frame: Baseline up to 24 Months
Spleen Volume
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
Time frame: From Baseline to End-of-Study (approximately 9.75 years)
Distance Walked, as Measured by Six Minute Walk Test (6MWT)
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)
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)
Time frame: Baseline to End-of-Study (approximately 9.75 years)
Adaptive Behavior, as Measured by the Vineland Adaptive Behavior Scales (VABS II)
Time frame: Baseline to End-of-Study (approximately 9.75 years)
Anti-Idursulfase Antibodies (ADA) in Serum
Time frame: From Baseline to End-of-Study (Approximately 9.75 years)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Apr 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Click here for more information about this trial in easy-to-understand language, including a Plain Language Summary of the results if the trial has been completed.Link provided by the sponsor.
- Individual participant dataSharing planned: 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
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Study Director study director
Shire
8 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Elaprase for intravenous (IV) infusion in people with Hunter Syndrome, run by Shire at 8 sites in 8 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 19 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 2 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in United States, Dominican Republic, Germany, Malaysia, Philippines and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 2 Apr 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Height Overall (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). The study lists 9 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Apr 2026; AzTrials last refreshed its copy on 31 Oct 2026.