Trial Outcomes & Findings for Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age (NCT NCT02455622)

Last Updated: 2026-04-02

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

21 participants

Primary outcome timeframe

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

Results posted on

2026-04-02

Participant Flow

Participants took part in the study at various investigative sites globally from 28 October 2015 to 29 July 2025.

Treatment-naïve participants with mucopolysaccharidosis II (MPS II) were enrolled in the study to receive weekly intravenous (IV) infusions of Elaprase starting at less than (\<) 6 years of age. Data for primary growth analyses was utilized from the Hunter Outcome Survey (HOS) registry participants for this study.

Participant milestones

Participant milestones
Measure	Prospective Set Participants received once-weekly IV infusions of Elaprase at a dose of 0.5 mg/kg and were 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 Set Treated participants from the Hunter Outcome Survey (HOS) patient registry, who met the criteria.	HOS Untreated Set Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.
Overall Study STARTED	21	19	26
Overall Study COMPLETED	17	19	26
Overall Study NOT COMPLETED	4	0	0

Reasons for withdrawal

Reasons for withdrawal
Measure	Prospective Set Participants received once-weekly IV infusions of Elaprase at a dose of 0.5 mg/kg and were 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 Set Treated participants from the Hunter Outcome Survey (HOS) patient registry, who met the criteria.	HOS Untreated Set Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.
Overall Study Death	1	0	0
Overall Study Non-compliance with study drug	2	0	0
Overall Study Reason Not Specified	1	0	0

Baseline Characteristics

Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Total n=66 Participants Total of all reporting groups	Prospective Set n=21 Participants Participants received once-weekly IV infusions of Elaprase at a dose of 0.5 mg/kg and were followed for a minimum of 5 years after initiation of Elaprase treatment, or until they reached their 10th birthday, whichever was longer.	HOS Treated Set n=19 Participants 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 Set n=26 Participants Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.
Age, Customized Less than (<)2 Years	19 Participants n=5 Participants	2 Participants n=5 Participants	2 Participants n=5 Participants	15 Participants n=10 Participants
Age, Customized Greater than equal to (>=) 2 Years	47 Participants n=5 Participants	19 Participants n=5 Participants	17 Participants n=5 Participants	11 Participants n=10 Participants
Sex: Female, Male Female	0 Participants n=5 Participants	0 Participants n=5 Participants	0 Participants n=5 Participants	0 Participants n=10 Participants
Sex: Female, Male Male	66 Participants n=5 Participants	21 Participants n=5 Participants	19 Participants n=5 Participants	26 Participants n=10 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=5 Participants	2 Participants n=5 Participants	0 Participants n=5 Participants	0 Participants n=10 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	9 Participants n=5 Participants	9 Participants n=5 Participants	0 Participants n=5 Participants	0 Participants n=10 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	55 Participants n=5 Participants	10 Participants n=5 Participants	19 Participants n=5 Participants	26 Participants n=10 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=5 Participants	0 Participants n=5 Participants	0 Participants n=10 Participants
Race (NIH/OMB) Asian	17 Participants n=5 Participants	15 Participants n=5 Participants	1 Participants n=5 Participants	1 Participants n=10 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=5 Participants	0 Participants n=5 Participants	0 Participants n=10 Participants
Race (NIH/OMB) Black or African American	3 Participants n=5 Participants	0 Participants n=5 Participants	1 Participants n=5 Participants	2 Participants n=10 Participants
Race (NIH/OMB) White	41 Participants n=5 Participants	4 Participants n=5 Participants	14 Participants n=5 Participants	23 Participants n=10 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=5 Participants	0 Participants n=5 Participants	0 Participants n=10 Participants
Race (NIH/OMB) Unknown or Not Reported	5 Participants n=5 Participants	2 Participants n=5 Participants	3 Participants n=5 Participants	0 Participants n=10 Participants

PRIMARY outcome

Timeframe: 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

Population: The Combined Set included data from all participants in both the Efficacy Set and the HOS Treated Set. The Efficacy Set consisted of all prospective participants, who had a baseline and at least 1 post-baseline efficacy assessment. The HOS Treated Set included all participants enrolled in the HOS registry that met the criteria. The HOS Untreated Set included untreated participants from the HOS registry that met the criteria.

Outcome measures

Outcome measures
Measure	Combined Set n=40 Participants The Combined Set included data from all participants in both the Efficacy Set and the HOS Treated Set. Efficacy Set included prospective participants who received once-weekly IV infusions of Elaprase at a dose of 0.5 mg/kg and were followed for a minimum of 5 years after initiation of Elaprase treatment, or until they reached their 10th birthday, whichever was longer.	HOS Untreated Set n=26 Participants Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.	HOS Untreated Set Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.
Height Overall	113.759 centimeter (cm) Standard Error 1.211	108.143 centimeter (cm) Standard Error 1.7668	—

PRIMARY outcome

Population: The Combined Set included data from all participants in both the Efficacy Set and the HOS Treated Set. The Efficacy Set consisted of all prospective participants, who had a baseline and at least 1 post-baseline efficacy assessment. The HOS Treated Patients included all participants enrolled in the HOS registry that met the criteria. The HOS Untreated Set included untreated participants from the HOS registry that met the criteria.

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.

Outcome measures

Outcome measures
Measure	Combined Set n=40 Participants The Combined Set included data from all participants in both the Efficacy Set and the HOS Treated Set. Efficacy Set included prospective participants who received once-weekly IV infusions of Elaprase at a dose of 0.5 mg/kg and were followed for a minimum of 5 years after initiation of Elaprase treatment, or until they reached their 10th birthday, whichever was longer.	HOS Untreated Set n=26 Participants Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.	HOS Untreated Set Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.
Weight Overall	25.164 kilograms (kg) Standard Error 0.8382	26.11 kilograms (kg) Standard Error 1.1647	—

PRIMARY outcome

Population: Combined Set included data from all the participants in both Efficacy Set \& HOS Treated Set. HOS Untreated Set included untreated participants from the HOS registry that met the criteria. Combined Set included treated participants enrolled in this study (prospective participants) as well as treated participants from the HOS registry. Overall number analyzed is the number of participants with data available for analysis for this outcome measure.

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.

Outcome measures

Outcome measures
Measure	Combined Set n=39 Participants The Combined Set included data from all participants in both the Efficacy Set and the HOS Treated Set. Efficacy Set included prospective participants who received once-weekly IV infusions of Elaprase at a dose of 0.5 mg/kg and were followed for a minimum of 5 years after initiation of Elaprase treatment, or until they reached their 10th birthday, whichever was longer.	HOS Untreated Set n=22 Participants Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.	HOS Untreated Set Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.
Change From Baseline in Height Measured by Z-score	-1.135 Z-score Standard Deviation 1.3480	-4.228 Z-score Standard Deviation 1.4616	—

PRIMARY outcome

Population: The Combined Set included data from all participants in both the Efficacy Set and the HOS Treated Set. The HOS Untreated Set included untreated participants from the HOS registry that met the criteria. The Combined Set included treated participants enrolled in this study (prospective participants) as well as treated participants from the HOS registry. Overall number analyzed is the number of participants with data available for analysis for this outcome measure.

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.

Outcome measures

Outcome measures
Measure	Combined Set n=39 Participants The Combined Set included data from all participants in both the Efficacy Set and the HOS Treated Set. Efficacy Set included prospective participants who received once-weekly IV infusions of Elaprase at a dose of 0.5 mg/kg and were followed for a minimum of 5 years after initiation of Elaprase treatment, or until they reached their 10th birthday, whichever was longer.	HOS Untreated Set n=26 Participants Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.	HOS Untreated Set Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.
Change From Baseline in Weight Measured by Z-score	-1.161 Z-score Standard Deviation 1.3259	-2.337 Z-score Standard Deviation 1.9014	—

PRIMARY outcome

Timeframe: From Screening to End-of-Study (approximately 9.75 years)

Population: The Safety Analysis Set consisted of all prospective participants who received any amount of investigational product (IP). As pre-specified in the SAP, descriptive analysis for this outcome measure was planned only for the reported arm, which included all treated participants in this study.

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).

Outcome measures

Outcome measures
Measure	Combined Set n=21 Participants The Combined Set included data from all participants in both the Efficacy Set and the HOS Treated Set. Efficacy Set included prospective participants who received once-weekly IV infusions of Elaprase at a dose of 0.5 mg/kg and were followed for a minimum of 5 years after initiation of Elaprase treatment, or until they reached their 10th birthday, whichever was longer.	HOS Untreated Set Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.	HOS Untreated Set Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.
Number of Participants With Clinical Significant Abnormal Neurological Examination	0 Participants	—	—

PRIMARY outcome

Timeframe: From screening to End-of-Study (approximately 9.75 years)

Population: The Safety Analysis Set consisted of all prospective participants who received any amount of IP. As pre-specified in the SAP, descriptive analysis for this outcome measure was planned only for the reported arm, which included all treated participants in this study.

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.

Outcome measures

Outcome measures
Measure	Combined Set n=21 Participants The Combined Set included data from all participants in both the Efficacy Set and the HOS Treated Set. Efficacy Set included prospective participants who received once-weekly IV infusions of Elaprase at a dose of 0.5 mg/kg and were followed for a minimum of 5 years after initiation of Elaprase treatment, or until they reached their 10th birthday, whichever was longer.	HOS Untreated Set Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.	HOS Untreated Set Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.
Number of Participants With Treatment-emergent Adverse Events (TEAE)	21 Participants	—	—

PRIMARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Combined Set n=21 Participants The Combined Set included data from all participants in both the Efficacy Set and the HOS Treated Set. Efficacy Set included prospective participants who received once-weekly IV infusions of Elaprase at a dose of 0.5 mg/kg and were followed for a minimum of 5 years after initiation of Elaprase treatment, or until they reached their 10th birthday, whichever was longer.	HOS Untreated Set Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.	HOS Untreated Set Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.
Number of Participants With Clinically Significant Abnormal Urinalysis Values Erythrocytes (/HPF)	1 Participants	—	—
Number of Participants With Clinically Significant Abnormal Urinalysis Values Glucose (milligrams per deciliter (mg/dl))	1 Participants	—	—
Number of Participants With Clinically Significant Abnormal Urinalysis Values Occult Blood	1 Participants	—	—
Number of Participants With Clinically Significant Abnormal Urinalysis Values Protein (mg/dL)	3 Participants	—	—
Number of Participants With Clinically Significant Abnormal Urinalysis Values Bacteria (/High-power field (HPF))	5 Participants	—	—
Number of Participants With Clinically Significant Abnormal Urinalysis Values Leukocyte Esterase	1 Participants	—	—
Number of Participants With Clinically Significant Abnormal Urinalysis Values Mucous Threads	1 Participants	—	—
Number of Participants With Clinically Significant Abnormal Urinalysis Values Nitrite	4 Participants	—	—
Number of Participants With Clinically Significant Abnormal Urinalysis Values Specimen Appearance	6 Participants	—	—
Number of Participants With Clinically Significant Abnormal Urinalysis Values Squamous Epithelial Cells (/HPF)	2 Participants	—	—
Number of Participants With Clinically Significant Abnormal Urinalysis Values Squamous Transitional Epithelial Cells (/HPF)	1 Participants	—	—
Number of Participants With Clinically Significant Abnormal Urinalysis Values Triple Phosphate Crystals (/HPF)	2 Participants	—	—
Number of Participants With Clinically Significant Abnormal Urinalysis Values Yeast Cells	1 Participants	—	—
Number of Participants With Clinically Significant Abnormal Urinalysis Values pH (pH)	3 Participants	—	—

PRIMARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Combined Set n=21 Participants The Combined Set included data from all participants in both the Efficacy Set and the HOS Treated Set. Efficacy Set included prospective participants who received once-weekly IV infusions of Elaprase at a dose of 0.5 mg/kg and were followed for a minimum of 5 years after initiation of Elaprase treatment, or until they reached their 10th birthday, whichever was longer.	HOS Untreated Set Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.	HOS Untreated Set Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.
Number of Participants With Clinically Significant Abnormal Serum Chemistry Values Alanine Aminotransferase (units per liter (U/L))	1 Participants	—	—
Number of Participants With Clinically Significant Abnormal Serum Chemistry Values Aspartate Aminotransferase (U/L)	1 Participants	—	—
Number of Participants With Clinically Significant Abnormal Serum Chemistry Values Gamma Glutamyl Transferase (U/L)	1 Participants	—	—

PRIMARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Combined Set n=21 Participants The Combined Set included data from all participants in both the Efficacy Set and the HOS Treated Set. Efficacy Set included prospective participants who received once-weekly IV infusions of Elaprase at a dose of 0.5 mg/kg and were followed for a minimum of 5 years after initiation of Elaprase treatment, or until they reached their 10th birthday, whichever was longer.	HOS Untreated Set Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.	HOS Untreated Set Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.
Number of Participants With Clinically Significant Abnormal Hematology Values Ery. Mean Corpuscular Hemoglobin (picogram (pg))	1 Participants	—	—
Number of Participants With Clinically Significant Abnormal Hematology Values Ery. Mean Corpuscular Volume (femtoliter (fL))	1 Participants	—	—
Number of Participants With Clinically Significant Abnormal Hematology Values Hemoglobin (gram per litre (g/L))	1 Participants	—	—

SECONDARY outcome

Timeframe: 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

Population: Efficacy Set: all prospective participants, who had a baseline and at least 1 post-baseline efficacy assessment. Combined Set included data from all participants in both Efficacy Set and HOS Treated Set. HOS Treated Set included all participants enrolled in HOS registry that met the criteria. HOS Untreated Set included untreated participants from HOS registry that met the criteria. Number analyzed is the number of participants with data available for the specified categories.

Height velocity was calculated as the difference in height, divided by the difference in age between consecutive study visits.

Outcome measures

Outcome measures
Measure	Combined Set n=40 Participants The Combined Set included data from all participants in both the Efficacy Set and the HOS Treated Set. Efficacy Set included prospective participants who received once-weekly IV infusions of Elaprase at a dose of 0.5 mg/kg and were followed for a minimum of 5 years after initiation of Elaprase treatment, or until they reached their 10th birthday, whichever was longer.	HOS Untreated Set n=21 Participants Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.	HOS Untreated Set n=26 Participants Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.
Observed Value of Height Velocity From Baseline to End of Study Overall Height Velocity: BL to < 6 Years of Age	6.556 centimeters (cm)/year Standard Deviation 2.6803	5.401 centimeters (cm)/year Standard Deviation 2.5815	6.557 centimeters (cm)/year Standard Deviation 2.5902
Observed Value of Height Velocity From Baseline to End of Study Overall Height Velocity: 6 Years of Age to EOS	3.571 centimeters (cm)/year Standard Deviation 1.8064	2.699 centimeters (cm)/year Standard Deviation 1.7610	1.761 centimeters (cm)/year Standard Deviation 1.2178

SECONDARY outcome

Timeframe: 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

Population: Efficacy Set: all prospective participants, who had a baseline and at least 1 post-baseline efficacy assessment. Combined Set included data from all participants in both Efficacy Set and HOS Treated Set. HOS Treated Set included all participants enrolled in HOS registry that met the criteria. HOS Untreated Set included untreated participants from HOS registry that met criteria. Number analyzed is the number of participants with data available for the specified categories.

Weight velocity was be calculated as the difference in weight, divided by the difference in age between consecutive study visits.

Outcome measures

Outcome measures
Measure	Combined Set n=40 Participants The Combined Set included data from all participants in both the Efficacy Set and the HOS Treated Set. Efficacy Set included prospective participants who received once-weekly IV infusions of Elaprase at a dose of 0.5 mg/kg and were followed for a minimum of 5 years after initiation of Elaprase treatment, or until they reached their 10th birthday, whichever was longer.	HOS Untreated Set n=21 Participants Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.	HOS Untreated Set n=26 Participants Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.
Observed Value of Weight Velocity From Baseline to End of Study Overall Velocity: Baseline to < 6 Years of Age	2.475 kilograms (kg)/year Standard Deviation 1.4550	1.772 kilograms (kg)/year Standard Deviation 1.2029	3.670 kilograms (kg)/year Standard Deviation 2.0005
Observed Value of Weight Velocity From Baseline to End of Study Overall Velocity: 6 Years of Age to EOS	2.305 kilograms (kg)/year Standard Deviation 1.7134	1.573 kilograms (kg)/year Standard Deviation 1.3198	1.445 kilograms (kg)/year Standard Deviation 0.8324

SECONDARY outcome

Timeframe: Baseline to End-of-Study (Approximately 9.75 years)

Population: The Efficacy Set is defined as all prospective participants who had a baseline and at least 1 post-baseline efficacy assessment. Overall number analyzed is the number of participants with data available for analysis for this outcome measure at the end-of-study. As pre-specified in the SAP, descriptive analysis for this outcome measure was planned only for the reported arm, which included all prospective participants in the Efficacy Set in this study.

Urinary GAG levels were normalized to urine creatinine (normalized uGAG) and reported as mg uGAG/ millimoles (mmol) creatinine.

Outcome measures

Outcome measures
Measure	Combined Set n=20 Participants The Combined Set included data from all participants in both the Efficacy Set and the HOS Treated Set. Efficacy Set included prospective participants who received once-weekly IV infusions of Elaprase at a dose of 0.5 mg/kg and were followed for a minimum of 5 years after initiation of Elaprase treatment, or until they reached their 10th birthday, whichever was longer.	HOS Untreated Set Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.	HOS Untreated Set Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.
Percent Change From Baseline for Urinary Glycosaminoglycans (uGAG) Levels Normalized to Urine Creatinine	-70.446 percent change Standard Deviation 17.8741	—	—

SECONDARY outcome

Timeframe: Baseline to End-of-Study (Approximately 9.75 years)

Population: The Efficacy Set is defined as all prospective participants who had a baseline and at least 1 post-baseline efficacy assessment. Number analyzed is the number of participants with data available for analysis for this outcome measure at the end-of-study. As pre-specified in the SAP, descriptive analysis for this outcome measure was planned only for the reported arm, which included all prospective participants in the Efficacy Set in this study.

Normalized uGAG was divided by the upper limit of normal for age (uGAG/ULN), where the ULN for uGAG was obtained from Mayo Clinic.

Outcome measures

Outcome measures
Measure	Combined Set n=21 Participants The Combined Set included data from all participants in both the Efficacy Set and the HOS Treated Set. Efficacy Set included prospective participants who received once-weekly IV infusions of Elaprase at a dose of 0.5 mg/kg and were followed for a minimum of 5 years after initiation of Elaprase treatment, or until they reached their 10th birthday, whichever was longer.	HOS Untreated Set Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.	HOS Untreated Set Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.
Normalized uGAG Divided by Upper Llimit of Normal for Age (uGAG/ULN) Every 12 Months Normalized uGAG/ULN: 72 Months	2.190 Normalized uGAG/ULN Standard Deviation 1.0945	—	—
Normalized uGAG Divided by Upper Llimit of Normal for Age (uGAG/ULN) Every 12 Months Normalized uGAG/ULN: 84 Months	0.830 Normalized uGAG/ULN Standard Deviation NA SD was not estimable for a single participant.	—	—
Normalized uGAG Divided by Upper Llimit of Normal for Age (uGAG/ULN) Every 12 Months Normalized uGAG/ULN: End of Study	2.055 Normalized uGAG/ULN Standard Deviation 1.1866	—	—
Normalized uGAG Divided by Upper Llimit of Normal for Age (uGAG/ULN) Every 12 Months Normalized uGAG/ULN: Baseline	5.052 Normalized uGAG/ULN Standard Deviation 1.0376	—	—
Normalized uGAG Divided by Upper Llimit of Normal for Age (uGAG/ULN) Every 12 Months Normalized uGAG/ULN: 12 Months	2.315 Normalized uGAG/ULN Standard Deviation 0.7655	—	—
Normalized uGAG Divided by Upper Llimit of Normal for Age (uGAG/ULN) Every 12 Months Normalized uGAG/ULN: 24 Months	2.096 Normalized uGAG/ULN Standard Deviation 0.7657	—	—
Normalized uGAG Divided by Upper Llimit of Normal for Age (uGAG/ULN) Every 12 Months Normalized uGAG/ULN: 36 Months	2.182 Normalized uGAG/ULN Standard Deviation 0.9339	—	—
Normalized uGAG Divided by Upper Llimit of Normal for Age (uGAG/ULN) Every 12 Months Normalized uGAG/ULN: 48 Months	2.063 Normalized uGAG/ULN Standard Deviation 0.6853	—	—
Normalized uGAG Divided by Upper Llimit of Normal for Age (uGAG/ULN) Every 12 Months Normalized uGAG/ULN: 60 Months	2.197 Normalized uGAG/ULN Standard Deviation 0.8870	—	—

SECONDARY outcome

Timeframe: Baseline up to 24 Months

Population: Efficacy Set consisted of all prospective participants, who had a baseline and at least 1 post-baseline efficacy assessment. Overall number analyzed is the number of participants with data available for analysis. Number analyzed is the number of participants with data available for analysis at the specified time points. As pre-specified in the SAP, descriptive analysis for this outcome measure was planned only for the reported arm, which included all prospective participants in the Efficacy Set.

Liver volume was assessed using abdominal ultrasonography.

Outcome measures

Outcome measures
Measure	Combined Set n=20 Participants The Combined Set included data from all participants in both the Efficacy Set and the HOS Treated Set. Efficacy Set included prospective participants who received once-weekly IV infusions of Elaprase at a dose of 0.5 mg/kg and were followed for a minimum of 5 years after initiation of Elaprase treatment, or until they reached their 10th birthday, whichever was longer.	HOS Untreated Set Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.	HOS Untreated Set Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.
Liver Volume Liver Volume: Baseline	105.855 milliliters (mL) Standard Deviation 16.8925	—	—
Liver Volume Liver Volume: 24 Months	87.811 milliliters (mL) Standard Deviation 7.0147	—	—

SECONDARY outcome

Timeframe: Baseline up to 24 Months

Spleen volume was assessed using abdominal ultrasonography.

Outcome measures

Outcome measures
Measure	Combined Set n=20 Participants The Combined Set included data from all participants in both the Efficacy Set and the HOS Treated Set. Efficacy Set included prospective participants who received once-weekly IV infusions of Elaprase at a dose of 0.5 mg/kg and were followed for a minimum of 5 years after initiation of Elaprase treatment, or until they reached their 10th birthday, whichever was longer.	HOS Untreated Set Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.	HOS Untreated Set Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.
Spleen Volume Spleen Volume: Baseline	104.593 mL Standard Deviation 29.9658	—	—
Spleen Volume Spleen Volume: 24 Months	98.427 mL Standard Deviation 47.8022	—	—

SECONDARY outcome

Timeframe: From Baseline to End-of-Study (approximately 9.75 years)

Population: Efficacy Set consisted of all prospective participants, who had a baseline and at least 1 post-baseline efficacy assessment. Overall number analyzed is the number of participants with data available for analysis for this outcome measure at the end-of-study. As pre-specified in the SAP, descriptive analysis for this outcome measure was planned only for the reported arm, which included all prospective participants in the Efficacy Set in this study.

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.

Outcome measures

Outcome measures
Measure	Combined Set n=20 Participants The Combined Set included data from all participants in both the Efficacy Set and the HOS Treated Set. Efficacy Set included prospective participants who received once-weekly IV infusions of Elaprase at a dose of 0.5 mg/kg and were followed for a minimum of 5 years after initiation of Elaprase treatment, or until they reached their 10th birthday, whichever was longer.	HOS Untreated Set Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.	HOS Untreated Set Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.
Joint Mobility, as Measured by Joint Range of Motion (JROM) Scores, Including Upper-Limb and Lower-Limb Joint Scores Upper Limb	104.24 percent score Standard Deviation 21.471	—	—
Joint Mobility, as Measured by Joint Range of Motion (JROM) Scores, Including Upper-Limb and Lower-Limb Joint Scores Lower Limb	94.50 percent score Standard Deviation 31.634	—	—

SECONDARY outcome

Timeframe: From Baseline to End-of-Study (approximately 9.75 years)

Population: Efficacy Set consisted of all prospective participants, who had a baseline and at least 1 post-baseline efficacy assessment. As pre-specified in the SAP, descriptive analysis for this outcome measure was planned only for the reported arm, which included all prospective participants in the Efficacy Set in this study.

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.

Outcome measures

Outcome measures
Measure	Combined Set n=21 Participants The Combined Set included data from all participants in both the Efficacy Set and the HOS Treated Set. Efficacy Set included prospective participants who received once-weekly IV infusions of Elaprase at a dose of 0.5 mg/kg and were followed for a minimum of 5 years after initiation of Elaprase treatment, or until they reached their 10th birthday, whichever was longer.	HOS Untreated Set Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.	HOS Untreated Set Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.
Distance Walked, as Measured by Six Minute Walk Test (6MWT)	224.2 meters Standard Deviation 178.42	—	—

SECONDARY outcome

Timeframe: Baseline to End-of-Study (Approximately 9.75 years)

Population: Efficacy Set consisted of all prospective participants, who had a baseline and at least 1 post-baseline efficacy assessment. Number analyzed is the number of participants with data available for analysis for the specified categories. As pre-specified in the SAP, descriptive analysis for this outcome measure was planned only for the reported arm, which included all prospective participants in the Efficacy Set in this study.

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.

Outcome measures

Outcome measures
Measure	Combined Set n=21 Participants The Combined Set included data from all participants in both the Efficacy Set and the HOS Treated Set. Efficacy Set included prospective participants who received once-weekly IV infusions of Elaprase at a dose of 0.5 mg/kg and were followed for a minimum of 5 years after initiation of Elaprase treatment, or until they reached their 10th birthday, whichever was longer.	HOS Untreated Set Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.	HOS Untreated Set Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.
Quality of Life, as Measured by Hunter-Syndrome Functional Outcome in Clinical Understanding Scale (HS-FOCUS) End of Study: Walking/Standing	1.19 score on a scale Standard Deviation 1.042	—	—
Quality of Life, as Measured by Hunter-Syndrome Functional Outcome in Clinical Understanding Scale (HS-FOCUS) End of Study: Grip/Reach	1.83 score on a scale Standard Deviation 0.986	—	—
Quality of Life, as Measured by Hunter-Syndrome Functional Outcome in Clinical Understanding Scale (HS-FOCUS) End of Study: School/Work	1.97 score on a scale Standard Deviation 1.166	—	—
Quality of Life, as Measured by Hunter-Syndrome Functional Outcome in Clinical Understanding Scale (HS-FOCUS) End of Study: Activities	1.68 score on a scale Standard Deviation 1.238	—	—
Quality of Life, as Measured by Hunter-Syndrome Functional Outcome in Clinical Understanding Scale (HS-FOCUS) End of Study: Breathing	1.00 score on a scale Standard Deviation 0.929	—	—

SECONDARY outcome

Timeframe: Baseline to End-of-Study (approximately 9.75 years)

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.

Outcome measures

Outcome measures
Measure	Combined Set n=21 Participants The Combined Set included data from all participants in both the Efficacy Set and the HOS Treated Set. Efficacy Set included prospective participants who received once-weekly IV infusions of Elaprase at a dose of 0.5 mg/kg and were followed for a minimum of 5 years after initiation of Elaprase treatment, or until they reached their 10th birthday, whichever was longer.	HOS Untreated Set Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.	HOS Untreated Set Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.
Impact of Illness on Ability to Function in Daily Life, as Measured by Childhood Health Assessment Questionnaire (CHAQ Parent Report) Disability Index	2.143 score on a scale Standard Deviation 0.7334	—	—
Impact of Illness on Ability to Function in Daily Life, as Measured by Childhood Health Assessment Questionnaire (CHAQ Parent Report) Discomfort Index	16.4 score on a scale Standard Deviation 25.57	—	—
Impact of Illness on Ability to Function in Daily Life, as Measured by Childhood Health Assessment Questionnaire (CHAQ Parent Report) Health Status Index	51.9 score on a scale Standard Deviation 33.12	—	—

SECONDARY outcome

Timeframe: Baseline to End-of-Study (approximately 9.75 years)

Population: Efficacy Set consisted of all prospective participants, who had a baseline and at least 1 post-baseline efficacy assessment. Overall number analyzed is the number of participants with data available for analysis for this outcome measure at the end-of-study. As pre-specified in the SAP, descriptive analysis for this outcome measure was planned only for the reported arm, which included all prospective participants in the Efficacy Set in this study.

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.

Outcome measures

Outcome measures
Measure	Combined Set n=16 Participants The Combined Set included data from all participants in both the Efficacy Set and the HOS Treated Set. Efficacy Set included prospective participants who received once-weekly IV infusions of Elaprase at a dose of 0.5 mg/kg and were followed for a minimum of 5 years after initiation of Elaprase treatment, or until they reached their 10th birthday, whichever was longer.	HOS Untreated Set Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.	HOS Untreated Set Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.
Adaptive Behavior, as Measured by the Vineland Adaptive Behavior Scales (VABS II) EOS: Social Domain Standard Score	53.6 score on a scale Standard Deviation 19.88	—	—
Adaptive Behavior, as Measured by the Vineland Adaptive Behavior Scales (VABS II) EOS:Adaptive Behavior Composite Standard Score	48.1 score on a scale Standard Deviation 19.07	—	—
Adaptive Behavior, as Measured by the Vineland Adaptive Behavior Scales (VABS II) EOS:Communication Domain Standard Score	44.4 score on a scale Standard Deviation 15.19	—	—
Adaptive Behavior, as Measured by the Vineland Adaptive Behavior Scales (VABS II) EOS:Daily Living Domain Standard Score	51.6 score on a scale Standard Deviation 22.53	—	—
Adaptive Behavior, as Measured by the Vineland Adaptive Behavior Scales (VABS II) EOS:Motor Skills Domain Standard Score	28.5 score on a scale Standard Deviation 13.22	—	—

SECONDARY outcome

Timeframe: From Baseline to End-of-Study (Approximately 9.75 years)

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).

Outcome measures

Outcome measures
Measure	Combined Set n=21 Participants The Combined Set included data from all participants in both the Efficacy Set and the HOS Treated Set. Efficacy Set included prospective participants who received once-weekly IV infusions of Elaprase at a dose of 0.5 mg/kg and were followed for a minimum of 5 years after initiation of Elaprase treatment, or until they reached their 10th birthday, whichever was longer.	HOS Untreated Set Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.	HOS Untreated Set Participants in the HOS patient registry, who were not treated, were used as the comparator in the Primary Growth Analysis for this study using their height and weight data.
Anti-Idursulfase Antibodies (ADA) in Serum ADA Positive (ADA+)	21 Participants	—	—
Anti-Idursulfase Antibodies (ADA) in Serum NAb Positive (NAb+)	15 Participants	—	—

Adverse Events

Prospective Set

Serious events: 11 serious events

Other events: 21 other events

Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure	Prospective Set n=21 participants at risk Participants received once-weekly IV infusions of Elaprase at a dose of 0.5 mg/kg and were followed for a minimum of 5 years after initiation of Elaprase treatment, or until they reached their 10th birthday, whichever was longer.
Cardiac disorders Bradycardia	4.8% 1/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Infections and infestations Bronchitis	14.3% 3/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Infections and infestations COVID-19	9.5% 2/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
General disorders Chills	4.8% 1/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Infections and infestations Dengue fever	4.8% 1/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Respiratory, thoracic and mediastinal disorders Dyspnoea	4.8% 1/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Skin and subcutaneous tissue disorders Eczema	4.8% 1/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Nervous system disorders Hydrocephalus	4.8% 1/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Metabolism and nutrition disorders Hypernatraemia	4.8% 1/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Infections and infestations Influenza	4.8% 1/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Gastrointestinal disorders Inguinal hernia	4.8% 1/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Respiratory, thoracic and mediastinal disorders Obstructive airways disorder	4.8% 1/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Eye disorders Papilloedema	4.8% 1/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Infections and infestations Pneumonia	28.6% 6/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Nervous system disorders Seizure	4.8% 1/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Infections and infestations Sepsis	4.8% 1/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Infections and infestations Skin infection	4.8% 1/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
General disorders Sudden death	4.8% 1/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Musculoskeletal and connective tissue disorders Systemic lupus erythematosus	4.8% 1/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.

Other adverse events

Other adverse events
Measure	Prospective Set n=21 participants at risk Participants received once-weekly IV infusions of Elaprase at a dose of 0.5 mg/kg and were followed for a minimum of 5 years after initiation of Elaprase treatment, or until they reached their 10th birthday, whichever was longer.
Blood and lymphatic system disorders Anaemia	9.5% 2/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Gastrointestinal disorders Anal fissure	9.5% 2/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Cardiac disorders Aortic valve incompetence	23.8% 5/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Eye disorders Blepharitis	9.5% 2/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Infections and infestations Bronchitis	52.4% 11/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Infections and infestations COVID-19	33.3% 7/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Infections and infestations Carbuncle	9.5% 2/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Nervous system disorders Carpal tunnel syndrome	9.5% 2/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Infections and infestations Conjunctivitis	23.8% 5/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Gastrointestinal disorders Constipation	14.3% 3/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Respiratory, thoracic and mediastinal disorders Cough	28.6% 6/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Skin and subcutaneous tissue disorders Dermatitis	9.5% 2/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Skin and subcutaneous tissue disorders Dermatitis allergic	19.0% 4/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Skin and subcutaneous tissue disorders Dermatitis atopic	9.5% 2/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Skin and subcutaneous tissue disorders Dermatitis diaper	9.5% 2/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Gastrointestinal disorders Diarrhoea	61.9% 13/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Infections and infestations Ear infection	9.5% 2/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Injury, poisoning and procedural complications Ear injury	9.5% 2/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Skin and subcutaneous tissue disorders Eczema	9.5% 2/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Skin and subcutaneous tissue disorders Erythema	14.3% 3/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Infections and infestations Furuncle	14.3% 3/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
General disorders Gait disturbance	9.5% 2/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Infections and infestations Gastroenteritis	19.0% 4/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Injury, poisoning and procedural complications Head injury	9.5% 2/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Infections and infestations Impetigo	9.5% 2/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Infections and infestations Influenza	14.3% 3/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Cardiac disorders Mitral valve incompetence	23.8% 5/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Infections and infestations Nasopharyngitis	66.7% 14/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	19.0% 4/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Infections and infestations Otitis media	9.5% 2/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Infections and infestations Otitis media acute	14.3% 3/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Infections and infestations Parotitis	9.5% 2/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Infections and infestations Pharyngitis	28.6% 6/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Infections and infestations Pharyngotonsillitis	9.5% 2/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Infections and infestations Pneumonia	19.0% 4/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Injury, poisoning and procedural complications Procedural pain	9.5% 2/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
General disorders Pyrexia	52.4% 11/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Skin and subcutaneous tissue disorders Rash	9.5% 2/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Skin and subcutaneous tissue disorders Rash macular	9.5% 2/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Infections and infestations Rhinitis	33.3% 7/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Respiratory, thoracic and mediastinal disorders Rhinitis allergic	19.0% 4/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	14.3% 3/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Nervous system disorders Seizure	9.5% 2/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Infections and infestations Sinusitis	14.3% 3/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Injury, poisoning and procedural complications Skin abrasion	14.3% 3/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Injury, poisoning and procedural complications Skin laceration	14.3% 3/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Psychiatric disorders Sleep disorder	14.3% 3/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Injury, poisoning and procedural complications Thermal burn	9.5% 2/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Infections and infestations Tonsillitis	23.8% 5/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Infections and infestations Upper respiratory tract infection	61.9% 13/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Infections and infestations Urinary tract infection	28.6% 6/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Skin and subcutaneous tissue disorders Urticaria	28.6% 6/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Infections and infestations Varicella	19.0% 4/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Infections and infestations Viral infection	33.3% 7/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Metabolism and nutrition disorders Vitamin D deficiency	9.5% 2/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Gastrointestinal disorders Vomiting	14.3% 3/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.
Respiratory, thoracic and mediastinal disorders Wheezing	9.5% 2/21 • From baseline until the end of study (up to approximately 9.75 years) The Safety Analysis Set consisted of all prospective participants who received any amount of IP. Adverse Event data only for participants receiving study drug in this study was collected.

Additional Information

Study Director

Takeda

Phone: +1-877-825-3327

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place