Registered outcomes15

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

1 to 15 of 15

List	Outcome	Time frame	Description
Primary	Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuation Due to TEAEs and Death	From start of the study drug administration up to Week 53	An adverse event (AE) was defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (example, a clinically significant abnormal laboratory value), symptom, or disease temporally associated with the use of a drug whether or not it is considered related to the drug. A serious TEAE was defined as any untoward medical occurrence that at any dose results in: death; is life-threatening: requires inpatient hospitalization or results in prolongation of existing hospitalization; persistent or significant disability/incapacity; leads to a congenital anomaly/birth defect or is an important medical event. Number of participants with TEAEs, serious TEAEs, discontinuation due to TEAEs, and death are reported.
Primary	Number of Participants With Adverse Drug Reactions (ADRs)	From start of the study drug administration up to Week 53	An ADR was defined as a response to a drug which was noxious and unintended, and which occurred at doses normally used in humans for prophylaxis, diagnosis, or therapy of disease, or for the modification of physiological function.
Primary	Number of Participants With Infusion-related Reactions (IRRs)	From start of the study drug administration up to Week 53	An IRR was defined as an AE that had been assessed as at least possibly related to treatment with elaprase and occurred during an infusion or up to 24 hours post-infusion.
Secondary	Change From Baseline in Percentage Forced Vital Capacity (%FVC) at Weeks 27 and 53	Baseline, Weeks 27 and 53	FVC is the amount of air that can be forcibly exhaled from your lungs after taking the deepest breath possible, as measured by spirometry. FVC is a measure of respiratory function.
Secondary	Change From Baseline in 6 Minute Walk Test (6MWT) at Weeks 27 and 53	Baseline, Weeks 27 and 53	6MWT is the distance covered over a time of 6 minutes and is a measure of physical functional capacity which is determined on a walking course.
Secondary	Change From Baseline in Cardiac Left Ventricular Mass Index (LVMI) at Weeks 27 and 53	Baseline, Weeks 27 and 53	Cardiac LVMI was measured by 2-dimensional (2D) echocardiography. Cardiac LVMI is the left ventricular mass (LVM) in grams indexed to body surface area (BSA), in square meters (m\^2). Cardiac LVMI (in grams per square meter \[g/m\^2\])=LVM divided by BSA.
Secondary	Change From Baseline in Left Ventricular Ejection Fraction (LVEF) at Weeks 27 and 53	Baseline, Weeks 27 and 53	The LVEF was measured by 2D echocardiography and considered a sufficiently sensitive measure to evaluate any changes in cardiac function.
Secondary	Change From Baseline in Liver Volume at Weeks 27 and 53	Baseline, Weeks 27 and 53	Liver volume was determined by Ultrasonography (USG).
Secondary	Change From Baseline in Spleen Volume at Weeks 27 and 53	Baseline, Weeks 27 and 53	Spleen volume was determined by USG.
Secondary	Change From Baseline in Normalized Urine Glycosaminoglycan (uGAG) Levels at Week 14, 27, 40, and 53	Baseline, Weeks 14, 27, 40, and 53	Normalized uGAG was analyzed using urine testing. The uGAG levels were normalized to urine creatinine and were reported as microgram glycosaminoglycan (GAG) per milligram creatinine (μg GAG/mg creatinine).
Secondary	Change From Baseline in Global Joint Range of Motion (JROM) Score at Weeks 27 and 53	Baseline, Weeks 27 and 53	Passive joint mobility is defined as the range of motion of the shoulder, elbow, wrist, hip, knee, and ankle joints, as assessed by one expert physician using universal goniometry method. Global JROM (% of normal range of motion) is the average of 11 ratios multiplied by 100. Ratios are left/right means of passive range of motion in shoulder (flexion/extension, abduction, internal/external rotation), elbow (flexion/extension), wrist (flexion/extension), index finger (flexion/extension \[combined metacarpophalangeal joint, proximal interphalangeal joint, distal interphalangeal joint motion\]), hip (flexion/extension, abduction, internal/external rotation), knee (flexion/extension), and ankle (dorsiflexion) divided by the normal range (American Academy of Orthopedic Surgeons and American Medical Association).
Secondary	Change From Baseline in Anthropometric Parameter: Height at Weeks 27 and 53	Baseline, Weeks 27 and 53	Change from baseline in height (centimeters \[cm\]) was assessed in participants less than (\<)18 years of age.
Secondary	Change From Baseline in Anthropometric Parameter: Weight at Weeks 27 and 53	Baseline, Weeks 27 and 53	Change from baseline in weight (kilograms\[kg\]) was assessed in all participants.
Secondary	Health-related Quality of Life (HRQoL) Based on Change From Baseline in the Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS) Domain Scores	Baseline, Weeks 27 and 53	HS-FOCUS is developed as disease-specific measure of the impact of Hunter syndrome on HRQL. The HS-FOCUS is designed to gather information on the participant's daily life and wellbeing, satisfaction with treatment, and hospitalizations, as well as on how Hunter syndrome impacts participant's general quality of life. HS-FOCUS includes 2 validated components: a parent version and a participant self-reported version for those over age 12 years. The HS-FOCUS (shortened version) questionnaire has 5 function domains (walking/standing, grip/reach, school/work, activities, and breathing). Items are scored using a response scale from 0 to 3, with 0 signifying an ability to complete the activity-related functions 'without any difficulty' and 3 denoting highest disability. Higher scores on each domain indicate greater disability.
Secondary	Change From Baseline in the HRQoL Based on Childhood Health Assessment Questionnaire (CHAQ) Domain Scores	Baseline, Weeks 27 and 53	The CHAQ was initially developed for assessing juvenile idiopathic arthritis, from the perspective of the parent or participant, and has been previously applied to other chronic disabling conditions such as Hunter syndrome. It is a 30-item instrument that measures functional capacity and independence in activities of daily life across eight domains: dressing and grooming, arising, eating, walking, reach, grip, hygiene, and activities. For each domain, there is a 4-level difficulty scale that is scored from 0 to 3, with 0 corresponding to 'without any difficulty' and 3 to 'unable to do'. Higher scores on each domain indicate greater disability.

Source ClinicalTrials.govtrials-2026-11-12-0.5.0

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

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