Outcome
Type |
Measure |
Description |
Time frame |
Safety issue |
Primary |
Number of Participants With Infusion-related Reactions (IRRs) |
An Infusion-related reaction (IRR) is an adverse event (AE) that occurs during or within 24 hours of an infusion and with evidence of a causal relationship with Elaprase. |
Baseline to year 17 |
|
Primary |
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) |
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 the registry, whether or not considered product-related. This includes an exacerbation of a pre-existing condition. An AE or adverse drug reaction (ADR) that meets one or more of the following criteria/outcomes is classified as serious whether considered to be related to the pharmaceutical product or not: death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalizations, a persistent or significant disability or incapacity, a congenital anomaly or birth defect and important medical events. |
Baseline to year 17 |
|
Primary |
Number of Participants With Positive Antibody Response |
Immunogenicity is determined by time to first positive antibody response (antibody level and isotype), antibody titer, isotype, and neutralizing antibodies. |
Baseline to year 17 |
|
Primary |
Change in Urinary Glycosaminoglycan (GAG) Levels |
Change in urinary GAG levels from the start of ERT is reported. |
Baseline to year 17 |
|
Primary |
Change in Height |
Change in height from the start of ERT will be reported. |
Baseline to year 17 |
|
Primary |
Change in Weight |
Change in weight from the start of ERT will be reported. |
Baseline to year 17 |
|
Primary |
Change in Head Circumference and Corresponding Calculated Z-scores |
Change in head circumference with the corresponding Z-scores from the start of ERT will be reported. |
Baseline to year 17 |
|
Primary |
Change in Distance Walked in the 6-minute Walk Test |
Change in distance walked in 6-minute walk test from the start of ERT is reported. |
Baseline to year 17 |
|
Primary |
Left Ventricular Mass Index (LVMI) |
Change in LVMI will be assessed as calculated by echocardiography. |
Baseline to year 17 |
|
Primary |
Change in Forced Expiratory Volume in 1 Second (FEV1) |
Change in pulmonary function from the start of ERT will be reported as measured by forced expiratory volume in 1 second (FEV1). |
Baseline to year 17 |
|
Primary |
Change in Forced Vital Capacity (FVC) |
Change in pulmonary function from the start of ERT will be reported as measured by forced vital capacity (FVC). |
Baseline to year 17 |
|
Primary |
Change in Liver and Spleen Size |
Change in liver and spleen size as estimated by palpation will be reported. |
Baseline to year 17 |
|
Primary |
Prevalence of Cardiac and Pulmonary-related Hospitalizations |
Prevalence of cardiac and pulmonary-related hospitalizations will be reported. |
Baseline to year 17 |
|
Primary |
Age at the Time of Death |
Age at the time of death will be reported. |
Baseline to year 17 |
|
Primary |
Cause of Death |
Causes of death will be reported |
Baseline to year 17 |
|
Secondary |
Natural History of Untreated Participants With Hunter Syndrome |
Evaluation of signs and symptoms for the natural history of disease: hepatosplenomegaly, central nervous system involvement, skeletal involvement, ear, nose, and throat signs and symptoms, pulmonary signs and symptoms and cardiac signs and symptoms will be reported. |
Baseline to year 17 |
|
Secondary |
Dosing Regimens of Elaprase for Prescribed Dose in Participants With Hunter Syndrome |
Dosing regiments of Elaprase will be evaluated for prescribed dose. |
Baseline to year 17 |
|
Secondary |
Dosing Regimens of Elaprase for Administered Dose in Participants With Hunter Syndrome |
Dosing regiments of Elaprase will be evaluated for administered dose. |
Baseline to year 17 |
|
Secondary |
Dosing Regimens of Elaprase for Total Infusion Time in Participants With Hunter Syndrome |
Dosing regiments of Elaprase will be evaluated for total infusion time. |
Baseline to year 17 |
|
Secondary |
Dosing Regimens of Elaprase for Missed Infusions in Participants With Hunter Syndrome |
Dosing regiments of Elaprase will be evaluated for missed infusions. |
Baseline to year 17 |
|
Secondary |
Dosing Regimens of Elaprase for Reason for Missed Infusions. |
Dosing regiments of Elaprase will be evaluated for reason for missed infusions. |
Baseline to year 17 |
|
Secondary |
Assessment of Hunter Syndrome on Health-related Quality of Life (HRQL) Using Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS) |
HS-FOCUS was 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 patient self-reported version for those over age 12 years. The HS-FOCUS Version 2.0 contains 6 functional status domains: Walking/Standing, Reach/Grip, Sleeping, Schooling/Work, Activities, and Breathing. Items are scored using a response scale from 0 to 4, with ="0" expressing being able to complete the activity-related functions "without any difficulty" and "4" as "unable to do so. Scores are averaged to calculate the 6 function domain scores and the Overall Function Score, with higher scores corresponding to a higher degree of incapacity. |
Baseline to year 17 |
|