The purpose of this study is to collect data that will increase understanding of Hunter syndrome. The data from HOS may provide guidance to healthcare professionals about disease treatment options.
73 studies on the registry are indexed under Mucopolysaccharidosis II; 8 are open to participants now.
This study's enrollment of 1,443 is above the median of 34 across 25 observational studies indexed under Mucopolysaccharidosis II.
Browse Mucopolysaccharidosis II studies →Shire is the lead sponsor of 351 studies on the registry; 2 are open to participants now.
Of its 48 completed or terminated interventional studies of FDA-regulated products, 48 (100%) have results posted.
First submitted before Shire became part of Takeda.
Counted across the registry records on this site, refreshed daily.
The HOS registry is open to all participants (both alive and deceased) with Hunter syndrome who are untreated or who are receiving/received treatment with Elaprase, including participants who are alive at HOS enrollment (referred to as "Prospective Patients") and participants who are deceased at HOS enrollment (referred to as "Historical Patients").
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Signed and dated written informed consent, as per either a or b below:
Prospective Participants: Signed and dated written informed consent from the participant or, for participants aged less than (\<) 18 years (\<16 years in Scotland), parent and/or participant's legally authorized representative (LAR), and assent of the minor where applicable.
informed consent must be obtained from LARs for cognitively impaired participants, where applicable.
OR
Exclusion Criteria:
Participants with Hunters Syndrome received or receiving treatment with Elaprase as prescribed by their physician following locally approved prescribing information.
Participants received no treatment for Hunters Syndrome.
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.
Time frame: Baseline to year 17
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.
Time frame: Baseline to year 17
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.
Time frame: Baseline to year 17
Change in Urinary Glycosaminoglycan (GAG) Levels
Change in urinary GAG levels from the start of ERT is reported.
Time frame: Baseline to year 17
Change in Height
Change in height from the start of ERT will be reported.
Time frame: Baseline to year 17
Change in Weight
Change in weight from the start of ERT will be reported.
Time frame: Baseline to year 17
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.
Time frame: Baseline to year 17
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.
Time frame: Baseline to year 17
Left Ventricular Mass Index (LVMI)
Change in LVMI will be assessed as calculated by echocardiography.
Time frame: Baseline to year 17
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).
Time frame: Baseline to year 17
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).
Time frame: Baseline to year 17
Change in Liver and Spleen Size
Change in liver and spleen size as estimated by palpation will be reported.
Time frame: Baseline to year 17
Prevalence of Cardiac and Pulmonary-related Hospitalizations
Prevalence of cardiac and pulmonary-related hospitalizations will be reported.
Time frame: Baseline to year 17
Age at the Time of Death
Age at the time of death will be reported.
Time frame: Baseline to year 17
Cause of Death
Causes of death will be reported
Time frame: Baseline to year 17
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.
Time frame: Baseline to year 17
Dosing Regimens of Elaprase for Prescribed Dose in Participants With Hunter Syndrome
Dosing regiments of Elaprase will be evaluated for prescribed dose.
Time frame: Baseline to year 17
Dosing Regimens of Elaprase for Administered Dose in Participants With Hunter Syndrome
Dosing regiments of Elaprase will be evaluated for administered dose.
Time frame: Baseline to year 17
Dosing Regimens of Elaprase for Total Infusion Time in Participants With Hunter Syndrome
Dosing regiments of Elaprase will be evaluated for total infusion time.
Time frame: Baseline to year 17
Dosing Regimens of Elaprase for Missed Infusions in Participants With Hunter Syndrome
Dosing regiments of Elaprase will be evaluated for missed infusions.
Time frame: Baseline to year 17
Dosing Regimens of Elaprase for Reason for Missed Infusions.
Dosing regiments of Elaprase will be evaluated for reason for missed infusions.
Time frame: Baseline to year 17
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.
Time frame: Baseline to year 17
This study is completed, as verified in Oct 2024. You cannot join it, but the record below documents what was studied.
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