- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03292887
Hunter Outcome Survey (HOS) (HOS)
Hunter Outcome Survey: A Global, Multi-Center, Long-Term, Observational Registry of Patients With Hunter Syndrome (Mucopolysaccharidosis Type II, MPS II)
Study Overview
Status
Conditions
Study Type
Enrollment (Actual)
Contacts and Locations
Study Locations
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Massachusetts
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Lexington, Massachusetts, United States, 02421
- Shire
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
- Child
- Adult
- Older Adult
Accepts Healthy Volunteers
Sampling Method
Study Population
Description
Inclusion Criteria:
- Diagnosis of Hunter syndrome (biochemically and/or genetically)
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
- Historical Participants: Signed and dated informed consent from the participant's LAR (where allowed by relevant individual country or site regulations/laws). .
Exclusion Criteria:
- Participants enrolled in an interventional clinical trial are not eligible. Participants may re-enroll once they have completed or withdrawn from the other clinical study.
- Participants receiving treatment for Hunter syndrome with an ERT product other than Elaprase are not eligible. Participants may enroll or re-enroll once they have stopped treatment with another ERT.
Study Plan
How is the study designed?
Design Details
- Observational Models: Other
- Time Perspectives: Prospective
Cohorts and Interventions
Group / Cohort |
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Elaprase Treated
Participants with Hunters Syndrome received or receiving treatment with Elaprase as prescribed by their physician following locally approved prescribing information.
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Elaprase Non-Treated
Participants received no treatment for Hunters Syndrome.
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Number of Participants With Infusion-related Reactions (IRRs)
Time Frame: Baseline to year 17
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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.
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Baseline to year 17
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Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time Frame: Baseline to year 17
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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.
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Baseline to year 17
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Number of Participants With Positive Antibody Response
Time Frame: Baseline to year 17
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Immunogenicity is determined by time to first positive antibody response (antibody level and isotype), antibody titer, isotype, and neutralizing antibodies.
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Baseline to year 17
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Change in Urinary Glycosaminoglycan (GAG) Levels
Time Frame: Baseline to year 17
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Change in urinary GAG levels from the start of ERT is reported.
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Baseline to year 17
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Change in Height
Time Frame: Baseline to year 17
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Change in height from the start of ERT will be reported.
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Baseline to year 17
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Change in Weight
Time Frame: Baseline to year 17
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Change in weight from the start of ERT will be reported.
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Baseline to year 17
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Change in Head Circumference and Corresponding Calculated Z-scores
Time Frame: Baseline to year 17
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Change in head circumference with the corresponding Z-scores from the start of ERT will be reported.
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Baseline to year 17
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Change in Distance Walked in the 6-minute Walk Test
Time Frame: Baseline to year 17
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Change in distance walked in 6-minute walk test from the start of ERT is reported.
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Baseline to year 17
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Left Ventricular Mass Index (LVMI)
Time Frame: Baseline to year 17
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Change in LVMI will be assessed as calculated by echocardiography.
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Baseline to year 17
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Change in Forced Expiratory Volume in 1 Second (FEV1)
Time Frame: Baseline to year 17
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Change in pulmonary function from the start of ERT will be reported as measured by forced expiratory volume in 1 second (FEV1).
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Baseline to year 17
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Change in Forced Vital Capacity (FVC)
Time Frame: Baseline to year 17
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Change in pulmonary function from the start of ERT will be reported as measured by forced vital capacity (FVC).
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Baseline to year 17
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Change in Liver and Spleen Size
Time Frame: Baseline to year 17
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Change in liver and spleen size as estimated by palpation will be reported.
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Baseline to year 17
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Prevalence of Cardiac and Pulmonary-related Hospitalizations
Time Frame: Baseline to year 17
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Prevalence of cardiac and pulmonary-related hospitalizations will be reported.
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Baseline to year 17
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Age at the Time of Death
Time Frame: Baseline to year 17
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Age at the time of death will be reported.
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Baseline to year 17
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Cause of Death
Time Frame: Baseline to year 17
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Causes of death will be reported
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Baseline to year 17
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Natural History of Untreated Participants With Hunter Syndrome
Time Frame: Baseline to year 17
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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.
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Baseline to year 17
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Dosing Regimens of Elaprase for Prescribed Dose in Participants With Hunter Syndrome
Time Frame: Baseline to year 17
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Dosing regiments of Elaprase will be evaluated for prescribed dose.
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Baseline to year 17
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Dosing Regimens of Elaprase for Administered Dose in Participants With Hunter Syndrome
Time Frame: Baseline to year 17
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Dosing regiments of Elaprase will be evaluated for administered dose.
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Baseline to year 17
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Dosing Regimens of Elaprase for Total Infusion Time in Participants With Hunter Syndrome
Time Frame: Baseline to year 17
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Dosing regiments of Elaprase will be evaluated for total infusion time.
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Baseline to year 17
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Dosing Regimens of Elaprase for Missed Infusions in Participants With Hunter Syndrome
Time Frame: Baseline to year 17
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Dosing regiments of Elaprase will be evaluated for missed infusions.
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Baseline to year 17
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Dosing Regimens of Elaprase for Reason for Missed Infusions.
Time Frame: Baseline to year 17
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Dosing regiments of Elaprase will be evaluated for reason for missed infusions.
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Baseline to year 17
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Assessment of Hunter Syndrome on Health-related Quality of Life (HRQL) Using Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS)
Time Frame: Baseline to year 17
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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.
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Baseline to year 17
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Collaborators and Investigators
Sponsor
Investigators
- Study Director: Study Director, Shire
Publications and helpful links
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Estimated)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Pathologic Processes
- Metabolic Diseases
- Nervous System Diseases
- Neurologic Manifestations
- Neurobehavioral Manifestations
- Disease
- Genetic Diseases, Inborn
- Genetic Diseases, X-Linked
- Connective Tissue Diseases
- Carbohydrate Metabolism, Inborn Errors
- Metabolism, Inborn Errors
- Lysosomal Storage Diseases
- Mucinoses
- Mental Retardation, X-Linked
- Intellectual Disability
- Heredodegenerative Disorders, Nervous System
- Syndrome
- Mucopolysaccharidosis II
- Mucopolysaccharidoses
Other Study ID Numbers
- HOS
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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