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Hunter Outcome Survey (HOS) (HOS)

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ClinicalTrials.gov Identifier: NCT03292887
Recruitment Status : Recruiting
First Posted : September 26, 2017
Last Update Posted : March 19, 2019
Sponsor:
Information provided by (Responsible Party):
Shire

Brief Summary:
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.

Condition or disease
Hunter Syndrome

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Study Type : Observational [Patient Registry]
Estimated Enrollment : 2000 participants
Observational Model: Other
Time Perspective: Prospective
Target Follow-Up Duration: 20 Years
Official Title: Hunter Outcome Survey: A Global, Multi-Center, Long-Term, Observational Registry of Patients With Hunter Syndrome (Mucopolysaccharidosis Type II, MPS II)
Actual Study Start Date : August 1, 2005
Estimated Primary Completion Date : September 1, 2022
Estimated Study Completion Date : September 1, 2022


Group/Cohort
Elaprase Treated
Participants with Hunters Syndrome received or receiving treatment with Elaprase as prescribed by their physician following locally approved prescribing information.
Elaprase Non-Treated
Participants received no treatment for Hunters Syndrome.



Primary Outcome Measures :
  1. Number of Participants With Infusion-related Reactions (IRRs) [ Time Frame: Baseline to year 17 ]
    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.

  2. Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Baseline to year 17 ]
    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.

  3. Number of Participants With Positive Antibody Response [ Time Frame: Baseline to year 17 ]
    Immunogenicity is determined by time to first positive antibody response (antibody level and isotype), antibody titer, isotype, and neutralizing antibodies.

  4. Change in Urinary Glycosaminoglycan (GAG) Levels [ Time Frame: Baseline to year 17 ]
    Change in urinary GAG levels from the start of ERT is reported.

  5. Change in Height [ Time Frame: Baseline to year 17 ]
    Change in height from the start of ERT will be reported.

  6. Change in Weight [ Time Frame: Baseline to year 17 ]
    Change in weight from the start of ERT will be reported.

  7. Change in Head Circumference and Corresponding Calculated Z-scores [ Time Frame: Baseline to year 17 ]
    Change in head circumference with the corresponding Z-scores from the start of ERT will be reported.

  8. Change in Distance Walked in the 6-minute Walk Test [ Time Frame: Baseline to year 17 ]
    Change in distance walked in 6-minute walk test from the start of ERT is reported.

  9. Left Ventricular Mass Index (LVMI) [ Time Frame: Baseline to year 17 ]
    Change in LVMI will be assessed as calculated by echocardiography.

  10. Change in Forced Expiratory Volume in 1 Second (FEV1) [ Time Frame: Baseline to year 17 ]
    Change in pulmonary function from the start of ERT will be reported as measured by forced expiratory volume in 1 second (FEV1).

  11. Change in Forced Vital Capacity (FVC) [ Time Frame: Baseline to year 17 ]
    Change in pulmonary function from the start of ERT will be reported as measured by forced vital capacity (FVC).

  12. Change in Liver and Spleen Size [ Time Frame: Baseline to year 17 ]
    Change in liver and spleen size as estimated by palpation will be reported.

  13. Prevalence of Cardiac and Pulmonary-related Hospitalizations [ Time Frame: Baseline to year 17 ]
    Prevalence of cardiac and pulmonary-related hospitalizations will be reported.

  14. Age at the Time of Death [ Time Frame: Baseline to year 17 ]
    Age at the time of death will be reported.

  15. Cause of Death [ Time Frame: Baseline to year 17 ]
    Causes of death will be reported


Secondary Outcome Measures :
  1. Natural History of Untreated Participants With Hunter Syndrome [ Time Frame: Baseline to year 17 ]
    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.

  2. Dosing Regimens of Elaprase for Prescribed Dose in Participants With Hunter Syndrome [ Time Frame: Baseline to year 17 ]
    Dosing regiments of Elaprase will be evaluated for prescribed dose.

  3. Dosing Regimens of Elaprase for Administered Dose in Participants With Hunter Syndrome [ Time Frame: Baseline to year 17 ]
    Dosing regiments of Elaprase will be evaluated for administered dose.

  4. Dosing Regimens of Elaprase for Total Infusion Time in Participants With Hunter Syndrome [ Time Frame: Baseline to year 17 ]
    Dosing regiments of Elaprase will be evaluated for total infusion time.

  5. Dosing Regimens of Elaprase for Missed Infusions in Participants With Hunter Syndrome [ Time Frame: Baseline to year 17 ]
    Dosing regiments of Elaprase will be evaluated for missed infusions.

  6. Dosing Regimens of Elaprase for Reason for Missed Infusions. [ Time Frame: Baseline to year 17 ]
    Dosing regiments of Elaprase will be evaluated for reason for missed infusions.

  7. 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 ]
    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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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
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").
Criteria

Inclusion Criteria:

  1. Diagnosis of Hunter syndrome (biochemically and/or genetically)
  2. Signed and dated written informed consent, as per either a or b below:

    1. 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

    2. Historical Participants: Signed and dated informed consent from the participant's LAR (where allowed by relevant individual country or site regulations/laws). .

Exclusion Criteria:

  1. 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.
  2. 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03292887


Contacts
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Contact: Shire Contact +1 866 842 5335 ClinicalTransparency@shire.com

Locations
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United States, Massachusetts
Shire Recruiting
Lexington, Massachusetts, United States, 02421
Contact: Central Contact       ClinicalTransparency@shire.com   
Principal Investigator: Shire PI         
Sponsors and Collaborators
Shire
Investigators
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Study Director: Study Director Shire

Additional Information:

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Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT03292887     History of Changes
Other Study ID Numbers: HOS
First Posted: September 26, 2017    Key Record Dates
Last Update Posted: March 19, 2019
Last Verified: March 2019

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Shire:
Mucopolysaccharidosis II
Hunter Syndrome
Enzyme Replacement Therapy
Lysosomal
Glycosaminoglycans

Additional relevant MeSH terms:
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Syndrome
Mucopolysaccharidosis II
Mucopolysaccharidoses
Disease
Pathologic Processes
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Genetic Diseases, X-Linked
Heredodegenerative Disorders, Nervous System