Hunter Outcome Survey (HOS)

Hunter Syndrome Clinical Trial

— HOS  

Official title:

Hunter Outcome Survey: A Global, Multi-Center, Long-Term, Observational Registry of Patients With Hunter Syndrome (Mucopolysaccharidosis Type II, MPS II)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03292887
• Other study ID #: HOS
• Status: Completed
• Start date: October 3, 2005
• Est. completion date: February 16, 2023

Study information

• Verified date: February 2024
• Source: Takeda
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 1332
• Est. completion date: February 16, 2023
• Est. primary completion date: February 16, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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 Participants may re-enroll once they have completed or withdrawn from the other clinical study.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.

Related Conditions & MeSH terms

• Hunter Syndrome
• Mucopolysaccharidoses
• Mucopolysaccharidosis II
• Syndrome

Locations

Country	Name	City	State
United States	Shire	Lexington	Massachusetts

Sponsors (1)

Lead Sponsor	Collaborator
Shire

Country where clinical trial is conducted

United States, 

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

External Links

•   To obtain more information on the study, click here/on this link