Hunter Outcome Survey (HOS)

Shire

Status: Completed

Conditions: Hunter Syndrome

Study type: Observational

Funder types: Industry

Identifiers

NCT03292887

HOS

Details and patient eligibility

About

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.

Enrollment: 1,443 patients

Sex: All

Volunteers: No Healthy Volunteers

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 Participants may re-enroll once they have completed or withdrawn from the other clinical study.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

Trial design

1,443 participants in 2 patient groups

Elaprase Treated

Description:

Participants with Hunters Syndrome received or receiving treatment with Elaprase as prescribed by their physician following locally approved prescribing information.

Elaprase Non-Treated

Description:

Participants received no treatment for Hunters Syndrome.