MPS II (Hunter Syndrome) Outcome Survey – The HOS Study

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

Protocol Description

The Hunter Syndrome Outcome Survey (HOS) is an ongoing observational, international, multi-center, long-term database of patients with Hunter syndrome. HOS collects information from patients about the course of their disease, their medical management, and the use of current or past medications or treatments, if any. The principal aim of HOS is to use the data collected to better understand the progression of the disease, its long-term response to treatment, and possibly improve how patients are cared for.

Eligibility Criteria

Patients of any age or gender with Hunter syndrome may participate in the study.
Males and Females: All ages


No experimental intervention is involved. Patients undergo clinical assessments and receive care as determined by the patients' treating physician, who is also responsible for recording relevant data into the HOS database. Participants may be asked to complete a Health-Related Quality of Life Questionnaire called the HS-FOCUS to assess changes in their physical functioning and quality of life.
Visits: None beyond normal clinical care
Duration: Ongoing

Status: Open to Enrollment

Source(s) of Support

Shire Human Genetic Therapies

Primary Investigator

Damara Ortiz, MD

Contact Information

For more information about the study or enrollment, please contact:
Nadene Henderson, MS, LCGC