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Observational Study to Evaluate Neurodevelopmental Status in Pediatric Patients With Hunter Syndrome (MPS II)

NCT01822184 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 100

Last updated 2021-03-17

No results posted yet for this study

Summary

Hunter syndrome (Mucopolysaccharidosis II, \[MPS II\]) is a rare, genetically linked lysosomal storage disease (LSD) caused by deficiency of the enzyme, iduronate-2-sulfatase (I2S). Most MPS II patients will present with some degree of neurodevelopmental involvement, ranging from severe cognitive impairment and behavioral problems to mildly impaired cognition. This is an observational study; no investigational treatment will be administered. The primary objective of this study is to evaluate the neurodevelopmental status of pediatric patients with MPS II over time and to gain information to guide future treatment studies in this patient population.

Conditions

Sponsors & Collaborators

  • Shire

    lead INDUSTRY

Principal Investigators

  • Study Director · Takeda

Eligibility

Min Age
2 Years
Max Age
18 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2013-01-18
Primary Completion
2016-10-05
Completion
2016-10-05

Countries

  • United States
  • Argentina
  • Mexico
  • Spain
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01822184 on ClinicalTrials.gov