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

Study Status: Completed
Recruit Status: Completed
Study Type: Observational

Official Title: A Prospective, Longitudinal, Observational Study to Evaluate Neurodevelopmental Status in Pediatric Patients With Hunter Syndrome (MPS II)

Brief 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.

Detailed Summary:
Sponsor: Shire

Current Primary Outcome:

  • Neurodevelopmental parameters of cognitive function over time in pediatric patients with MPS II [ Time Frame: 24 months ]
  • Neurodevelopmental parameters of adaptive function over time in pediatric patients with MPS II [ Time Frame: 24 months ]


Original Primary Outcome:

  • Neurodevelopmental parameters of cognitive function over time in pediatric patients with MPS II [ Time Frame: 24 months ]
  • Neurodevelopmental parameters of motor function over time in pediatric patients with MPS II [ Time Frame: 24 months ]
  • Neurodevelopmental parameters of behavioral function over time in pediatric patients with MPS II [ Time Frame: 24 months ]
  • Neurodevelopmental parameters of adaptive function over time in pediatric patients with MPS II [ Time Frame: 24 months ]


Current Secondary Outcome:

  • Reported adverse events [ Time Frame: 24 months ]
    Type and severity measurements
  • Medication usage [ Time Frame: 24 months ]
  • Quality of life [ Time Frame: 24 months ]


Original Secondary Outcome: Same as current

Information By: Shire

Dates:
Date Received: March 12, 2013
Date Started: January 2013
Date Completion:
Last Updated: October 19, 2016
Last Verified: October 2016