Clinical Trial: An Observational Study of Pediatric Subjects With Globoid Cell Leukodystrophy (GLD)
Study Status: Withdrawn
Recruit Status: Withdrawn
Study Type: Observational
Official Title: A Multicenter, Prospective, Longitudinal, Observational Study of Pediatric Subjects With Globoid Cell Leukodystrophy (Krabbe Disease)
Brief Summary: The objective of this study is to evaluate the natural history of disease progression in infants with globoid cell leukodystrophy (GLD).
Detailed Summary: This study is being conducted to gather prospective data on disease progression in infants diagnosed with GLD. This study will be performed using protocol-defined, standardized assessments including clinical, developmental, and neurologic measures. All study visits will be conducted in the subject's home. No travel to the study site is necessary.
Sponsor: Shire
Current Primary Outcome:
- To measure the change from baseline in growth parameters (eg, weight gain, linear growth, head circumference). [ Time Frame: 1 year ]
- To determine the onset date of inadequate oral nutrition, hydration, and/or ventilation as a biomarker for survival [ Time Frame: 1 year ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- To assess the change from baseline in clinical parameters of GLD disease progression from a standardized infant neurological examination and infant distress scales. [ Time Frame: 1 year ]
- To assess the change from baseline in clinical parameters described in Hagberg's clinical staging. [ Time Frame: 1 year ]
- To measure the time to absolute survival [ Time Frame: 1 year ]
- To assess the AE experience in this patient population [ Time Frame: 1 year ]
Original Secondary Outcome: Same as current
Information By: Shire
Dates:
Date Received: March 24, 2010
Date Started:
Date Completion:
Last Updated: February 19, 2014
Last Verified: February 2014
