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| Sponsor: | Shire Human Genetic Therapies, Inc. |
|---|---|
| Collaborators: |
PharmaNet Nextrials, Inc. |
| Information provided by: | Shire Human Genetic Therapies, Inc. |
| ClinicalTrials.gov Identifier: | NCT01093105 |
Purpose
The objective of this study is to evaluate the natural history of disease progression in infants with globoid cell leukodystrophy (GLD).
| Condition |
|---|
|
Leukodystrophy, Globoid Cell |
| Study Type: | Observational |
| Study Design: | Observational Model: Cohort Time Perspective: Prospective |
| Official Title: | A Multicenter, Prospective, Longitudinal, Observational Study of Pediatric Subjects With Globoid Cell Leukodystrophy (Krabbe Disease) |
| Estimated Enrollment: | 24 |
| Study Start Date: | April 2010 |
| Estimated Study Completion Date: | September 2012 |
| Estimated Primary Completion Date: | May 2012 (Final data collection date for primary outcome measure) |
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.
Eligibility| Ages Eligible for Study: | up to 21 Months |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
| Sampling Method: | Non-Probability Sample |
Subjects with a documented diagnosis of GLD and clinical signs and symptoms consistent with that diagnosis
Inclusion Criteria:
Subjects must meet all of the following criteria to be considered eligible for this study:
The subject must have clinical signs and symptoms consistent with the diagnosis of infantile GLD including at least 2 of the following:
Exclusion Criteria: Subjects who meet any of the following criteria are not eligible for this study:
Contacts and Locations
More Information
| Responsible Party: | Lawrence Charnas, MD, PhD/Medical Director, Shire Human Genetic Therapies, Inc. |
| ClinicalTrials.gov Identifier: | NCT01093105 History of Changes |
| Other Study ID Numbers: | HGT-GLD-056 |
| Study First Received: | March 24, 2010 |
| Last Updated: | October 6, 2010 |
| Health Authority: | United States: Food and Drug Administration United States: Institutional Review Board |
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Leukodystrophy, Globoid Cell Classic Globoid Cell Leukodystrophy Early-Onset Globoid Cell Leukodystrophy Globoid Cell Leukodystrophy Globoid Cell Leukoencephalopathy Infantile Globoid Cell Leukodystrophy Late-Onset Globoid Cell Leukodystrophy Leukodystrophy, Globoid Cell, Classic Leukodystrophy, Globoid Cell, Early-Onset |
Leukodystrophy, Globoid Cell, Infantile Leukodystrophy, Globoid Cell, Late-Onset Psychosine galactosylsphingosine (psychosine) beta-galactosidase Psychosine-UDP galactosyltransferase Krabbe Disease Krabbe Leukodystrophy Krabbe's Disease Krabbe's Leukodystrophy |
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Leukodystrophy, Globoid Cell Hereditary Central Nervous System Demyelinating Diseases Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases Nervous System Diseases Sphingolipidoses Lysosomal Storage Diseases, Nervous System |
Leukoencephalopathies Demyelinating Diseases Metabolism, Inborn Errors Genetic Diseases, Inborn Lipidoses Lipid Metabolism, Inborn Errors Lysosomal Storage Diseases Metabolic Diseases Lipid Metabolism Disorders |