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The objective of this study is to evaluate the natural history of disease progression in infants with globoid cell leukodystrophy (GLD).
Full description
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.
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Inclusion and exclusion criteria
Inclusion Criteria:
Subjects must meet all of the following criteria to be considered eligible for this study:
The subject has a documented diagnosis of GLD as evidenced by GALC enzyme activity or a GALC genotype that is predictive of GLD.
The subject must have clinical signs and symptoms consistent with the diagnosis of infantile GLD including at least 2 of the following:
The subject has documented onset of signs and symptoms consistent with GLD at <12 months of age and is <21 months of age at time of study entry.
The subject was born at a gestational age of 35-41 weeks.
The subject had a birth weight of ≥2 kg.
At study entry, the subject must be able to maintain oral nutrition and hydration without the use of supportive measures, defined as use of a feeding tube.
At study entry, the subject must be able to maintain ventilation without the use of invasive supportive measures, defined as use of a breathing tube.
The subject must be able, in the opinion of the Investigator, to accommodate the protocol requirements, including feasibility of study visits.
The subject's parent(s) or legal guardian must have voluntarily signed an Institutional Review Board/Independent Ethics Committee-approved informed consent form after all relevant aspects of the study have been explained and discussed with the subject's parent(s), or legal guardian.
Exclusion Criteria: Subjects who meet any of the following criteria are not eligible for this study:
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Data sourced from clinicaltrials.gov
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