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This observational study investigates the relationship between genetic makeup and disease features in late infantile neuronal ceroid lipofuscinosis, an inherited brain disease. It enrolled children aged 2-18 with a definitive clinical and genetic diagnosis. This study had no assigned phase and has been completed.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria. 1. Definitive diagnosis of LINCL, based on clinical phenotype and genotype. 2. The subject must be between the age of 2 and 18 years. 3. The subject will not previously have participated in a gene transfer or stem cell study. 4. Parents of study participants must agree to comply in good faith with the conditions of the study, including attending all of the required baseline and follow-up assessments, and both parents or legal guardians must give consent for their child's participation. Exclusion criteria. 1. Presence of other significant medical or neurological conditions may disqualify the subject from participation in this study e.g.,malignancy, congenital heart disease, liver or renal failure. 2. Subjects without adequate control of seizures. 3. Subjects with heart disease that would be a risk for anesthesia or a history of major risk factors for hemorrhage. 4. Subjects who cannot participate in MRI studies. 5. Concurrent participation in any other FDA approved Investigational New Drug. 6. Subjects with history of prolonged bleeding or abnormal platelet function or taking aspirin.