This study examined a lymphocyte gene therapy in which the iduronate-2-sulfatase gene was transferred into the patient's lymphocytes using a retroviral method, in people with mucopolysaccharidosis II (mild Hunter syndrome). Patients whose mild Hunter syndrome was confirmed by clinical, biochemical, and genetic findings could take part. The study was carried out in Phase 1/Phase 2 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.
PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Mucopolysaccharidosis II (mild Hunter syndrome) as defined by the following: * Characteristic coarse facial features, hepatosplenomegaly, and radiographic evidence of dysostosis multiplex * Elevated urinary excretion of glycosaminoglycans in 3 urine specimens * Deficient iduronate-2-sulfatase enzyme activity as measured in plasma and leukocytes * Mutation consistent with mild Hunter syndrome must have either: A single base substitution of the coding sequence not previously associated with severe Hunter syndrome phenotype OR An exon-skipping mutation that would allow for occasional production of (minimal amounts of) normal protein --Patient Characteristics-- Cardiovascular: No severe cardiac disease Pulmonary: No severe respiratory disease Other: * Must have IQ score of 80 or higher * Effective contraception required of all fertile patients