Retroviral-Mediated Transfer of Iduronate-2-Sulfatase Gene for Mild Hunter Syndrome
lymphocyte gene therapy
Carbohydrate Metabolism, Inborn Errors+20
+ Connective Tissue Diseases
+ Intellectual Disability
Treatment Study
Summary
Study start date: October 1, 1996
Actual date on which the first participant was enrolled.PROTOCOL OUTLINE: Peripheral blood lymphocytes are harvested from patient by apheresis, stimulated to initiate the growth of T-lymphocytes, transduced with retrovirus L2SN containing iduronate-2-sulfatase, and reinfused into the patient. Patients receive 12 monthly infusions of these retroviral-mediated gene transduced lymphocytes with the first three infusions in a dose escalation format. Patients are monitored for at least 2 hours after completion of each infusion. Patients are followed at 1 year after treatment, and then until death.
Protocol
This section provides details of the study plan, including how the study is designed and what the study is measuring.2 patients to be enrolled
Total number of participants that the clinical trial aims to recruit.Treatment Study
Eligibility
Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.Any sex
Biological sex of participants that are eligible to enroll.Over 18 Years
Range of ages for which participants are eligible to join.Healthy volunteers not allowed
If individuals who are healthy and do not have the condition being studied can participate.Conditions
Pathology
Criteria
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
Study Centers
These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.This study has 1 location
University of Minnesota Medical School
Minneapolis, United StatesOpen University of Minnesota Medical School in Google Maps