Phase I/II Study of Retroviral-Mediated Transfer of Iduronate-2-Sulfatase Gene Into Lymphocytes of Patients With Mucopolysaccharidosis II (Mild Hunter Syndrome)
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|ClinicalTrials.gov Identifier: NCT00004454|
Recruitment Status : Completed
First Posted : October 19, 1999
Last Update Posted : June 24, 2005
OBJECTIVES: I. Evaluate the safety and feasibility of treating mucopolysaccharidosis II (mild Hunter syndrome) by lymphocyte gene therapy.
II. Determine the levels of iduronate-2-sulfatase enzyme in these patients attained by infusing increasing doses of lymphocytes transduced with a retroviral vector designed for insertion and expression of this iduronate-2-sulfatase gene (L2SN).
III. Determine the duration of survival of these transduced cells in these patients.
IV. Determine whether monthly infusion of L2SN-transduced lymphocytes accomplishes metabolic correction (as measured by glycosaminoglycan excretion), decrease in liver or spleen volume, any therapeutic effect upon cardiac and pulmonary dysfunction, or any other effects from treatment.
|Condition or disease||Intervention/treatment||Phase|
|Mucopolysaccharidosis II||Genetic: lymphocyte gene therapy||Phase 1 Phase 2|
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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||2 participants|
|Study Start Date :||October 1996|
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00004454
|United States, Minnesota|
|University of Minnesota Medical School|
|Minneapolis, Minnesota, United States, 55455|
|Study Chair:||Chester B. Whitley||University of Minnesota - Clinical and Translational Science Institute|