Phase I/II Study of Retroviral-Mediated Transfer of Iduronate-2-Sulfatase Gene Into Lymphocytes of Patients With Mucopolysaccharidosis II (Mild Hunter Syndrome)

Completed · Phase 1/Phase 2

Conditions studied: Mucopolysaccharidosis II

In brief

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.

Key facts

Study ID

NCT00004454

Run by

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

People needed

2

Starts

1996-10-01

Last updated by the study team

2005-06-24

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

Where it is running

• University of Minnesota Medical School — Minneapolis, Minnesota, United States

Full record on ClinicalTrials.gov

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