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Hunter Syndrome (MPS II) Clinical Trials

A listing of Hunter Syndrome (MPS II) medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.


Found (4) clinical trials

Safety and Dose Ranging Study of Insulin Receptor MoAb-IDS Fusion Protein in Patients With Hunter Syndrome

This is a sequential, open-label, dose escalation, multi-dose study in adults with Hunter syndrome. Two dose levels, assuming tolerability, are planned sequentially, with safety data from the previous cohort being reviewed prior to escalation to the next higher dose cohort. Subjects will receive weekly doses of AGT-182 for 8 weeks ...


Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II

The objectives of the study are to provide long term expression of IDS and improve the current clinical outcome of enzyme replacement therapy (ERT) in subjects with MPS II, a recessive lysosomal storage disorder that results from mutations in the gene encoding IDS. SB-913 is a therapeutic for ZFN-mediated genome ...


Mucopolysaccharidosis Type II Natural History

Mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, is caused by a deficiency of iduronate-2-sulfatase (IDS) leading to an accumulation of glycosaminoglycans (GAGs) in tissues of MPS II patients, resulting in characteristic storage lesions and diverse disease sequelae, and in patients with the more severe form of the ...

Phase N/A

RGX-121 Gene Therapy in Patients With MPS II (Hunter Syndrome)

MPS II is a rare X-linked recessive genetic disease caused by mutations in the iduronate-2-sulfatase (IDS) gene . Enzyme replacement therapy (ERT) with recombinant idursulfase (ELAPRASE) is the only approved product for the treatment of Hunter syndrome, however, ERT as currently administered does not cross the Blood Brain Barrier and ...