Last updated on November 2018

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

Brief description of study

The purpose of the study is to evaluate the safety, tolerability and effect on leukocyte and plasma Iduronate 2-Sulfatase (IDS) enzyme activity of ascending doses of SB-913. SB-913 is an intravenously delivered Zinc Finger Nuclease (ZFN) Therapeutic for genome editing. It inserts a correct copy of the IDS gene into the Albumin locus in hepatocytes with the goal of lifelong therapeutic production of the IDS enzyme.

Detailed Study Description

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 editing which will be delivered by adeno-associated virus (AAV)-derived vectors. SB-913 is intended to function by placement of the corrective copy of IDS transgene into the genome of the subject's own hepatocytes, under the control of the highly expressed endogenous albumin locus, and is expected to provide permanent, liver-specific expression of Iduronate 2-Sulfatase for the lifetime of an MPS II patient.

Clinical Study Identifier: NCT03041324

Contact Investigators or Research Sites near you

Start Over

Paul Harmatz, MD

UCSF Benioff Children's Hospital Oakland
Oakland, CA United States
  Connect »

Barbara Burton, MD

Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, IL United States
  Connect »

Chester Whitley, MD, PhD

University of Minnesota
Minneapolis, MN United States
  Connect »

Joseph Muenzer, MD

University of North Carolina
Chapel Hill, NC United States
  Connect »

Can Ficicioglu, MD

Children's Hospital of Philadelphia
Philadelphia, PA United States
  Connect »

Heather A. Lau, M.D.

NYU School of Medicine, Neurogenetics Division
New York, NY United States
  Connect »

Carlos E Prada, MD

Cincinnati Children's Hospital Medical Center
Cincinnati, OH United States
  Connect »

Recruitment Status: Open

Brief Description Eligibility Contact Research Team

Receive Emails About New Clinical Trials!

Sign up for our FREE service to receive email notifications when clinical trials are posted in the medical category of interest to you.