A Phase I / 2, Multicenter, Open-label, Single-dose, Dose-ranging Study to Assess the Safety and Tolerability of SB-913, a rAAV2/6-based Gene Transfer in Subjects With Mucopolysaccharidosis II (MPS II)

Who is this study for? Patients with mucopolysaccharidosis II
What treatments are being studied? SB-913
Status: Terminated
Location: See all (5) locations...
Intervention Type: Biological
Study Type: Interventional
Study Phase: Phase 1/Phase 2
SUMMARY

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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 5
Maximum Age: 65
Healthy Volunteers: No
View:

• Male or female 5 years to 65 years of age.

• Clinical diagnosis of MPS II (based on evidence of hepatosplenomegaly, dysostosis multiplex by X-ray, valvular heart disease, or obstructive airway disease) IDS deficiency confirmed by gene sequencing.

Locations
United States
California
UCSF Benioff Children's Hospital Oakland
Oakland
Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago
North Carolina
University of North Carolina
Chapel Hill
New York
NYU School of Medicine, Neurogenetics Division
New York
Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati
Time Frame
Start Date: May 11, 2017
Completion Date: May 7, 2021
Participants
Target number of participants: 9
Treatments
Experimental: Cohort 1 SB-913 Starting Dose 5.00E+12 vg/kg
A single dose of each of the three components of SB-913 [zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)] administered via intravenous (IV) infusion.
Experimental: Cohort 2 SB-913 at Next Ascending Dose 1.00E+13 vg/kg
A single dose of each of the three components of SB-913 [zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)] administered via intravenous (IV) infusion.
Experimental: Cohort 3 SB-913 at Next Ascending Dose 5.00E+13 vg/kg
A single dose of each of the three components of SB-913 [zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)] administered via intravenous (IV) infusion.
Sponsors
Leads: Sangamo Therapeutics

This content was sourced from clinicaltrials.gov

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