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



Status:Recruiting
Conditions:Metabolic, Metabolic
Therapuetic Areas:Pharmacology / Toxicology
Healthy:No
Age Range:5 - Any
Updated:1/31/2019
Start Date:May 11, 2017
End Date:February 2022
Contact:Medical Monitor
Email:clinicaltrials@sangamo.com
Phone:send email

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A Phase I, 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)

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.

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.

Inclusion Criteria:

- Male or female ≥ 5 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.

Exclusion Criteria:

- Known to be unresponsive to ERT

- Neutralizing antibodies to AAV 2/6

- Serious intercurrent illness or clinically significant organic disease (unless
secondary to MPS II)

- Receiving antiviral therapy for hepatitis B or C, or with active hepatitis B or
hepatitis C or HIV 1/2

- Lack of tolerance to idursulfase treatment with significant IARs or occurrence of
anaphylaxis

- Markers of hepatic dysfunction

- Creatinine ≥ 1.5 mg/dL

- Contraindication to the use of corticosteroids for immunosuppression

- Current treatment with systemic (IV or oral) immunomodulatory agent or steroid use
(topical treatment allowed)

- Participation in prior investigational drug or medical device study within the
previous 3 months

- Prior treatment with a gene therapy product

- Elevated or abnormal circulating α-fetoprotein (AFP)

- Weight < 20 kg at Screening Visit
We found this trial at
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sites
South 34th Street
Philadelphia, Pennsylvania 19104
 215-590-1000
Principal Investigator: Can Ficicioglu, MD
Phone: 267-426-1368
Children's Hospital of Philadelphia Since its start in 1855 as the nation's first hospital devoted...
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3333 Burnet Avenue # Mlc3008
Cincinnati, Ohio 45229
 1-513-636-4200 
Principal Investigator: Carlos E Prada, MD
Phone: 513-636-4507
Cincinnati Children's Hospital Medical Center Patients and families from across the region and around the...
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Minneapolis, Minnesota 55455
(612) 625-5000
Principal Investigator: Chester Whitley, MD, PhD
Phone: 612-672-5151
Univ of Minnesota With a flagship campus in the heart of the Twin Cities, and...
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Chapel Hill, North Carolina 27599
(919) 962-2211
Principal Investigator: Joseph Muenzer, MD
Phone: 919-843-5731
Univ of North Carolina Carolina’s vibrant people and programs attest to the University’s long-standing place...
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225 E Chicago Ave
Chicago, Illinois 60611
(312) 227-4000
Principal Investigator: Barbara Burton, MD
Phone: 312-227-6764
Ann & Robert H. Lurie Children's Hospital of Chicago Ann & Robert H. Lurie Children
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160 East 34th Street
New York, New York 10016
Principal Investigator: Heather A. Lau, M.D.
Phone: 212-263-0139
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Oakland, California 94609
Principal Investigator: Paul Harmatz, MD
Phone: 510-428-3885
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