Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I



Status:Recruiting
Conditions:Metabolic, Metabolic
Therapuetic Areas:Pharmacology / Toxicology
Healthy:No
Age Range:18 - Any
Updated:6/9/2018
Start Date:May 24, 2017
End Date:January 2022
Contact:Medical Monitor
Email:clinicaltrials@sangamo.com
Phone:510-307-7266

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A Phase I, Multicenter, Open-label, Single-dose, Dose-ranging Study to Assess the Safety and Tolerability of SB-318, a rAAV2/6-based Gene Transfer in Subjects With Mucopolysaccharidosis I (MPS I)

The purpose of the study is to evaluate the safety, tolerability of ascending doses of
SB-318. SB-318 is an intravenously delivered Zinc Finger Nuclease (ZFN) Therapeutic for
genome editing. It inserts a correct copy of the α-L-iduronidase (IDUA) gene into the Albumin
locus in hepatocytes with the goal of lifelong therapeutic production of the IDUA enzyme.

The objectives of the study are to provide long term expression of IDUA and improve the
current clinical outcome of enzyme replacement therapy (ERT) or hematopoietic stem cell
transplantation (HSCT) therapy in subjects with attenuated MPS I, a recessive lysosomal
storage disorder that results from mutations in the gene encoding IDUA. SB-318 is a
therapeutic for ZFN-mediated genome editing which will be delivered by adeno-associated virus
(AAV)-derived vectors. SB-318 is intended to function by placement of the corrective copy of
the IDUA 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 iduronidase for the lifetime of an MPS I patient.

Inclusion Criteria:

- Male or female >18 years of age

- Clinical diagnosis of attenuated MPS I deficiency (Hurler-Scheie, Scheie, or Hurlers
status post-HSCT)

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 I)

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

- Lack of tolerance to laronidase 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)
We found this trial at
6
sites
201 Dowman Dr
Atlanta, Georgia 30303
(404) 727-6123
Principal Investigator: William Wilcox, M.D.
Phone: 404-778-8421
Emory University Emory University, recognized internationally for its outstanding liberal artscolleges, graduate and professional schools,...
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3333 Burnet Avenue # Mlc3008
Cincinnati, Ohio 45229
 1-513-636-4200 
Principal Investigator: Nancy Leslie, M.D.
Phone: 513-636-4507
Cincinnati Children's Hospital Medical Center Patients and families from across the region and around the...
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Cincinnati, OH
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Gainesville, Florida 32610
(352) 392-3261
Principal Investigator: Coy Heldermon, M.D., Ph.D.
Phone: 352-294-8283
University of Florida The University of Florida (UF) is a major, public, comprehensive, land-grant, research...
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Minneapolis, Minnesota 55455
(612) 625-5000
Principal Investigator: Chester B Whitley, PhD, MD
Phone: 612-625-1594
Univ of Minnesota With a flagship campus in the heart of the Twin Cities, and...
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New York, New York 10016
Principal Investigator: Heather A Lau, MD
Phone: 212-263-0139
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New York, NY
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Oakland, California 94609
Principal Investigator: Paul Harmatz, MD
Phone: 510-428-3885
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Oakland, CA
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