Ascending Dose Study of Genome Editing by Zinc Finger Nuclease Therapeutic SB-FIX in Subjects With Severe Hemophilia B



Status:Recruiting
Conditions:Hematology
Therapuetic Areas:Hematology
Healthy:No
Age Range:18 - Any
Updated:2/14/2019
Start Date:November 15, 2016
End Date:January 2021
Contact:Medical Monitor
Email:clinicaltrials@sangamo.com

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A Phase I, Open-Label, Ascending Dose Study to Assess the Safety and Tolerability of AAV2/6 Factor IX Gene Therapy Via Zinc Finger Nuclease (ZFN) Mediated Targeted Integration of SB-FIX in Adult Subjects With Severe Hemophilia B

The purpose of the study is to evaluate the safety, tolerability and effect on FIX antigen
and activity levels of ascending doses of SB-FIX. SB-FIX is an intravenously delivered Zinc
Finger Nuclease (ZFN) Therapeutic for genome editing. It inserts a correct copy of the Factor
9 gene into the albumin locus in hepatocytes with the goal of lifelong therapeutic production
of the Factor IX clotting factor.

The objective of the study is to provide long term expression of Factor IX in subjects with
severe hemophilia B. SB-FIX is a therapeutic for ZFN-mediated genome editing which will be
delivered by adeno-associated virus (AAV)-derived vectors. SB-FIX is intended to function by
placement of a corrective copy of the Factor IX 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 Factor IX for the lifetime of a
hemophilia B subject.

Inclusion Criteria:

- Male >18 years of age

- Severe hemophilia B (native circulating FIX activity <1%, with or without cross
reactive material)

Exclusion Criteria:

- Presence of neutralizing antibodies

- History of hypersensitivity response or an allergic reaction to FIX or FIX products

- Currently receiving long acting FIX replacement therapy

- FIX mutations known to be associated with FIX inhibitors

- Polymorphisms in the ZFN target region

- Presence of any liver mass on MRI, or elevated alpha-fetoprotein (AFP)

- Any contraindication to the use of corticosteroids for immunosuppression

- Currently receiving antiviral therapy for hepatitis B or C or with history or active
hepatitis B or hepatitis C or HIV-1 or HIV1/2 antibody positive.

- Chronic anemia, leukopenia, or thrombocytopenia

- Past medical history of active tuberculosis or significant fungal disease

- Symptomatic cardiovascular disease as a co-morbid condition

- Markers of hepatic inflammation or overt or occult cirrhosis

- History of chronic renal disease or creatinine ≥ 1.5 mg/dL

- Systemic (iv or oral) immunomodulatory agent or steroid use (topical treatment is
allowed)

- History of chronic infection or other chronic disorder considered an unacceptable risk

- History of malignancy except for treated basal cell or squamous cell carcinoma

- History of alcohol or substance abuse

- Previously received gene therapy product

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

- History of therapeutic non-adherence

- Any other reason that, in the opinion of the Investigator or Medical Monitor, would
render the subject unsuitable for participation in the study
We found this trial at
8
sites
Indianapolis, Indiana 46260
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1801 Inwood Rd
Dallas, Texas 75390
(214) 645-3300
University of Texas Southwestern Medical Center UT Southwestern is an academic medical center, world-renowned for...
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2799 W Grand Blvd
Detroit, Michigan 48202
(313) 916-2600
Principal Investigator: Philip Kuriakose, MD
Phone: 313-916-1784
Henry Ford Hospital Founded in 1915 by auto pioneer Henry Ford and now one of...
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Detroit, MI
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1500 East Duarte Road
Duarte, California 91010
626-256-HOPE (4673)
Principal Investigator: Nadia P Ewing, MD
City of Hope National Medical Center City of Hope is dedicated to making a difference...
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Edgbaston, Birmingham
Phone: +44 (0121) 371 6843
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403 West Adams Boulevard
Los Angeles, California 90007
Principal Investigator: Doris Quon, MD
Phone: 213-742-1402
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San Francisco, California 94115
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3800 Reservoir Rd NW
Washington, District of Columbia 20007
(202) 687-7695
Phone: 202-687-4868
Georgetown University Medical Center Georgetown University Medical Center is committed to excellence in research, education...
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