Ascending Dose Study of Genome Editing by Zinc Finger Nuclease Therapeutic SB-FIX in Subjects With Severe Hemophilia B
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
Doctors at research locations in DC, IN, and TX seek male patients with Hemophilia B currently under factor IX prophylaxis treatment to trial a new therapy called SB-FIX. SB-FIX is a genome editing-drug that inserts a correct copy of the Factor 9 gene into the liver with the goal of lifelong therapeutic production of the Factor IX clotting factor.
Study participants will receive a single infusion of the investigatory gene therapy drug. Follow up will be assessing for adverse reactions and for FIX activity levels in the blood and will last for ~3 years.
- Be male and 18 years or older
- Have severe hemophilia B (native circulating FIX activity <1%, with or without cross reactive material)
- Not have received previous gene therapy treatment
- Be HIV-negative
THE STUDY INVOLVES:
- Screening before the treatment
- A single infusion of SB-FIX will be administered
- Monitoring of the patient for a few hours after dosing, then minimal follow up for the next 3 years.
LOCATIONS AND CONTACTS:
The three study site locations are in DC, Indiana, and Texas:
Georgetown University Medical Center in Washington, DC, Map.
Indiana Hemophilia and Thrombosis Center in Indianapolis, IN, Map.
University of Texas Southwestern Medical Center in Dallas, TX, Map.
Medical Monitor | [email protected]
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