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Hemophilia B, Severe - Healing Genes
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Hemophilia B, Severe

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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


Phase 1

DESCRIPTION:

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.


PATIENT MUST:

  • 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:

  1. Screening before the treatment
  2. A single infusion of SB-FIX will be administered
  3. 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.
 
Contact:
Medical Monitor  |  [email protected]
 
SPONSOR INFORMATION:
Sangamo Therapeutics
 
Or go online:
https://clinicaltrials.gov/ct2/show/NCT02695160

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