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Hemophilia A (Severe) - Healing Genes
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Hemophilia A (Severe)

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Dose-Ranging Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 in Subjects With Severe Hemophilia A

A Phase 1/2, Open-Label, Adaptive, Dose-Ranging Study to Assess the Safety and Tolerability of SB-525 (Recombinant AAV2/6 Human Factor 8 Gene Therapy) in Adult Subjects With Severe Hemophilia A


Phase 1 / 2

DESCRIPTION:
Doctors at five research hospitals seek male patients with severe Hemophilia A (with Factor VIII Inhibitors) to trial a new product, SB-525, an investigatory gene therapy. This therapy utilizes a harmless virus to deliver a functional copy of the factor VIII (F8) gene to the liver, which may permanently provide sufficient blood coagulation and reduce the need for transfusions.

The treatment requires a single intravenous injection of SB-525 and follow up will involve lab assessments and check-ins over the following year and for up to 3 years.


PATIENT MUST:

  • Be male and 18 years or older
  • Have congenital severe or moderately severe hemophilia A
  • Not have participated in a gene transfer trial within the last 52 weeks or in a clinical trial with an investigational product within the last 12 weeks

THE STUDY INVOLVES:

  1. Screening before the treatment
  2. A single infusion of the gene therapy drug will be administered
  3. Monitoring of the patient for a few hours after dosing, with periodic lab assessments required for 3 years.

LOCATIONS AND CONTACTS:
Trials will take place at 13 locations across the United States. See the list of locations.
 
Contact: Medical Monitor  |  510.307.7266  |  [email protected]
 
SPONSOR INFORMATION:
Sangamo Therapeutics
 
Or go online:
https://clinicaltrials.gov/ct2/show/NCT03061201

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