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Hemophilia A - Healing Genes
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Hemophilia A

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Gene Therapy for Haemophilia A. (GO-8)

GO-8: Gene Therapy for Haemophilia A Using a Novel Serotype 8 Capsid Pseudotyped Adeno-associated Viral Vector Encoding Factor VIII-V3


Phase 1
DESCRIPTION:

Based on success with Hemophilia B gene therapy, St. Jude Children’s Research Hospital is now exploring a similar gene therapy for Hemophilia A. They seek male patients with Hemophilia A currently under clotting factor prophylaxis treatment.

Study participants will receive a single infusion of an investigatory gene therapy drug. This new treatment utilizes a harmless virus to deliver a gene designed to help the patient’s body produce factor VIII and reduce bleeding events. The virus works to transfer the gene into the patient’s DNA and cannot cause infection. For the first year, follow up will be minimal, tracking annualized bleeding rate and use of Factor VIII.


PATIENT MUST:

  • Be male and 18 years or older
  • Have congenital severe or moderately severe hemophilia A
  • Not have received previous gene therapy treatment
  • Be HIV- negative

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 lab assessments required frequently in the first 12 weeks after the treatment. Annual reviews will be required for up to 15 years.

LOCATIONS AND CONTACTS:

Trials will take place at St. Jude’s Children’s Research Hospital in Memphis, TN. Map. Coordinating trials will also be held in the United Kingdom.

Contact: Mark Philips  |  [email protected]  | +442 078302068 ext 33768 (this is an international number)

SPONSOR INFORMATION:

University College, London

Medical Research Council

Or go online:

https://clinicaltrials.gov/ct2/show/NCT03001830

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