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Hemophilia A-Gene Transfer - Healing Genes
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Hemophilia A-Gene Transfer

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A Gene Transfer Study for Hemophilia A

Gene Transfer, Dose-Finding Safety, Tolerability, and Efficacy Study of SPK-8011 [a Recombinant Adeno-Associated Viral Vector With Human Factor VIII Gene] in Individuals With Hemophilia A


Phase 1 / 2

DESCRIPTION:
Doctors at three research hospitals seek male patients with Hemophilia A (with Factor VIII Inhibitors) to trial a new product, SPK-8011, an investigatory gene therapy. This therapy utilizes a harmless virus to deliver a functional copy of the Factor VIII (F8) gene which may provide sufficient blood coagulation and reduce need for transfusions.

The treatment requires a single outpatient intravenous injection of SPK-8016 and follow up will involve lab assessments and check-ins over the following year.


PATIENT MUST:

  • Be male and 18 years or older
  • Have congenital severe or moderately severe hemophilia A
  • Not have received gene therapy treatment in the prior year
  • 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 periodic lab assessments required for 52 weeks.

LOCATIONS AND CONTACTS:
Trials will take place at 9 locations. Find the location nearest to you.
 
Contact: Clinical Director  |  215-220-9300  |  [email protected]
 
SPONSOR INFORMATION:
Spark Therapeutics
 
Or go online:
https://clinicaltrials.gov/ct2/show/NCT03003533

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