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
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.
- 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:
- Screening before the treatment
- A single infusion of the gene therapy drug will be administered.
- 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]
Or go online: