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

About this study

This study will test the safety of a new study drug in patients with hemophilia A. This is an unproven treatment, and it will be the first time the drug has been used in humans.

Hemophilia A is a blood disorder that is found mostly in men. It is also known as factor VIII or factor 8 deficiency. Hemophilia A is a genetic disorder, which means it can be inherited. People with hemophilia A do not make enough factor VIII in their blood. Factor VIII helps the blood clot, or stop bleeding. People with hemophilia A bruise easily and often bleed into their joints and soft tissues.

Standard treatment for hemophilia A is regular intravenous (through a vein) infusions of factor VIII protein concentrate to help the blood clot normally.

In this study, doctors will use a new procedure to help you make your own factor VIII. They will take a factor VIII gene and carry it to the liver using a vector. A vector is a virus that has been inactivated and can no longer cause an infection. This type of treatment is called gene therapy.

Eligibility overview

  • Male
  • At least 18 years old
  • Severe hemophilia A

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Overview

Full title:

Gene Therapy for Hemophilia A Using a Novel Serotype 8 Capsid Pseudotyped Adeno-Associated Viral Vector Encoding Factor VIII-V3

Study goal:

The main goal of this study is to test the safety and effectiveness of the study drug at different doses to find out what effects, if any, it has on people.

Diagnosis:

Hemophilia A

Age:

Males at least 18 years old

Clinical trials categories:

Blood Disorders Hemophilia

For physicians and researchers

Patients accepted to St. Jude must be referred by a physician or other qualified medical professional. Learn how St. Jude can partner with you to care for your patient.

 

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