Trial Information

Summary: Gene Transfer for Patients with Fanconi Anemia, Genotype A: A Pilot Study

What is the purpose of this study?

The purpose of this study is to determine the safety and side effects of inserting the normal Fanconi anemia gene into your blood producing cells. The actual gene insertion will take place in a laboratory and the cells will then be infused back into you.

Who can participate?

Patients with Fanconi anemia, Genotype A, can participate in this study. Patients must be between 1 and 35 years of age, and weigh more than 22 pounds. A separate study and consent (Collection of Hematopoietic Cells from Patients with Fanconi Anemia for Future Autologous Reinfusion and Research) describes in greater detail the procedures used to collect your blood producing cells.

What is involved?

The normal gene for Fanconi anemia will be transferred in your blood producing cells, known as CD34+ cells using an experimental technology called "retroviral mediated gene transfer", which is done in the laboratory. These cells will then be re-infused back into you either through a vein or central line (if you have one). There is extensive follow-up for this study including blood tests and bone marrow examinations for up to 15 years.

What are the benefits?

It is possible that you may not benefit directly from this study and there will be no improvement in your bone marrow failure.

What are the risks?

The risks associated with gene transfer are extensive and include the risk of infection, immune reaction, development of cancer and death. The risks of this study will be explained in detail with you.

Will I be paid for participating in this study?

No.

If you would like to learn more about participating in this study, please send an e-mail message using the form below.