Living with

Hemophilia A?

The Alta Study is testing a potential
one-time lasting treatment

ALTA is a clinical research study that will test an investigational gene therapy called SB-525 for severe hemophilia A. Listed below are some questions and answers about the study. If you have general questions about clinical research, please visit the Clinical Research FAQ tab.

How do I qualify for this study?

You may qualify for the ALTA study if you:

  • Are a male age 18 or older
  • Have severe hemophilia A and are currently free of inhibitor
  • Have had factor VIII replacement therapy for at least 150 days

To learn more about this study opportunity, please contact clinicaltrials@sangamo.com.

What is SB-525?

The ALTA clinical research study is looking for men age 18 and over with hemophilia A to test SB-525, an investigational gene therapy. (“Investigational” means it isn’t approved by the FDA for general use by the public.)

SB-525 consists of a substance (called a vector or carrier) intended to carry a working copy of the gene for factor VIII and insert it into the DNA of your liver cells. Study participants will receive a single infusion of SB-525 into a vein. This will allow the working copy of the gene for factor VIII to travel through the bloodstream to the liver.

Doctors are studying SB-525 to see if it’s safe and tolerable, and learn more about its effectiveness at enabling your body to produce factor VIII on its own and improve blood clotting.

Click on About Gene Therapy to learn more about SB-525 and gene therapy

Will all study patients receive SB-525?

Yes. Study participants will be assigned to receive 1 of approximately 7 dose levels of SB-525. This study is open-label, which means that you and your doctor will know which dose of SB-525 you received.

Will study participation cost me anything?

No. All study medication and study-related care will be at no cost to you. You’ll also receive reimbursement for travel. Additionally, transportation assistance is available.

How long will study participation last?

After you receive the infusion of SB-525, your study staff will follow your health for up to 3 years. You’ll make regular study appointments with the study staff, and the staff will work with you to
find convenient times for these visits. At these visits, the study staff will check your health, including taking blood, urine, saliva, stool, and semen samples to study the effects of the investigational therapy. If you need to leave the study for any reason, you may do so at any time.

Do I have to stop using factor VIII concentrates?

No. You can continue receiving factor VIII concentrates during the study.

doctor testing

You may qualify for the ALTA clinical research study if you:

  • Are a male age 18 or older
  • Have severe hemophilia A and are currently free of inhibitor
  • Have had factor VIII replacement therapy for at least 150 days

People who qualify will receive:

  • Investigational gene therapy (SB-525) for severe hemophilia A
  • Study-related medical care

Think the ALTA study may be for you? Contact us.