Enrollment Finishes Early for a Late Stage Hemophilia B Gene Therapy Clinical Trial

According to a story from Hemophilia News Today, the drug developer uniQure has completed its goals for patient enrollment ahead of time for is phase 3 clinical trial. This clinical trial is evaluating the company’s investigational gene therapy AMT-061 as a treatment for patients with hemophilia B. The results of this trial could have breakthrough implications for the treatment of this bleeding disorder. The experimental gene therapy has already earned Breakthrough Therapy designation from the US Food and Drug Administration (FDA) as well as priority medicines, or PRIME, designation from the European Medicines Agency (EMA).

About Hemophilia

Hemophilia is a genetic disorder which affects the ability of the blood to form clots, a process that is vital for stopping bleeding after a wound is sustained. The severity of symptoms can vary widely. The disorder is caused by a mutation found on the X chromosome. Symptoms include bleeding for a long time after an injury, risk of bleeding in the brain and joints, and easy bruising. Bleeding in the joints can cause permanent damage and brain bleeding can lead to headaches, decreased consciousness, and seizures. There are multiple types of hemophilia, with the most common types being type A and type B, which are distinguished by having deficiencies in different clotting factors. Treatment involves replacing the missing clotting factor. Drugs that thin the blood should be avoided. To learn more about hemophilia, click here.

AMT-061 and the Clinical Trial

AMT-061 uses an AAV5 viral delivery mechanism, which will transport a specially modified version of clotting factor IX (which is deficient in hemophilia B) which is projected to boost factor activity by as much as nine times. A total of 56 hemophilia B patients will be participating in the ongoing phase 3 trial. The study includes an observational period lasting six months in which patients are expected to continue their periodic treatment regimens. Then the patients will be dosed with AMT-061 and monitored for a period of five years.

The main endpoint will be changes to factor IX activity 26 weeks following dosing; secondary endpoints will include rate of adverse events, bleed rates, and dependence on factor IX replacement therapy. Prior study data has indicated that the therapy can boost factor activity to as much as 54 percent of normal levels.


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