Gene Therapy for Hemophilia B

Palo Alto (17 mi)

Age: 18+

Sex: Male

Travel: May be covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: Baxalta now part of Shire

No Placebo Group

Trial Summary

What is the purpose of this trial?This trial tests a gene therapy for adults with Hemophilia B. The therapy aims to help their bodies produce a missing protein needed for blood clotting by delivering a healthy gene to their liver cells. Gene therapy for hemophilia B has shown promise in previous studies.

Eligibility Criteria

This trial is for adult males aged 18-75 with Hemophilia B, experiencing frequent bleeding episodes or requiring regular treatment. Participants must have low plasma FIX activity and no history of inhibitors to FIX protein. They should not have Hepatitis C, detectable AAV8 antibodies, significant liver inflammation, cirrhosis signs, or allergies to any FIX product.

Inclusion Criteria

I do not have an active Hepatitis C infection.

I have hemophilia B and need treatment often due to frequent bleeding.

I am a man aged between 18 and 75.

Exclusion Criteria

I have a family history or personal evidence of inhibitors to the FIX protein.

I have signs of liver problems or damage.

Treatment Details

The study tests the safety of different doses of AskBio009, a gene therapy delivered through IV aiming to treat Hemophilia B by providing a functional copy of the Factor IX gene responsible for blood clotting.

1Treatment groups

Experimental Treatment

Group I: AskBio009 Dose EscalationExperimental Treatment1 Intervention

Single Dose of a Self-Complementing Optimized Adeno-associated Virus (AAV) Serotype 8 Factor IX Gene Therapy