What side effects are associated with this type of intervention?

The most common treatments for Hemophilia B, such as recombinant factor IX products and gene therapy, generally have a good safety profile but can have some side effects. Recombinant factor IX products, like rFIX-FP and rFIX-GP, may cause allergic reactions, headaches, dizziness, and injection site reactions. Gene therapy for Hemophilia B, which involves introducing a functional Factor IX gene, can lead to immune responses against the viral vector used for gene delivery, liver enzyme elevations, and other mild to moderate adverse effects. Long-term data on gene therapy are still being collected to fully understand its safety profile.

What prior FDA approvals exist?

The FDA has approved several treatments for Hemophilia B, including recombinant factor IX products such as Idelvion (recombinant factor IX-albumin fusion) and Rebinyn (glyco-PEGylated recombinant factor IX). These treatments aim to replace the deficient factor IX in patients, thereby preventing and controlling bleeding episodes. Idelvion, approved in 2016, offers a prolonged half-life, allowing for less frequent dosing. Rebinyn, approved in 2017, also provides extended protection due to its PEGylation. These treatments are similar to the investigational Factor IX (FIX) Gene Therapy, which introduces a functional factor IX gene to correct the deficiency in Hemophilia B patients, potentially offering a long-term solution.

What other types of research exist?

Promising novel alternatives to Factor IX (FIX) Gene Therapy for Hemophilia B patients include: 1) RNA interference (RNAi) therapy targeting antithrombin, such as fitusiran, which aims to rebalance hemostasis by reducing antithrombin levels. 2) Anti-tissue factor pathway inhibitor (TFPI) monoclonal antibodies like concizumab, which enhance thrombin generation. 3) Oral gene therapy approaches using chitosan-formulated FIX mutants, which offer a non-viral delivery method for FIX. These alternatives are in various stages of clinical development and show potential for effective management of Hemophilia B.

← Back to Search

Virus Therapy

Gene Therapy for Hemophilia B

Phase 1 & 2

Waitlist Available

Research Sponsored by Baxalta now part of Shire

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Negative for active Hepatitis C virus (HCV), defined as Hepatitis C virus antibody negative and negative (undetectable) PCR test for plasma Hepatitis C virus ribonucleic acid (RNA) OR if Hepatitis C virus antibody positive must have ≥2 consecutive negative (undetectable) PCR tests for plasma HCV RNA at least 3 months apart, and negative at screening

Established hemophilia B with ≥3 hemorrhages per year requiring treatment with exogenous FIX OR use of FIX prophylaxis because of history of frequent bleeding episodes

Must not have

Family history of inhibitor to FIX protein or personal laboratory evidence of having developed inhibitors to FIX protein at any time (>0.6 Bethesda Units on any single test)

Markers of hepatic inflammation or overt or occult cirrhosis as evidenced by one or more of the following: Platelet count <175,000/μL, Albumin ≤3.5 g/dL, Total bilirubin >1.5 x ULN and direct bilirubin ≥0.5 mg/dL, Alkaline phosphatase >2.0 x ULN, ALT or AST >2.0 x ULN (except for subjects who are HIV infected), Liver biopsy in the past indicating moderate or severe fibrosis (Metavir staging of 2 or greater), History of ascites, varices, variceal hemorrhage or hepatic encephalopathy

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

No Placebo-Only Group

Summary

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.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Potential side effects are not explicitly listed but may include typical gene therapy-related reactions such as immune responses to the viral vector (like fever), potential liver enzyme elevations indicating liver stress or damage, and other infusion-related reactions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I do not have an active Hepatitis C infection.

Select...

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

Select...

I am a man aged between 18 and 75.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

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

Select...

I have signs of liver problems or damage.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

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

0 / 5Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Hemophilia B primarily include replacement therapies and gene therapies. Replacement therapies involve administering recombinant or plasma-derived Factor IX to compensate for the deficient clotting factor. Gene therapy, such as the one using adeno-associated virus serotype 8 (AAV8) to introduce a functional Factor IX gene, aims to correct the genetic defect by enabling the patient's cells to produce Factor IX internally. This is significant for Hemophilia B patients as it offers the potential for a long-term solution, reducing bleeding episodes and improving overall quality of life.