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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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up at multiple timepoints from pre-dose through up to 5 years post-dose
Awards & highlights

Study Summary

This trial is testing a new gene therapy for hemophilia B, to see if it is safe.

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.Check my eligibility
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.See study design
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
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I do not have an active Hepatitis C infection.
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I have hemophilia B and need treatment often due to frequent bleeding.
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I am a man aged between 18 and 75.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~at multiple timepoints from pre-dose through up to 5 years post-dose
This trial's timeline: 3 weeks for screening, Varies for treatment, and at multiple timepoints from pre-dose through up to 5 years post-dose for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Change from baseline in clinical laboratory evaluations
Number of patients experiencing treatment-related adverse events by dose group
Secondary outcome measures
Changes from Baseline in FIX activity levels, FIX protein levels, and Bleeding Episode Severity & Frequency
Detection of AskBio009 genomes in blood, saliva, urine, stool, and semen
Immune Response to AskBio009

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

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Who is running the clinical trial?

Baxalta now part of ShireLead Sponsor
110 Previous Clinical Trials
9,074 Total Patients Enrolled
15 Trials studying Hemophilia B
827 Patients Enrolled for Hemophilia B
Study DirectorStudy DirectorShire
1,221 Previous Clinical Trials
499,944 Total Patients Enrolled
18 Trials studying Hemophilia B
927 Patients Enrolled for Hemophilia B

Media Library

AskBio009 (Virus Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT01687608 — Phase 1 & 2
Hemophilia B Research Study Groups: AskBio009 Dose Escalation
Hemophilia B Clinical Trial 2023: AskBio009 Highlights & Side Effects. Trial Name: NCT01687608 — Phase 1 & 2
AskBio009 (Virus Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT01687608 — Phase 1 & 2
~10 spots leftby Jan 2030
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