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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 weeks3 visits

Treatment ~ Varies

Follow Up ~ at multiple timepoints from pre-dose through up to 5 years post-dose

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

0 / 3Eligibility criteria met

Find a Location

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

1,207 Previous Clinical Trials

489,650 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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How widespread is this clinical trial's implementation?

"Presently, there are 15 active clinical trial sites participating in this project. These locations range from Houston to Seattle and many other cities besides. To reduce the need for extensive travel, it is advisable for those considering enrollment to select a clinic close by."

Answered by AI

Are fresh participants being accepted for this research endeavor?

"Data displayed on clinicaltrials.gov reveals that this particular medical experiment is not currently enrolling patients, despite being initially posted in February 2013 and updated as recently as September 2022. However, there are 89 other trials actively recruiting participants at the present moment."

Answered by AI

Is it possible to join this investigation?

"The requisite qualifications for this trial necessitates that applicants must possess hemophilia b and be aged between 18-75. As of now, 30 participants are being accepted."

Answered by AI