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Virus Therapy

Gene Therapy for Hemophilia B

Phase 1

Waitlist Available

Led By Ulrike Reiss, MD

Research Sponsored by St. Jude Children's Research Hospital

Eligibility Criteria Checklist

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

Must have

A negative family history for the development of an inhibitor

Males ≥ 18 years of age with established severe HB (FIX:C<1u/dl)

Must not have

Hypertension with systolic BP ≥ 140 mmHg or diastolic BP ≥ 90 mmHg

Poor performance status (WHO performance status score >1)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 15 years

Awards & highlights

Summary

This trial is testing the safety of giving a normal factor IX gene to people with an abnormal or no factor IX gene, in order to treat hemophilia B.

Who is the study for?

Adult males (18+) with severe Hemophilia B, treated with FIX products for over 10 years or have had at least 50 exposure days. Candidates must average a minimum of three bleeding episodes per year requiring FIX infusions and be free of inhibitors to FIX protein. They should not have active hepatitis, HIV infections under control are eligible, and must agree to use barrier contraception.

What is being tested?

The trial is testing the safety of a gene transfer treatment using scAAV2/8-LP1-hFIXco vector in adults with Hemophilia B. It aims to provide a normal factor IX gene to those who lack it due to genetic mutations causing their condition.

What are the potential side effects?

Potential side effects may include immune reactions against the new gene or viral vector, liver enzyme elevations indicating liver stress or damage, and general symptoms like fatigue or fever as the body responds to the treatment.

Eligibility Criteria

Inclusion Criteria

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

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My family does not have a history of developing inhibitors.

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I am a man over 18 with severe Hemophilia B.

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I have used Factor IX products for over 10 years or on 50 different days.

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I need regular infusions due to bleeding episodes.

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I have never had inhibitors to FIX protein.

Exclusion Criteria

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

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My blood pressure is high, at or above 140/90 mmHg.

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My health limits my daily activities significantly.

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I am on antiviral therapy for Hepatitis B or C.

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My liver isn't working properly.

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I have coronary artery disease.

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I have had tuberculosis, fungal disease, or another long-term infection.

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My liver ultrasound showed possible signs of cancer.

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I have not received gene therapy in the last 6 months.

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I have lung nodules that may be cancerous.

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I do not have an active Hepatitis B or C infection.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 15 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~15 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and 15 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

To assess the safety of systemic administration of a novel self-complementary AAV vector in adults with severe hemophilia B at up to four different dosage levels.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Group 1Experimental Treatment2 Interventions

All participants who meet the eligibility requirements. Intervention: Gene Transfer and drug (scAAV2/8-LP1-hFIXco).

0 / 15Eligibility criteria met

Find a Location

Who is running the clinical trial?

Children's Hospital of PhiladelphiaOTHER

721 Previous Clinical Trials

8,604,313 Total Patients Enrolled

3 Trials studying Hemophilia B

23 Patients Enrolled for Hemophilia B

University College, LondonOTHER

857 Previous Clinical Trials

38,830,766 Total Patients Enrolled

2 Trials studying Hemophilia B

20 Patients Enrolled for Hemophilia B

St. Jude Children's Research HospitalLead Sponsor

442 Previous Clinical Trials

5,308,855 Total Patients Enrolled

2 Trials studying Hemophilia B

158 Patients Enrolled for Hemophilia B

~5 spots leftby Jun 2032

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