Gene Therapy for Hemophilia B

Not currently recruiting at 7 trial locations

Age: 18+

Sex: Male

Trial Phase: Phase 1

Sponsor: St. Jude Children's Research Hospital

Must be taking: Factor IX concentrates

Must not be taking: Antiviral therapy

Disqualifiers: Hepatitis, HIV, Liver dysfunction, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 1

Duration: 1 day

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to test the safety of a new gene therapy for individuals with hemophilia B, a condition where blood doesn't clot properly due to a faulty or missing Factor IX gene. The treatment, scAAV2/8-LP1-hFIXco, uses a specially designed virus to deliver a normal Factor IX gene into the body, potentially reducing the need for regular infusions. It suits adults with severe hemophilia B who experience frequent bleeding episodes and have used Factor IX products for at least ten years. Participants should not have active hepatitis, anti-Factor IX antibodies, or a history of certain infections or liver issues. As a Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this innovative therapy.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications. However, if you are on antiviral therapy for Hepatitis B or C, you may not be eligible to participate.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown that the scAAV2/8-LP1-hFIXco gene therapy for hemophilia B is promising in terms of safety. In studies, a single dose led to long-lasting production of factor IX, a protein essential for blood clotting. Importantly, no new safety issues emerged long after treatment. Another study tracked patients for over 10 years and found stable, beneficial levels of factor IX without major safety concerns. These findings suggest that the treatment is well-tolerated and has not caused significant side effects in recipients. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard of care for hemophilia B, which typically involves regular infusions of clotting factor IX, the gene therapy scAAV2/8-LP1-hFIXco introduces a new approach by delivering a functional copy of the FIX gene directly into the patient's cells. This method aims to provide a long-term solution by enabling the body to produce its own clotting factor continuously, potentially reducing or eliminating the need for frequent infusions. Researchers are excited about this treatment because it targets the root cause of the condition, offering the possibility of sustained improvement and a significant enhancement in the quality of life for patients with hemophilia B.

What evidence suggests that this gene therapy could be an effective treatment for hemophilia B?

Research has shown that a new gene therapy, scAAV2/8-LP1-hFIXco, could help treat hemophilia B. In this trial, all participants will receive this gene therapy. Studies have found that just one treatment can lead to long-lasting production of factor IX, a protein needed for blood clotting. Specifically, patients have maintained stable levels of this protein for over ten years, resulting in fewer bleeding episodes and noticeable improvements in daily life. Early research also indicates no major safety concerns over the years. These findings suggest that this treatment could be an effective long-term solution for hemophilia B. ^{1 3 4 5 6}

Who Is on the Research Team?

Ulrike Reiss, MD

Principal Investigator

St. Jude Children's Research Hospital

Are You a Good Fit for This Trial?

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.

Inclusion Criteria

Able to give informed consent and comply with requirements of the trial

My family does not have a history of developing inhibitors.

I agree to use barrier contraception until 3 tests show no vector genomes.

Exclusion Criteria

Subjects who are unwilling to provide the required semen samples

Creatinine ≥ 1.5 mg/dl

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

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a gene transfer using a self-complementary AAV vector to deliver the normal factor IX gene

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after gene transfer

15 years

What Are the Treatments Tested in This Trial?

Interventions

• scAAV2/8-LP1-hFIXco

Trial Overview 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.

How Is the Trial Designed?

Group I: Group 1 Experimental Treatment 2 Interventions

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

Who Is Running the Clinical Trial?

St. Jude Children's Research Hospital

Lead Sponsor

Trials

451

Recruited

5,326,000+

Children's Hospital of Philadelphia

Collaborator

Trials

749

Recruited

11,400,000+

University College, London

Collaborator

Trials

884

Recruited

38,770,000+

Hemophilia of Georgia, Inc.

Collaborator

Trials

2

Recruited

3,000+

National Heart, Lung, and Blood Institute (NHLBI)

Collaborator

Trials

3,987

Recruited

47,860,000+

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40499172/

Sustained Clinical Benefit of AAV Gene Therapy in Severe ... A single administration of scAAV2/8-LP1-hFIXco gene therapy resulted in durable factor IX expression, sustained clinical benefit, and no late-onset safety ...

2. ashpublications.org ashpublications.org/blood/article/142/Supplement%201/1056/502498/Stable-Therapeutic-Transgenic-FIX-Levels-for-More

Stable Therapeutic Transgenic FIX Levels for More Than 10 ... Stable therapeutic transgenic FIX levels for more than 10 years in subjects with severe hemophilia B who received scAAV2/8-LP1-Hfixco adeno-associated virus ...

3. hematologyadvisor.com hematologyadvisor.com/news/hemophilia-gene-therapy-long-term-efficacy-safety-treatment-risk/

Hemophilia B Gene Therapy Demonstrates Long-Term ... A 13-year follow-up study has demonstrated that a single administration of gene therapy for hemophilia B resulted in durable factor IX expression, sustained ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT00979238

NCT00979238 | Dose-Escalation Study Of A Self ... The purpose of this study is to determine the safety of giving a normal factor IX gene to treat individuals who have an abnormal or no factor IX gene.

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC7617823/

Sustained clinical benefit of AAV gene therapy in severe ... Here, we present safety and efficacy data from ten men with severe hemophilia B treated with a single bolus infusion of scAAV2/8-LP1-hFIXco, ...

6. nejm.org nejm.org/doi/abs/10.1056/NEJMoa2414783

Sustained Clinical Benefit of AAV Gene Therapy in Severe ... A single administration of scAAV2/8-LP1-hFIXco gene therapy resulted in durable factor IX expression, sustained clinical benefit, and no late-onset safety ...