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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)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 15 years
Awards & highlights

Study 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.Check my eligibility
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.See study design
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.

Timeline

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
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).

Find a Location

Who is running the clinical trial?

Children's Hospital of PhiladelphiaOTHER
707 Previous Clinical Trials
8,581,277 Total Patients Enrolled
3 Trials studying Hemophilia B
23 Patients Enrolled for Hemophilia B
University College, LondonOTHER
833 Previous Clinical Trials
38,910,375 Total Patients Enrolled
2 Trials studying Hemophilia B
20 Patients Enrolled for Hemophilia B
St. Jude Children's Research HospitalLead Sponsor
427 Previous Clinical Trials
5,306,563 Total Patients Enrolled
2 Trials studying Hemophilia B
158 Patients Enrolled for Hemophilia B

Frequently Asked Questions

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

What deleterious effects have been observed in individuals undergoing this treatment?

"With limited clinical data to draw from, this treatment was rated a 1 for safety by the team at Power. This is in accordance with Phase I trial expectations as it does not contain sufficient evidence of efficacy or safety."

Answered by AI

Are there any health care facilities in Canada that are conducting this experiment?

"Admittance to this clinical trial is offered at St. Jude Children's Research Hospital in Memphis, Tennessee; Oregon Health and Science University in Portland, Oregon; Stanford Medical School in Stanford, California as well as seven other medical centres."

Answered by AI

Are there any sign-up opportunities for this research project?

"Clinicaltrials.gov reports that this medical study, which was first uploaded on February 22nd 2010 and last updated on November 17th 2022, is not currently open to recruitment of participants. Nonetheless, there are 89 other trials in need of volunteers at present."

Answered by AI
Recent research and studies
New England Journal of MedicineJournal
Long-term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B
Nathwani, Amit C., Ulreke M. Reiss, Edward G.D. Tuddenham, Cecilia Rosales, Pratima Chowdary, Jenny McIntosh, Marco Della Peruta, et al.. 2014. “Long-term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B”. New England Journal of Medicine. Massachusetts Medical Society. doi:10.1056/nejmoa1407309.
New England Journal of MedicineJournal
Adenovirus-associated Virus Vector–mediated Gene Transfer in Hemophilia B
Nathwani, Amit C., Edward G.D. Tuddenham, Savita Rangarajan, Cecilia Rosales, Jenny McIntosh, David C. Linch, Pratima Chowdary, et al.. 2011. “Adenovirus-associated Virus Vector–mediated Gene Transfer in Hemophilia B”. New England Journal of Medicine. Massachusetts Medical Society. doi:10.1056/nejmoa1108046.
New England Journal of MedicineJournal
Adenovirus-associated Virus Vector–mediated Gene Transfer in Hemophilia B
Nathwani, Amit C., Edward G.D. Tuddenham, Savita Rangarajan, Cecilia Rosales, Jenny McIntosh, David C. Linch, Pratima Chowdary, et al.. 2011. “Adenovirus-associated Virus Vector–mediated Gene Transfer in Hemophilia B”. New England Journal of Medicine. Massachusetts Medical Society. doi:10.1056/nejmoa1108046.
clinicaltrials.govStudy
Dose-Escalation Study Of A Self Complementary Adeno-Associated Viral Vector For Gene Transfer in Hemophilia B
2010. "Dose-Escalation Study Of A Self Complementary Adeno-Associated Viral Vector For Gene Transfer in Hemophilia B". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT00979238.
All > Hemophilia A > Hemophilia
~5 spots leftby Jun 2032
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