Treatment for Hemophilia B

Phase-Based Progress Estimates
Hemophilia B+1 More
What conditions do you have?

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.

Eligible Conditions
  • Hemophilia B

Treatment Effectiveness

Study Objectives

1 Primary · 0 Secondary · Reporting Duration: 15 years

15 years
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 Safety

Trial Design

0 Treatment Group

14 Total Participants · 0 Treatment Group

Primary Treatment: Treatment · No Placebo Group · Phase 1

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 15 years

Who is running the clinical trial?

Children's Hospital of PhiladelphiaOTHER
644 Previous Clinical Trials
7,847,713 Total Patients Enrolled
3 Trials studying Hemophilia B
23 Patients Enrolled for Hemophilia B
St. Jude Children's Research HospitalLead Sponsor
395 Previous Clinical Trials
5,296,097 Total Patients Enrolled
1 Trials studying Hemophilia B
60 Patients Enrolled for Hemophilia B
Hemophilia of Georgia, Inc.OTHER
1 Previous Clinical Trials
3,000 Total Patients Enrolled
University College, LondonOTHER
752 Previous Clinical Trials
38,687,656 Total Patients Enrolled
2 Trials studying Hemophilia B
20 Patients Enrolled for Hemophilia B
National Heart, Lung, and Blood Institute (NHLBI)NIH
3,576 Previous Clinical Trials
46,968,567 Total Patients Enrolled
Ulrike Reiss, MDPrincipal InvestigatorSt. Jude Children's Research Hospital
3 Previous Clinical Trials
681 Total Patients Enrolled
1 Trials studying Hemophilia B
550 Patients Enrolled for Hemophilia B

Eligibility Criteria

Age 18+ · Male Participants · 7 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:
You are willing to practice a reliable barrier method of contraception until 3 consecutive samples are negative for vector genomes using our PCR assay.
You have a male with established severe HB.
You have a minimum of 3 bleeding episodes per year requiring transfusions of Factor VIII or Factor IX.
You have been treated with (or have been exposed to) products containing the factor VIII inhibitor, factor VIII.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 8th, 2021

Last Reviewed: October 20th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.