Global Hemophilia B Gene Therapy Trial

St. Jude Children’s Research Hospital and the World Federation of Hemophilia (WFH) are collaborating on an international gene therapy clinical trial for older adolescents and adults with hemophilia B. About 75% of hemophilia B (factor IX deficiency) patients live in low- and middle-income countries (LMICs) and have very limited access to factor IX concentrate for treatment. This can lead to early disability or even death from untreated bleeding.

Our research suggests that gene therapy with a single-infusion of a factor IX gene-carrying vector can achieve long-term factor IX levels. This can prevent most, or possibly all, bleeding without the need for repeated administration of factor concentrate. The goal of this trial is to create proof-of-principle for establishing gene therapy programs in LMICs and help move this treatment option to countries that may not have the resources to develop and support this technology by themselves.

This global trial will evaluate how a gene therapy program can be established in LMICs as well as the safety, efficacy and impact of the treatment. The trial will use a unique vector designed to facilitate therapeutic factor IX levels. This vector will be manufactured at the Children’s GMP, LLC, a Good Manufacturing Practices facility on the St. Jude campus in Memphis, Tennessee in the United States.

The trial will be conducted at a limited number of LMIC sites and will be open for enrollment for patients from these sites and within the US. The trial will include an upfront education program for patients and providers, as well as technical and logistical support of the sites. Eligible participants will first receive treatment at St. Jude in Memphis before later being monitored at their local site. For subsequent patients, both vector administration and follow-up monitoring will be done at the local center in the LMIC.

Who is eligible: This trial will recruit patients who have severe hemophilia B or moderate hemophilia B with chronic hemophilic arthropathy from specific centers in LMICs and the US. Eligible patients should be experiencing chronic hemophilic arthropathy (chronic joint pain, joint destruction, and loss of range of motion). Trial preparations are ongoing. The trial is projected to begin enrolling patients late 2020.

Read more about the global trial >

Gene therapy developed at St. Jude, University College London and the Royal Free Hospital has transformed life for trial participants with severe hemophilia. The therapy provides a safe, reliable source of the blood clotting protein factor IX that has allowed some patients to adopt a more active lifestyle. The results of the study were published in 2011 and 2014 in the New England Journal of Medicine.

Access the 2014 NEJM article >

Access the 2011 NEJM article >

Andrew Davidoff, MD

Senior author, hemophilia B gene therapy study
St. Jude Children’s Research Hospital
Chair, Department of Surgery

Ulrike M. Reiss, MD

St. Jude Children's Research Hospital
Director, Hemophilia Treatment Center

Carlos Rodriguez-Galindo, MD

St. Jude Children's Research Hospital
Director, St. Jude Global