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.
About this trial
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.
Gene therapy for hemophilia
St. Jude has been a pioneer in gene therapy since 1997. Recently, our doctors helped develop a successful gene transfer treatment that improved the quality of life for adult patients with severe hemophilia B.
Gene therapy provides life-changing relief from hemophilia
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.
Read the research highlight >
"One of the beauties of the gene therapy approach is that it corrects the abnormality at a very basic level, at the gene level, but also has the potential, after just a single intervention, to correct the disease process long-term for the patients. Just a single administration of the viral vector can lead to a long-term cure. And that is a novel, groundbreaking approach."
Andrew Davidoff, MD
Senior author, hemophilia B gene therapy study
St. Jude Children’s Research Hospital
Chair, Department of Surgery
"All participants with hemophilia B have had persistent benefit from the factor IX gene transfer with continuously measurable factor IX levels, decrease in bleeding events and less use of factor concentrates for several years now. It has been a significant change for their quality of life – an amazing experience for the whole team, participants and researchers included."
Ulrike M. Reiss, MD
St. Jude Children's Research Hospital
Director, Hemophilia Treatment Center
"The successes of our gene therapy trials at St. Jude have given children, adolescents and young adults living with hemophilia in the United States and Europe reason to hope for a better future. With this trial as a proof of concept, we mean to expand this hope to reach every child with any genetic disorder who may benefit from gene therapy in the future, regardless of where they were born or receive treatment."
Carlos Rodriguez-Galindo, MD
St. Jude Children's Research Hospital
Director, St. Jude Global
Hemophilia Treatment Team at St. Jude
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Bleeding disorders, thrombosis, bone marrow failure
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Member, St. Jude Faculty
Chair, Surgery Department
Director, Surgical Research -
Assistant Member, St. Jude Faculty
Director, Sub-Saharan Africa Region, St. Jude Global
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Ulrike M. Reiss, MD
Associate Member, St. Jude Faculty
Department of Hematology
St. Jude Children Research Hospital