Presentation Details
| ATHN Transcends – Hemophilia Gene Therapy Outcomes Arm: A Long-Term Follow-Up Study on Safety and Effectiveness of Gene Therapy in People with Hemophilia Staber Janice1, Blanca Salazar2, Roberto Guillen-Gonzalez2, Michael Recht3, 4, Tammuella Chrisentery-Singleton5, Ulrike Reiss6. 1Iowa Hemophilia and Thrombosis Center University of Iowa Stead Family Children’s Hospital, Iowa City, IA, USA.2CSL Behring, King of Prussia, PA, USA.3National Bleeding Disorders Foundation, New York, NY, USA.4Yale University School of Medicine, New Haven, CT, USA.5American Thrombosis and Hemostasis Network, Rochester, NY, USA.6Hemophilia Treatment Center St.Jude’s Children’s Research Hospital, Memphis, TN, USA |
Abstract
BACKGROUND: American Thrombosis and Hemostasis Network (ATHN) Transcends (NCT04398628) assesses the safety and effectiveness of therapies for people with inherited bleeding and clotting disorders. The study arm “Hemophilia Gene Therapy Outcomes” assesses these same end points in person after receiving gene therapy. A new module within the Hemophilia Gene Therapy Outcomes Arm, the HEMGENIX® (etranacogene dezaparvovec-drlb) Module, is an observational, post-authorization, long-term, follow-up, multicenter study to investigate the short- and long-term effectiveness and safety of HEMGENIX® treatment in adults with hemophilia B. Etranacogene dezaparvovec-drlb is a somatic gene therapy product that aims to deliver a factor IX (FIX) (Padua variant) gene expression cassette to the liver of patients with hemophilia B. OBJECTIVES: Hemophilia Gene Therapy Outcomes Arm: (1) to determine the safety of adeno-associated viral vector (AAV)- or lentiviral vector (LV)-mediated factor VIII (FVIII) and factor IX (FIX) therapies used for participants with hemophilia. (2) to describe hemophilia gene therapy medication dosing regimens, the effectiveness of gene therapy treatment as measured by factor levels and bleeds, and the impact of gene therapy on health care utilization and health-related quality of life. HEMGENIX® Module: (1) to investigate the short- and long-term effectiveness profile of HEMGENIX® by following adults with hemophilia B who are treated with HEMGENIX® or are on continuous FIX prophylaxis for 15 years. (2) to investigate the short- and long-term safety profile of (HEMGENIX®) by following adults with hemophilia B treated with etranacogene dezaparvovec-drlb or are on continuous FIX prophylaxis for 15 years. METHODS: Data will be collected at time of enrollment, then every 3 months up to a year, and then every 6 months, and annually thereafter until year 15 after vector infusion. Eligibility criteria: Includes participants from trials, people considering gene therapy, and people treated with approved product.
The HEMGENIX® Module will collect data from 2 patient cohorts for analysis: (a) people with hemophilia B treated with HEMGENIX® in the US where HEMGENIX is approved for commercial use. (b) People with hemophilia B on FIX prophylaxis and enrolled in the ATHN Transcends study. Enrollment in the HEMGENIX Module will be 5 years after receiving HEMGENIX treatment and providing study consent. The total study duration is 20 years. Additional modules may be added in the future. Modules offer the mechanism to collect post-marketing required evaluations on FDA-approved gene therapy products. ATHN Transcends has received central IRB approval and is currently being rolled out across participating American Thrombosis and Hemostasis Network (ATHN) Affiliates in the US. Enrollment is now open. CONCLUSION: ATHN Transcends: Hemophilia Gene Therapy Outcomes Arm is the only comprehensive follow-up registry study in the US for people with hemophilia considering or after having received gene therapy. Long-term follow-up after gene therapy is critical to confirm short-term findings from trials and identify the unknowns.
No part of this publication may be reproduced, distributed, or transmitted in any form or by any means, including photocopying, recording, or other electronic or mechanical methods, without the prior written permission of the author.
The HEMGENIX® Module will collect data from 2 patient cohorts for analysis: (a) people with hemophilia B treated with HEMGENIX® in the US where HEMGENIX is approved for commercial use. (b) People with hemophilia B on FIX prophylaxis and enrolled in the ATHN Transcends study. Enrollment in the HEMGENIX Module will be 5 years after receiving HEMGENIX treatment and providing study consent. The total study duration is 20 years. Additional modules may be added in the future. Modules offer the mechanism to collect post-marketing required evaluations on FDA-approved gene therapy products. ATHN Transcends has received central IRB approval and is currently being rolled out across participating American Thrombosis and Hemostasis Network (ATHN) Affiliates in the US. Enrollment is now open. CONCLUSION: ATHN Transcends: Hemophilia Gene Therapy Outcomes Arm is the only comprehensive follow-up registry study in the US for people with hemophilia considering or after having received gene therapy. Long-term follow-up after gene therapy is critical to confirm short-term findings from trials and identify the unknowns.
No part of this publication may be reproduced, distributed, or transmitted in any form or by any means, including photocopying, recording, or other electronic or mechanical methods, without the prior written permission of the author.
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