Presentation Details
| Design of a Prospective, Observational, Multicenter Study of the Effectiveness of Efanesoctocog Alfa on Long-term Joint Health in Patients With Hemophilia A in the United States and Japan Jonathan C.Roberts1, Spencer Sullivan2, Eric F.Grabowski3, Doris Quon4, Jennifer Dumont5, Annemieke Willemze6, Nicole Tsao5. 1Bleeding & Clotting Disorders Institute, Peoria, IL, USA.2Mississippi Center for Advanced Medicine, Madison, MS, USA.3Massachusetts General Hospital and Harvard Medical School, Boston, MA, USA.4Orthopedic Hemophilia Treatment Center, Los Angeles, CA, USA.5Sanofi, Cambridge, MA, USA.6Sanofi, Amsterdam, Netherlands |
Abstract
Background
Efanesoctocog alfa (formerly BIVV001) is a first-in-class high-sustained factor VIII (FVIII) replacement therapy, designed to decouple FVIII from endogenous von Willebrand factor, thereby extending FVIII half-life. Clinical trials have established efficacy and safety of efanesoctocog alfa but use in real-world clinical practice has not been studied.
Objectives
To investigate effectiveness, usage, patient-reported outcomes (PROs), and safety with efanesoctocog alfa treatment in routine practice to understand real-world outcomes.
Methods
This prospective, observational study (NCT05911763) in the United States and Japan, will assess prophylactic (cohort A) and on-demand (cohort B) efanesoctocog alfa treatment (Figure 1). Efanesoctocog alfa is commercially available in both countries and participants are currently being enrolled. Approximately 200 participants will be enrolled over 2 years and observed up to 5 years.
Inclusion criteria include diagnosis of hemophilia A (mild, moderate, severe), and initiation of efanesoctocog alfa ≤1 month before enrollment. Male and female patients of all ages can enroll. Participants with severe hemophilia (endogenous FVIII level <1 IU/dL [1%]) with joint imaging using Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) or Joint Tissue Activity and Damage Exam (JADE) ≤6 months before initiating treatment, or ≤3 months after initiating treatment, will enter sub-cohort A1. Participants with severe hemophilia ≤6 years old and no joint damage will enter sub-cohort A2. Participants will be excluded if diagnosed with another bleeding disorder, participating in an investigational product trial, or receiving an investigational product within ≤3 months of study inclusion, or diagnosed with an FVIII inhibitor (titer ≥0.60 BU/mL) at screening.
Results
Primary endpoints will be change from baseline in annualized joint bleed rate and target joint development and/or resolution, or recurrence (cohort A) (Figure 2). Selected secondary endpoints for all cohorts will include the number of injections and dose to treat a bleeding episode, all adverse and serious adverse events, inhibitor development, change from baseline in PRO measures, and changes in healthcare resource utilization.
In cohort A, secondary endpoints will include change from baseline in Hemophilia Joint Health Scores, annualized bleed rates, percentage of participants with zero joint bleeds, and annualized factor consumption per kg body weight.
Sub-cohort A1 endpoints will include change from baseline in HEAD-US total/domain scores or JADE musculoskeletal ultrasound and synovial hypertrophy, assessed at yearly intervals.
Percentage of bleeding episodes treated with a single injection and changes in treatment regimen will be recorded for the on-demand cohort. Perioperative use–specific outcomes will be recorded across cohorts A and B.
Conclusions
The real-world experience of participants receiving efanesoctocog alfa in this study will provide insight into the long-term clinical impact of high-sustained factor levels including joint health. This study will also provide data on participants previously underrepresented in clinical trials.
Study funded by Sanofi. Editorial assistance provided by Ella Ewins, PhD, of Fishawack Communications Ltd., part of Avalere Health; support funded by Sanofi.
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.
Efanesoctocog alfa (formerly BIVV001) is a first-in-class high-sustained factor VIII (FVIII) replacement therapy, designed to decouple FVIII from endogenous von Willebrand factor, thereby extending FVIII half-life. Clinical trials have established efficacy and safety of efanesoctocog alfa but use in real-world clinical practice has not been studied.
Objectives
To investigate effectiveness, usage, patient-reported outcomes (PROs), and safety with efanesoctocog alfa treatment in routine practice to understand real-world outcomes.
Methods
This prospective, observational study (NCT05911763) in the United States and Japan, will assess prophylactic (cohort A) and on-demand (cohort B) efanesoctocog alfa treatment (Figure 1). Efanesoctocog alfa is commercially available in both countries and participants are currently being enrolled. Approximately 200 participants will be enrolled over 2 years and observed up to 5 years.
Inclusion criteria include diagnosis of hemophilia A (mild, moderate, severe), and initiation of efanesoctocog alfa ≤1 month before enrollment. Male and female patients of all ages can enroll. Participants with severe hemophilia (endogenous FVIII level <1 IU/dL [1%]) with joint imaging using Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) or Joint Tissue Activity and Damage Exam (JADE) ≤6 months before initiating treatment, or ≤3 months after initiating treatment, will enter sub-cohort A1. Participants with severe hemophilia ≤6 years old and no joint damage will enter sub-cohort A2. Participants will be excluded if diagnosed with another bleeding disorder, participating in an investigational product trial, or receiving an investigational product within ≤3 months of study inclusion, or diagnosed with an FVIII inhibitor (titer ≥0.60 BU/mL) at screening.
Results
Primary endpoints will be change from baseline in annualized joint bleed rate and target joint development and/or resolution, or recurrence (cohort A) (Figure 2). Selected secondary endpoints for all cohorts will include the number of injections and dose to treat a bleeding episode, all adverse and serious adverse events, inhibitor development, change from baseline in PRO measures, and changes in healthcare resource utilization.
In cohort A, secondary endpoints will include change from baseline in Hemophilia Joint Health Scores, annualized bleed rates, percentage of participants with zero joint bleeds, and annualized factor consumption per kg body weight.
Sub-cohort A1 endpoints will include change from baseline in HEAD-US total/domain scores or JADE musculoskeletal ultrasound and synovial hypertrophy, assessed at yearly intervals.
Percentage of bleeding episodes treated with a single injection and changes in treatment regimen will be recorded for the on-demand cohort. Perioperative use–specific outcomes will be recorded across cohorts A and B.
Conclusions
The real-world experience of participants receiving efanesoctocog alfa in this study will provide insight into the long-term clinical impact of high-sustained factor levels including joint health. This study will also provide data on participants previously underrepresented in clinical trials.
Study funded by Sanofi. Editorial assistance provided by Ella Ewins, PhD, of Fishawack Communications Ltd., part of Avalere Health; support funded by Sanofi.
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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