A Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With Hemophilia A

NCT ID: NCT05911763

Last Updated: 2025-10-02

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Total Enrollment

200 participants

Study Classification

OBSERVATIONAL

Study Start Date

2023-06-30

Study Completion Date

2030-09-17

Brief Summary

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This is a prospective, observational, multi-center longitudinal cohort study to describe the real-world effectiveness, safety and treatment usage of efanesoctocog alfa in patients with hemophilia A treated per standard of care in the US and Japan.

Patients will be enrolled in the study after the introduction of efanesoctocog alfa in the hemophilia treatment landscape in each study country. Decision to initiate treatment with commercially available efanesoctocog alfa will be made by the treating physician independently from the decision to include patients in the study. No study medication is provided. The data related to efanesoctocog alfa effectiveness, safety and usage will be collected prospectively during routine visits (expected annual/semi-annual visits) for up to 5 years following enrollment /treatment initiation.

Detailed Description

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Conditions

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Hemophilia A

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Cohort A (Prophylactic treatment)

All participants on efanesoctocog alfa prophylactic treatment fulfilling the overall study inclusion/exclusion criteria. The prophylactic cohort will include the following sub-cohorts:

Sub-cohort A1 (Joint imaging): Participants with severe hemophilia A and joint imaging by Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) or Joint Tissue Activity and Damage Exam (JADE) protocol performed within 6 months of initiating treatment with efanesoctocog alfa or within 3 months after initiating treatment with efanesoctocog alfa available.

Sub-cohort A2 (Children with no prior joint damage):Participants with severe hemophilia A who have no prior joint damage

Efanesoctocog Alfa BIVV001

Intervention Type DRUG

Given per investigator's discretion

Cohort B (On-Demand treatment)

Participants receiving on-demand treatment with efanesoctocog alfa who fulfil the overall study inclusion/exclusion criteria

Efanesoctocog Alfa BIVV001

Intervention Type DRUG

Given per investigator's discretion

Interventions

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Efanesoctocog Alfa BIVV001

Given per investigator's discretion

Intervention Type DRUG

Other Intervention Names

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ALTUVIIIO

Eligibility Criteria

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Inclusion Criteria

* Have a diagnosis of hemophilia A
* Patients starting efanesoctocog alfa treatment as per standard of care no more than one month prior to the enrollment date, for either on demand or prophylaxis. Patients starting efanesoctocog alfa treatment for a surgery event may also be enrolled only if the treatment is prescribed at enrollment.
* Physician's decision to treat the patient with efanesoctocog alfa is made prior to and independently of participation in the study.
* Signed and dated informed consent provided by the patient, or by the patient's legally acceptable representative for patients under the legal age before any study-related activities are undertaken. Assent should be obtained for pediatric patients according to local regulations.

Exclusion Criteria

Diagnosed with other known bleeding disorder

* Participation in an investigational medicinal product trial at enrollment visit, or intake of an Investigational Medicinal Product within 3 months prior to inclusion in this study
* Current diagnosis of a FVIII inhibitor, defined as inhibitor titer ≥0.60 BU/mL

"The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial."
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Sanofi

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Clinical Sciences & Operations

Role: STUDY_DIRECTOR

Sanofi

Locations

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Banner MD Anderson Cancer Center- Site Number : 8400008

Gilbert, Arizona, United States

Site Status

Loma Linda University- Site Number : 8400015

Loma Linda, California, United States

Site Status

Orthopedic Institute for Children- Site Number : 8400004

Los Angeles, California, United States

Site Status

The Center for Inherited Blood Disorders- Site Number : 8400001

Orange, California, United States

Site Status

University of Colorado Hemophilia and Thrombosis Center- Site Number : 8400019

Aurora, Colorado, United States

Site Status

William A. Shands Hospital at the University of Florida- Site Number : 8400032

Gainesville, Florida, United States

Site Status

Johns Hopkins All Childrens Hospital - Outpatient Care Center - PIN- Site Number : 8400025

St. Petersburg, Florida, United States

Site Status

Rush University Medical Center -1725 W Harrison St- Site Number : 8400014

Chicago, Illinois, United States

Site Status

The Bleeding and Clotting Disorders Institute- Site Number : 8400005

Peoria, Illinois, United States

Site Status

Indiana Hemophilia and Thrombosis Center- Site Number : 8400013

Indianapolis, Indiana, United States

Site Status

University Of Iowa Hospitals And Clinics- Site Number : 8400026

Iowa City, Iowa, United States

Site Status

~Massachusetts General Hospital- Site Number : 8400017

Boston, Massachusetts, United States

Site Status

Dana Farber and Boston Children's Hospital- Site Number : 8400031

Boston, Massachusetts, United States

Site Status

University of Michigan Hospital - 1500 E Medical Center Dr- Site Number : 8400023

Ann Arbor, Michigan, United States

Site Status

M Health Fairview- Masonic Cancer Clinic - Clinics- Site Number : 8400010

Minneapolis, Minnesota, United States

Site Status

Mayo Clinic - PPDS- Site Number : 8400035

Rochester, Minnesota, United States

Site Status

Mississippi Center For Advanced Medicine - 7731 Old Canton Rd- Site Number : 8400016

Madison, Mississippi, United States

Site Status

University of Nebraska Medical Center - 985400 Nebraska Medical Center- Site Number : 8400009

Omaha, Nebraska, United States

Site Status

Children's Specialty Center - Las Vegas- Site Number : 8400007

Las Vegas, Nevada, United States

Site Status

Weill Cornell Medicine-New York Presbyterian Hospital- Site Number : 8400021

New York, New York, United States

Site Status

Cincinnati Children's Hospital Medical Center - PIN- Site Number : 8400006

Cincinnati, Ohio, United States

Site Status

Hemophilia Center of Western Pennsylvania- Site Number : 8400012

Pittsburgh, Pennsylvania, United States

Site Status

Vanderbilt University Medical Center- Site Number : 8400003

Nashville, Tennessee, United States

Site Status

University of Texas Southwestern Medical Center-2001 Inwood Rd- Site Number : 8400020

Dallas, Texas, United States

Site Status

University of Texas Southwestern Medical Center- Site Number : 8400011

Dallas, Texas, United States

Site Status

Gulf States Hemophilia and Thrombophilia Center- Site Number : 8400029

Houston, Texas, United States

Site Status

Washington Institute for Coagulation- Site Number : 8400022

Seattle, Washington, United States

Site Status

Blood Center of Wisconnsin- Site Number : 8400027

Milwaukee, Wisconsin, United States

Site Status

Countries

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United States

Other Identifiers

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U1111-1281-8840

Identifier Type: REGISTRY

Identifier Source: secondary_id

OBS17523

Identifier Type: -

Identifier Source: org_study_id

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