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

Running, not enrolling

Conditions studied: Hemophilia A

In brief

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.

Key facts

Study ID

NCT05911763

Run by

Sanofi

People needed

200

Starts

2023-06-30

Expected to finish

2030-09-17

Last updated by the study team

2025-10-02

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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."

Where it is running

• Banner MD Anderson Cancer Center- Site Number : 8400008 — Gilbert, Arizona, United States
• Loma Linda University- Site Number : 8400015 — Loma Linda, California, United States
• Orthopedic Institute for Children- Site Number : 8400004 — Los Angeles, California, United States
• The Center for Inherited Blood Disorders- Site Number : 8400001 — Orange, California, United States
• University of Colorado Hemophilia and Thrombosis Center- Site Number : 8400019 — Aurora, Colorado, United States
• William A. Shands Hospital at the University of Florida- Site Number : 8400032 — Gainesville, Florida, United States
• Johns Hopkins All Childrens Hospital - Outpatient Care Center - PIN- Site Number : 8400025 — St. Petersburg, Florida, United States
• Rush University Medical Center -1725 W Harrison St- Site Number : 8400014 — Chicago, Illinois, United States
• The Bleeding and Clotting Disorders Institute- Site Number : 8400005 — Peoria, Illinois, United States
• Indiana Hemophilia and Thrombosis Center- Site Number : 8400013 — Indianapolis, Indiana, United States
• University Of Iowa Hospitals And Clinics- Site Number : 8400026 — Iowa City, Iowa, United States
• ~Massachusetts General Hospital- Site Number : 8400017 — Boston, Massachusetts, United States
• Dana Farber and Boston Children's Hospital- Site Number : 8400031 — Boston, Massachusetts, United States
• University of Michigan Hospital - 1500 E Medical Center Dr- Site Number : 8400023 — Ann Arbor, Michigan, United States
• M Health Fairview- Masonic Cancer Clinic - Clinics- Site Number : 8400010 — Minneapolis, Minnesota, United States
• Mayo Clinic - PPDS- Site Number : 8400035 — Rochester, Minnesota, United States
• Mississippi Center For Advanced Medicine - 7731 Old Canton Rd- Site Number : 8400016 — Madison, Mississippi, United States
• University of Nebraska Medical Center - 985400 Nebraska Medical Center- Site Number : 8400009 — Omaha, Nebraska, United States
• Children's Specialty Center - Las Vegas- Site Number : 8400007 — Las Vegas, Nevada, United States
• Weill Cornell Medicine-New York Presbyterian Hospital- Site Number : 8400021 — New York, New York, United States
• Cincinnati Children's Hospital Medical Center - PIN- Site Number : 8400006 — Cincinnati, Ohio, United States
• Hemophilia Center of Western Pennsylvania- Site Number : 8400012 — Pittsburgh, Pennsylvania, United States
• Vanderbilt University Medical Center- Site Number : 8400003 — Nashville, Tennessee, United States
• University of Texas Southwestern Medical Center-2001 Inwood Rd- Site Number : 8400020 — Dallas, Texas, United States
• University of Texas Southwestern Medical Center- Site Number : 8400011 — Dallas, Texas, United States

Full record on ClinicalTrials.gov

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