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Clinical Trials in the USA / NCT05911763
Active, not recruiting Observational

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

NCT05911763 · tracked via the Priya Life Science USA tracker
Sponsor
Sanofi
Phase
Observational
Started
2023-06-30
Last updated
2026-09-29

Condition(s) studied

Hemophilia A

Investigational drug(s) / intervention(s)

Efanesoctocog Alfa BIVV001 →

Efanesoctocog Alfa BIVV001: Given per investigator's discretion

Study summary

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.

Eligibility

Sex
ALL
Min age
—
Max age
—
Healthy volunteers
No
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."

Primary outcome measure(s)

  • Change from baseline in Annualized joint bleeding rate (AjBR) for treated bleeds — Up to 5 years
    Data will be reported for the effectiveness of efanesoctocog alfa prophylaxis on clinical joint status over 5-years for prophylactic cohort
  • Change from baseline in Annualized joint bleeding rate (AjBR) for all (treated and untreated) bleeds — Up to 5 years
    Data will be reported for the effectiveness of efanesoctocog alfa prophylaxis on clinical joint status over 5-years for prophylactic cohort
  • Number of Target joint development, resolution and/or recurrence — Up to 5 years
    Data will be reported for the effectiveness of efanesoctocog alfa prophylaxis on clinical joint status over 5-years for prophylactic coho

Trial sites (28)

FacilityCityRegionStatus
Banner MD Anderson Cancer Center- Site Number : 8400008 Gilbert Arizona
Loma Linda University- Site Number : 8400015 Loma Linda California
Orthopedic Institute for Children- Site Number : 8400004 Los Angeles California
The Center for Inherited Blood Disorders- Site Number : 8400001 Orange California
University of Colorado Hemophilia and Thrombosis Center- Site Number : 8400019 Aurora Colorado
William A. Shands Hospital at the University of Florida- Site Number : 8400032 Gainesville Florida
Johns Hopkins All Childrens Hospital - Outpatient Care Center - PIN- Site Number : 8400025 St. Petersburg Florida
Rush University Medical Center -1725 W Harrison St- Site Number : 8400014 Chicago Illinois
The Bleeding and Clotting Disorders Institute- Site Number : 8400005 Peoria Illinois
Indiana Hemophilia and Thrombosis Center- Site Number : 8400013 Indianapolis Indiana
University Of Iowa Hospitals And Clinics- Site Number : 8400026 Iowa City Iowa
~Massachusetts General Hospital- Site Number : 8400017 Boston Massachusetts
Dana Farber and Boston Children's Hospital- Site Number : 8400031 Boston Massachusetts
University of Michigan Hospital - 1500 E Medical Center Dr- Site Number : 8400023 Ann Arbor Michigan
M Health Fairview- Masonic Cancer Clinic - Clinics- Site Number : 8400010 Minneapolis Minnesota
Mayo Clinic - PPDS- Site Number : 8400035 Rochester Minnesota
Mississippi Center For Advanced Medicine - 7731 Old Canton Rd- Site Number : 8400016 Madison Mississippi
University of Nebraska Medical Center - 985400 Nebraska Medical Center- Site Number : 8400009 Omaha Nebraska
Children's Specialty Center - Las Vegas- Site Number : 8400007 Las Vegas Nevada
Weill Cornell Medicine-New York Presbyterian Hospital- Site Number : 8400021 New York New York
Cincinnati Children's Hospital Medical Center - PIN- Site Number : 8400006 Cincinnati Ohio
Hemophilia Center of Western Pennsylvania- Site Number : 8400012 Pittsburgh Pennsylvania
Vanderbilt University Medical Center- Site Number : 8400003 Nashville Tennessee
University of Texas Southwestern Medical Center-2001 Inwood Rd- Site Number : 8400020 Dallas Texas
University of Texas Southwestern Medical Center- Site Number : 8400011 Dallas Texas
Gulf States Hemophilia and Thrombophilia Center- Site Number : 8400029 Houston Texas
Washington Institute for Coagulation- Site Number : 8400022 Seattle Washington
Blood Center of Wisconnsin- Site Number : 8400027 Milwaukee Wisconsin

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Other trials for the same condition

Evaluating Effectiveness and Long Term Safety of Damoctocog Alfa Pegol in Patients, Who Have Been Diagnosed With Hemophilia A Active, not recruiting
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A Recruiting
A Study of Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SR604 in Two Participants Groups (Part A: Healthy Participants, and Part B: Participants With Hemophilia A or Hemophilia B or Factor VII Deficiency) Recruiting
Long-Term Safety and Efficacy of Spark-Sponsored Gene Therapies in Males With Hemophilia A Active, not recruiting
A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401) Enrolling by invitation
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05911763 on ClinicalTrials.gov ↗ ← All trials in the USA