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

NCT05911763 | Active Not Recruiting FDA Drug

• 2 other identifiers: OBS17523U1111-1281-8840
• Study Type: observational
• Target: 200
• Locations: 1 country
• Sites: 28

Brief 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.

Conditions

Hemophilia A

Outcome Measures

Primary Outcomes (3)

• Change from baseline in Annualized joint bleeding rate (AjBR) for treated bleeds

  Data will be reported for the effectiveness of efanesoctocog alfa prophylaxis on clinical joint status over 5-years for prophylactic cohort

  Up to 5 years

• Change from baseline in Annualized joint bleeding rate (AjBR) for all (treated and untreated) bleeds

  Data will be reported for the effectiveness of efanesoctocog alfa prophylaxis on clinical joint status over 5-years for prophylactic cohort

  Up to 5 years

• Number of Target joint development, resolution and/or recurrence

  Data will be reported for the effectiveness of efanesoctocog alfa prophylaxis on clinical joint status over 5-years for prophylactic coho

  Up to 5 years

Secondary Outcomes (34)

• Change from baseline in the Hemophilia Joint Health Score (HJHS v2.1) total/domains scores

  At 1, 2, 3, 4, 5 years

• Annual Bleeding Rate (ABR) by type for treated bleeds

  At 1, 2, 3, 4, 5 years

• ABR by type all (treated and untreated) bleeds

  At 1, 2, 3, 4, 5 years

• ABR by location for treated bleeds

  At 1, 2, 3, 4, 5 years

• ABR by location for all (treated and untreated) bleeds

  At 1, 2, 3, 4, 5 years

Study Arms (2)

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

Drug: Efanesoctocog Alfa BIVV001

Cohort B (On-Demand treatment)

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

Drug: Efanesoctocog Alfa BIVV001

Interventions

Efanesoctocog Alfa BIVV001 DRUG

Given per investigator's discretion

Also known as: ALTUVIIIO

Cohort A (Prophylactic treatment); Cohort B (On-Demand treatment)

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Approximately 200 patients from sites in the US and Japan will be enrolled in the study after the introduction of efanesoctocog alfa in the hemophilia treatment landscape in each of these two countries. To generate data for the broad real-world population of individuals with hemophilia A, eligible patients will include all ages, sexes and hemophilia A severities, whether on prophylactic or on-demand therapy or if efanesoctocog alfa is taken perioperatively.

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
• 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."

Sponsors & Collaborators

• Sanofi: lead

Study Sites (28)

Banner MD Anderson Cancer Center- Site Number : 8400008

Gilbert, Arizona, 85234-2165, United States

Location

Loma Linda University- Site Number : 8400015

Loma Linda, California, 92354-3450, United States

Location

Orthopedic Institute for Children- Site Number : 8400004

Los Angeles, California, 90007, United States

Location

The Center for Inherited Blood Disorders- Site Number : 8400001

Orange, California, 92868, United States

Location

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

Aurora, Colorado, 80045-7202, United States

Location

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

Gainesville, Florida, 32610-3003, United States

Location

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

St. Petersburg, Florida, 33701-4804, United States

Location

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

Chicago, Illinois, 60612-3863, United States

Location

The Bleeding and Clotting Disorders Institute- Site Number : 8400005

Peoria, Illinois, 61615, United States

Location

Indiana Hemophilia and Thrombosis Center- Site Number : 8400013

Indianapolis, Indiana, 46260-1920, United States

Location

University Of Iowa Hospitals And Clinics- Site Number : 8400026

Iowa City, Iowa, 52242-1009, United States

Location

~Massachusetts General Hospital- Site Number : 8400017

Boston, Massachusetts, 02114, United States

Location

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

Boston, Massachusetts, 02215-5418, United States

Location

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

Ann Arbor, Michigan, 48109-5000, United States

Location

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

Minneapolis, Minnesota, 55455-4800, United States

Location

Mayo Clinic - PPDS- Site Number : 8400035

Rochester, Minnesota, 55905-0001, United States

Location

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

Madison, Mississippi, 39110-6115, United States

Location

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

Omaha, Nebraska, 68114-4113, United States

Location

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

Las Vegas, Nevada, 89109, United States

Location

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

New York, New York, 10065-4870, United States

Location

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

Cincinnati, Ohio, 45229-3026, United States

Location

Hemophilia Center of Western Pennsylvania- Site Number : 8400012

Pittsburgh, Pennsylvania, 15213-4306, United States

Location

Vanderbilt University Medical Center- Site Number : 8400003

Nashville, Tennessee, 37232-0011, United States

Location

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

Dallas, Texas, 75390-7202, United States

Location

University of Texas Southwestern Medical Center- Site Number : 8400011

Dallas, Texas, 75390-8565, United States

Location

Gulf States Hemophilia and Thrombophilia Center- Site Number : 8400029

Houston, Texas, 77030, United States

Location

Washington Institute for Coagulation- Site Number : 8400022

Seattle, Washington, 98101-3932, United States

Location

Blood Center of Wisconnsin- Site Number : 8400027

Milwaukee, Wisconsin, 53233-2121, United States

Location

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Clinical Sciences & Operations

  Sanofi

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 12, 2023
• First Posted: June 22, 2023
• Study Start: June 30, 2023
• Primary Completion (Estimated): June 6, 2028
• Study Completion (Estimated): September 17, 2030
• Last Updated: October 2, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will share

Locations

US (28)