ACTIVE_NOT_RECRUITING

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

Conditions

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

Prospective, Observational , Multicenter Study of Effectiveness of Efanesoctocog Alfa on Long-term Joint Health in Patients With Hemophilia A

Quick Facts

Study Start:2023-06-30
Study Completion:2030-09-17
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:ACTIVE_NOT_RECRUITING

Study ID

NCT05911763

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:Not specified
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Have a diagnosis of hemophilia A
  2. * 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.
  3. * Physician's decision to treat the patient with efanesoctocog alfa is made prior to and independently of participation in the study.
  4. * 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.
  1. * 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
  2. * Current diagnosis of a FVIII inhibitor, defined as inhibitor titer ≥0.60 BU/mL

Contacts and Locations

Principal Investigator

Clinical Sciences & Operations
STUDY_DIRECTOR
Sanofi

Study Locations (Sites)

Banner MD Anderson Cancer Center- Site Number : 8400008
Gilbert, Arizona, 85234-2165
United States
Loma Linda University- Site Number : 8400015
Loma Linda, California, 92354-3450
United States
Orthopedic Institute for Children- Site Number : 8400004
Los Angeles, California, 90007
United States
The Center for Inherited Blood Disorders- Site Number : 8400001
Orange, California, 92868
United States
University of Colorado Hemophilia and Thrombosis Center- Site Number : 8400019
Aurora, Colorado, 80045-7202
United States
William A. Shands Hospital at the University of Florida- Site Number : 8400032
Gainesville, Florida, 32610-3003
United States
Johns Hopkins All Childrens Hospital - Outpatient Care Center - PIN- Site Number : 8400025
St. Petersburg, Florida, 33701-4804
United States
Rush University Medical Center -1725 W Harrison St- Site Number : 8400014
Chicago, Illinois, 60612-3863
United States
The Bleeding and Clotting Disorders Institute- Site Number : 8400005
Peoria, Illinois, 61615
United States
Indiana Hemophilia and Thrombosis Center- Site Number : 8400013
Indianapolis, Indiana, 46260-1920
United States
University Of Iowa Hospitals And Clinics- Site Number : 8400026
Iowa City, Iowa, 52242-1009
United States
~Massachusetts General Hospital- Site Number : 8400017
Boston, Massachusetts, 02114
United States
Dana Farber and Boston Children's Hospital- Site Number : 8400031
Boston, Massachusetts, 02215-5418
United States
University of Michigan Hospital - 1500 E Medical Center Dr- Site Number : 8400023
Ann Arbor, Michigan, 48109-5000
United States
M Health Fairview- Masonic Cancer Clinic - Clinics- Site Number : 8400010
Minneapolis, Minnesota, 55455-4800
United States
Mayo Clinic - PPDS- Site Number : 8400035
Rochester, Minnesota, 55905-0001
United States
Mississippi Center For Advanced Medicine - 7731 Old Canton Rd- Site Number : 8400016
Madison, Mississippi, 39110-6115
United States
University of Nebraska Medical Center - 985400 Nebraska Medical Center- Site Number : 8400009
Omaha, Nebraska, 68114-4113
United States
Children's Specialty Center - Las Vegas- Site Number : 8400007
Las Vegas, Nevada, 89109
United States
Weill Cornell Medicine-New York Presbyterian Hospital- Site Number : 8400021
New York, New York, 10065-4870
United States
Cincinnati Children's Hospital Medical Center - PIN- Site Number : 8400006
Cincinnati, Ohio, 45229-3026
United States
Hemophilia Center of Western Pennsylvania- Site Number : 8400012
Pittsburgh, Pennsylvania, 15213-4306
United States
Vanderbilt University Medical Center- Site Number : 8400003
Nashville, Tennessee, 37232-0011
United States
University of Texas Southwestern Medical Center-2001 Inwood Rd- Site Number : 8400020
Dallas, Texas, 75390-7202
United States
University of Texas Southwestern Medical Center- Site Number : 8400011
Dallas, Texas, 75390-8565
United States
Gulf States Hemophilia and Thrombophilia Center- Site Number : 8400029
Houston, Texas, 77030
United States
Washington Institute for Coagulation- Site Number : 8400022
Seattle, Washington, 98101-3932
United States
Blood Center of Wisconnsin- Site Number : 8400027
Milwaukee, Wisconsin, 53233-2121
United States

Collaborators and Investigators

Sponsor: Sanofi

  • Clinical Sciences & Operations, STUDY_DIRECTOR, Sanofi

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2023-06-30
Study Completion Date2030-09-17

Study Record Updates

Study Start Date2023-06-30
Study Completion Date2030-09-17

Terms related to this study

Additional Relevant MeSH Terms

  • Hemophilia A