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NCT05911763Active, not recruiting

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

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

Source: ClinicalTrials.gov · Last updated 29 Sept 2026

Brief summary (registry, in English)

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.

Status (registry)
Active, not recruiting
Study type
Observational
Enrollment
200 (estimated)
First submitted
12 May 2023
Start date
30 Jun 2023
Primary completion date
6 Jun 2028
Lead sponsor
Sanofi
Diseases
Haemophilia
NCT number
NCT05911763

What you commit to

  • Study type: Observational
  • Planned participants: 200
  • From 30 Jun 2023 to the primary completion date, 6 Jun 2028
  • 28 sites in 1 country
  • Visits and tests: ask the trial team, the registry does not list them

Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.

Who cannot take part

Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.

Prior treatment

  • 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."

Other illness

  • Diagnosed with other known bleeding disorder

Who the study is looking for

  • 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.

Sex: All · Does not accept healthy volunteers

Full criteria on ClinicalTrials.gov

Sites (28)

Sites in registry order. Pick your city on the disease page to sort them by distance.

SiteCityCountrySite status
University of Michigan Hospital - 1500 E Medical Center Dr- Site Number : 8400023
Coordinator of this site? Confirm its status
Ann ArborUnited StatesUnknown status
University of Colorado Hemophilia and Thrombosis Center- Site Number : 8400019
Coordinator of this site? Confirm its status
AuroraUnited StatesUnknown status
~Massachusetts General Hospital- Site Number : 8400017
Coordinator of this site? Confirm its status
BostonUnited StatesUnknown status
Dana Farber and Boston Children's Hospital- Site Number : 8400031
Coordinator of this site? Confirm its status
BostonUnited StatesUnknown status
Rush University Medical Center -1725 W Harrison St- Site Number : 8400014
Coordinator of this site? Confirm its status
ChicagoUnited StatesUnknown status
Cincinnati Children's Hospital Medical Center - PIN- Site Number : 8400006
Coordinator of this site? Confirm its status
CincinnatiUnited StatesUnknown status
University of Texas - Southwestern Medical Center- Site Number : 8400011
Coordinator of this site? Confirm its status
DallasUnited StatesUnknown status
University of Texas Southwestern Medical Center-2001 Inwood Rd- Site Number : 8400020
Coordinator of this site? Confirm its status
DallasUnited StatesUnknown status
William A. Shands Hospital at the University of Florida- Site Number : 8400032
Coordinator of this site? Confirm its status
GainesvilleUnited StatesUnknown status
Banner MD Anderson Cancer Center- Site Number : 8400008
Coordinator of this site? Confirm its status
GilbertUnited StatesUnknown status
Gulf States Hemophilia and Thrombophilia Center- Site Number : 8400029
Coordinator of this site? Confirm its status
HoustonUnited StatesUnknown status
Indiana Hemophilia and Thrombosis Center- Site Number : 8400013
Coordinator of this site? Confirm its status
IndianapolisUnited StatesUnknown status
University Of Iowa Hospitals And Clinics- Site Number : 8400026
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Iowa CityUnited StatesUnknown status
Children's Specialty Center - Las Vegas- Site Number : 8400007
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Las VegasUnited StatesUnknown status
Loma Linda University- Site Number : 8400015
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Loma LindaUnited StatesUnknown status
Orthopedic Institute for Children- Site Number : 8400004
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Los AngelesUnited StatesUnknown status
Mississippi Center For Advanced Medicine - 7731 Old Canton Rd- Site Number : 8400016
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MadisonUnited StatesUnknown status
Blood Center of Wisconnsin- Site Number : 8400027
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MilwaukeeUnited StatesUnknown status
M Health Fairview- Masonic Cancer Clinic - Clinics- Site Number : 8400010
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MinneapolisUnited StatesUnknown status
Vanderbilt University Medical Center- Site Number : 8400003
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NashvilleUnited StatesUnknown status
Weill Cornell Medicine-New York Presbyterian Hospital- Site Number : 8400021
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New YorkUnited StatesUnknown status
University of Nebraska Medical Center - 985400 Nebraska Medical Center- Site Number : 8400009
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OmahaUnited StatesUnknown status
The Center for Inherited Blood Disorders- Site Number : 8400001
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OrangeUnited StatesUnknown status
The Bleeding and Clotting Disorders Institute- Site Number : 8400005
Coordinator of this site? Confirm its status
PeoriaUnited StatesUnknown status
Hemophilia Center of Western Pennsylvania- Site Number : 8400012
Coordinator of this site? Confirm its status
PittsburghUnited StatesUnknown status
Mayo Clinic - PPDS- Site Number : 8400035
Coordinator of this site? Confirm its status
RochesterUnited StatesUnknown status
Washington Institute for Coagulation- Site Number : 8400022
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SeattleUnited StatesUnknown status
Johns Hopkins All Childrens Hospital - Outpatient Care Center - PIN- Site Number : 8400025
Coordinator of this site? Confirm its status
St. PetersburgUnited StatesUnknown status

This page is information, not medical advice. Discuss any trial with your doctor.