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Une étude pour évaluer l'impact d'Efanesoctocog Alfa sur la santé articulaire à long terme chez les participants atteints d'hémophilie A

24 septembre 2026 mis à jour par: Sanofi

Étude prospective, observationnelle et multicentrique de l'efficacité d'Efanesoctocog Alfa sur la santé articulaire à long terme chez les patients atteints d'hémophilie A

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.

Aperçu de l'étude

Statut

Actif, ne recrute pas

Les conditions

Intervention / Traitement

Type d'étude

Observationnel

Inscription (Estimé)

200

Contacts et emplacements

Cette section fournit les coordonnées de ceux qui mènent l'étude et des informations sur le lieu où cette étude est menée.

Lieux d'étude

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

Critères de participation

Les chercheurs recherchent des personnes qui correspondent à une certaine description, appelée critères d'éligibilité. Certains exemples de ces critères sont l'état de santé général d'une personne ou des traitements antérieurs.

Critère d'éligibilité

Âges éligibles pour étudier

  • Enfant
  • Adulte
  • Adulte plus âgé

Accepte les volontaires sains

Non

Sexes éligibles pour l'étude

Tout

Méthode d'échantillonnage

Échantillon non probabiliste

Population étudiée

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.

La description

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."

Plan d'étude

Cette section fournit des détails sur le plan d'étude, y compris la façon dont l'étude est conçue et ce que l'étude mesure.

Comment l'étude est-elle conçue ?

Détails de conception

  • Modèles d'observation: COHORT
  • Perspectives temporelles: PROSPECTIVE

Cohortes et interventions

Groupe / Cohorte
Intervention / Traitement
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
Médicament: Efanesoctocog Alfa BIVV001

Given per investigator's discretion

Autres noms:
  • ALTUIIIO
Cohort B (On-Demand treatment)

Participants receiving on-demand treatment with efanesoctocog alfa who fulfil the overall study inclusion/exclusion criteria
Médicament: Efanesoctocog Alfa BIVV001

Given per investigator's discretion

Autres noms:
  • ALTUIIIO

Que mesure l'étude ?

Principaux critères de jugement

Mesure des résultats
Description de la mesure
Délai
Change from baseline in Annualized joint bleeding rate (AjBR) for treated bleeds
Délai: 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
Up to 5 years
Change from baseline in Annualized joint bleeding rate (AjBR) for all (treated and untreated) bleeds
Délai: 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
Up to 5 years
Number of Target joint development, resolution and/or recurrence
Délai: 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
Up to 5 years

Mesures de résultats secondaires

Mesure des résultats
Description de la mesure
Délai
Change from baseline in the Hemophilia Joint Health Score (HJHS v2.1) total/domains scores
Délai: At 1, 2, 3, 4, 5 years

HJHS total/domain scores will be reported for prophylactic cohorts (A1 ad A2)
At 1, 2, 3, 4, 5 years
Annual Bleeding Rate (ABR) by type for treated bleeds
Délai: At 1, 2, 3, 4, 5 years

Data will be reported for prophylactic cohorts (A1 and A2)
At 1, 2, 3, 4, 5 years
ABR by type all (treated and untreated) bleeds
Délai: At 1, 2, 3, 4, 5 years

Data will be reported for prophylactic cohorts (A1 and A2)
At 1, 2, 3, 4, 5 years
ABR by location for treated bleeds
Délai: At 1, 2, 3, 4, 5 years

Data will be reported for prophylactic cohorts (A1 and A2)
At 1, 2, 3, 4, 5 years
ABR by location for all (treated and untreated) bleeds
Délai: At 1, 2, 3, 4, 5 years

Data will be reported for prophylactic cohorts (A1 and A2)
At 1, 2, 3, 4, 5 years
ABR for all bleeding episodes (including untreated bleeding episodes)
Délai: At 1, 2, 3, 4, 5 years

Data will be reported for prophylactic cohorts (A1 and A2)
At 1, 2, 3, 4, 5 years
Percentage of patients with zero joint bleeds
Délai: At 1, 2, 3, 4, 5 years

Data will be reported for prophylactic cohorts (A1 and A2)
At 1, 2, 3, 4, 5 years
Annualized factor consumption per participant (IU/kg) assessed by prescription during the follow-up period.
Délai: At 1, 2, 3, 4, 5 years

Data will be reported for prophylactic cohorts (A1 and A2)
At 1, 2, 3, 4, 5 years
Annualized injection frequency per participant (assessed by prescription) during the follow- up period
Délai: At 1, 2, 3, 4, 5 years

Data will be reported for prophylactic cohorts (A1 and A2)
At 1, 2, 3, 4, 5 years
Treatment adherence (%) as judged by the physician during the follow-up period
Délai: At 1, 2, 3, 4, 5 years

Data will be reported for prophylactic cohorts (A1 and A2)
At 1, 2, 3, 4, 5 years
Number of injections of efanesoctocog alfa to treat a bleeding episode.
Délai: At 1, 2, 3, 4, 5 years

Data will be reported for prophylactic cohorts (Sub Cohorts A1 and A2)
At 1, 2, 3, 4, 5 years
Total dose of efanesoctocog alfa to treat a bleeding episode.
Délai: At 1, 2, 3, 4, 5 years

Data will be reported for prophylactic cohorts (Sub Cohorts A1 and A2)
At 1, 2, 3, 4, 5 years
Number of injections of efanesoctocog alfa to treat a bleeding episode
Délai: At 1, 2, 3, 4, 5 years

Data will be reported for On-demand cohort
At 1, 2, 3, 4, 5 years
Total dose of efanesoctocog alfa to treat a bleeding episode
Délai: At 1, 2, 3, 4, 5 years

Data will be reported for On-demand cohort
At 1, 2, 3, 4, 5 years
Percentage of bleeding episodes treated with a single injection of efanesoctocog alfa.
Délai: At 1, 2, 3, 4, 5 years

Data will be reported for On-demand cohort
At 1, 2, 3, 4, 5 years
Occurrence of a change in treatment regimen (on-demand to prophylactic or prophylactic to on-demand) at baseline and follow-up.
Délai: At 1, 2, 3, 4, 5 years

Data will be reported for On-demand cohort
At 1, 2, 3, 4, 5 years
Change from baseline in Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) total/domain scores
Délai: At 1, 2, 3, 4 and 5 years

HEAD-US total/domain scores will be reported for joint imaging sub cohort
At 1, 2, 3, 4 and 5 years
Change from baseline in Hemophilia Early Arthropathy Detection with Joint Tissue Activity and Damage exam (JADE) musculoskeletal ultrasound (MSKUS) (JADE MSKUS)
Délai: At 1, 2, 3, 4 and 5 years

Data will be reported for consenting participants from the joint imaging sub-cohort
At 1, 2, 3, 4 and 5 years
Change from baseline in synovial hypertrophy by change in mm thickness AND/OR HEAD-US synovitis domain, AND/OR by JADE MSKUS synovial hypertrophy +/- power doppler signal
Délai: At 6 months, 1, 2, 3, 4 and 5 years

Data will be reported from the consenting participants from the joint imaging sub-cohort
At 6 months, 1, 2, 3, 4 and 5 years
Occurrence of adverse events (AEs) and serious adverse events (SAEs)
Délai: Over 5 year period

The safety and tolerability of efanesoctocog alfa over 5 years evaluated in all participants
Over 5 year period
Development of inhibitors (neutralizing antibodies directed against factor FVIII as determined via the Nijmegen modified Bethesda assay.
Délai: Over 5 year period

The safety and tolerability of efanesoctocog alfa over 5 years assessed in all participants
Over 5 year period
Change from baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) pain intensity 3a questionnaire (adults)
Délai: At 3 months, 6 months, 1, 2, 3, 4 and 5 years

Effectiveness of efanesoctocog alfa assessed per participants' PROs in all participants
At 3 months, 6 months, 1, 2, 3, 4 and 5 years
Change from baseline in Canadian Hemophilia Outcomes-Kids' Life Assessment Tool (CHO-KLAT) 3.0 (boys ≤18 years old) questionnaire (including Parent Proxy)
Délai: At 1, 2, 3, 4 and 5 years

Effectiveness of efanesoctocog alfa assessed per participants' PROs in all participants
At 1, 2, 3, 4 and 5 years
Change from baseline in the occurrence of inpatient and outpatient visits, and length of hospital stay related to hemophilia A
Délai: At 1, 2, 3, 4 and 5 years

Healthcare resource use (HCRU) in efanesoctocog alfa related to hemophilia A over a 5-year period
At 1, 2, 3, 4 and 5 years
Hemostatic response/physician reported during peri-operative period for surgery (major, minor) with efanesoctocog alfa.
Délai: Over a 5 year period

Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Over a 5 year period
Number of injections required to maintain hemostasis during perioperative period for surgery (major, minor)
Délai: Over 5 year period

Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Over 5 year period
Dose per injection required to maintain hemostasis during perioperative period for surgery (major, minor)
Délai: Over 5 year period

Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Over 5 year period
Total efanesoctocog alfa consumption (IU) during perioperative period for surgery (major, minor)
Délai: Over 5 year period

Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Over 5 year period
Number of blood component transfusions used during perioperative period for surgery
Délai: Over 5 year Period

Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Over 5 year Period
Type of blood component transfusions used during perioperative period for surgery
Délai: Over 5 year period

Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Over 5 year period
Estimated blood loss (ml) (intraoperative and post-operative period) for major surgery
Délai: Over 5 year period

Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Over 5 year period
Number of transfusions required for surgery (intraoperative and post-operative period)
Délai: Over 5 year period

Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Over 5 year period
Duration of hospitalization (major, minor)
Délai: Over 5 years period

Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period.
Over 5 years period
Dose per injection required to maintain hemostasis during perioperative period for surgery (major, minor)
Délai: Over a 5 year period

Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period for On-demand Cohort
Over a 5 year period

Collaborateurs et enquêteurs

C'est ici que vous trouverez les personnes et les organisations impliquées dans cette étude.

Parrainer

Sanofi

Les enquêteurs

  • Directeur d'études: Clinical Sciences & Operations, Sanofi

Dates d'enregistrement des études

Ces dates suivent la progression des dossiers d'étude et des soumissions de résultats sommaires à ClinicalTrials.gov. Les dossiers d'étude et les résultats rapportés sont examinés par la Bibliothèque nationale de médecine (NLM) pour s'assurer qu'ils répondent à des normes de contrôle de qualité spécifiques avant d'être publiés sur le site Web public.

Dates principales de l'étude

Début de l'étude (Réel)

30 juin 2023

Achèvement primaire (Estimé)

6 juin 2028

Achèvement de l'étude (Estimé)

17 septembre 2030

Dates d'inscription aux études

Première soumission

12 mai 2023

Première soumission répondant aux critères de contrôle qualité

19 juin 2023

Première publication (Réel)

22 juin 2023

Mises à jour des dossiers d'étude

Dernière mise à jour publiée (Réel)

29 septembre 2026

Dernière mise à jour soumise répondant aux critères de contrôle qualité

24 septembre 2026

Dernière vérification

1 septembre 2026

Plus d'information

Termes liés à cette étude

Termes MeSH pertinents supplémentaires

Autres numéros d'identification d'étude

  • OBS17523
  • U1111-1281-8840 (Identificateur de registre: ICTRP)

Plan pour les données individuelles des participants (IPD)

Prévoyez-vous de partager les données individuelles des participants (DPI) ?

OUI

Description du régime IPD

Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

Informations sur les médicaments et les dispositifs, documents d'étude

Étudie un produit pharmaceutique réglementé par la FDA américaine

Oui

Étudie un produit d'appareil réglementé par la FDA américaine

Non

Ces informations ont été extraites directement du site Web clinicaltrials.gov sans aucune modification. Si vous avez des demandes de modification, de suppression ou de mise à jour des détails de votre étude, veuillez contacter register@clinicaltrials.gov. Dès qu'un changement est mis en œuvre sur clinicaltrials.gov, il sera également mis à jour automatiquement sur notre site Web .

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