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Uno studio per valutare l'impatto di Efanesoctocog Alfa sulla salute delle articolazioni a lungo termine nei partecipanti con emofilia A

24 settembre 2026 aggiornato da: Sanofi

Studio prospettico, osservazionale e multicentrico sull'efficacia di Efanesoctocog Alfa sulla salute delle articolazioni a lungo termine nei pazienti con emofilia 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.

Panoramica dello studio

Stato

Attivo, non reclutante

Condizioni

Intervento / Trattamento

Tipo di studio

Osservativo

Iscrizione (Stimato)

200

Contatti e Sedi

Questa sezione fornisce i recapiti di coloro che conducono lo studio e informazioni su dove viene condotto lo studio.

Luoghi di studio

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

Criteri di partecipazione

I ricercatori cercano persone che corrispondano a una certa descrizione, chiamata criteri di ammissibilità. Alcuni esempi di questi criteri sono le condizioni generali di salute di una persona o trattamenti precedenti.

Criteri di ammissibilità

Età idonea allo studio

  • Bambino
  • Adulto
  • Adulto più anziano

Accetta volontari sani

No

Sessi ammissibili allo studio

Tutto

Metodo di campionamento

Campione non probabilistico

Popolazione di studio

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.

Descrizione

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."

Piano di studio

Questa sezione fornisce i dettagli del piano di studio, compreso il modo in cui lo studio è progettato e ciò che lo studio sta misurando.

Come è strutturato lo studio?

Dettagli di progettazione

  • Modelli osservazionali: COHORT
  • Prospettive temporali: PROSPECTIVE

Coorti e interventi

Gruppo / Coorte
Intervento / Trattamento
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
Droga: Efanesoctocog Alfa BIVV001

Given per investigator's discretion

Altri nomi:
  • ALTUVIII
Cohort B (On-Demand treatment)

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

Given per investigator's discretion

Altri nomi:
  • ALTUVIII

Cosa sta misurando lo studio?

Misure di risultato primarie

Misura del risultato
Misura Descrizione
Lasso di tempo
Change from baseline in Annualized joint bleeding rate (AjBR) for treated bleeds
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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

Misure di risultato secondarie

Misura del risultato
Misura Descrizione
Lasso di tempo
Change from baseline in the Hemophilia Joint Health Score (HJHS v2.1) total/domains scores
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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)
Lasso di tempo: 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
Lasso di tempo: 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.
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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.
Lasso di tempo: 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.
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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.
Lasso di tempo: 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.
Lasso di tempo: 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
Lasso di tempo: 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)
Lasso di tempo: 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
Lasso di tempo: 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)
Lasso di tempo: 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.
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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
Lasso di tempo: 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.
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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

Collaboratori e investigatori

Qui è dove troverai le persone e le organizzazioni coinvolte in questo studio.

Sponsor

Sanofi

Investigatori

  • Direttore dello studio: Clinical Sciences & Operations, Sanofi

Studiare le date dei record

Queste date tengono traccia dell'avanzamento della registrazione dello studio e dell'invio dei risultati di sintesi a ClinicalTrials.gov. I record degli studi e i risultati riportati vengono esaminati dalla National Library of Medicine (NLM) per assicurarsi che soddisfino specifici standard di controllo della qualità prima di essere pubblicati sul sito Web pubblico.

Studia le date principali

Inizio studio (Effettivo)

30 giugno 2023

Completamento primario (Stimato)

6 giugno 2028

Completamento dello studio (Stimato)

17 settembre 2030

Date di iscrizione allo studio

Primo inviato

12 maggio 2023

Primo inviato che soddisfa i criteri di controllo qualità

19 giugno 2023

Primo Inserito (Effettivo)

22 giugno 2023

Aggiornamenti dei record di studio

Ultimo aggiornamento pubblicato (Effettivo)

29 settembre 2026

Ultimo aggiornamento inviato che soddisfa i criteri QC

24 settembre 2026

Ultimo verificato

1 settembre 2026

Maggiori informazioni

Termini relativi a questo studio

Termini MeSH pertinenti aggiuntivi

Altri numeri di identificazione dello studio

  • OBS17523
  • U1111-1281-8840 (Identificatore di registro: ICTRP)

Piano per i dati dei singoli partecipanti (IPD)

Hai intenzione di condividere i dati dei singoli partecipanti (IPD)?

SÌ

Descrizione del piano 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

Informazioni su farmaci e dispositivi, documenti di studio

Studia un prodotto farmaceutico regolamentato dalla FDA degli Stati Uniti

Sì

Studia un dispositivo regolamentato dalla FDA degli Stati Uniti

No

Queste informazioni sono state recuperate direttamente dal sito web clinicaltrials.gov senza alcuna modifica. In caso di richieste di modifica, rimozione o aggiornamento dei dettagli dello studio, contattare register@clinicaltrials.gov. Non appena verrà implementata una modifica su clinicaltrials.gov, questa verrà aggiornata automaticamente anche sul nostro sito web .

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