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Eine Studie zur Bewertung der Auswirkungen von Efanesoctocog Alfa auf die langfristige Gelenkgesundheit bei Teilnehmern mit Hämophilie A

24. September 2026 aktualisiert von: Sanofi

Prospektive, beobachtende, multizentrische Studie zur Wirksamkeit von Efanesoctocog Alfa auf die langfristige Gelenkgesundheit bei Patienten mit Hämophilie 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.

Studienübersicht

Status

Aktiv, nicht rekrutierend

Bedingungen

Intervention / Behandlung

Studientyp

Beobachtungs

Einschreibung (Geschätzt)

200

Kontakte und Standorte

Dieser Abschnitt enthält die Kontaktdaten derjenigen, die die Studie durchführen, und Informationen darüber, wo diese Studie durchgeführt wird.

Studienorte

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

Teilnahmekriterien

Forscher suchen nach Personen, die einer bestimmten Beschreibung entsprechen, die als Auswahlkriterien bezeichnet werden. Einige Beispiele für diese Kriterien sind der allgemeine Gesundheitszustand einer Person oder frühere Behandlungen.

Zulassungskriterien

Studienberechtigtes Alter

  • Kind
  • Erwachsene
  • Älterer Erwachsener

Akzeptiert gesunde Freiwillige

Nein

Studienberechtigte Geschlechter

Alle

Probenahmeverfahren

Nicht-Wahrscheinlichkeitsprobe

Studienpopulation

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.

Beschreibung

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."

Studienplan

Dieser Abschnitt enthält Einzelheiten zum Studienplan, einschließlich des Studiendesigns und der Messung der Studieninhalte.

Wie ist die Studie aufgebaut?

Designdetails

  • Beobachtungsmodelle: COHORT
  • Zeitperspektiven: PROSPECTIVE

Kohorten und Interventionen

Gruppe / Kohorte
Intervention / Behandlung
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
Arzneimittel: Efanesoctocog Alfa BIVV001

Given per investigator's discretion

Andere Namen:
  • ALTUVIIO
Cohort B (On-Demand treatment)

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

Given per investigator's discretion

Andere Namen:
  • ALTUVIIO

Was misst die Studie?

Primäre Ergebnismessungen

Ergebnis Maßnahme
Maßnahmenbeschreibung
Zeitfenster
Change from baseline in Annualized joint bleeding rate (AjBR) for treated bleeds
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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

Sekundäre Ergebnismessungen

Ergebnis Maßnahme
Maßnahmenbeschreibung
Zeitfenster
Change from baseline in the Hemophilia Joint Health Score (HJHS v2.1) total/domains scores
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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)
Zeitfenster: 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
Zeitfenster: 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.
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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.
Zeitfenster: 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.
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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.
Zeitfenster: 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.
Zeitfenster: 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
Zeitfenster: 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)
Zeitfenster: 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
Zeitfenster: 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)
Zeitfenster: 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.
Zeitfenster: 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)
Zeitfenster: 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)
Zeitfenster: 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
Zeitfenster: 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.
Zeitfenster: 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)
Zeitfenster: 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)
Zeitfenster: 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)
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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)
Zeitfenster: 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)
Zeitfenster: 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)
Zeitfenster: 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

Mitarbeiter und Ermittler

Hier finden Sie Personen und Organisationen, die an dieser Studie beteiligt sind.

Sponsor

Sanofi

Ermittler

  • Studienleiter: Clinical Sciences & Operations, Sanofi

Studienaufzeichnungsdaten

Diese Daten verfolgen den Fortschritt der Übermittlung von Studienaufzeichnungen und zusammenfassenden Ergebnissen an ClinicalTrials.gov. Studienaufzeichnungen und gemeldete Ergebnisse werden von der National Library of Medicine (NLM) überprüft, um sicherzustellen, dass sie bestimmten Qualitätskontrollstandards entsprechen, bevor sie auf der öffentlichen Website veröffentlicht werden.

Haupttermine studieren

Studienbeginn (Tatsächlich)

30. Juni 2023

Primärer Abschluss (Geschätzt)

6. Juni 2028

Studienabschluss (Geschätzt)

17. September 2030

Studienanmeldedaten

Zuerst eingereicht

12. Mai 2023

Zuerst eingereicht, das die QC-Kriterien erfüllt hat

19. Juni 2023

Zuerst gepostet (Tatsächlich)

22. Juni 2023

Studienaufzeichnungsaktualisierungen

Letztes Update gepostet (Tatsächlich)

29. September 2026

Letztes eingereichtes Update, das die QC-Kriterien erfüllt

24. September 2026

Zuletzt verifiziert

1. September 2026

Mehr Informationen

Begriffe im Zusammenhang mit dieser Studie

Zusätzliche relevante MeSH-Bedingungen

Andere Studien-ID-Nummern

  • OBS17523
  • U1111-1281-8840 (Registrierungskennung: ICTRP)

Plan für individuelle Teilnehmerdaten (IPD)

Planen Sie, individuelle Teilnehmerdaten (IPD) zu teilen?

JA

Beschreibung des IPD-Plans

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

Arzneimittel- und Geräteinformationen, Studienunterlagen

Studiert ein von der US-amerikanischen FDA reguliertes Arzneimittelprodukt

Ja

Studiert ein von der US-amerikanischen FDA reguliertes Geräteprodukt

Nein

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