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En studie for å evaluere effekten av Efanesoctocog Alfa på langsiktig leddhelse hos deltakere med hemofili A

24. september 2026 oppdatert av: Sanofi

Prospektiv, observasjons-, multisenterstudie av effektiviteten av Efanesoctocog Alfa på langsiktig leddhelse hos pasienter med hemofili 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.

Studieoversikt

Status

Aktiv, ikke rekrutterende

Forhold

Intervensjon / Behandling

Studietype

Observasjonsmessig

Registrering (Antatt)

200

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiesteder

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

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

  • Barn
  • Voksen
  • Eldre voksen

Tar imot friske frivillige

Nei

Kjønn som er kvalifisert for studier

Alle

Prøvetakingsmetode

Ikke-sannsynlighetsprøve

Studiepopulasjon

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.

Beskrivelse

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."

Studieplan

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

  • Observasjonsmodeller: COHORT
  • Tidsperspektiver: PROSPECTIVE

Kohorter og intervensjoner

Gruppe / Kohort
Intervensjon / Behandling
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
Legemiddel: Efanesoctocog Alfa BIVV001

Given per investigator's discretion

Andre navn:
  • ALTUVIIIO
Cohort B (On-Demand treatment)

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

Given per investigator's discretion

Andre navn:
  • ALTUVIIIO

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Change from baseline in Annualized joint bleeding rate (AjBR) for treated bleeds
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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

Sekundære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Change from baseline in the Hemophilia Joint Health Score (HJHS v2.1) total/domains scores
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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)
Tidsramme: 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
Tidsramme: 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.
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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.
Tidsramme: 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.
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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.
Tidsramme: 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.
Tidsramme: 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
Tidsramme: 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)
Tidsramme: 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
Tidsramme: 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)
Tidsramme: 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.
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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
Tidsramme: 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.
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Sponsor

Sanofi

Etterforskere

  • Studieleder: Clinical Sciences & Operations, Sanofi

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart (Faktiske)

30. juni 2023

Primær fullføring (Antatt)

6. juni 2028

Studiet fullført (Antatt)

17. september 2030

Datoer for studieregistrering

Først innsendt

12. mai 2023

Først innsendt som oppfylte QC-kriteriene

19. juni 2023

Først lagt ut (Faktiske)

22. juni 2023

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

29. september 2026

Siste oppdatering sendt inn som oppfylte QC-kriteriene

24. september 2026

Sist bekreftet

1. september 2026

Mer informasjon

Begreper knyttet til denne studien

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

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

Plan for individuelle deltakerdata (IPD)

Planlegger du å dele individuelle deltakerdata (IPD)?

JA

IPD-planbeskrivelse

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

Legemiddel- og utstyrsinformasjon, studiedokumenter

Studerer et amerikansk FDA-regulert medikamentprodukt

Ja

Studerer et amerikansk FDA-regulert enhetsprodukt

Nei

Denne informasjonen ble hentet direkte fra nettstedet clinicaltrials.gov uten noen endringer. Hvis du har noen forespørsler om å endre, fjerne eller oppdatere studiedetaljene dine, vennligst kontakt register@clinicaltrials.gov. Så snart en endring er implementert på clinicaltrials.gov, vil denne også bli oppdatert automatisk på nettstedet vårt. .

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