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EFANESOCTOCOG ALFA Profylakse hos pasienter med hemofili A med synovial hypertrofi (SYNOVIIIUS)

6. november 2026 oppdatert av: Sanofi

Prospektiv intervensjonell studie av effektiviteten av efanesoctocog alfa profylakse på synovial hypertrofi hos pasienter med hemofili A

The objective of the study is to assess the improvement of synovial hypertrophy during the 12 months of efanesoctocog alfa prophylaxis once per week (QW) in joints with existing evidence of synovial hypertrophy in participants with hemophilia A.

The study duration for each participant is approximately 12 months.

Studieoversikt

Status

Rekruttering

Forhold

Intervensjon / Behandling

Studietype

Intervensjonell

Registrering (Antatt)

35

Fase

  • Fase 4

Kontakter og plasseringer

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

Studiekontakt

  • Navn: Trial Transparency email recommended (Toll free for US & Canada)
  • Telefonnummer: option 6 800-633-1610
  • E-post: Contact-US@sanofi.com

Studiesteder

  • Australia
    • Western Australia
      • Murdoch, Western Australia, Australia, 6150
        • Fullført
        • Investigational Site Number : 0360001
  • Canada
    • Alberta
      • Calgary, Alberta, Canada, T2N 1N4
        • Rekruttering
        • Investigational Site Number : 1240002
    • Newfoundland and Labrador
      • St. John's, Newfoundland and Labrador, Canada, A1B 3V6
        • Rekruttering
        • Investigational Site Number : 1240001
    • Ontario
      • Hamilton, Ontario, Canada, L8N 3Z5
        • Rekruttering
        • Investigational Site Number : 1240003
      • Hamilton, Ontario, Canada, L8N 3Z5
        • Fullført
        • Investigational Site Number : 1240004
  • Forente stater
    • California
      • Los Angeles, California, Forente stater, 90007
        • Fullført
        • Orthopedic Institute for Children- Site Number : 8400009
      • Sacramento, California, Forente stater, 95817-1399
        • Rekruttering
        • University of California Davis Medical Center-Site Number: 8400005
    • Indiana
      • Indianapolis, Indiana, Forente stater, 46260
        • Rekruttering
        • Indiana Hemophilia and Thrombosis Center- Site Number : 8400006
    • Iowa
      • Iowa City, Iowa, Forente stater, 52242
        • Rekruttering
        • University of Iowa- Site Number : 8400004
    • Ohio
      • Columbus, Ohio, Forente stater, 43203
        • Fullført
        • The Ohio State University Wexner Medical Center - CarePoint East- Site Number : 8400001
    • Oregon
      • Portland, Oregon, Forente stater, 97239-3011
        • Rekruttering
        • Oregon Health and Science University-Site Number: 8400002
    • Washington
      • Seattle, Washington, Forente stater, 98101-3932
        • Rekruttering
        • Washington Center for Bleeding Disorders- Site Number : 8400008
  • Japan
      • Hiroshima, Japan, 734-8551
        • Fullført
        • Investigational Site Number : 3920002
      • Saitama, Japan, 330-8777
        • Rekruttering
        • Investigational Site Number : 3920001
      • Tokyo, Japan, 160-0023
        • Fullført
        • Investigational Site Number : 3920004
    • Kanagawa
      • Kawasaki, Kanagawa, Japan, 216-8511
        • Rekruttering
        • Investigational Site Number : 3920003
  • Taiwan
      • Kaohsiung City, Taiwan, 80756
        • Rekruttering
        • Investigational Site Number: 1580003
      • Taichung, Taiwan, 407
        • Rekruttering
        • Investigational Site Number: 1580001
      • Taipei, Taiwan, 114
        • Rekruttering
        • Investigational Site Number: 1580002

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

12 år og eldre (Barn, Voksen, Eldre voksen)

Tar imot friske frivillige

Nei

Kjønn som er kvalifisert for studier

Alle

Beskrivelse

Inclusion Criteria:

  • Participant is diagnosed with moderate to severe hemophilia A (defined as less than or equal to (≤)5 percent (%) of endogenous FVIII clotting activity) at the time of consent/assent.

  • Participant is more than or equal to (≥)12 years old at the time of consent/assent.

  • Participant has existing synovial hypertrophy, defined as at least 1 eligible* joint by the HEAD-US score (Synovitis score: 1 or 2) at the time of consent/assent.

  • Participant has at least 1 eligible* joint without planned future major orthopedic procedures (example, arthroscopic synovectomy, radioisotopic or chemical synoviorthesis), or major orthopedic procedures in the past 3 months prior to the screening visit (Visit 1).

    *An eligible joint is a joint with existing synovial hypertrophy, as defined by a HEAD-US synovitis score of 1 or 2, considering hypertrophic synovium as an indication of the presence of synovitis

  • Participant has received prophylactic treatment with hemophilia prophylaxis prescriptions in the last 12 months prior to the baseline visit (Visit 2).

  • Participant is capable of understanding the written Informed Consent Form (ICF)/assent form, provides a signed and witnessed written ICF/assent form, and agrees to comply with the protocol requirements.

  • If male, no contraceptive measures are required for this study.

  • If female, is not pregnant or breastfeeding, and one of the following conditions applies:

  • Is a women of nonchildbearing potential (WONCBP)

  • Is a WOCBP and agrees to use a highly effective contraceptive method, with a failure rate of less than (<)1 percent during the study treatment period (at least until the Week 52/ End of Treatment [EoT] visit).

  • A WOCBP must have a negative serum pregnancy test at the screening visit (Visit 1)

Exclusion Criteria:

  • Participants are excluded from the study if any of the following criteria apply:
  • Has other associated clotting disorders at the time of consent/assent.
  • Is already under efanesoctocog alfa treatment.
  • Has a current diagnosis of an factor VIII (FVIII) inhibitor, defined as inhibitor titer ≥0.60 BU/mL.
  • Has ITI within the last 2 years prior to the baseline visit (Visit 2).
  • Has been enrolled in a concurrent clinical interventional study or exposed to other investigational drug(s) within 3 months prior to screening for this study.
  • Is currently in an institution because of regulatory or legal order (that is, is a prisoner or a patient who is legally institutionalized).
  • Is not suitable for participation, whatever the reason, as judged by the investigator, including medical or clinical conditions, or patients potentially at risk of noncompliance to study procedures.
  • Is an employee or family member of the investigator or site personnel.
  • Is involved in a specific situation during study implementation or the course of the study that may raise ethics considerations.
  • Has hypersensitivity to efanesoctocog alfa or its components or any of its excipients that, in the opinion of the investigator, contraindicates participation in the study.

The above information is not intended to contain all considerations relevant to a patient's 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

  • Primært formål: Behandling
  • Tildeling: Ikke-randomisert
  • Intervensjonsmodell: Enkeltgruppeoppdrag
  • Masking: Ingen (Open Label)

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Eksperimentell: Efanesoctocog Alfa

Participants will receive Efanesoctocog Alfa at 50 international unit per kilogram (IU/kg) intravenous (IV) QW
Legemiddel: Efanesoctocog alfa

Pharmaceutical form:Lyophilized powder in a sterile vial that requires reconstitution with sterile water for injection (diluent)-Route of administration:Intravenous

Andre navn:
  • BIVV001
  • Altuviiio®

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Proportion of joints with improvement in the Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) synovitis domain score
Tidsramme: From Baseline up to Week 52

HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment.
From Baseline up to Week 52

Sekundære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Change from baseline in the distribution of joint HEAD-US synovitis score
Tidsramme: From Baseline up to Week 52

HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment.
From Baseline up to Week 52
Number of joints with the HEAD-US synovitis domain score that has remained unchanged or worsened
Tidsramme: From Baseline up to Week 52

HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment.
From Baseline up to Week 52
Percentage of joints with the HEAD-US synovitis domain score that has remained unchanged or worsened
Tidsramme: From Baseline up to Week 52

HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment.
From Baseline up to Week 52
Number of index joint type with the HEAD-US synovitis domain score that has improved, remained unchanged, or worsened
Tidsramme: From Baseline up to Week 52

HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment.
From Baseline up to Week 52
Percentage of index joint type with the HEAD-US synovitis domain score that has improved, remained unchanged, or worsened
Tidsramme: From Baseline up to Week 52

HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment.
From Baseline up to Week 52
Number of participants with at least 1 joint with the HEAD-US synovitis domain score that has improved, remained unchanged, or worsened
Tidsramme: From Baseline up to Week 52

HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment.
From Baseline up to Week 52
Percentage of participants with at least 1 joint with the HEAD-US synovitis domain score that has improved, remained unchanged, or worsened
Tidsramme: From Baseline up to Week 52

HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment.
From Baseline up to Week 52
Change from baseline in average HEAD-US synovitis domain score across all index joints
Tidsramme: From Baseline up to Week 52

HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment.
From Baseline up to Week 52
Change from baseline in total/ domain scores of the HEAD-US
Tidsramme: From Baseline up to Week 26

HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment.
From Baseline up to Week 26
Change from baseline in total/domain scores of the HJHS
Tidsramme: From Baseline up to Week 26

HJHS comprises an assessment of specific features, or items, of the 6 index joints and an assessment of global gait. For each of the 6 joints, the following items are scored: swelling (0 to 3), duration of swelling (0 to 1), muscle atrophy (0 to 2), crepitus on motion (0 to 2), flexion loss (0 to 3), extension loss (0 to 3), joint pain (0 to 2), and strength (0 to 4). The maximum score for an individual index joint is 20. Gait is scored from 0 to 4. The maximum HJHS total score is 124, with a higher score indicating worse joint health.
From Baseline up to Week 26
Change from baseline in total/domain scores of the HJHS
Tidsramme: From Baseline up to Week 52

HJHS comprises an assessment of specific features, or items, of the 6 index joints and an assessment of global gait. For each of the 6 joints, the following items are scored: swelling (0 to 3), duration of swelling (0 to 1), muscle atrophy (0 to 2), crepitus on motion (0 to 2), flexion loss (0 to 3), extension loss (0 to 3), joint pain (0 to 2), and strength (0 to 4). The maximum score for an individual index joint is 20. Gait is scored from 0 to 4. The maximum HJHS total score is 124, with a higher score indicating worse joint health.
From Baseline up to Week 52
Change from baseline in total/ domain scores of magnetic resonance imaging (MRI)
Tidsramme: From Baseline up to Week 52

MRI will be used to detect changes in synovial hypertrophy in index joints of participants.
From Baseline up to Week 52
Change from baseline in patient-reported outcome (PRO)
Tidsramme: From Baseline up to Week 52

Change from baseline in PRO will be assessed by the EQ-5D 5-level (EQ-5D-5L), Patient-Reported Outcomes Measurement Information System - Short Form (PROMIS-SF) version 2.0 Pain Intensity 3a, PROMIS-SF version 1.1 Pain Interference 6a, and PROMIS-SF version 2.0 Physical Function 6b.
From Baseline up to Week 52
Patient reported treatment preference and satisfaction
Tidsramme: At Week 52

Patient reported treatment preference and satisfaction will be assessed through surveys and exit interviews.
At Week 52
Change from baseline in annualized bleeding rate (ABR) (spontaneous, traumatic)
Tidsramme: At Week 52

The ABR will be defined as (number of bleeding episodes occurring during the treatment period)/(duration of the treatment period [days]) × 365.25.
At Week 52
Change from baseline in annualized joint bleeding rate (AjBR) (spontaneous, traumatic)
Tidsramme: At Week 52

The AjBR will be defined as (number of joint bleeding episodes occurring during the treatment period)/(duration of the treatment period [days]) × 365.25.
At Week 52
Change from baseline in target joint resolution, recurrence, or development
Tidsramme: At Week 52

Change from baseline in target joint resolution, recurrence, or development will be assessed.
At Week 52
The occurrence of Adverse Events (AEs), all AEs leading to treatment discontinuation, Serious Adverse Events (SAEs), and Adverse Event of Special Interest (AESIs)
Tidsramme: Up to Week 52

The safety and tolerability of efanesoctocog alfa evaluated in all participants
Up to Week 52

Samarbeidspartnere og etterforskere

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

Sponsor

Sanofi

Publikasjoner og nyttige lenker

Den som er ansvarlig for å legge inn informasjon om studien leverer frivillig disse publikasjonene. Disse kan handle om alt relatert til studiet.

Hjelpsomme linker

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)

23. september 2025

Primær fullføring (Antatt)

25. oktober 2027

Studiet fullført (Antatt)

25. oktober 2027

Datoer for studieregistrering

Først innsendt

16. april 2025

Først innsendt som oppfylte QC-kriteriene

16. april 2025

Først lagt ut (Faktiske)

24. april 2025

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

9. november 2026

Siste oppdatering sendt inn som oppfylte QC-kriteriene

6. november 2026

Sist bekreftet

1. november 2026

Mer informasjon

Begreper knyttet til denne studien

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

  • LPS18168
  • U1111-1311-1701 (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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