Questa pagina è stata tradotta automaticamente e l'accuratezza della traduzione non è garantita. Si prega di fare riferimento al Versione inglese per un testo di partenza.

Profilassi di efanesoctocog alfa in pazienti con emofilia A con ipertrofia sinoviale (SYNOVIIIUS)

6 novembre 2026 aggiornato da: Sanofi

Studio interventistico prospettico sull'efficacia dell'efanesoctocog alfa profilassi sull'ipertrofia sinoviale nei pazienti con emofilia 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.

Panoramica dello studio

Stato

Reclutamento

Condizioni

Intervento / Trattamento

Tipo di studio

Interventistico

Iscrizione (Stimato)

35

Fase

  • Fase 4

Contatti e Sedi

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

Contatto studio

  • Nome: Trial Transparency email recommended (Toll free for US & Canada)
  • Numero di telefono: option 6 800-633-1610
  • Email: Contact-US@sanofi.com

Luoghi di studio

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

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

12 anni e precedenti (Bambino, Adulto, Adulto più anziano)

Accetta volontari sani

No

Sessi ammissibili allo studio

Tutto

Descrizione

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.

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

  • Scopo principale: Trattamento
  • Assegnazione: Non randomizzato
  • Modello interventistico: Assegnazione di gruppo singolo
  • Mascheramento: Nessuno (etichetta aperta)

Armi e interventi

Gruppo di partecipanti / Arm
Intervento / Trattamento
Sperimentale: Efanesoctocog Alfa

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

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

Altri nomi:
  • BIVV001
  • Altuviiio®

Cosa sta misurando lo studio?

Misure di risultato primarie

Misura del risultato
Misura Descrizione
Lasso di tempo
Proportion of joints with improvement in the Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) synovitis domain score
Lasso di tempo: 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

Misure di risultato secondarie

Misura del risultato
Misura Descrizione
Lasso di tempo
Change from baseline in the distribution of joint HEAD-US synovitis score
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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
Lasso di tempo: 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)
Lasso di tempo: Up to Week 52

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

Collaboratori e investigatori

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

Sponsor

Sanofi

Pubblicazioni e link utili

La persona responsabile dell'inserimento delle informazioni sullo studio fornisce volontariamente queste pubblicazioni. Questi possono riguardare qualsiasi cosa relativa allo studio.

Collegamenti utili

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)

23 settembre 2025

Completamento primario (Stimato)

25 ottobre 2027

Completamento dello studio (Stimato)

25 ottobre 2027

Date di iscrizione allo studio

Primo inviato

16 aprile 2025

Primo inviato che soddisfa i criteri di controllo qualità

16 aprile 2025

Primo Inserito (Effettivo)

24 aprile 2025

Aggiornamenti dei record di studio

Ultimo aggiornamento pubblicato (Effettivo)

9 novembre 2026

Ultimo aggiornamento inviato che soddisfa i criteri QC

6 novembre 2026

Ultimo verificato

1 novembre 2026

Maggiori informazioni

Termini relativi a questo studio

Termini MeSH pertinenti aggiuntivi

Altri numeri di identificazione dello studio

  • LPS18168
  • U1111-1311-1701 (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 .

Sottoscrivi