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Une étude pour tester un médicament (Fitusiran) pour prévenir les hémorragies chez les personnes atteintes d'hémophilie sévère ayant déjà reçu un traitement préventif avec l'émicizumab

18 septembre 2026 mis à jour par: Sanofi

Une étude de traitement ouverte à un seul bras pour étudier l'innocuité et la tolérabilité du passage de l'émicizumab à la prophylaxie par le fitusiran chez des participants masculins âgés de ≥ 18 ans atteints d'hémophilie A sévère, avec ou sans inhibiteurs

This is an exploratory, single group, Phase 4, study to assess treatment with fitusiran prophylaxis after switching from emicizumab prophylaxis.

This study aims to evaluate the safety and tolerability of switching to fitusiran after a transition period from the last dose of emicizumab. The study will be conducted in male participants with severe hemophilia A, with or without inhibitors, aged ≥12 years, who were previously receiving emicizumab prophylaxis.

Study details include:

  • The study duration will be up to approximately 28 months:

    • There will be an approximately 2-month screening period.
    • There will be an approximately 2-month period before fitusiran treatment starts (pre-fitusiran treatment period)
    • The fitusiran treatment duration will be up to 18-months (fitusiran treatment period)
    • The antithrombin (AT) follow-up (FU) period will be approximately 6 months after the last dose of fitusiran (during which the AT activity level will be monitored at approximately monthly intervals following the final fitusiran dose until AT activity levels return to at least 60%).

  • The study site visits are scheduled at monthly/ every 2 months intervals of 28 days (4 weeks) / 56 days (8 weeks), respectively, during the fitusiran treatment period.

Aperçu de l'étude

Statut

Recrutement

Les conditions

Intervention / Traitement

Type d'étude

Interventionnel

Inscription (Estimé)

20

Phase

  • Phase 4

Contacts et emplacements

Cette section fournit les coordonnées de ceux qui mènent l'étude et des informations sur le lieu où cette étude est menée.

Coordonnées de l'étude

  • Nom: Trial Transparency email recommended (Toll free for US & Canada)
  • Numéro de téléphone: option 6 800-633-1610
  • E-mail: Contact-US@sanofi.com

Lieux d'étude

  • Taïwan
      • Taipei, Taïwan, 100
        • Recrutement
        • Investigational Site Number : 1580001
  • États-Unis
    • California
      • Los Angeles, California, États-Unis, 90027
        • Recrutement
        • Children's Hospital Los Angeles- Site Number : 8400005
    • Mississippi
      • Flowood, Mississippi, États-Unis, 39232
        • Recrutement
        • Southern Specialty Research- Site Number : 8400011

Critères de participation

Les chercheurs recherchent des personnes qui correspondent à une certaine description, appelée critères d'éligibilité. Certains exemples de ces critères sont l'état de santé général d'une personne ou des traitements antérieurs.

Critère d'éligibilité

Âges éligibles pour étudier

12 ans et plus (Enfant, Adulte, Adulte plus âgé)

Accepte les volontaires sains

Non

Sexes éligibles pour l'étude

Homme

La description

Inclusion Criteria:

  • Male participants must be ≥12 years of age inclusive, at the time of signing the informed consent

  • Diagnosis of severe congenital hemophilia A (FVIII < 1%) as evidenced by a central laboratory measurement at screening or documented medical record evidence.

    • Inhibitor titer of ≥0.6 BU/mL at Screening, or
    • Inhibitor titer of <0.6 BU/mL at Screening with medical record evidence of 2 consecutive titers ≥0.6 BU/mL, or
    • Inhibitor titer of <0.6 BU/mL at Screening with medical record evidence of anamnestic response.

  • Participants who are currently on the full labeled dose of emicizumab prophylaxis, irrespective of inhibitor/non-inhibitor status.

  • Signed informed consent/assent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.

Exclusion Criteria:

Participants are excluded from the study if any of the following criteria apply:

  • Known coexisting bleeding disorders
  • History of antiphospholipid antibody syndrome.
  • History of arterial or venous thromboembolism, atrial fibrillation, significant valvular disease, myocardial infarction, angina, transient ischemic attack, or stroke. Participants who have experienced thrombosis associated with indwelling venous access may be enrolled.
  • Presence of clinically significant liver disease
  • Current or prior participation in a fitusiran trial
  • Current or prior participation in a gene therapy trial
  • AT activity <60% at Screening, as determined by central laboratory measurement
  • Coexisting thrombophilic disorder - Hepatitis C virus antibody positive, except participants who have negative Hepatitis C viral load and no evidence of cirrhosis
  • Presence of acute hepatitis, ie, hepatitis A, hepatitis E.
  • Presence of acute or chronic hepatitis B infection
  • Known to be HIV positive with CD4 count <200 cells/μL.
  • Reduced renal function

The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Plan d'étude

Cette section fournit des détails sur le plan d'étude, y compris la façon dont l'étude est conçue et ce que l'étude mesure.

Comment l'étude est-elle conçue ?

Détails de conception

  • Objectif principal: Traitement
  • Répartition: N / A
  • Modèle interventionnel: Affectation à un seul groupe
  • Masquage: Aucun (étiquette ouverte)

Armes et Interventions

Groupe de participants / Bras
Intervention / Traitement
Expérimental: fitusiran

The pre-fitusiran treatment period is defined as the transition period up to the first fitusiran administration. Participants will receive on demand or prophylactic treatment with intravenous clotting factor concentrates (IV CFCs) or bypassing agents (BPAs) from Month-2 until Day 1.

Fitusiran treatment period: Participants will receive subcutaneous (SC) fitusiran prophylaxis once every 2 months (Q2M) or once monthly (QM) from Day 1 until Month18.

Participants may receive IV antithrombin concentrate (ATIIIC) upon investigator's judgement.

AT FU period: Participants will be followed up until AT activity levels recover to at least 60% (per central laboratory).

In case of bleeding events participants will receive IV CFCs or BPAs. Participants may receive IV ATIIIC upon investigator's judgement.
Médicament: Fitusiran (SAR439774)

Pharmaceutical form:Solution for injection-Route of administration:Subcutaneous (SC) injection

Autres noms:
  • SAR439774, QFITLIA®
Biologique: Clotting factor concentrates (CFC) or bypassing agents (BPA)

Pharmaceutical form: Solution for injection-Route of administration: Intravenous (IV) injection

Biologique: Antithrombin concentrate (ATIIIC)

Pharmaceutical form:Solution for injection-Route of administration:Intravenous (IV) injection

Biologique: Emicizumab

Pharmaceutical form:Solution for injection-Route of administration:SC injection

Que mesure l'étude ?

Principaux critères de jugement

Mesure des résultats
Description de la mesure
Délai
Number of participants with Adverse events (AEs) during the fitusiran treatment
Délai: From Day 1 up to Month 4

Incidence, severity, and seriousness, of AEs occurred during fitusiran treatment period will be reported
From Day 1 up to Month 4

Mesures de résultats secondaires

Mesure des résultats
Description de la mesure
Délai
The peak thrombin generation (TG) - pre fitusiran treatment
Délai: From Month -2 up to Day 1

Central laboratory assessments (peak TG) will be reported for pre-fitusiran period
From Month -2 up to Day 1
The peak TG during fitusiran treatment
Délai: From Day 1 up to Month 4

Central laboratory assessments (peak TG) will be reported for fitusiran treatment period
From Day 1 up to Month 4
The antithrombin (AT) levels during pre-fitusiran treatment
Délai: From Month -2 up to Day 1

Central laboratory assessments (AT) will be reported for pre-fitusiran treatment period
From Month -2 up to Day 1
The AT levels during fitusiran prophylaxis
Délai: From Day 1 up to Month 4

Central laboratory assessments (AT) will be reported for fitusiran treatment
From Day 1 up to Month 4
Emicizumab concentrations in plasma
Délai: Up to Month 4 of fitusiran treatment

Emicizumab concentrations in plasma will be reported
Up to Month 4 of fitusiran treatment
Number of participants with AEs from Day 1 to Month 18 of fitusiran treatment
Délai: From Day 1 up to Month 18

Incidence, severity, and seriousness of AEs will be reported
From Day 1 up to Month 18
Change in participants' overall treatment satisfaction during the fitusiran treatment period assessed via the Treatment Satisfaction Questionnaire for Medication (TSQM-9) domain scores
Délai: From approximately Month -2 to Month 19 [end of study (EoS) visit]

The three domains of effectiveness, convenience, and global satisfaction will be reported
From approximately Month -2 to Month 19 [end of study (EoS) visit]
Participants' treatment preferences (via the Preference Questionnaire)
Délai: At Month 12

The Preference questionnaire Data will be reported
At Month 12
Change in adult participants' pain intensity during the fitusiran treatment period over time
Délai: From approximately Month - 2 to Month 19 (EoS) visit

The PROMIS (Patient-Reported Outcomes Measurement Information System) Pain Intensity 3a v2.0 consists of three items measuring the worst, average, and current intensity of pain with a recall period of 7 days. Each question has five response options ranging from one ('no pain') to five ('very severe').

Adult participants (age ≥ 18 years) will self-complete the PROMIS Pain Intensity 3a v2.0 questionnaire.
From approximately Month - 2 to Month 19 (EoS) visit
Change in adolescent participants' pain intensity during the fitusiran treatment period over time
Délai: From approximately Month - 2 to Month 19 (EoS) visit

The PROMIS Pediatric Numeric Rating Scale v.1.0 - Pain Intensity 1a consists of one item measuring the average intensity of pain with a recall period of 7 days. The question has ten response options ranging from one ('no pain') to ten ('worst pain').

Adolescent participants (age ≥12 years and <17 years) will self-complete the PROMIS Pediatric Pain Intensity 1a v.1.0.
From approximately Month - 2 to Month 19 (EoS) visit
Change in participants' physical functioning and physical activity during the fitusiran treatment period (via the International Physical Activity Questionnaire [IPAQ]) over time
Délai: From approximately Month - 2 to Month 19 (EoS) visit

Participants' physical functioning and physical activity data during the fitusiran treatment period will be collected via the International Physical Activity Questionnaire and reported
From approximately Month - 2 to Month 19 (EoS) visit
Change in participants' joint health (via the Hemophilia Joint Health Score [HJHS]) during the fitusiran treatment period over time
Délai: From approximately Month - 2 to Month 19 (EoS) visit

HJHS during the fitusiran treatment period will be reported
From approximately Month - 2 to Month 19 (EoS) visit
Annualized Bleeding Rate (ABR) while receiving fitusiran prophylaxis
Délai: From Month 4 up to Month 18 (14-month extension period)

The frequency of treated bleeding episodes will be reported
From Month 4 up to Month 18 (14-month extension period)

Collaborateurs et enquêteurs

C'est ici que vous trouverez les personnes et les organisations impliquées dans cette étude.

Parrainer

Sanofi

Les enquêteurs

  • Directeur d'études: Clinical Sciences & Operations, Sanofi

Publications et liens utiles

La personne responsable de la saisie des informations sur l'étude fournit volontairement ces publications. Il peut s'agir de tout ce qui concerne l'étude.

Liens utiles

Dates d'enregistrement des études

Ces dates suivent la progression des dossiers d'étude et des soumissions de résultats sommaires à ClinicalTrials.gov. Les dossiers d'étude et les résultats rapportés sont examinés par la Bibliothèque nationale de médecine (NLM) pour s'assurer qu'ils répondent à des normes de contrôle de qualité spécifiques avant d'être publiés sur le site Web public.

Dates principales de l'étude

Début de l'étude (Réel)

1 mars 2024

Achèvement primaire (Estimé)

28 mai 2028

Achèvement de l'étude (Estimé)

30 janvier 2030

Dates d'inscription aux études

Première soumission

17 novembre 2023

Première soumission répondant aux critères de contrôle qualité

17 novembre 2023

Première publication (Réel)

24 novembre 2023

Mises à jour des dossiers d'étude

Dernière mise à jour publiée (Réel)

23 septembre 2026

Dernière mise à jour soumise répondant aux critères de contrôle qualité

18 septembre 2026

Dernière vérification

1 septembre 2026

Plus d'information

Termes liés à cette étude

Termes MeSH pertinents supplémentaires

Autres numéros d'identification d'étude

  • SFY17741
  • 2022-502414-84 (Identificateur de registre: CTIS)
  • U1111-1280-7227 (Identificateur de registre: ICTRP)

Plan pour les données individuelles des participants (IPD)

Prévoyez-vous de partager les données individuelles des participants (DPI) ?

OUI

Description du régime 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

Informations sur les médicaments et les dispositifs, documents d'étude

Étudie un produit pharmaceutique réglementé par la FDA américaine

Oui

Étudie un produit d'appareil réglementé par la FDA américaine

Non

Ces informations ont été extraites directement du site Web clinicaltrials.gov sans aucune modification. Si vous avez des demandes de modification, de suppression ou de mise à jour des détails de votre étude, veuillez contacter register@clinicaltrials.gov. Dès qu'un changement est mis en œuvre sur clinicaltrials.gov, il sera également mis à jour automatiquement sur notre site Web .

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