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En studie för att testa ett läkemedel (Fitusiran) för att förebygga blödningar hos personer med svår hemofili som tidigare fått förebyggande behandling med Emicizumab

18 september 2026 uppdaterad av: Sanofi

En öppen, enarmad behandlingsstudie för att undersöka säkerheten och toleransen av att byta från Emicizumab till Fitusiran-profylax hos manliga deltagare i åldern ≥18 år med svår hemofili A, med eller utan inhibitorer

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.

Studieöversikt

Status

Rekrytering

Betingelser

Intervention / Behandling

Studietyp

Interventionell

Inskrivning (Beräknad)

20

Fas

  • Fas 4

Kontakter och platser

Det här avsnittet innehåller kontaktuppgifter för dem som genomför studien och information om var denna studie genomförs.

Studiekontakt

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

Studieorter

  • Förenta staterna
    • California
      • Los Angeles, California, Förenta staterna, 90027
        • Rekrytering
        • Children's Hospital Los Angeles- Site Number : 8400005
    • Mississippi
      • Flowood, Mississippi, Förenta staterna, 39232
        • Rekrytering
        • Southern Specialty Research- Site Number : 8400011
  • Taiwan
      • Taipei, Taiwan, 100
        • Rekrytering
        • Investigational Site Number : 1580001

Deltagandekriterier

Forskare letar efter personer som passar en viss beskrivning, så kallade behörighetskriterier. Några exempel på dessa kriterier är en persons allmänna hälsotillstånd eller tidigare behandlingar.

Urvalskriterier

Åldrar som är berättigade till studier

12 år och äldre (Barn, Vuxen, Äldre vuxen)

Tar emot friska volontärer

Nej

Kön som är behöriga för studier

Manlig

Beskrivning

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.

Studieplan

Det här avsnittet ger detaljer om studieplanen, inklusive hur studien är utformad och vad studien mäter.

Hur är studien utformad?

Designdetaljer

  • Primärt syfte: Behandling
  • Tilldelning: N/A
  • Interventionsmodell: Enskild gruppuppgift
  • Maskning: Ingen (Open Label)

Vapen och interventioner

Deltagargrupp / Arm
Intervention / Behandling
Experimentell: 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.
Läkemedel: Fitusiran (SAR439774)

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

Andra namn:
  • SAR439774, Qfitlia®
Biologisk: Clotting factor concentrates (CFC) or bypassing agents (BPA)

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

Biologisk: Antithrombin concentrate (ATIIIC)

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

Biologisk: Emicizumab

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

Vad mäter studien?

Primära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Number of participants with Adverse events (AEs) during the fitusiran treatment
Tidsram: 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

Sekundära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
The peak thrombin generation (TG) - pre fitusiran treatment
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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)
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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)

Samarbetspartners och utredare

Det är här du hittar personer och organisationer som är involverade i denna studie.

Sponsor

Sanofi

Utredare

  • Studierektor: Clinical Sciences & Operations, Sanofi

Publikationer och användbara länkar

Den som ansvarar för att lägga in information om studien tillhandahåller frivilligt dessa publikationer. Dessa kan handla om allt som har med studien att göra.

Användbara länkar

Studieavstämningsdatum

Dessa datum spårar framstegen för inlämningar av studieposter och sammanfattande resultat till ClinicalTrials.gov. Studieposter och rapporterade resultat granskas av National Library of Medicine (NLM) för att säkerställa att de uppfyller specifika kvalitetskontrollstandarder innan de publiceras på den offentliga webbplatsen.

Studera stora datum

Studiestart (Faktisk)

1 mars 2024

Primärt slutförande (Beräknad)

28 maj 2028

Avslutad studie (Beräknad)

30 januari 2030

Studieregistreringsdatum

Först inskickad

17 november 2023

Först inskickad som uppfyllde QC-kriterierna

17 november 2023

Första postat (Faktisk)

24 november 2023

Uppdateringar av studier

Senaste uppdatering publicerad (Faktisk)

23 september 2026

Senaste inskickade uppdateringen som uppfyllde QC-kriterierna

18 september 2026

Senast verifierad

1 september 2026

Mer information

Termer relaterade till denna studie

Ytterligare relevanta MeSH-villkor

Andra studie-ID-nummer

  • SFY17741
  • 2022-502414-84 (Registeridentifierare: CTIS)
  • U1111-1280-7227 (Registeridentifierare: ICTRP)

Plan för individuella deltagardata (IPD)

Planerar du att dela individuella deltagardata (IPD)?

JA

IPD-planbeskrivning

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

Läkemedels- och apparatinformation, studiedokument

Studerar en amerikansk FDA-reglerad läkemedelsprodukt

Ja

Studerar en amerikansk FDA-reglerad produktprodukt

Nej

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