A Study to Test a Medicine (Fitusiran) for Preventing Bleeds in People With Severe Hemophilia Who Previously Received Preventive Treatment With Emicizumab

Part of paid clinical trials in Los Angeles, California.

Sponsor
Sanofi
Study ID
NCT06145373
Phase
PHASE4
Status
Recruiting
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Conditions

  • Hemophilia A

Eligibility Criteria

Sex
MALE
Age
12 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • Fitusiran (SAR439774) — DRUG
    Pharmaceutical form:Solution for injection-Route of administration:Subcutaneous (SC) injection
  • Clotting factor concentrates (CFC) or bypassing agents (BPA) — BIOLOGICAL
    Pharmaceutical form: Solution for injection-Route of administration: Intravenous (IV) injection
  • Antithrombin concentrate (ATIIIC) — BIOLOGICAL
    Pharmaceutical form:Solution for injection-Route of administration:Intravenous (IV) injection
  • Emicizumab — BIOLOGICAL
    Pharmaceutical form:Solution for injection-Route of administration:SC injection

Study Details

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.

Key Dates

Start date
Mar 1, 2024
Status verified
Jun 2026
Primary completion
May 28, 2028
Completion
Jan 30, 2030

Study Design

Enrollment
20 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: 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.

Primary Outcome Measure

Number of participants with Adverse events (AEs) during the fitusiran treatment [ Time Frame: From Day 1 up to Month 4 ]

Central Contacts

  • Trial Transparency email recommended (Toll free for US & Canada)
    800-633-1610
    [email protected]

Locations (1)

FacilityCityStateZIPSite coordinators
Children's Hospital Los Angeles- Site Number : 8400005Los AngelesCalifornia90027-

Find similar trials in Los Angeles, CA

By research site
Children's Hospital Los Angeles· Los Angeles, CA

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