A Study to Test a Medicine (Fitusiran) Injected Under the Skin for Preventing Bleeding Episodes in Male Adolescent or Adult Participants With Severe Hemophilia

Condition(s) studied

Investigational drug(s) / intervention(s)

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

Clotting factor concentrates (CFC) or bypassing agents (BPA): * Coagulation factor VIII (ATC code: B02BD02) * Coagulation factor IX (ATC code: B02BD04) * Coagulation factor VIIa (ATC code: B02BD08) * Factor VIII inhibitor bypassing activity (ATC code: B02BD03) Pharmaceutical form: Solution for infusion Route of administration: Intravenous (IV)

Antithrombin concentrate (ATIIIC): Antithrombin III (ATC code: B01AB02) Pharmaceutical form: Solution for infusion Route of administration: Intravenous (IV)

Study summary

This is a multicenter, multinational, open-label, one-way cross-over, Phase 3, single-arm study for treatment of hemophilia.

The purpose of this study is to measure the frequency of treated bleeding episodes with fitusiran in male adult and adolescent (≥12 years old) participants with hemophilia A or B, with or without inhibitory antibodies to factor VIII or IX who have switched from their prior standard of care treatment.

The total study duration will be up to approximately 50 months (200 weeks, 1 study month is equivalent to 4 weeks) and will include:

* A screening period up to approximately 60 days,
* A standard of care (SOC) period of approximately 6 study months (24 weeks),
* A fitusiran treatment period of approximately 36 study months (144 weeks),
* An antithrombin (AT) follow-up period of approximately 6 study months (24 weeks) but may be shorter or longer depending on individual participants AT recovery.

The frequency for telephone visits will be approximately every 2 weeks. For site visits the frequency will be approximately every 8 weeks during the SOC period and approximately every 4 weeks during the fitusiran treatment period. If applicable and if allowed by local regulation, home and/or remote visits may be conducted during the study

Eligibility

Primary outcome measure(s)

• Annualized bleeding rate (ABR) in the fitusiran primary efficacy period — Day 169 to Day 505 (since the first dose of fitusiran)
  A bleeding episode is defined as any occurrence of hemorrhage that requires administration of CFCs or BPAs, e.g., hemarthrosis, muscle, or mucosal bleeding.

Trial sites (50)

+ 10 more sites — see the full list on the official registry below.

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