Emicizumab for Hemophilia A | Hemophilia A without inhibitors

Inclusion criteria

• {"criterion_text":"- 1. No history of documented FVIII inhibitor (i.e., < 0.6 BU/mL), FVIII drug-elimination half-life < 6 hours, or FVIII recovery < 66%\n- 2. Mandatory receipt of vitamin K prophylaxis according to local standard practice\n- 3. Diagnosis of severe congenital hemophilia A (intrinsic FVIII level < 1%)\n- 4. A negative test for FVIII inhibitor (i.e., < 0.6 Bethesda units [BU]/mL) locally assessed during the 2-week screening period for all patients\n- 5. Previously untreated patients (PUPs) or minimally treated patient (MTPs) (i.e., up to 5 days of exposure with hemophilia-related treatments, such as plasma-derived FVIII, recombinant FVIII, fresh frozen plasma, cryoprecipitate, or whole blood products)\n- 6. Documentation of the details of the hemophilia-related treatments received since birth and documentation of the details of the bleeding episodes since birth"}

Exclusion criteria

• {"criterion_text":"- 1. Inherited or acquired bleeding disorder other than severe hemophilia A\n- 2. Use of systemic immunomodulators (e.g., interferon) at enrollment or planned use during the study\n- 3. Current active severe bleed, such as intracranial hemorrhage (ICH)\n- 4. History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection\n- 5. Patients who are at high risk for thrombotic microangiopathy (TMA) (e.g., have a previous medical or family history of TMA, such as thrombotic thrombocytopenic purpura, atypical hemolytic uremic syndrome) in the investigator's judgment\n- 6. Previous or current treatment for thromboembolic disease or signs of thromboembolic disease"}

Primary endpoints

• {"endpoint_text":"- 1. Number of treated bleeds over time","definition_or_measurement_approach":""}
• {"endpoint_text":"- 2. Number of all bleeds over time","definition_or_measurement_approach":""}
• {"endpoint_text":"- 3. Number of treated spontaneous bleeds over time","definition_or_measurement_approach":""}
• {"endpoint_text":"- 4. Number of treated joint bleeds over time","definition_or_measurement_approach":""}
• {"endpoint_text":"- 5. Joint health, as assessed through use of the Hemophilia Joint Health Score (HJHS) and magnetic resonance imaging (MRI) score of specific joints at specified timepoints only during the 7-year long-term follow-up (LTFU) period","definition_or_measurement_approach":"Assessed using Hemophilia Joint Health Score (HJHS) and MRI scoring of specified joints at specified timepoints during the 7-year LTFU period."}
• {"endpoint_text":"- 6. Incidence and severity of adverse events, with severity determined according to WHO Toxicity Grading Scale","definition_or_measurement_approach":"Severity determined according to WHO Toxicity Grading Scale."}
• {"endpoint_text":"- 7. Incidence of thromboembolic events and thrombotic microangiopathy (TMA)","definition_or_measurement_approach":""}
• {"endpoint_text":"- 8. Change from baseline in physical examination findings","definition_or_measurement_approach":""}
• {"endpoint_text":"- 9. Change from baseline in vital signs","definition_or_measurement_approach":""}
• {"endpoint_text":"- 10. Incidence of laboratory abnormalities","definition_or_measurement_approach":""}
• {"endpoint_text":"- 11. Incidence and severity of injection-site reactions","definition_or_measurement_approach":""}
• {"endpoint_text":"- 12. Incidence of adverse events leading to study drug discontinuation","definition_or_measurement_approach":""}
• {"endpoint_text":"- 13. Incidence of severe hypersensitivity, anaphylaxis, and anaphylactoid events","definition_or_measurement_approach":""}
• {"endpoint_text":"- 14. Plasma trough concentrations (Ctrough) of emicizumab prior to study drug administration","definition_or_measurement_approach":"Measurement: plasma trough concentration (Ctrough) sampling prior to study drug administration."}
• {"endpoint_text":"- 15. Effect of emicizumab on PD parameters, including aPTT, thrombin generation (TG), and reported FVIII activity, as well as FIX antigen and FX antigen (emicizumab substrates) levels prior to study drug","definition_or_measurement_approach":"PD parameters measured include aPTT, thrombin generation (TG), reported FVIII activity, and FIX and FX antigen levels prior to study drug."}

Secondary endpoints

• {"endpoint_text":"- Not applicable","definition_or_measurement_approach":"Not applicable"}

Austria

Latest Decision Or Authorization Date

08-04-2024

Number Of Sites

1

Number Of Participants

4

Italy

Latest Decision Or Authorization Date

20-03-2024

Number Of Sites

4

Number Of Participants

7

Spain

Latest Decision Or Authorization Date

06-03-2024

Number Of Sites

3

Number Of Participants

6

Germany

Latest Decision Or Authorization Date

07-03-2024

Number Of Sites

1

Number Of Participants

3

France

Latest Decision Or Authorization Date

08-03-2024

Number Of Sites

1

Number Of Participants

9

Belgium

Latest Decision Or Authorization Date

21-03-2024

Number Of Sites

2

Number Of Participants

2

Primary sponsor

Full Name

F. Hoffmann-La Roche AG

Organisation Type

Pharmaceutical company

Country Of Registered Address

Switzerland

Third parties

• {"country":"United States","full_name":"Worldcare Clinical LLC","duties_or_roles":"sponsorDuties code 4","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United States","full_name":"Labcorp Central Laboratory Services LP","duties_or_roles":"sponsorDuties code 4","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"sponsorDuties code 7","organisation_type":"Pharmaceutical company"}
• {"country":"Belgium","full_name":"S-Clinica","duties_or_roles":"sponsorDuties code 15 (Randomization)","organisation_type":"Pharmaceutical company"}