Hemophilia A
Conditions
Interventions
Emicizumab will be administered at 3 mg/kg Q2W for a period of 52 weeks. After
1 year of treatment, patients will continue to receive emicizumab (1.5 mg/kg
QW, 3 mg/kg Q2W, or 6 mg/kg Q4W) over a 7-
Sponsors
Roche Nederland B.V.
Eligibility
Age
2 Years to 11 Years
Inclusion criteria
Inclusion criteria: Inclusion criteria: * Age from birth to
Exclusion criteria
Exclusion criteria: Exclusion criteria * Inherited or acquired bleeding disorder other than severe hemophilia A * Use of systemic immunomodulators (e.g., interferon) at enrollment or planned use during the study * Receipt of any of the following: * An investigational drug to treat or reduce the risk of hemophilic bleeds within 5 drug-elimination half-lives of last drug administration * A non-hemophilia-related investigational drug within the last 30 days or 5 drug-elimination half-lives, whichever is shorter * An investigational drug concurrently * Current active severe bleed, such as ICH * Planned surgery during the study * History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection * Previous or current treatment for thromboembolic disease or signs of thromboembolic disease * Any hereditary or acquired maternal condition that may predispose the patient to thrombotic events * Other diseases (e.g., certain autoimmune diseases) that may increase risk of bleeding or thrombosis * Known infection with HIV, hepatitis B virus, or hepatitis C virus * Serious infection requiring antibiotics or antiviral treatments within 14 days prior to screening * Concurrent disease, treatment, abnormality in clinical laboratory tests, vital signs measurements, or physical examination findings that could interfere with the conduct of the study or that would, in the opinion of the investigator or Sponsor, preclude the patient*s safe participation in and completion of the study or interpretation of the study results * Unwillingness of the parent or caregiver to allow receipt of blood or blood products, or any standard-of-care treatment for a life-threatening condition * Any other medical, social, or other condition that may prevent adequate compliance with the study protocol in the opinion of the investigator
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The efficacy objective for this study is to evaluate the efficacy of emicizumab on the basis of the following endpoints: - Number of treated bleeds over time (i.e., treated bleed rate) - Number of all bleeds over time (i.e., all bleed rate) - Number of treated spontaneous bleeds over time (i.e., treated spontaneous bleed rate) - Number of treated joint bleeds over time (i.e., treated joint bleed rate) - 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 LTFU period | — |
Secondary
| Measure | Time frame |
|---|---|
| The safety objective for this study is to evaluate the safety of emicizumab on the basis of the following endpoints: - Incidence and severity of adverse events, with severity determined according to WHO Toxicity Grading Scale (see Appendix 5) - Incidence of thromboembolic events - Incidence of thrombotic microangiopathy (TMA) - Change from baseline in physical examination findings - Change from baseline in vital signs - Incidence of laboratory abnormalities - Incidence and severity of injection-site reactions - Incidence of adverse events leading to drug discontinuation - Incidence of severe hypersensitivity, anaphylaxis, and anaphylactoid events See protocol section 2 for Pharmacokinetic, biomarker and Immunogenicity objectives | — |
Countries
Netherlands
Outcome results
None listed