Hemophilia A MedDRA version: 20.0 Level: LLT Classification code 10053753 Term: Hemophilia A without inhibitors System Organ Class: 100000004850
Conditions
Sponsors
F. Hoffmann-La Roche Ltd
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Inclusion criteria: • Age from birth to = 3 kg at time of informed consent • Mandatory receipt of vitamin K prophylaxis according to local standard practice • Diagnosis of severe congenital hemophilia A (intrinsic FVIII level =65 years) no F.1.3.1 Number of subjects for this age range 0
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 |
|---|---|
| Main Objective: Main objectives: •To evaluate the efficacy of emicizumab •To evaluate the safety of emicizumab •To characterize the emicizumab pharmacokinetic (PK) profile •To investigate the effect of emicizumab on pharmacodynamics (PD) parameters •To evaluate the immune response to treatment ;Secondary Objective: Not applicable;Primary end point(s): Primary endpoints: 1. Number of treated bleeds over time 2. Number of all bleeds over time 3. Number of treated spontaneous bleeds over time 4. Number of treated joint bleeds over time 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 LTFU period 6. Incidence and severity of adverse events, with severity determined according to WHO Toxicity Grading Scale 7. Incidence of thromboembolic events 8. Incidence of thrombotic microangiopathy (TMA) 9. Change from baseline in physical examination findings 10. Change from baseline in vital signs 11. Incidence of laboratory abnormalities 12. Incidence and severity of injection-site reactions 13. Incidence of adverse events leading to drug discontinuation 14. Incidence of severe hypersensitivity, anaphylaxis, and anaphylactoid events 15. Plasma trough concentrations (Ctrough) of emicizumab prior to study drug administration 16. aPTT levels prior to study drug administration 17. Thrombin generation (TG) prior to study drug administration 18. Reported FVIII activity prior to study drug administration 19. FIX antigen and FX antigen (emicizumab substrates) levels prior to study drug administration ;Timepoint(s) of evaluation of this end point: 1-8. Up to 8 years 9-10. Baseline (Day 1) to 8 years 11-14. Up to 8 years 15-19. Every 2 weeks during Weeks 1-9 and every 4 weeks during Weeks 13-53 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Not applicable;Timepoint(s) of evaluation of this end point: Not applicable | — |
Countries
Australia, Austria, Belgium, Brazil, Canada, France, Germany, Israel, Italy, Netherlands, South Africa, Spain, Switzerland, Turkey, United Kingdom, United States
Contacts
Public ContactTrial Information Support Line-TISL
F. Hoffmann-La Roche Ltd
Outcome results
None listed