Severe hemophilia A without factor VIII inhibitors 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
Male
Inclusion criteria
Inclusion criteria: • Males with diagnosis of severe congenital hemophilia A without FVIII inhibitor (FVIII level =12-17 years the hemophilia associated caregiver burden scale (HEMOCAB) questionnaire will be answered by the caregiver • Patients with previous inhibitors should have no documented inhibitors to FVIII (i.e., =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: • Patients with FVIII inhibitors • Inherited or acquired bleeding disorder other than hemophilia A • Previous (in the past 18 months) or current treatment for thromboembolic disease (with the exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing) or signs of thromboembolic disease • Other conditions (e.g., certain autoimmune diseases) that may increase the risk of bleeding or thrombosis according to investigator’s clinical judgment • History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection • Inability to comply with the study protocol in the opinion of the investigator • Planned surgery (excluding, minor procedures such as tooth extraction or incision and drainage) during the study • Current drug or alcohol dependence • Females with severe hemophilia A
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: • To evaluate the impact of emicizumab on aspects of health-related quality of life (HRQoL);Secondary Objective: • To evaluate impact of emicizumab on physical activity (PA) • To evaluate the impact of emicizumab on joint health • To evaluate the impact of emicizumab on bleeds, and compared with previous factor VIII (FVIII) prophylaxis • To evaluate the impact of emicizumab on health status • To assess the treatment preference for emicizumab according to the emicizumab preference (EmiPref) questionnaire • To evaluate the impact of emicizumab on caregiver burden • To evaluate the safety of emicizumab in patients with severe hemophilia A without FVIII inhibitors ;Primary end point(s): 1. Change from baseline in the following domains: risk perception of recreational activities, restrictions experienced in recreational activities, preoccupation with disease, impact of treatment burden on HRQoL, and pain severity as measured by the Comprehensive Assessment Tool of Challenges in Hemophilia (CATCH) questionnaire ;Timepoint(s) of evaluation of this end point: 1 Baseline (Week 24 (MO42590)), Weeks 25 and 49 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Level of PA during study measured with an activity tracker 2. Time and intensity levels of PA as measured by the International Physical Activity Questionnaire-Short Form (IPAQ-SF) 3. Levels of PA measured with activity tracker compared with data collected from the preceding non-interventional standard of care study MO42590 4. Hemophilia Early Arthropathy Detection with ultrasound (HEAD-US) 5. Hemophilia Joint Health Score 2.1 (HJHS) 6. Number of treated bleeds over time in patients 7. Percentage of patients with zero-treated bleeds 8. Number of all bleeds over time in patients 9. Number of treated spontaneous or traumatic bleeds over time in patients 10. Number of treated joint bleeds over time in patients 11. Number of treated target joint bleeds over time in patients 12. Number of FVIII doses and total amount of units to treat a breakthrough bleed in patients 13. Change from baseline in health status as measured by the EuroQoL Five-Dimension-Five Level Questionnaire (EQ-5D-5L) 14. Patient preference for the emicizumab regimen as measured by the emicizumab preference (EmiPref) questionnaire 15. caregiver burden using Hemophilia Associated Caregiver Burden (HEMOCAB) scale (ages 12-17) 16. Incidence and severity of adverse events ;Timepoint(s) of evaluation of this end point: 1. Weeks 9-16, 25-32, and 41-48 2. Baseline (Week 1), Weeks 13, 25, 37 and 49 3. Week 1-8 (MO42590), Week 17-24 (MO42590), Week 25-32 and Weeks 41-48 4-5. Baseline (Week 1) and Weeks 49 6-12. Up to 49 Weeks 13. Baseline (Week 1), Weeks 25 and 49 14. Weeks 25 and 49 15. Baseline (Week 1) and Week 49 16. Up to Week 49 | — |
Countries
Denmark, Finland, Norway, Sweden
Contacts
Public ContactTrial Information Support Line-TISL
F. Hoffmann-La Roche Ltd
Outcome results
None listed