Haemophilia 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
All
Inclusion criteria
Inclusion criteria: - Aged 12 years or older at the time of informed consent - Body weight >= 40 kg at the time of screening - Diagnosis of severe congenital haemophilia A (intrinsic FVIII level = 5 bleeds in the last 24 weeks prior to study entry - Patients who were on FVIII prophylaxis for at least the last 24 weeks, can be enrolled regardless of the number of bleeds during this period. Eligibility will be based on investigator's attestation of adequate prophylaxis regimen. - At least 40 patients who were on FVIII prophylaxis pre-enrolment will have been enrolled for minimum of 24 weeks in Study BH29768 (non-interventional) - Adequate haematologic function, defined as platelet count >= 100,000/ microliter and haemoglobin >= 8 gram/decilitre (4.97 millimoles/liter) at the time of screening - Adequate hepatic and renal function - Agreement to remain abstinent or use contraceptive methods specified in the study Are the trial subjects under 18? yes Number of subjects for this age range: 7 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 140 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 5
Exclusion criteria
Exclusion criteria: - Inherited or acquired bleeding disorder other than haemophilia A - Previous (in the past 12 months) or current treatment for thromboembolic disease (except 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 risk of bleeding or thrombosis - Known human immunodeficiency virus infection with CD4 count = 48 weeks of non-therapy induced amenorrhea) or surgically sterile must have a negative pregnancy test result within 7 days prior to initiation of study drug
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: •To evaluate the efficacy of prophylactic emicizumab (1.5 mg/kg/week or 3 mg/kg/2weeks) compared with no prophylaxis in patients with haemophilia A without FVIII inhibitors on the basis of the number of bleeds over time (primary endpoint is based on treated bleeds).;Secondary Objective: • To evaluate the efficacy of prophylactic emicizumab on the basis of number of bleeds over time compared with the patient's historical bleed rate (treated and all bleeds) • To evaluate the efficacy of prophylactic emicizumab (1.5 mg/kg/week or 3 mg/kg/2weeks) on the basis of: - All bleeds over time - Spontaneous bleeds over time (spontaneous bleed rate) - Joint bleeds over time - Target joint bleeds over time - Health-related quality of life (HRQoL) of patients according to Haem A QoL (aged >= 18) or Haemo-QoL-Short Form (aged 12-17) scores after 24 weeks - Health status of patients according to EuroQoL Five Dimension Five Levels Questionnaire (EQ-5D-5L) scores after 24 weeks • Maintaining adequate control of bleeding in patients previously treated with factor VIII prophylaxis by evaluation of the bleed rate (treated and all bleeds) • To evaluate overall safety of prophylactic emicizumab in patients with haem. A without inhibitors • To evaluate pharmacokinetic of emicizumab;Primary end point(s): 1. Number of bleeds over time (i.e., bleed rate) ;Timepoint(s) of evaluation of this end point: 1. Up to 6 years | — |
Secondary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: 1-20. Up to 6 years;Secondary end point(s): Efficacy: 1. Change in the number of bleeds over time compared with the patient's historical bleed rate over the last 24 weeks prior to study entry 2. All bleeds over time (treated and not treated) 3. Spontaneous bleeds over time (spontaneous bleed rate) 4. Joint bleeds over time 5. Target joint bleeds over time 6. HRQoL of patients according to Haem-A-QoL (aged >=18) or Haemo- QoL-Short Form (aged 12-17) scores 7. Health status of patients according to EQ-5D-5L scores 8. Maintaining adequate control of bleeding by evaluation of the bleed rate in patients previously on factor VIII prophylaxis Safety: 9. Incidence of adverse events 10. Incidence of thromboembolic events 11. Incidence of new physical examination abnormalities 12. Incidence of new vital signs abnormalities 13. Incidence of laboratory abnormalities 14. Incidence injection-site reactions 15. Incidence of adverse events leading to drug discontinuation 16. Incidence of severe hypersensitivity, anaphylaxis, or anaphylactoid reactions 17. Incidence and severity of thrombotic microangiopathy 18. Incidence and clinical significance of anti-emicizumab antibodies 19. Incidence of de novo development of FVIII inhibitors in patients receiving emicizumab prophylaxis Pharmacokinetic (PK): 20. Trough plasma concentrations of emicizumab | — |
Countries
Australia, Costa Rica, France, Germany, Ireland, Italy, Japan, Korea, Republic of, Poland, South Africa, Spain, Taiwan, United Kingdom, United States
Contacts
Public ContactTrial Information Support Line-TISL
F.Hoffmann-La Roche Ltd
Outcome results
None listed