Health Condition 1: null- Hemophilia A patients with inhibitors Health Condition 2: D66- Hereditary factor VIII deficiency
Conditions
Interventions
Intervention1: Emicizumab: 3 mg/kg/week subcutaneously for 4 weeks when initiating treatment, followed by 1.5 mg/kg/week subcutaneously for the remainder of the 2-year treatment period
Control Interve
Sponsors
F HoffmannLa Roche Ltd
Roche Products India Pvt Ltd
Eligibility
Inclusion criteria
Inclusion criteria: 1. Signed Informed Consent Form 2. Willingness and ability to comply with scheduled visits, treatment plans, laboratory tests, and other study procedures, including the PRO questionnaires and bleed diaries through the use of an electronic device, as per the investigatorâ??s judgment 3. Aged 12 years or older at the time of informed consent 4. Body weight >=40 kg at the time of screening 5. Diagnosis of congenital hemophilia A with persistent inhibitors against FVIII 6. Adequate hematologic function 7. Adequate hepatic function 8. Adequate renal function
Exclusion criteria
Exclusion criteria: 1. Inherited or acquired bleeding disorder other than hemophilia A 2. Ongoing (or plan to receive during the study) ITI therapy (prophylaxis regimens with FVIII and/or bypassing agents must be discontinued prior to enrollment). 3. History of illicit drug or alcohol abuse within 12 months prior to screening, as per the investigatorâ??s judgment 4. Other conditions (e.g., certain autoimmune diseases) that may increase the risk of bleeding or thrombosis 5. History of clinically significant hypersensitivity reaction associated with monoclonal antibody therapies 6. Known human immunodeficiency virus (HIV) infection 7. Concurrent disease, treatment, or abnormality in clinical laboratory tests that could interfere with the conduct of the study or that would, in the opinion of the investigator or Sponsor, preclude the patientâ??s safe participation in and completion of the study or interpretation of the study results
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To capture the incidence and severity of adverse events, including thromboembolic, TMA, systemic hypersensitivity, anaphylaxis, and anaphylactoid events.Timepoint: The primary safety analysis will occur at the end of the study. | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. To evaluate the efficacy of prophylactic administration of emicizumab on the basis of the number of bleeds over time 2. To evaluate the HRQoL of patients according to Haem-A-QoL (â?¥18 y) or Haemo-QoL-SF (ages 12â??17) scores over time 3. To evaluate the health status of patients according to EQ-5D-5L scores over time 4. To assess patient preference for the emicizumab regimen compared with the previous regimen usedTimepoint: 1. The patients will record the information on bleed or hemophilia medication use, at least every week. 2. HRQoL (using the Haem-A-QoL or the Haemo-QoL-SF), health status (using the EQ-5D-5L) will be assessed at week 1, 3 month, 6 month, 12 month, 18 month & at study completion. The HRQoL scale scores for all patients will be evaluated after 6 months of treatment. 3. Patient treatment preference will be assessed at 3 month | — |
Countries
Australia, Belgium, Brazil, Canada, Colombia, Denmark, Finland, Germany, Guatemala, Hungary, India, Israel, Italy, Mexico, Netherlands, Panama, Poland, Portugal, Romania, Russian Federation, Saudi Arabia, Spain, Sweden, Switzerland, United Kingdom
Contacts
Public ContactJitendra Soni
Roche Products (India) Pvt. Ltd.
Outcome results
None listed