Health Condition 1: null- â?¢Hemophilia A â?¢Hemophilia B Health Condition 2: D689- Coagulation defect, unspecified
Conditions
Interventions
Intervention1: Fitusiran (SAR439774 [formerly Alnylam ALN-AT3SC]): In Fitusiran treatment arm, fitusiran 80 mg administered subcutaneously as prophylaxis once monthly, with use of on demand bypassing
Sponsors
Sanofi Genzyme
PPD Pharmaceuticals India Private Limited
Eligibility
Inclusion criteria
Inclusion criteria: 1. Males, >=12 years of age 2. Severe hemophilia A or B with inhibitors â?¦(Severity confirmed by a central laboratory where FVIII level is =0.6 BU/mL or as evidenced by medical records) 3. A minimum of 6 bleeding episodes requiring bypassing agent treatment within the last 6 months prior to Screening 4. Willing and able to comply with the study requirements and to provide written informed consent and assent
Exclusion criteria
Exclusion criteria: 1. Known co-existing bleeding disorders other than hemophilia A or B 2. Antithrombin (AT) activity 3. Co-existing thrombophilic disorder 4. Clinically significant liver disease 5. Active HCV infection 6. HIV positive with a CD4 count of 7. History of arterial or venous thromboembolism 8. Inadequate renal function 9. History of multiple drug allergies or history of allergic reaction to an oligonucleotide or N-Acetylgalactosamine (GalNAc) 10. History of intolerance to SC injection(s) 11. Any other conditions or comorbidities that would make the patient unsuitable for enrollment or could interfere with participation in or completion of the study, per Investigator judgement
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Annualized bleeding rate (ABR)Timepoint: Annualized bleeding rate (ABR): 9 months | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Annualized spontaneous bleeding rate 2. Annualized joint bleeding rate 3. Quality of Life (QOL) as measured by Haem-A-QOL Questionnaire score on a scale of 0-100 with higher scores representing greater impairmentTimepoint: 9 months 9 months 9 months | — |
Countries
Australia, Bulgaria, Canada, China, Denmark, France, Germany, Hungary, India, Israel, Italy, Japan, Malaysia, Netherlands, Portugal, Republic of Korea, Russian Federation, South Africa, Spain, Taiwan, Turkey, Ukraine, United Kingdom, United States of America
Contacts
Public ContactRashmi Chitgupi
PPD Pharmaceutical Development India Private Limited
Outcome results
None listed