Hemophilia A or Hemophilia B MedDRA version: 20.0 Level: LLT Classification code 10060613 Term: Hemophilia A (Factor VIII) System Organ Class: 100000004850 MedDRA version: 20.0 Level: LLT Classification code 10060614 Term: Hemophilia B (Factor IX) System Organ Class: 100000004850
Conditions
Sponsors
Genzyme Corporation
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: Males =12 years of age. Severe hemophilia A or B with inhibitors evidenced by: a.A central laboratory measurement or documented medical record evidence of FVIII =65 years) yes F.1.3.1 Number of subjects for this age range 3
Exclusion criteria
Exclusion criteria: Patients with known co-existing bleeding disorders other than hemophilia A or B Patients with clinically significant liver disease Patients known to be HIV positive and have a CD4 count <200 cells/µL Patients with a history of arterial or venous thromboembolism Estimated glomerular filtration rate =45 mL/min/1.73m2 (using the Modification of Diet in Renal Disease [MDRD] formula) Patients with a co-existing thrombophilic disorder Patients with a history of multiple drug allergies or history of allergic reaction to an oligonucleotide or GalNAc Patients with a history of intolerance to SC injection(s) Patients with an anticipated or planned need for surgery during the study 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 |
|---|---|
| Main Objective: To evaluate the efficacy of fitusiran compared to on-demand treatment with BPAs (bypassing agents), as determined by the frequency of bleeding episodes;Secondary Objective: To evaluate the efficacy of fitusiran compared to on-demand treatment with BPAs, as determined by the frequency of spontaneous bleeding episodes, the frequency of joint bleeding episodes in patients, and health related quality of life (HRQOL) in patients receiving fitusiran.;Primary end point(s): Annualized bleeding rate (ABR) ;Timepoint(s) of evaluation of this end point: Through 9 months | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): ABR in the treatment period Annualized spontaneous bleeding rate in the efficacy period Annualized joint bleeding rate in the efficacy period Change in Haem-A-QOL physical health score and total score in the treatment period ABR in the onset period;Timepoint(s) of evaluation of this end point: Through 9 months | — |
Countries
Australia, Bulgaria, Canada, China, Denmark, France, Germany, Hungary, India, Israel, Italy, Japan, Korea, Republic of, Malaysia, Netherlands, Portugal, Russian Federation, South Africa, Spain, Taiwan, Turkey, Ukraine, United Kingdom, United States
Contacts
Public ContactProject Management
PPD Global
Outcome results
None listed