Hemophilia A or Hemophilia B MedDRA version: 20.0 Level: LLT Classification code 10060613 Term: Hemophilia A (Factor VIII) System Organ Class: 100000011915 MedDRA version: 20.0 Level: LLT Classification code 10060614 Term: Hemophilia B (Factor IX) System Organ Class: 100000011913
Conditions
Interventions
Product Name: fitusiran
Product Code: ALN-AT3SC
Pharmaceutical Form: Solution for injection
INN or Proposed INN: fitusiran
CAS Number: 1609016-97-8
Current Sponsor code: ALN-AT3SC
Concentration unit:
Sponsors
Alnylam Pharmaceuticals, Inc.
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: Males =12 years of age. Severe hemophilia A or B (evidenced by a central laboratory FVIII =65 years) yes F.1.3.1 Number of subjects for this age range 5
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 characterize the frequency of bleeding episodes while receiving fitusiran treatment, relative to the frequency of bleeding episodes while receiving factor or bypassing agent prophylaxis.;Secondary Objective: To characterize the frequency of spontaneous bleeding episodes, the frequency of joint bleeding episodes in patients, and health related quality of life (HRQOL) in patients while receiving fitusiran treatment, relative to receiving factor or bypassing agent prophylaxis.;Primary end point(s): Annualized bleeding rate (ABR);Timepoint(s) of evaluation of this end point: Through 13 months | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Annualized spontaneous bleeding rate Annualized joint bleeding rate Haem-A-QOL score;Timepoint(s) of evaluation of this end point: Through 13 months | — |
Countries
Australia, Brazil, Bulgaria, Canada, China, Denmark, France, Germany, Hungary, Ireland, Israel, Italy, Japan, Korea, Republic of, Malaysia, Netherlands, Portugal, Russian Federation, South Africa, Spain, Switzerland, Taiwan, Turkey, Ukraine, United Kingdom, United States
Contacts
Public ContactDorota Jusza
PPD Global
Outcome results
None listed