A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors
Hemophilia A, Hemophilia B
About this trial
This is an interventional treatment trial for Hemophilia A focused on measuring RNAi therapeutic, Hemophilia A, Hemophilia B, Hemophilia A, Severe, Hemophilia B, Severe, Blood Coagulation Disorders, Inherited, Blood Coagulation Disorders, Hematologic Diseases, Coagulation Protein Disorders, Hemorrhagic Disorders, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, Factor VIII, Factor IX, Coagulants, Fitusiran
Eligibility Criteria
Inclusion Criteria:
- Males, >=12 years of age.
Severe hemophilia A or B without inhibitors.
- Severity confirmed by a central laboratory where FVIII level was less than (<) 1 percent (%) or Factor IX (FIX) level was less than or equal to (<=) 2% at Screening.
On-demand use of factor concentrate to manage bleeding episodes for at least the last 6 months prior to Screening, and meet each of the following criterion:
- Nijmegen modified Bethesda assay inhibitor titer of <0.6 Bethesda units per milliliter (BU/mL) at Screening.
- No use of Bypassing agents to treat bleeding episodes for at least the last 6 months prior to Screening.
- No history of immune tolerance induction therapy within the last 3 years prior to Screening.
- A minimum of 6 bleeding episodes requiring factor concentrate treatment within the last 6 months prior to Screening.
- Willing and complied with the study requirements and to provide written informed consent and assent.
Exclusion Criteria:
- Known co-existing bleeding disorders other than hemophilia A or B, i.e., Von Willebrand's disease, additional factor deficiencies, or platelet disorders.
- Antithrombin (AT) activity <60% at Screening.
- Co-existing thrombophilic disorder.
- Clinically significant liver disease.
- Active hepatitis C virus infection.
- HIV positive with a cluster of differentiation-4 count of <200 cells/microliter.
- History of arterial or venous thromboembolism.
- Inadequate renal function.
- History of multiple drug allergies or history of allergic reaction to an oligonucleotide or N-Acetylgalactosamine (GalNAc).
- History of intolerance to SC injection(s).
- Any other conditions or comorbidities that would make the participant unsuitable for enrollment or could interfere with participation in or completion of the study, per Investigator judgment.
Sites / Locations
- Investigational Site Number 0140
- Investigational Site Number 128
- Investigational Site Number 103
- Investigational Site Number 102
- Investigational Site Number 119
- Investigational Site Number 125
- Investigational Site Number 111
- Investigational Site Number 110
- Investigational Site Number 6101
- Investigational Site Number 6103
- Investigational Site Number 6104
- Investigational Site Number 8604
- Investigational Site Number 8602
- Investigational Site Number 8605
- Investigational Site Number 8603
- Investigational Site Number 8601
- Investigational Site Number 4501
- Investigational Site Number 3303
- Investigational Site Number 3305
- Investigational Site Number 3301
- Investigational Site Number 4904
- Investigational Site Number 4905
- Investigational Site Number 4906
- Investigational Site Number 3602
- Investigational Site Number 9102
- Investigational Site Number 9104
- Investigational Site Number 9106
- Investigational Site Number 9109
- Investigational Site Number 9108
- Investigational Site Number 9111
- Investigational Site Number 9103
- Investigational Site Number 9105
- Investigational Site Number 9701
- Investigational Site Number 3904
- Investigational Site Number 8105
- Investigational Site Number 8104
- Investigational Site Number 8201
- Investigational Site Number 8202
- Investigational Site Number 8204
- Investigational Site Number 6004
- Investigational Site Number 6002
- Investigational Site Number 6003
- Investigational Site Number 2701
- Investigational Site Number 2702
- Investigational Site Number 3402
- Investigational Site Number 8807
- Investigational Site Number 8805
- Investigational Site Number 8804
- Investigational Site Number 8801
- Investigational Site Number 8808
- Investigational Site Number 9002
- Investigational Site Number 9004
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- Investigational Site Number 9005
- Investigational Site Number 9003
- Investigational Site Number 9009
- Investigational Site Number 9006
- Investigational Site Number 8001
- Investigational Site Number 8003
- Investigational Site Number 8002
- Investigational Site Number 8005
- Investigational Site Number 4402
- Investigational Site Number 4407
- Investigational Site Number 4401
Arms of the Study
Arm 1
Arm 2
Experimental
Experimental
Factor On-demand
Fitusiran 80 mg Prophylaxis
Participants received on-demand factor concentrates (as needed, for episodic bleeding episodes, and not on a regular regimen intended to prevent spontaneous bleeding) per Investigator discretion for the treatment of breakthrough bleeding episodes from Day 1 up to a total of 9 months.
Participants received open-label fitusiran 80 milligram (mg) administered subcutaneously (SC) as prophylaxis once monthly from Day 1 up to a total of 9 months. Participants received on-demand factor concentrates (per investigator's discretion and within bleeding dosing guidelines) for the treatment of breakthrough bleeding episodes.