Status:
COMPLETED
A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors
Lead Sponsor:
Genzyme, a Sanofi Company
Conditions:
Hemophilia A
Hemophilia B
Eligibility:
MALE
12+ years
Phase:
PHASE3
Brief Summary
The purpose of this study was to determine the frequency of bleeding episodes in participants receiving fitusiran as prophylactic treatment of hemophilia compared to participants who were assigned to ...
Detailed Description
The duration of treatment with fitusiran was 9 months. The estimated total time on study, inclusive of screening, for each participant was up to 11 months for all participants who enroll in the extens...
Eligibility Criteria
Inclusion
- Males, greater than or equal to (\>=) 12 years of age.
- Severe hemophilia A or B with inhibitors.
- (Severity confirmed by a central laboratory where coagulation factor VIII (FVIII) level was less than (\<)1% or factor IX (FIX) level was less than or equal to \[\<=\]2% at Screening; Inhibitors defined as inhibitor titer of \>=0.6 Bethesda units per milliliter \[BU/mL\] or as evidenced by medical records).
- A minimum of 6 bleeding episodes requiring BPA treatment within the last 6 months prior to screening.
- Willing and able to comply with the study requirements and to provide written informed consent and assent.
Exclusion
- Known co-existing bleeding disorders other than hemophilia A or B.
- 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.
- 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 judgement.
Key Trial Info
Start Date :
February 14 2018
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
June 23 2021
Estimated Enrollment :
60 Patients enrolled
Trial Details
Trial ID
NCT03417102
Start Date
February 14 2018
End Date
June 23 2021
Last Update
March 28 2022
Active Locations (58)
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1
Investigational Site Number 0117
Phoenix, Arizona, United States, 85016
2
Investigational Site Number 0139
Los Angeles, California, United States, 90027
3
Investigational Site Number 0135
Orange, California, United States, 92868
4
Investigational Site Number 0137
San Diego, California, United States, 92123