Status:
COMPLETED
A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors
Lead Sponsor:
Genzyme, a Sanofi Company
Conditions:
Hemophilia A
Hemophilia B
Eligibility:
MALE
12+ years
Phase:
PHASE3
Brief Summary
Primary Objective: -To evaluate the efficacy of fitusiran compared to on-demand treatment with factor concentrates, as determined by the frequency of bleeding episodes. Secondary Objectives: * To e...
Detailed Description
The duration of treatment with fitusiran was 9 months. The estimated total time on study, inclusive of screening, was up to 11 months for all participants in the factor on-demand arm and for participa...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
March 1 2018
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
July 14 2021
Estimated Enrollment :
120 Patients enrolled
Trial Details
Trial ID
NCT03417245
Start Date
March 1 2018
End Date
July 14 2021
Last Update
March 28 2022
Active Locations (64)
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1
Investigational Site Number 0140
Little Rock, Arkansas, United States, 72202
2
Investigational Site Number 128
Gainesville, Florida, United States, 32610
3
Investigational Site Number 103
Tampa, Florida, United States, 33607
4
Investigational Site Number 102
Chicago, Illinois, United States, 60612-3833