Status and phase
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Treatments
Study type
Funder types
Identifiers
About
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 evaluate the efficacy of fitusiran compared to on-demand treatment with factor concentrates, as determined by:
To determine the frequency of bleeding episodes during the onset period.
To determine the safety and tolerability of fitusiran.
Full 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 participants in the fitusiran arm who enrolled in the extension study (LTE15174). The estimated total time on the study was up to 17 months for participants in the fitusiran treatment arm who did not enroll in the extension study due to the requirement for up to an additional 6 months of follow-up monitoring for antithrombin levels.
Participants who completed the study will be eligible for an open-label extension study LTE15174 (NCT03754790).
Enrollment
Sex
Ages
Volunteers
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:
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
Primary purpose
Allocation
Interventional model
Masking
120 participants in 2 patient groups
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Data sourced from clinicaltrials.gov
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