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Active (not recruiting) Phase 3 NCT03754790

Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX

Phase 3 – large-scale trial before approval
Conditions: Hemophilia

Sponsor: Genzyme, a Sanofi Company

trial.available_in: БГ
Overview
Primary Objective: To characterize the long-term safety and tolerability of fitusiran Secondary Objectives: * To characterize the efficacy and long-term efficacy of fitusiran as assessed by the frequency of: * Bleeding episodes * Spontaneous bleeding episodes * Joint bleeding episodes * To characterize the effects of fitusiran on health-related quality of life (HRQOL) measures in participants ≥17 years of age
Description
The estimated total time on the study for a participant is up to 90 months (including screening, treatment period and safety follow up period).
Who can participate
Inclusion criteria : * Participant must be at least 12 years of age inclusive, at the time of signing the informed consent * Participants with severe hemophilia A or B who have completed a Phase 3 fitusiran clinical trial * Male * Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol. In countries where legal age of majority is above 18 years, a specific ICF must also be signed by the participant's legally authorized representative Exclusion criteria: * Completion of a surgical procedure within 14 days prior to screening, or currently receiving additional factor concentrate or BPA infusion for postoperative hemostasis * Current participation in immune tolerance induction treatment (ITI) * Current use of factor concentrates or bypassing agents (BPAs) as regularly administered prophylaxis designed to prevent spontaneous bleeding episodes except for participants requiring factor concentrates or BPAs prophylaxis during the study dosing pause period * Use of compounds other than factor concentrates or BPAs for hemophilia treatment * Current or prior participation in a gene therapy trial * Alanine aminotransferase (ALT) and/or Aspartate aminotransferase (AST) \>1.5 × upper limit of normal reference range (ULN) for patients who are naïve to fitusiran at study start; ALT and/or AST \> 5 x ULN for patients who were in the fitusiran arm in the parent study * Additional exclusions for participants not currently participating in a fitusiran trial at the time of enrollment in the lower dose cohort: * Clinically significant liver disease * History of arterial or venous thromboembolism The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Locations 19
Australia (1)
Investigational Site Number : 0360001
Camperdown , New South Wales
Canada (1)
Investigational Site Number : 1240001
Montreal , Quebec
China (1)
Investigational Site Number : 1560004
Beijing
Denmark (1)
Investigational Site Number : 2080001
Copenhagen
France (1)
Investigational Site Number : 2500002
Lyon
Hungary (1)
Investigational Site Number : 3480002
Budapest
India (1)
Investigational Site Number : 3560001
Bangalore
Ireland (1)
Investigational Site Number : 3720001
Crumlin , Dublin
Israel (1)
Investigational Site Number : 3760001
Ramat Gan
Italy (1)
Investigational Site Number : 3800002
Milan , Lombardy
Japan (1)
Investigational Site Number : 3920002
Nishinomiya , Hyōgo
Malaysia (1)
Investigational Site Number : 4580003
Ampang
South Africa (1)
Investigational Site Number : 7100002
Port Elizabeth
South Korea (1)
Investigational Site Number : 4100001
Busan , Busan
Taiwan (1)
Investigational Site Number : 1580002
Changhua
Turkey (1)
Investigational Site Number : 7920002
Adana
Ukraine (1)
Investigational Site Number : 8040001
Kyiv
United Kingdom (1)
Investigational Site Number : 8260001
London , London, City of
United States (1)
Phoenix Childrens Hospital Site Number : 8400009
Phoenix , Arizona
Technical details
Status
Active (not recruiting)
Phase
Phase 3
Study type
INTERVENTIONAL
Sex
Male only
Minimum age
12 Years
Healthy volunteers
No
Start date
09.01.2019
Completion date
05.11.2026
Registry ID
NCT03754790
Source
anzctr
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Information is automatically extracted from ClinicalTrials.gov. Consult your doctor before taking action.