Clareo Health | 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
Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Genzyme, a Sanofi Company
The estimated total time on the study for a participant is up to 90 months (including screening, treatment period and safety follow up period).
Age
12–any
Sex
MALE
Healthy volunteers
Not accepted
You may be eligible if
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
You may not be if
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.
