Brief Title
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
Official Title
An Open-label, 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
Brief Summary
Primary Objective: To characterize the long-term safety and tolerability of fitusiran Secondary Objectives: - To characterize the long-term efficacy of fitusiran as assessed by the frequency of: - Bleeding episodes - Spontaneous bleeding episodes - Target joint bleeding episodes - To characterize the effects of fitusiran on health-related quality of life (HRQOL) measures in participants ≥17 years of age
Detailed Description
The estimated total time on study for each participant is up to 55 months which consists of a screening period of up to 30 days, an open label treatment period of up to 48 months and a follow up period of up to 6-month after the last dose of fitusiran
Study Phase
Phase 3
Study Type
Interventional
Primary Outcome
Number of patients with adverse events
Secondary Outcome
Annualized bleeding rate (ABR)
Condition
Hemophilia
Intervention
Fitusiran
Study Arms / Comparison Groups
Fitusiran
Description: Fitusiran fixed dose, once-monthly subcutaneous injection for 48 months
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
244
Start Date
January 9, 2019
Completion Date
January 2026
Primary Completion Date
January 2026
Eligibility Criteria
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 - 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 The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Gender
Male
Ages
12 Years - N/A
Accepts Healthy Volunteers
No
Contacts
Clinical Sciences & Operations, 800-633-1610, [email protected]
Location Countries
Australia
Location Countries
Australia
Administrative Informations
NCT ID
NCT03754790
Organization ID
LTE15174
Secondary IDs
2018-002880-25
Responsible Party
Sponsor
Study Sponsor
Genzyme, a Sanofi Company
Study Sponsor
Clinical Sciences & Operations, Study Director, Sanofi
Verification Date
November 2020