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

JRCT ID: jRCT2080225111

Registered date:04/03/2020

Basic Information

• Recruitment status: completed
• Health condition(s) or Problem(s) studied: Hemophilia
• Date of first enrollment: 10/04/2020
• Target sample size: 244
• Countries of recruitment: Australia,Canada,China,Denmark,France,Germany,Hungary,India,Ireland,Israel,Italy,Republic of Korea,Malaysia,South Africa,Taiwan,Turkey,Ukraine,United Kingdom,United States,Japan
• Study type: Interventional

Intervention(s)

Drug: Fitusiran
Pharmaceutical form: solution for injection Route of administration: subcutaneous

Outcome(s)

Primary Outcome

1. Number of participants with treatment emergent adverse events (TEAEs)
[Time Frame: from study baseline (day 1) up to maximum 88 months]
The number of participants experiencing any TEAEs, serious TEAEs, discontinuation due to TEAEs and death will be reported

Secondary Outcome

1. Annualized bleeding rate (ABR)
[Time frame: from first ever dose of fitusiran (day 1) up to maximum 78 months]
Annualized bleeding rate (ABR) in the treatment period

2. Annualized spontaneous bleeding rate
[Time frame: from first ever dose of fitusiran (day 1) up to maximum 78 months]
Annualized spontaneous bleeding rate in the treatment period

3. Annualized joint bleeding rate
[Time frame: from first ever dose of fitusiran (day 1) up to maximum 78 months]
Annualized joint bleeding rate in the treatment period

4. Changes in Haem-A-quality of life (QoL) score
[Time frame: from first ever dose of fitusiran (day 1) up to maximum 78 months]
Change in Haem-A-QoL physical health score in the treatment period (in participants >=17 years of age) from first ever dose of fitusiran (day 1) to month 48

Key inclusion & exclusion criteria

• Age minimum: >= 12age old
• Age maximum: Not applicable
• Gender: Male

Include 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

Exclude 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 x upper limit of normal reference range (ULN) for patients who are naive 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

Related Information

• Primary Sponsor: Sanofi K.K.
• Secondary Sponsor: -
• Source(s) of Monetary Support: NA
• Secondary ID(s): NCT03754790,2018-002880-25,