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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
EU CTIS ID: 2023-508884-59-00
What this study is testing
To characterize the long-term safety and tolerability of fitusiran
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 01. Participant must be at least 12 years of age inclusive, at the time of signing the informed consent
- 02. Participants with severe hemophilia A or B who have completed a Phase 3 fitusiran clinical trial
- 03. Male
- 04. Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF and in this 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 likely can't join if
- 01. Completion of a surgical procedure within 14 days prior to screening, or currently receiving additional factor concentrate or BPA infusion for postoperative hemostasis
- 02. Current participation in tolerance induction treatment (ITI)
- 03. Current use of factor concentrates or BPAs as regularly administered prophylaxis designed to prevent spontaneous bleeding episodes. However, participants requiring factor concentrates or BPAs prophylaxis during the study dosing pause period
- 04. Use of compounds other than factor concentrates or BPAs for hemophilia treatment
- 05. Current or prior participation in a gene therapy trial
- 06. ALT and/or 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× ULN for patients who were in the fitusiran arm in the parent study.
See the full eligibility criteria
- 01. Participant must be at least 12 years of age inclusive, at the time of signing the informed consent
- 02. Participants with severe hemophilia A or B who have completed a Phase 3 fitusiran clinical trial
- 03. Male
- 04. Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF and in this 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.
- 01. Completion of a surgical procedure within 14 days prior to screening, or currently receiving additional factor concentrate or BPA infusion for postoperative hemostasis
- 02. Current participation in tolerance induction treatment (ITI)
- 03. Current use of factor concentrates or BPAs as regularly administered prophylaxis designed to prevent spontaneous bleeding episodes. However, participants requiring factor concentrates or BPAs prophylaxis during the study dosing pause period
- 04. Use of compounds other than factor concentrates or BPAs for hemophilia treatment
- 05. Current or prior participation in a gene therapy trial
- 06. ALT and/or 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× ULN for patients who were in the fitusiran arm in the parent study.
- 07Additional 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 study team makes the final eligibility decision.
Where it's taking place
- United Kingdom
- Malaysia
- India
- Ukraine
- China
- Taiwan
- United States
- Korea, Republic of
- Australia
- Israel
- South Africa
- Canada
- Turkey
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 65+ years, 0-17 years, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United Kingdom; Malaysia; India; Ukraine; China; Taiwan and 7 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.