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

STATUS

Recruiting
End date

Jan 12, 2026
participants needed

244
sponsor

Genzyme, a Sanofi Company

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Updated on 19 February 2024

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hemophilia

hemarthrosis

antihemophilic factor

fitusiran

haemophilia a

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

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

Details

Condition	Hemophilia A, hemophilia, hemophilia
Age	12years - 100years
Treatment	Fitusiran
Clinical Study Identifier	NCT03754790
Sponsor	Genzyme, a Sanofi Company
Last Modified on	19 February 2024

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Eligibility		Yes	No	Not Sure
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 nave 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

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Step 2 Get screened

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Step 3 Enroll in the clinical study

If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Step 4 Get your study results

Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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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