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 (ATLAS-OLE)

STATUS

Recruiting
End date

Oct 19, 2026
participants needed

244
sponsor

Genzyme, a Sanofi Company

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Updated on 11 May 2022

See if I qualify

hemarthrosis

severe haemophilia a

antihemophilic factor

fitusiran

Summary

Primary Objective:

To characterize the long-term safety and tolerability of fitusiran

Secondary Objectives:

To characterize the efficacy and long-term efficacy of fitusiran as assessed by the frequency of:
Bleeding episodes
Spontaneous bleeding episodes
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 56 months which consists of a screening period of up to 60 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. The duration of the treatment period and overall that of study participation, may exceed the intervals stated above for patient enrolled in the lower-dose cohort, to ensure a minimum of 18-month administration at this dosing regimen

Details

Condition	Hemophilia
Treatment	fitusiran
Clinical Study Identifier	NCT03754790
Sponsor	Genzyme, a Sanofi Company
Last Modified on	11 May 2022

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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 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

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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 (ATLAS-OLE)