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

Send

Updated on 19 February 2024

See if I qualify

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

How understandable was the trial content above?

Hard to understand

Easy to understand

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

How to participate?

Step 1 Connect with a study center

Select a site
Send a message

You have contacted , on

Your message has been sent to the study team at ,

A copy of the message has been sent to your email

What happens next?

You can expect the study team to contact you via email or phone in the next few days.
Sign up as volunteer to help accelerate the development of new treatments and to get notified about similar trials.

You are contacting

Primary Contact

I am at least 18 years old.

By sending a message, I agree to share my information with the study team for the selected site and consent to receive emails and text messages for trial updates, notifications, and related communication. TrialX values your privacy; your information will be handled in accordance with our Privacy Policy

Step 2 Get screened

Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

Learn more

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.

Learn more

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.

Learn more

Similar trials to consider

Browse trials for

hemophilia

hemarthrosis

antihemophilic factor

fitusiran

haemophilia a

Not finding what you're looking for?

Every year hundreds of thousands of volunteers step forward to participate in research. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.

Study Definition

Wikipedia

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