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 IdentifierNCT03754790
SponsorGenzyme, a Sanofi Company
Last Modified on19 February 2024

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
Clear my responses
Read more

How to participate?

Step 1 Connect with a study center
We have submitted the contact information you provided to the research team at {{SITE_NAME}}. A copy of the message has been sent to your email for your records.
Would you like to be notified about other trials? Sign up for Patient Notification Services.
Sign up

Send a message

Enter your contact details to connect with study team

Investigator Avatar

Primary Contact

First name*
Last name*
Email*
Phone number*
Other language

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

Learn more

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

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.

Sign up as volunteer
Add a private note
  • abc Select a piece of text.
  • Add notes visible only to you.
  • Send it to people through a passcode protected link.