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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 | Clinical Trials | Clareo Health

ACTIVE_NOT_RECRUITINGPHASE3

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

An Open-label, 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

NCT03754790•Genzyme, a Sanofi Company

View on ClinicalTrials.gov

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

Detailed Description

The estimated total time on the study for a participant is up to 90 months (including screening, treatment period and safety follow up period).

Eligibility

Age

12 - No limit years

Sex

MALE

Healthy Volunteers

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.

Locations (79)

Phoenix Childrens Hospital Site Number : 8400009

Phoenix, Arizona, United States

Children's Hospital Los Angeles Site Number : 8400019

Los Angeles, California, United States

Center for Inherited Blood Disorders (CIBD) Site Number : 8400016

Orange, California, United States

Nemours Children's Clinic Site Number : 8400008

Jacksonville, Florida, United States

St Joseph's Children's Hospital of Tampa Site Number : 8400002

Tampa, Florida, United States

Rush University Medical Center -1725 W Harrison St Site Number : 8400001

Chicago, Illinois, United States

~Massachusetts General Hospital Site Number : 8400011

Boston, Massachusetts, United States

University of Michigan Hospital - 1500 E Medical Center Dr Site Number : 8400012

Ann Arbor, Michigan, United States

Alliance for Childhood Diseases Site Number : 8400007

Las Vegas, Nevada, United States

Childrens Hospital Medical Center of Akron Site Number : 8400006

Akron, Ohio, United States

Key Dates

Start Date

January 9, 2019

Primary Completion Date

November 5, 2026

Completion Date

November 5, 2026

Last Updated

December 17, 2025

Enrollment

281

ACTUAL participants

Conditions

Hemophilia

Interventions

Fitusiran

DRUG

A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B

NCT06379789

Hemophilia B

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RECRUITINGPHASE1/PHASE2

A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A

NCT07226206

Hemophilia A

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RECRUITINGPHASE1/PHASE2

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: December 17, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions

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

Inclusion Criteria

Exclusion Criteria

A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B

A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A

An Observational Cohort Study to Characterize the Effectiveness and Safety of HEMGENIX® in Patients With Hemophilia B

National Longitudinal Cohort of Hematological Diseases

Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia B

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

Inclusion Criteria

Exclusion Criteria

A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B

A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A

An Observational Cohort Study to Characterize the Effectiveness and Safety of HEMGENIX® in Patients With Hemophilia B

National Longitudinal Cohort of Hematological Diseases

Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to &lt; 18 Years of Age) With Severe or Moderately Severe Hemophilia B

Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia B