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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

NCT03754790 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 281

Last updated 2025-12-17

No results posted yet for this study

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

Conditions

Interventions

DRUG

Fitusiran

Pharmaceutical form:solution for injection Route of administration: subcutaneous

Sponsors & Collaborators

  • Genzyme, a Sanofi Company

    lead INDUSTRY

Principal Investigators

  • Clinical Sciences & Operations · Sanofi

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
12 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-01-09
Primary Completion
2026-11-05
Completion
2026-11-05
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Canada
  • China
  • Denmark
  • France
  • Germany
  • Hungary
  • India
  • Ireland
  • Israel
  • Italy
  • Japan
  • Malaysia
  • South Africa
  • South Korea
  • Taiwan
  • Turkey (Türkiye)
  • Ukraine
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03754790 on ClinicalTrials.gov