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A Study of Fitusiran in Severe Hemophilia A and B Patients Previously Receiving Factor or Bypassing Agent Prophylaxis

NCT03549871 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 80

Last updated 2023-02-06

Study results available
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Summary

Primary Objective:

To characterize the frequency of bleeding episodes (BE) while receiving fitusiran treatment, relative to the frequency of bleeding episodes while receiving factor concentrate or bypassing agent (BPA) prophylaxis.

Secondary Objectives:

* To characterize the following while receiving fitusiran treatment, relative to receiving factor or BPA prophylaxis:
* the frequency of spontaneous bleeding episodes
* the frequency of joint bleeding episodes
* health related quality of life (HRQOL) in participants greater than or equal to (\>=) 17 years of age
* To characterize the frequency of bleeding episodes during the onset and treatment periods in participants receiving fitusiran.
* To characterize the safety and tolerability of fitusiran.
* To characterize the annualized weight-adjusted consumption of factor/BPA while receiving fitusiran treatment, relative to receiving factor or BPA prophylaxis.

Conditions

Interventions

DRUG

Fitusiran

Pharmaceutical form: solution for injection Route of administration: subcutaneous

DRUG

BPA prophylaxis

Pharmaceutical form: solution for injection Route of administration: Intravenous

DRUG

Factor (FVIII or FIX) prophylaxis

Pharmaceutical form: solution for injection Route of administration: Intravenous

Sponsors & Collaborators

  • Genzyme, a Sanofi Company

    lead INDUSTRY

Principal Investigators

  • Clinical Sciences & Operations · Sanofi

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
12 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-07-25
Primary Completion
2022-01-20
Completion
2022-03-25
FDA Drug
Yes

Countries

  • United States
  • Australia
  • China
  • Denmark
  • France
  • Ireland
  • Israel
  • Italy
  • Japan
  • Malaysia
  • Mexico
  • South Korea
  • 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 NCT03549871 on ClinicalTrials.gov