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A Study to Test a Medicine (Fitusiran) Injected Under the Skin for Preventing Bleeding Episodes in Male Adolescent or Adult Participants With Severe Hemophilia

NCT05662319 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 91

Last updated 2026-02-04

No results posted yet for this study

Summary

This is a multicenter, multinational, open-label, one-way cross-over, Phase 3, single-arm study for treatment of hemophilia.

The purpose of this study is to measure the frequency of treated bleeding episodes with fitusiran in male adult and adolescent (≥12 years old) participants with hemophilia A or B, with or without inhibitory antibodies to factor VIII or IX who have switched from their prior standard of care treatment.

The total study duration will be up to approximately 50 months (200 weeks, 1 study month is equivalent to 4 weeks) and will include:

* A screening period up to approximately 60 days,
* A standard of care (SOC) period of approximately 6 study months (24 weeks),
* A fitusiran treatment period of approximately 36 study months (144 weeks),
* An antithrombin (AT) follow-up period of approximately 6 study months (24 weeks) but may be shorter or longer depending on individual participants AT recovery.

The frequency for telephone visits will be approximately every 2 weeks. For site visits the frequency will be approximately every 8 weeks during the SOC period and approximately every 4 weeks during the fitusiran treatment period. If applicable and if allowed by local regulation, home and/or remote visits may be conducted during the study

Conditions

Interventions

DRUG

Fitusiran

Pharmaceutical form: Solution for injection Route of administration: Subcutaneous (SC)

DRUG

Clotting factor concentrates (CFC) or bypassing agents (BPA)

* Coagulation factor VIII (ATC code: B02BD02) * Coagulation factor IX (ATC code: B02BD04) * Coagulation factor VIIa (ATC code: B02BD08) * Factor VIII inhibitor bypassing activity (ATC code: B02BD03) Pharmaceutical form: Solution for infusion Route of administration: Intravenous (IV)

DRUG

Antithrombin concentrate (ATIIIC)

Antithrombin III (ATC code: B01AB02) Pharmaceutical form: Solution for infusion Route of administration: Intravenous (IV)

Sponsors & Collaborators

Principal Investigators

  • Clinical Sciences & Operations · Sanofi

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
CROSSOVER

Eligibility

Min Age
12 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-02-01
Primary Completion
2027-03-25
Completion
2029-01-25
FDA Drug
Yes

Countries

  • United States
  • Canada
  • China
  • France
  • Germany
  • Greece
  • India
  • Italy
  • Japan
  • Mexico
  • Poland
  • Saudi Arabia
  • South Africa
  • South Korea
  • Spain
  • Taiwan
  • Turkey (Türkiye)

Study Locations

More Related Trials

Entities

Diseases
Companies

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 NCT05662319 on ClinicalTrials.gov