A Phase 3 study to Evaluate the Efficacy and Safety of Fitusiran in Patients with Hemophilia A or B, with Inhibitory Antibodies to Factor VIII or IX

Phase 3

• Conditions: Health Condition 1: null- â?¢Hemophilia Aâ?¢Hemophilia BHealth Condition 2: D689- Coagulation defect, unspecified

• Registration Number: CTRI/2018/06/014682
• Lead Sponsor: Sanofi Genzyme

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Last Update Posted: 24 November 2021

Recruitment & Eligibility

• Status: Closed to Recruitment of Participants
• Sex: Not specified
• Target Recruitment: 0

Inclusion Criteria

1. Males, >=12 years of age

2. Severe hemophilia A or B with inhibitors

â?¦(Severity confirmed by a central laboratory where FVIII level is <1% or FIX level is <=2% at Screening; Inhibitors defined as inhibitor titer of >=0.6 BU/mL or as evidenced by medical records)

3. A minimum of 6 bleeding episodes requiring bypassing agent treatment within the last 6 months prior to Screening

4. Willing and able to comply with the study requirements and to provide written informed consent and assent

Exclusion Criteria

1. Known co-existing bleeding disorders other than hemophilia A or B

2. Antithrombin (AT) activity <60% at Screening

3. Co-existing thrombophilic disorder

4. Clinically significant liver disease

5. Active HCV infection

6. HIV positive with a CD4 count of <200 cells/μL

7. History of arterial or venous thromboembolism

8. Inadequate renal function

9. History of multiple drug allergies or history of allergic reaction to an oligonucleotide or N-Acetylgalactosamine (GalNAc)

10. History of intolerance to SC injection(s)

11. Any other conditions or comorbidities that would make the patient unsuitable for enrollment or could interfere with participation in or completion of the study, per Investigator judgement

Study & Design

• Study Type: Interventional
• Study Design: Not specified