Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B

Phase 2

• Conditions: Health Condition 1: D66- Hereditary factor VIII deficiency

• Registration Number: CTRI/2020/07/026641
• Lead Sponsor: Sanofi Healthcare India Private Limited

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Last Update Posted: 6 February 2023

Recruitment & Eligibility

• Status: Open to Recruitment
• Sex: Not specified
• Target Recruitment: 0

Inclusion Criteria

Participant must be 1 to less than 12 years of age at the time of enrollment

- Severe hemophilia A or B (FVIII <1% or FIX <=2%)

- Participants must have inhibitory antibodies to FVIII or FIX and must meet one of the following Nijmegen-modified Bethesda assay results criteria.

1. ­Inhibitor titer of >=0.6 BU/mL at screening

2. ­Inhibitor titer of <0.6 BU/mL at screening with medical record evidence of 2 consecutive titers >=0.6 BU/mL

3. ­Inhibitor titer of <0.6 BU/mL at screening with medical record evidence of anamnestic response

Exclusion Criteria

- Known co-existing bleeding disorders other than hemophilia A or B, ie, von Willebrand disease, additional factor deficiencies, or platelet disorders

- AT activity <60% at screening, as determined by central laboratory analysis.

- Presence of clinically significant liver disease, or as indicated by any of the conditions below

1. International normalized ratio (INR) >1.2

2. ALT and/or AST >2 Ã? ULN reference range

3. Total bilirubin >ULN ( >2 Ã? ULN in participants with Gilbertâ??s syndrome

4. History of portal hypertension, esophageal varices, or hepatic encephalopathy

5. Presence of ascites by physical examination

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
­Lowering of plasma antithrombin (AT) activity levelTimepoint: Day 1 to Day 85

Secondary Outcome Measures

Name	Time	Method
­Number of participants reported with adverse eventsTimepoint: 160 weeks;­Pharmacokinetics (PK): CtroughTimepoint: Day 1, Day 29, Day 57;­Pharmacokinetics (PK): Maximum plasma concentration (Cmax)Timepoint: Day 1, Day 29, Day 57;­Pharmacokinetics (PK): Time to reach maximum plasma concentration (Tmax)Timepoint: Day 1, Day 29, Day 57