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

ATLAS-PEDS: An open-label, multinational study of fitusiran prophylaxis in male pediatric subjects aged 1 to less than 12 years with hemophilia A or B - ATLAS-PEDS

Status

Recruiting

Phases

Phase 2Phase 3

Study type

Interventional

Source

CTRI

Registry ID

CTRI/2020/07/026641

Enrollment

Registered

2020-07-17

Start date

Unknown

Completion date

Unknown

Last updated

2023-02-06

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Health Condition 1: D66- Hereditary factor VIII deficiency

Interventions

Intervention1: Fitusiran: Fitusiran solution for injection in Phosphate buffered saline (Subcutaneous use) supplied as a sterile solution. Dosages administered as per the weight of the participants Co

Eligibility

Inclusion criteria

Inclusion criteria: -Participant must be 1 to less than 12 years of age at the time of enrollment - Severe hemophilia A or B (FVIII - Participants must have inhibitory antibodies to FVIII or FIX and must meet one of the following Nijmegen-modified Bethesda assay results criteria. 1. ÂInhibitor titer of >=0.6 BU/mL at screening 2. ÂInhibitor titer of =0.6 BU/mL 3. ÂInhibitor titer of <0.6 BU/mL at screening with medical record evidence of anamnestic response

Exclusion criteria

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 - 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

Design outcomes

Primary

Measure	Time frame
ÂLowering of plasma antithrombin (AT) activity levelTimepoint: Day 1 to Day 85	—

Secondary

Measure	Time frame
ÂNumber of participants reported with adverse eventsTimepoint: 160 weeks;ÂPharmacokinetics (PK): CtroughTimepoint: Day 1, Day 29, Day 57;ÂPharmacokinetics (PK): Maximum plasma concentration (Cmax)Timepoint: Day 1, Day 29, Day 57;ÂPharmacokinetics (PK): Time to reach maximum plasma concentration (Tmax)Timepoint: Day 1, Day 29, Day 57	—

Countries

Canada, India, Italy, Spain, Turkey, United States of America

Contacts

Public ContactNishant Kharb

Sanofi Healthcare India Private Limited

Godhuli.Chatterjee@sanofi.com919930151289

Outcome results

None listed

Source: CTRI (via WHO ICTRP) · Data processed: Feb 4, 2026