FITUSIRAN for Severe hemophilia A | Severe hemophilia B

Inclusion criteria

• {"criterion_text":"- Male, aged 1 to <12 years at the time of enrollment.\n- Severe hemophilia A or B (Factor VIII (FVIII) <1% or Factor IX (FIX) ≤2%)\n- Participants must have inhibitory antibodies to FVIII or FIX and must meet one of the following Nijmegen-modified Bethesda assay results criteria: -\tInhibitor titer of ≥0.6 BU/mL at screening, OR -\tInhibitor titer of <0.6 BU/mL at screening with medical record evidence of 2 consecutive titers ≥0.6 BU/mL, OR -\tInhibitor titer of <0.6 BU/mL at screening with medical record evidence of 1 inhibitor titer ≥0.6 BU/mL and a history of anamnestic response or severe allergic reaction (anaphylaxis or nephrotic syndrome)\n- Adequate peripheral venous access, as determined by the Investigator, to allow the blood draws required by the study protocol\n- Weight requirements at the time of enrollment: 8 to <45 kg\n- Willing and able to comply with the study requirements and to provide signed written informed consent obtained from parent(s)/legal guardian (hereinafter the “parent”) and written or oral assent obtained from participant, per local and national requirements"}

Exclusion criteria

• {"criterion_text":"- Known co-existing bleeding disorders other than hemophilia A or B\n- Antithrombin (AT) activity <60% at Screening\n- Co-existing thrombophilic disorder\n- Clinically significant liver disease\n- Active Hepatitis C virus infection\n- Acute or chronic Hepatitis B virus infection\n- Acute Hepatitis A or hepatitis E infection\n- HIV positive with a CD4 count of <400 cells/μL\n- History of arterial or venous thromboembolism, unrelated to an indwelling venous access\n- History of multiple drug allergies or history of allergic reaction to an oligonucleotide or N-Acetylgalactosamine (GalNAc)\n- Subjects with central or peripheral indwelling catheters, with history of venous access complications leading to hospitalization and/or systemic anticoagulation therapy.\n- History of intolerance to subcutaneous (SC) injection(s)\n- Use of emicizumab (Hemlibra®) within 6 months prior to screening\n- 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 judgment"}

Primary endpoints

• {"endpoint_text":"- Plasma antithrombin (AT) activity levels","definition_or_measurement_approach":""}

Secondary endpoints

• {"endpoint_text":"- Number of participants reported with adverse events","definition_or_measurement_approach":""}
• {"endpoint_text":"- Fitusiran plasma concentrations","definition_or_measurement_approach":"Plasma concentrations measured at selected time points (as per secondary objective: To determine fitusiran plasma concentrations at selected time points)."}

Spain

Earliest CTIS Part Ii Submission Date

05-03-2024

Latest Decision Or Authorization Date

04-06-2024

Processing Time Days

91

Number Of Sites

1

Number Of Participants

1

Italy

Earliest CTIS Part Ii Submission Date

05-03-2024

Latest Decision Or Authorization Date

10-06-2024

Processing Time Days

97

Number Of Sites

2

Number Of Participants

2

Primary sponsor

Full Name

Genzyme Corp.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

Icon Clinical Research Limited

Responsibilities

Home Health Care / Nursing

Name

Pharmaceutical Product Development LLC

Responsibilities

code 4

Name

Almac Clinical Technologies LLC

Responsibilities

code 3

Name

Charles River Laboratories Montreal ULC

Responsibilities

code 4

Third parties

• {"country":"Netherlands","full_name":"CoagScope B.V.","duties_or_roles":"code 4","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Pyxant Labs Inc.","duties_or_roles":"code 4","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"Clinical Outcomes Assessment Instrument (eCOA)","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Pharmaceutical Product Development LLC","duties_or_roles":"code 4","organisation_type":"Pharmaceutical company"}
• {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"Home Health Care / Nursing","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Azenta US Inc.","duties_or_roles":"code 4","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"Fisher Clinical Services UK Limited","duties_or_roles":"code 14","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Greenphire LLC","duties_or_roles":"Patient Reimbursement","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Almac Clinical Technologies LLC","duties_or_roles":"code 3","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"ESMS Global Limited","duties_or_roles":"Centralized 24-Hour Emergency System: eSMS","organisation_type":"Pharmaceutical company"}
• {"country":"Italy","full_name":"Depo-pack S.r.l.","duties_or_roles":"code 14","organisation_type":"Pharmaceutical company"}
• {"country":"Canada","full_name":"Charles River Laboratories Montreal ULC","duties_or_roles":"code 4","organisation_type":"Pharmaceutical company"}