Hemophilia Clinical Trial
— ATLAS-PEDSOfficial title:
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
Verified date | January 2024 |
Source | Sanofi |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
Primary Objective: - To confirm appropriate dose levels of fitusiran when administered to male pediatric participants (ages 1 to <12 years of age) with severe hemophilia A or B Secondary Objectives: - To characterize the safety and tolerability - To determine fitusiran plasma concentrations at selected time points
Status | Active, not recruiting |
Enrollment | 32 |
Est. completion date | August 1, 2028 |
Est. primary completion date | February 15, 2028 |
Accepts healthy volunteers | No |
Gender | Male |
Age group | 1 Year to 11 Years |
Eligibility | Inclusion Criteria: - Male, aged 1 to <12 years at the time of enrollment. - Severe hemophilia A or B (Factor VIII (FVIII) <1% or Factor IX (FIX) =2%) - Participants must have inhibitory antibodies to FVIII or FIX and must meet one of the following Nijmegen-modified Bethesda assay results criteria: - Inhibitor titer of =0.6 BU/mL at screening, OR - Inhibitor titer of <0.6 BU/mL at screening with medical record evidence of 2 consecutive titers =0.6 BU/mL, OR - Inhibitor 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) - Adequate peripheral venous access, as determined by the Investigator, to allow the blood draws required by the study protocol - Weight requirements at the time of enrollment: 8 to <45 kg - 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: Participants are excluded from the study if any of the following criteria apply: - Known co-existing bleeding disorders other than hemophilia A or B - Antithrombin (AT) activity <60% at Screening - Co-existing thrombophilic disorder - Clinically significant liver disease - Active Hepatitis C virus infection - Acute or chronic Hepatitis B virus infection - Acute Hepatitis A or hepatitis E infection - HIV positive with a CD4 count of <400 cells/µL - History of arterial or venous thromboembolism, unrelated to an indwelling venous access - Inadequate renal function - History of multiple drug allergies or history of allergic reaction to an oligonucleotide or N-Acetylgalactosamine (GalNAc) - Subjects with central or peripheral indwelling catheters, with history of venous access complications leading to hospitalization and/or systemic anticoagulation therapy. - History of intolerance to subcutaneous (SC) injection(s) - Use of emicizumab (Hemlibra®) within 6 months prior to screening - 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 The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial. |
Country | Name | City | State |
---|---|---|---|
Canada | Investigational Site Number : 1240001 | Hamilton | Ontario |
Canada | Investigational Site Number : 1240002 | Ottawa | Ontario |
India | Investigational Site Number : 3560006 | Bangalore | |
India | Investigational Site Number : 3560002 | Mumbai | |
India | Investigational Site Number : 3560001 | Pune-411011 | |
India | Investigational Site Number : 3560004 | Vellore | |
Italy | Investigational Site Number : 3800002 | Firenze | |
Italy | Investigational Site Number : 3800001 | Milano | |
Spain | Investigational Site Number : 7240002 | Madrid | Madrid, Comunidad De |
Turkey | Investigational Site Number : 7920001 | Adana | |
Turkey | Investigational Site Number : 7920002 | Istanbul | |
Turkey | Investigational Site Number : 7920003 | Izmir | |
United States | University Hospitals of Cleveland Site Number : 8400007 | Cleveland | Ohio |
United States | Hackensack University Medical Center Site Number : 8400008 | Hackensack | New Jersey |
United States | Penn State Milton S. Hershey Medical Center Site Number : 8400006 | Hershey | Pennsylvania |
United States | Children's Hospital Los Angeles Site Number : 8400002 | Los Angeles | California |
Lead Sponsor | Collaborator |
---|---|
Genzyme, a Sanofi Company |
United States, Canada, India, Italy, Spain, Turkey,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Plasma antithrombin (AT) activity levels | Characterize the AT activity at the optimal therapeutic dose | Day 1 to the AT analysis time point at the optimal therapeutic dose (approximately 256 weeks) | |
Secondary | Number of participants reported with adverse events | Number of participants reported with treatment-emergent adverse events (TEAEs) | Up to 280 weeks (up to 256 weeks of treatment + up to 24 weeks of AT follow up) | |
Secondary | Fitusiran plasma concentrations | Plasma samples will be collected for measurement of plasma concentrations of fitusiran at specific time points post dose on Day 1 and pre dose on Day 85 | Day 1 and Day 85 |
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