Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B (ATLAS-PEDS)
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT03974113 |
Recruitment Status :
Recruiting
First Posted : June 4, 2019
Last Update Posted : February 8, 2021
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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 Objective:
- To characterize the safety and tolerability
- To characterize the pharmacokinetics (PK)
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Hemophilia | Drug: Fitusiran | Phase 2 Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 12 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official 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 |
Actual Study Start Date : | January 28, 2020 |
Estimated Primary Completion Date : | April 2024 |
Estimated Study Completion Date : | September 2025 |

Arm | Intervention/treatment |
---|---|
Experimental: Fitusiran
Participants will receive a selected dose of fitusiran on regular intervals, as per study protocol
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Drug: Fitusiran
Pharmaceutical form:Solution for injection Route of administration: Subcutaneous |
- Lowering of plasma antithrombin (AT) activity level [ Time Frame: Day 1 to Day 85 ]Lowering of plasma antithrombin (AT) activity level from Day 1 pre-fitusiran dose to Day 85
- Number of participants reported with adverse events [ Time Frame: 160 weeks ]Number of participants reported with treatment-emergent adverse events (TEAEs)
- Pharmacokinetics (PK): Maximum plasma concentration (Cmax) [ Time Frame: Day 1, Day 29, Day 57 ]Plasma samples will be collected for measurement of plasma concentrations of fitusiran such as Cmax.
- Pharmacokinetics (PK): Time to reach maximum plasma concentration (Tmax) [ Time Frame: Day 1, Day 29, Day 57 ]To evaluate time to reach Cmax
- Pharmacokinetics (PK): Ctrough [ Time Frame: Day 1, Day 29, Day 57 ]To evaluate concentration observed just before investigational medicinal product (IMP) administration during repeated dosing (Ctrough)

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Ages Eligible for Study: | 1 Year to 11 Years (Child) |
Sexes Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
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%)
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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 anamnestic response.
- 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:
- 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)
- 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.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03974113
Contact: Trial Transparency email recommended (Toll free number for US & Canada) | 800-633-1610 ext Option 6 | Contact-US@sanofi.com |
United States, California | |
Investigational Site Number 8400002 | Recruiting |
Los Angeles, California, United States, 90027 | |
Investigational Site Number 8400010 | Recruiting |
Orange, California, United States, 92868 | |
United States, New Jersey | |
Investigational Site Number 8400008 | Recruiting |
Hackensack, New Jersey, United States, 07601 | |
United States, Ohio | |
Investigational Site Number 8400007 | Recruiting |
Cleveland, Ohio, United States, 44106 | |
United States, Pennsylvania | |
Investigational Site Number 8400006 | Recruiting |
Hershey, Pennsylvania, United States, 17033 | |
Canada | |
Investigational Site Number 1240001 | Recruiting |
Hamilton, Canada, L8N 3Z5 | |
India | |
Investigational Site Number 3560001 | Recruiting |
Pune-411011, India, 411 011 | |
Italy | |
Investigational Site Number 3800002 | Recruiting |
Firenze, Italy, 50134 | |
Investigational Site Number 3800001 | Recruiting |
Milano, Italy, 20121 | |
Spain | |
Investigational Site Number 7240001 | Recruiting |
Barcelona, Spain, 08035 | |
Investigational Site Number 7240002 | Recruiting |
Madrid, Spain, 28046 |
Study Director: | Clinical Sciences & Operations | Sanofi |
Responsible Party: | Genzyme, a Sanofi Company |
ClinicalTrials.gov Identifier: | NCT03974113 |
Other Study ID Numbers: |
EFC15467 2019-000679-18 ( EudraCT Number ) U1111-1223-4368 ( Other Identifier: UTN ) |
First Posted: | June 4, 2019 Key Record Dates |
Last Update Posted: | February 8, 2021 |
Last Verified: | February 2021 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | Yes |
Plan Description: | Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://www.clinicalstudydatarequest.com/ |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Hemophilia A Blood Coagulation Disorders, Inherited Blood Coagulation Disorders Hematologic Diseases |
Coagulation Protein Disorders Hemorrhagic Disorders Genetic Diseases, Inborn |