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Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B (ATLAS-PEDS)

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 20, 2020
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Study Description
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Brief Summary:

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

Detailed Description:
Study duration per participant is approximately 160 weeks, including a 12-week fitusiran efficacy period
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 12 participants
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 : February 2023
Estimated Study Completion Date : June 2024

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia

Arms and Interventions
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Arm Intervention/treatment
Experimental: Fitusiran
Participants will receive a selected dose of fitusiran on regular intervals, as per study protocol
 Drug: Fitusiran
Pharmaceutical form:Solution for injection Route of administration: Subcutaneous


Outcome Measures
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Primary Outcome Measures :
  1. 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


Secondary Outcome Measures :
  1. Number of participants reported with adverse events [ Time Frame: 160 weeks ]
    Number of participants reported with treatment-emergent adverse events (TEAEs)

  2. 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.

  3. Pharmacokinetics (PK): Time to reach maximum plasma concentration (Tmax) [ Time Frame: Day 1, Day 29, Day 57 ]
    To evaluate time to reach Cmax

  4. 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)


Eligibility Criteria
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Ages Eligible for Study:   1 Year to 11 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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 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.

Contacts and Locations
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Information from the National Library of Medicine

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


Contacts
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Contact: Trial Transparency email recommended (Toll free number for US & Canada) 800-633-1610 ext Option 6 Contact-US@sanofi.com

Locations
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United States, New Jersey
Investigational Site Number 8400008 Recruiting
Hackensack, New Jersey, United States, 07601
Canada
Investigational Site Number 1240001 Recruiting
Hamilton, Canada, L8N 3Z5
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
Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
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Study Director: Clinical Sciences & Operations Sanofi
More Information
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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 20, 2020
Last Verified: February 2020
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/

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
 Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Coagulants