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

Brief Title

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

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

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 determine fitusiran plasma concentrations at selected time points

Detailed Description

      The estimated total time on study is up to 160 weeks in participants who roll over into the
      extension study and up to 184 weeks in participants who do not roll over into the extension
      study (due to the requirement for up to an additional 6 months of follow-up for monitoring of
      AT levels).

Study Phase

Phase 2/Phase 3

Study Type

Interventional

Primary Outcome

Plasma antithrombin (AT) activity levels

Secondary Outcome

 Number of participants reported with adverse events

Condition

Hemophilia

Intervention

Fitusiran

Study Arms / Comparison Groups

 Fitusiran

Description:  Participants will receive a selected dose of fitusiran on regular intervals, as per study protocol

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status

Drug

Estimated Enrollment

Start Date

January 28, 2020

Completion Date

December 2026

Primary Completion Date

August 9, 2023

Eligibility 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 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:

          -  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)

          -  Participants 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)

          -  The 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.

Gender

Male

Ages

1 Year - 11 Years

Accepts Healthy Volunteers

No

Contacts

Clinical Sciences & Operations, 800-633-1610, [email protected]

Location Countries

Canada

Location Countries

Canada

Administrative Informations

NCT ID

NCT03974113

Organization ID

EFC15467

Secondary IDs

2019-000679-18

Responsible Party

Sponsor

Study Sponsor

Genzyme, a Sanofi Company

Study Sponsor

Clinical Sciences & Operations, Study Director, Sanofi

Verification Date

April 2022