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

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ClinicalTrials.gov Identifier: NCT03974113
Recruitment Status : Recruiting
First Posted : June 4, 2019
Last Update Posted : March 26, 2021
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Tracking Information
First Submitted Date  ICMJE June 3, 2019
First Posted Date  ICMJE June 4, 2019
Last Update Posted Date March 26, 2021
Actual Study Start Date  ICMJE January 28, 2020
Estimated Primary Completion Date April 2024   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 3, 2019)
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
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: June 3, 2019)
  • 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)
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B
Official Title  ICMJE 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 characterize the pharmacokinetics (PK)
Detailed Description Study duration per participant is approximately 160 weeks, including a 12-week fitusiran efficacy period
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Phase 3
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Hemophilia
Intervention  ICMJE Drug: Fitusiran
Pharmaceutical form:Solution for injection Route of administration: Subcutaneous
Study Arms  ICMJE Experimental: Fitusiran
Participants will receive a selected dose of fitusiran on regular intervals, as per study protocol
Intervention: Drug: Fitusiran
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: June 3, 2019)
12
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 2026
Estimated Primary Completion Date April 2024   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.

Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 1 Year to 11 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Trial Transparency email recommended (Toll free number for US & Canada) 800-633-1610 ext Option 6 Contact-US@sanofi.com
Listed Location Countries  ICMJE Canada,   India,   Italy,   Spain,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03974113
Other Study ID Numbers  ICMJE EFC15467
2019-000679-18 ( EudraCT Number )
U1111-1223-4368 ( Other Identifier: UTN )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
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/
Responsible Party Sanofi ( Genzyme, a Sanofi Company )
Study Sponsor  ICMJE Genzyme, a Sanofi Company
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Clinical Sciences & Operations Sanofi
PRS Account Sanofi
Verification Date March 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP