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Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX (ATLAS-OLE)

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: NCT03754790
Recruitment Status : Recruiting
First Posted : November 27, 2018
Last Update Posted : May 9, 2022
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Study Description
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Brief Summary:

Primary Objective:

To characterize the long-term safety and tolerability of fitusiran

Secondary Objectives:

  • To characterize the efficacy and long-term efficacy of fitusiran as assessed by the frequency of:

    • Bleeding episodes
    • Spontaneous bleeding episodes
    • Joint bleeding episodes
  • To characterize the effects of fitusiran on health-related quality of life (HRQOL) measures in participants ≥17 years of age

Condition or disease Intervention/treatment Phase
Hemophilia Drug: Fitusiran Phase 3

Detailed Description:
The estimated total time on study for each participant is up to 56 months which consists of a screening period of up to 60 days, an open label treatment period of up to 48 months and a follow up period of up to 6-month after the last dose of fitusiran. The duration of the treatment period and overall that of study participation, may exceed the intervals stated above for patient enrolled in the lower-dose cohort, to ensure a minimum of 18-month administration at this dosing regimen
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 244 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-label, Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX
Actual Study Start Date : January 9, 2019
Estimated Primary Completion Date : October 19, 2026
Estimated Study Completion Date : October 19, 2026

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 be administered fitusiran as an subcutaneous injection once monthly or every other month for 48 months.However, for participants enrolled in the lower-dose cohort, duration of fitusiran administration may exceed 48 months, to ensure a minimum of 18-month administration at this dose level.
 Drug: Fitusiran
Pharmaceutical form:solution for injection Route of administration: subcutaneous


Outcome Measures
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Primary Outcome Measures :
  1. Number of participants with treatment emergent adverse events (TEAEs) [ Time Frame: from study baseline (day 1) up to 54 months (48 months treatment +6 months follow up) ]
    The number of participants experiencing any TEAEs, serious TEAEs, discontinuation due to TEAEs and death will be reported


Secondary Outcome Measures :
  1. Annualized bleeding rate (ABR) [ Time Frame: from first ever dose of fitusiran (day 1) to Month 49 ]
    Annualized bleeding rate (ABR) in the treatment period

  2. Annualized spontaneous bleeding rate [ Time Frame: from first ever dose of fitusiran (day 1) to Month 49 ]
    Annualized spontaneous bleeding rate in the treatment period

  3. Annualized joint bleeding rate [ Time Frame: from first ever dose of fitusiran (day 1) to Month 49 ]
    Annualized joint bleeding rate in the treatment period

  4. Changes in Haem-A-quality of life (QoL) score [ Time Frame: from first ever dose of fitusiran (day 1) to Month 48 ]
    Change in Haem A QoL physical health score and total score in the treatment period (in participants ≥17 years of age) from first ever dose of fitusiran (day 1) to month 48


Eligibility Criteria
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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion criteria :

  • Participant must be at least 12 years of age inclusive, at the time of signing the informed consent
  • Participants with severe hemophilia A or B who have completed a Phase 3 fitusiran clinical trial
  • Male
  • Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol. In countries where legal age of majority is above 18 years, a specific ICF must also be signed by the participant's legally authorized representative

Exclusion criteria:

  • Completion of a surgical procedure within 14 days prior to screening, or currently receiving additional factor concentrate or BPA infusion for postoperative hemostasis
  • Current participation in immune tolerance induction treatment (ITI)
  • Current use of factor concentrates or bypassing agents (BPAs) as regularly administered prophylaxis designed to prevent spontaneous bleeding episodes except for participants requiring factor concentrates or BPAs prophylaxis during the study dosing pause period
  • Use of compounds other than factor concentrates or BPAs for hemophilia treatment
  • Current or prior participation in a gene therapy trial
  • Alanine aminotransferase (ALT) and/or Aspartate aminotransferase (AST) >1.5 × upper limit of normal reference range (ULN) for patients who are naïve to fitusiran at study start; ALT and/or AST > 5 x ULN for patients who were in the fitusiran arm in the parent study

  • Additional exclusions for participants not currently participating in a fitusiran trial at the time of enrollment in the lower dose cohort:

    • Clinically significant liver disease
    • History of arterial or venous thromboembolism

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): NCT03754790


Contacts
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Contact: Trial Transparency email recommended (Toll free number for US & Canada) 800-633-1610 ext 1 then # Contact-US@sanofi.com

Locations
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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: NCT03754790    
Other Study ID Numbers: LTE15174
2018-002880-25 ( EudraCT Number )
U1111-1210-0018 ( Registry Identifier: ICTRP )
First Posted: November 27, 2018    Key Record Dates
Last Update Posted: May 9, 2022
Last Verified: May 4, 2022
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://vivli.org

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: 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