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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 : 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 characterize the long-term safety and tolerability of fitusiran

Secondary Objectives:

  • To characterize the long-term efficacy of fitusiran as assessed by the frequency of:
  • Bleeding episodes
  • Spontaneous bleeding episodes
  • Target 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 55 months which consists of a screening period of up to 30 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
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 244 participants
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 2025
Estimated Study Completion Date : October 2025

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
Fitusiran fixed dose, once-monthly subcutaneous injection for 48 months
 Drug: Fitusiran
Pharmaceutical form:solution for injection Route of administration: subcutaneous


Outcome Measures
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Primary Outcome Measures :
  1. Number of patients with adverse events [ Time Frame: From day -30 (screening) to day 673 ]

Secondary Outcome Measures :
  1. Annualized bleeding rate (ABR) [ Time Frame: From day 29 to day 673 ]
    Annualized bleeding rate (ABR) in the treatment period

  2. Annualized spontaneous bleeding rate [ Time Frame: From day 29 to day 673 ]
    Annualized spontaneous bleeding rate in the treatment period

  3. Annualized joint bleeding rate [ Time Frame: From day 29 to day 673 ]
    Annualized joint bleeding rate in the treatment period

  4. Changes in Haem-A-quality of life (QoL) score [ Time Frame: On day 1 (baseline), day 337 and day 673 ]
    Change in Haem A QoL physical health score and total score in the treatment period (in participants ≥17 years of age) from baseline (day 1) to day 337 and day 673


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
  • 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

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 ( Other Identifier: UTN )
First Posted: November 27, 2018    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
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
Factor VIII
Antibodies
Immunologic Factors
Physiological Effects of Drugs
Coagulants