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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 : November 25, 2020
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Tracking Information
First Submitted Date  ICMJE November 25, 2018
First Posted Date  ICMJE November 27, 2018
Last Update Posted Date November 25, 2020
Actual Study Start Date  ICMJE January 9, 2019
Estimated Primary Completion Date January 2026   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 26, 2018)
Number of patients with adverse events [ Time Frame: From day -30 (screening) to day 673 ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: November 26, 2018)
  • Annualized bleeding rate (ABR) [ Time Frame: From day 29 to day 673 ]
    Annualized bleeding rate (ABR) in the treatment period
  • Annualized spontaneous bleeding rate [ Time Frame: From day 29 to day 673 ]
    Annualized spontaneous bleeding rate in the treatment period
  • Annualized joint bleeding rate [ Time Frame: From day 29 to day 673 ]
    Annualized joint bleeding rate in the treatment period
  • 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
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 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
Official Title  ICMJE 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
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
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 Type  ICMJE Interventional
Study Phase  ICMJE 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
Fitusiran fixed dose, once-monthly subcutaneous injection for 48 months
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: November 26, 2018)
244
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE January 2026
Estimated Primary Completion Date January 2026   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.

Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 12 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Trial Transparency email recommended (Toll free number for US & Canada) 800-633-1610 ext 1 then # Contact-US@sanofi.com
Listed Location Countries  ICMJE Australia,   Canada,   Denmark,   France,   Germany,   Hungary,   India,   Ireland,   Israel,   Italy,   Japan,   Korea, Republic of,   Malaysia,   South Africa,   Taiwan,   Turkey,   Ukraine,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03754790
Other Study ID Numbers  ICMJE LTE15174
2018-002880-25 ( EudraCT Number )
U1111-1210-0018 ( 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
Product Manufactured in and Exported from the U.S.: 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 November 2020

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