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A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors (ATLAS-INH)

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ClinicalTrials.gov Identifier: NCT03417102
Recruitment Status : Recruiting
First Posted : January 31, 2018
Last Update Posted : May 20, 2019
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Tracking Information
First Submitted Date  ICMJE January 25, 2018
First Posted Date  ICMJE January 31, 2018
Last Update Posted Date May 20, 2019
Actual Study Start Date  ICMJE February 14, 2018
Estimated Primary Completion Date February 10, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 30, 2018)
Annualized bleeding rate (ABR) [ Time Frame: 9 months ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03417102 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: February 6, 2018)
  • Annualized spontaneous bleeding rate [ Time Frame: 9 months ]
  • Annualized joint bleeding rate [ Time Frame: 9 months ]
  • Quality of Life (QOL) as measured by Haem-A-QOL Questionnaire score on a scale of 0-100 with higher scores representing greater impairment. [ Time Frame: 9 months ]
Original Secondary Outcome Measures  ICMJE
 (submitted: January 30, 2018)
  • Annualized spontaneous bleeding rate [ Time Frame: 9 months ]
  • Annualized joint bleeding rate [ Time Frame: 9 months ]
  • Haem-A-QOL Questionnaire score [ Time Frame: 9 months ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors
Official Title  ICMJE ATLAS-INH: A Phase 3 Study to Evaluate the Efficacy and Safety of Fitusiran in Patients With Hemophilia A or B, With Inhibitory Antibodies to Factor VIII or IX
Brief Summary The purpose of this study is to determine the frequency of bleeding episodes in patients receiving fitusiran as prophylactic treatment of hemophilia compared with patients who are assigned to continue with their regular medication. In addition, the study will assess safety, quality of life, pharmacodynamics (PD), and pharmacokinetics (PK).
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Hemophilia A
  • Hemophilia B
Intervention  ICMJE
  • Drug: fitusiran
    by subcutaneous (SC) injection
  • Drug: recombinant Factor VIIa and FEIBA
    by intravenous (IV) injection
Study Arms  ICMJE
  • Experimental: fitusiran
    Intervention: Drug: fitusiran
  • Active Comparator: On demand bypassing agents
    Intervention: Drug: recombinant Factor VIIa and FEIBA
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: January 30, 2018)
54
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE August 10, 2020
Estimated Primary Completion Date February 10, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Males, ≥12 years of age
  • Severe hemophilia A or B with inhibitors

    • (Severity confirmed by a central laboratory where FVIII level is <1% or FIX level is ≤2% at Screening; Inhibitors defined as inhibitor titer of ≥0.6 BU/mL or as evidenced by medical records)
  • A minimum of 6 bleeding episodes requiring bypassing agent treatment within the last 6 months prior to Screening
  • Willing and able to comply with the study requirements and to provide written informed consent and assent

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 HCV infection
  • HIV positive with a CD4 count of <200 cells/μL
  • History of arterial or venous thromboembolism
  • Inadequate renal function
  • History of multiple drug allergies or history of allergic reaction to an oligonucleotide or N-Acetylgalactosamine (GalNAc)
  • History of intolerance to 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 judgement
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,   Bulgaria,   France,   Germany,   India,   Israel,   Korea, Republic of,   Taiwan,   Turkey,   Ukraine,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03417102
Other Study ID Numbers  ICMJE EFC14768
2016-001463-36 ( EudraCT Number )
ALN-AT3SC-003 ( Other Identifier: Alnylam )
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, MD Sanofi
PRS Account Sanofi
Verification Date May 2019

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