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A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors

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. Identifier: NCT03417245
Recruitment Status : Recruiting
First Posted : January 31, 2018
Last Update Posted : May 19, 2020
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

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).

Condition or disease Intervention/treatment Phase
Hemophilia A Hemophilia B Drug: fitusiran Drug: factor VIII or factor IX Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 120 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: ATLAS-A/B: A Phase 3 Study to Evaluate the Efficacy and Safety of Fitusiran in Patients With Hemophilia A or B, Without Inhibitory Antibodies to Factor VIII or IX
Actual Study Start Date : March 1, 2018
Estimated Primary Completion Date : April 2021
Estimated Study Completion Date : August 2021

Arm Intervention/treatment
Experimental: fitusiran Drug: fitusiran
by subcutaneous (SC) injection

Active Comparator: On Demand factor VIII or IX Drug: factor VIII or factor IX
by intravenous (IV) injection

Primary Outcome Measures :
  1. Annualized bleeding rate (ABR) [ Time Frame: 9 months ]

Secondary Outcome Measures :
  1. Annualized spontaneous bleeding rate [ Time Frame: 9 months ]
  2. Annualized joint bleeding rate [ Time Frame: 9 months ]
  3. 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 ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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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

Inclusion Criteria:

  • Males, ≥12 years of age
  • Severe hemophilia A or B without inhibitors

    • (Severity confirmed by a central laboratory where FVIII level is <1% or FIX level is ≤2% at Screening; Inhibitors defined as Nijmegen modified Bethesda assay inhibitor titer of <0.6 BU/mL at Screening)
  • A minimum of 6 bleeding episodes requiring factor concentrate 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 judgment

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 identifier (NCT number): NCT03417245

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

Show Show 88 study locations
Sponsors and Collaborators
Genzyme, a Sanofi Company
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Study Director: Clinical Sciences & Operations, MD Sanofi
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Responsible Party: Genzyme, a Sanofi Company Identifier: NCT03417245    
Other Study ID Numbers: EFC14769
ALN-AT3SC-004 ( Other Identifier: Alnylam )
2016-001464-11 ( EudraCT Number )
First Posted: January 31, 2018    Key Record Dates
Last Update Posted: May 19, 2020
Last Verified: April 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:

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Sanofi ( Genzyme, a Sanofi Company ):
RNAi therapeutic
Hemophilia A
Hemophilia B
Hemophilia A, Severe
Hemophilia B, Severe
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Factor VIII
Factor IX
Additional relevant MeSH terms:
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Hemophilia A
Hemophilia B
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Factor VIII