ClinicalTrials.gov
ClinicalTrials.gov Menu

A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With 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.
ClinicalTrials.gov Identifier: NCT03417102
Recruitment Status : Recruiting
First Posted : January 31, 2018
Last Update Posted : June 25, 2018
Sponsor:
Collaborator:
Genzyme, a Sanofi Company
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

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: recombinant Factor VIIa and FEIBA Phase 3

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 54 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : March 22, 2018
Estimated Primary Completion Date : June 2019
Estimated Study Completion Date : July 2019

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia

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

Active Comparator: On demand bypassing agents Drug: recombinant Factor VIIa and FEIBA
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.


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

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

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): NCT03417102


Contacts
Contact: Alnylam Clinical Trial Information Line 1-877-ALNYLAM clinicaltrials@alnylam.com
Contact: Alnylam Clinical Trial Information Line 1-877-256-9526

Locations
United States, Arizona
Clinical Trial Site Recruiting
Phoenix, Arizona, United States, 85016
United States, California
Clinical Trial Site Recruiting
Orange, California, United States, 92868
Clinical Trial Site Recruiting
San Diego, California, United States, 92123
United States, Florida
Clinical Trial Site Recruiting
Tampa, Florida, United States, 33607
United States, Louisiana
Clinical Trial Site Recruiting
New Orleans, Louisiana, United States, 70112
United States, Nevada
Clinical Trial Site Recruiting
Las Vegas, Nevada, United States, 89109
United States, North Carolina
Clinical Trial Site Recruiting
Chapel Hill, North Carolina, United States, 27517
United States, Ohio
Clinical Trial Site Recruiting
Akron, Ohio, United States, 44308
Australia
Clinical Trial Site Recruiting
Melbourne, Australia, 3004
Sponsors and Collaborators
Alnylam Pharmaceuticals
Genzyme, a Sanofi Company
Investigators
Study Director: Kate Madigan, MD Alnylam Pharmaceuticals, Inc.

Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03417102     History of Changes
Other Study ID Numbers: ALN-AT3SC-003
First Posted: January 31, 2018    Key Record Dates
Last Update Posted: June 25, 2018
Last Verified: June 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Alnylam Pharmaceuticals:
Fitusiran
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
Inhibitor
Bypassing agents
Coagulants

Additional relevant MeSH terms:
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
Coagulants