Brief Title
A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors
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
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
The duration of treatment with fitusiran is 9 months. The estimated total time on study,
inclusive of Screening, for each patient is up to 11 months for all patients who enroll in
the extension study and patients in the on-demand arm who do not enroll in the extension
study. The estimated total time on the study may be up to 17 months in fitusiran treatment
arm patients who do not enroll in the extension study due to the requirement for an
additional 6 months of follow-up monitoring for antithrombin levels.
Study Phase
Phase 3
Study Type
Interventional
Primary Outcome
Annualized bleeding rate (ABR)
Secondary Outcome
Annualized bleeding rate (ABR)
Condition
Hemophilia A
Intervention
fitusiran
Study Arms / Comparison Groups
Fitusiran
Description: Fitusiran administered subcutaneously (SC) as prophylaxis once monthly, with use of on-demand BPAs for treatment of breakthrough bleeding episodes. All patients will be treated for a total of 9 months
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
59
Start Date
February 14, 2018
Completion Date
June 23, 2021
Primary Completion Date
November 25, 2020
Eligibility 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
Gender
Male
Ages
12 Years - N/A
Accepts Healthy Volunteers
No
Contacts
Clinical Sciences & Operations, MD, ,
Location Countries
Australia
Location Countries
Australia
Administrative Informations
NCT ID
NCT03417102
Organization ID
EFC14768
Secondary IDs
2016-001463-36
Responsible Party
Sponsor
Study Sponsor
Genzyme, a Sanofi Company
Study Sponsor
Clinical Sciences & Operations, MD, Study Director, Sanofi
Verification Date
July 2021