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 was to determine the frequency of bleeding episodes in participants
receiving fitusiran as prophylactic treatment of hemophilia compared to participants who were
assigned to continue with their regular medication. In addition, the study assessed safety,
quality of life, pharmacodynamics (PD), and pharmacokinetics (PK).
Detailed Description
The duration of treatment with fitusiran was 9 months. The estimated total time on study,
inclusive of screening, for each participant was up to 11 months for all participants who
enroll in the extension study and participants in the on-demand arm who did not enroll in the
extension study. The estimated total time on study was up to 17 months in fitusiran treatment
arm participants who did not enroll in the extension study due to the requirement for up to
an additional 6 months of follow-up monitoring for antithrombin levels.
Study Phase
Phase 3
Study Type
Interventional
Primary Outcome
Estimated Annualized Bleeding Rate (ABR) for Treated Bleeds During the Efficacy Period
Secondary Outcome
Estimated Annualized Bleeding Rate (ABR) for Treated Bleeds During the Treatment Period
Condition
Hemophilia A
Intervention
fitusiran
Study Arms / Comparison Groups
Bypassing Agents (BPA) On-demand
Description: Participants received On-demand BPAs (use of these agents, as needed, for episodic bleeding episodes, and not on a regular regimen intended to prevent spontaneous bleeding) per Investigator discretion from Day 1 for treatment of breakthrough bleeding episodes, up to 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
60
Start Date
February 14, 2018
Completion Date
June 23, 2021
Primary Completion Date
November 25, 2020
Eligibility Criteria
Inclusion Criteria:
- Males, greater than or equal to (>=) 12 years of age.
- Severe hemophilia A or B with inhibitors.
- (Severity confirmed by a central laboratory where coagulation factor VIII (FVIII)
level was less than (<)1% or factor IX (FIX) level was less than or equal to
[<=]2% at Screening; Inhibitors defined as inhibitor titer of >=0.6 Bethesda
units per milliliter [BU/mL] or as evidenced by medical records).
- A minimum of 6 bleeding episodes requiring BPA 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 hepatitis C virus infection.
- HIV positive with a cluster of differentiation-4 count of <200 cells/microliter.
- 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.
- History of intolerance to SC injection(s).
- Any other conditions or comorbidities that would make the participant 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
March 2022