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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT03754790
Registration number
NCT03754790
Ethics application status
Date submitted
25/11/2018
Date registered
27/11/2018
Date last updated
26/03/2024
Titles & IDs
Public title
Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX
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Scientific title
An Open-label, Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX
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Secondary ID [1]
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U1111-1210-0018
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Secondary ID [2]
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LTE15174
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Universal Trial Number (UTN)
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Trial acronym
ATLAS-OLE
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Hemophilia
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Condition category
Condition code
Blood
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Clotting disorders
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Fitusiran
Experimental: Fitusiran - Participants will be administered fitusiran as a subcutaneous injection once monthly or every other month for up to 48 months post initiation of modified IMP dose/frequency or until fitusiran becomes commercially available, whichever comes first.
Treatment: Drugs: Fitusiran
Pharmaceutical form:solution for injection Route of administration: subcutaneous
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Number of participants with treatment emergent adverse events (TEAEs)
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Assessment method [1]
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The number of participants experiencing any TEAEs, serious TEAEs, discontinuation due to TEAEs and death will be reported
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Timepoint [1]
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from study baseline (day 1) up to maximum 88 months
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Secondary outcome [1]
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Annualized bleeding rate (ABR)
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Assessment method [1]
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Annualized bleeding rate (ABR) in the treatment period
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Timepoint [1]
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from first ever dose of fitusiran (day 1) up to maximum 78 months
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Secondary outcome [2]
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Annualized spontaneous bleeding rate
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Assessment method [2]
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Annualized spontaneous bleeding rate in the treatment period
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Timepoint [2]
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from first ever dose of fitusiran (day 1) up to maximum 78 months
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Secondary outcome [3]
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Annualized joint bleeding rate
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Assessment method [3]
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Annualized joint bleeding rate in the treatment period
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Timepoint [3]
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from first ever dose of fitusiran (day 1) up to maximum 78 months
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Secondary outcome [4]
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Changes in Haem-A-quality of life (QoL) score
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Assessment method [4]
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Change in Haem A QoL physical health score and total score in the treatment period (in participants =17 years of age) from first ever dose of fitusiran (day 1) to month 48
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Timepoint [4]
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from first ever dose of fitusiran (day 1) up to maximum 78 months
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Eligibility
Key inclusion criteria
Inclusion criteria :
- Participant must be at least 12 years of age inclusive, at the time of signing the
informed consent
- Participants with severe hemophilia A or B who have completed a Phase 3 fitusiran
clinical trial
- Male
- Capable of giving signed informed consent which includes compliance with the
requirements and restrictions listed in the informed consent form (ICF) and in the
protocol. In countries where legal age of majority is above 18 years, a specific ICF
must also be signed by the participant's legally authorized representative
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Minimum age
12
Years
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Maximum age
No limit
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Sex
Males
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Can healthy volunteers participate?
No
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Key exclusion criteria
Exclusion criteria:
- Completion of a surgical procedure within 14 days prior to screening, or currently
receiving additional factor concentrate or BPA infusion for postoperative hemostasis
- Current participation in immune tolerance induction treatment (ITI)
- Current use of factor concentrates or bypassing agents (BPAs) as regularly
administered prophylaxis designed to prevent spontaneous bleeding episodes except for
participants requiring factor concentrates or BPAs prophylaxis during the study dosing
pause period
- Use of compounds other than factor concentrates or BPAs for hemophilia treatment
- Current or prior participation in a gene therapy trial
- Alanine aminotransferase (ALT) and/or Aspartate aminotransferase (AST) >1.5 × upper
limit of normal reference range (ULN) for patients who are naïve to fitusiran at study
start; ALT and/or AST > 5 x ULN for patients who were in the fitusiran arm in the
parent study
- Additional exclusions for participants not currently participating in a fitusiran
trial at the time of enrollment in the lower dose cohort:
- Clinically significant liver disease
- History of arterial or venous thromboembolism
The above information is not intended to contain all considerations relevant to a patient's
potential participation in a clinical trial.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
N/A
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Active, not recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
9/01/2019
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
5/11/2026
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Actual
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Sample size
Target
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Accrual to date
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Final
281
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Recruitment in Australia
Recruitment state(s)
NSW,VIC,WA
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Recruitment hospital [1]
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Investigational Site Number : 0360001 - Camperdown
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Recruitment hospital [2]
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Investigational Site Number : 0360003 - Prahran
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Recruitment hospital [3]
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Investigational Site Number : 0360002 - Murdoch
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Recruitment postcode(s) [1]
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2050 - Camperdown
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Recruitment postcode(s) [2]
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3181 - Prahran
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Recruitment postcode(s) [3]
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6961 - Murdoch
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Recruitment outside Australia
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Arizona
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Beijing
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Changsha
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Chengdu
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Guangzhou
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China
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Guiyang
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China
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Hangzhou
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China
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Jinan
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China
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Kunming
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China
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Lanzhou
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China
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Qingdao
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Lyon
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Paris
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Berlin
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Germany
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Leipzig
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Lucknow
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Ramat Gan
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Lviv
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Genzyme, a Sanofi Company
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Address
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Ethics approval
Ethics application status
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Summary
Brief summary
Primary Objective:
To characterize the long-term safety and tolerability of fitusiran
Secondary Objectives:
- To characterize the efficacy and long-term efficacy of fitusiran as assessed by the
frequency of:
- Bleeding episodes
- Spontaneous bleeding episodes
- Joint bleeding episodes
- To characterize the effects of fitusiran on health-related quality of life (HRQOL)
measures in participants =17 years of age
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Trial website
https://clinicaltrials.gov/ct2/show/NCT03754790
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Clinical Sciences & Operations
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Address
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Sanofi
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT03754790
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