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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03754790




Registration number
NCT03754790
Ethics application status
Date submitted
25/11/2018
Date registered
27/11/2018

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
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
Secondary ID [1] 0 0
U1111-1210-0018
Secondary ID [2] 0 0
LTE15174
Universal Trial Number (UTN)
Trial acronym
ATLAS-OLE
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hemophilia 0 0
Condition category
Condition code
Blood 0 0 0 0
Clotting disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
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
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of participants with treatment emergent adverse events (TEAEs)
Timepoint [1] 0 0
from study baseline (day 1) up to maximum 88 months
Secondary outcome [1] 0 0
Annualized bleeding rate (ABR)
Timepoint [1] 0 0
from first ever dose of fitusiran (day 1) up to maximum 78 months
Secondary outcome [2] 0 0
Annualized spontaneous bleeding rate
Timepoint [2] 0 0
from first ever dose of fitusiran (day 1) up to maximum 78 months
Secondary outcome [3] 0 0
Annualized joint bleeding rate
Timepoint [3] 0 0
from first ever dose of fitusiran (day 1) up to maximum 78 months
Secondary outcome [4] 0 0
Changes in Haem-A-quality of life (QoL) score
Timepoint [4] 0 0
from first ever dose of fitusiran (day 1) up to maximum 78 months

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
Minimum age
12 Years
Maximum age
No limit
Sex
Males
Can healthy volunteers participate?
No
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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
9/01/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
5/11/2026
Actual
Sample size
Target
Accrual to date
Final
281
Recruitment in Australia
Recruitment state(s)
NSW,VIC,WA
Recruitment hospital [1] 0 0
Investigational Site Number : 0360001 - Camperdown
Recruitment hospital [2] 0 0
Investigational Site Number : 0360003 - Prahran
Recruitment hospital [3] 0 0
Investigational Site Number : 0360002 - Murdoch
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment postcode(s) [2] 0 0
3181 - Prahran
Recruitment postcode(s) [3] 0 0
6961 - Murdoch
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Illinois
Country [5] 0 0
United States of America
State/province [5] 0 0
Massachusetts
Country [6] 0 0
United States of America
State/province [6] 0 0
Michigan
Country [7] 0 0
United States of America
State/province [7] 0 0
Nevada
Country [8] 0 0
United States of America
State/province [8] 0 0
Ohio
Country [9] 0 0
Canada
State/province [9] 0 0
Quebec
Country [10] 0 0
China
State/province [10] 0 0
Beijing
Country [11] 0 0
China
State/province [11] 0 0
Changsha
Country [12] 0 0
China
State/province [12] 0 0
Chengdu
Country [13] 0 0
China
State/province [13] 0 0
Guangzhou
Country [14] 0 0
China
State/province [14] 0 0
Guiyang
Country [15] 0 0
China
State/province [15] 0 0
Hangzhou
Country [16] 0 0
China
State/province [16] 0 0
Jinan
Country [17] 0 0
China
State/province [17] 0 0
Kunming
Country [18] 0 0
China
State/province [18] 0 0
Lanzhou
Country [19] 0 0
China
State/province [19] 0 0
Qingdao
Country [20] 0 0
China
State/province [20] 0 0
Shanghai
Country [21] 0 0
China
State/province [21] 0 0
Suzhou
Country [22] 0 0
Denmark
State/province [22] 0 0
Copenhagen
Country [23] 0 0
France
State/province [23] 0 0
Lyon
Country [24] 0 0
France
State/province [24] 0 0
Paris
Country [25] 0 0
Germany
State/province [25] 0 0
Berlin
Country [26] 0 0
Germany
State/province [26] 0 0
Leipzig
Country [27] 0 0
Hungary
State/province [27] 0 0
Budapest
Country [28] 0 0
India
State/province [28] 0 0
Bangalore
Country [29] 0 0
India
State/province [29] 0 0
Lucknow
Country [30] 0 0
India
State/province [30] 0 0
Mumbai
Country [31] 0 0
India
State/province [31] 0 0
Pune
Country [32] 0 0
India
State/province [32] 0 0
Ranipet
Country [33] 0 0
Ireland
State/province [33] 0 0
Dublin
Country [34] 0 0
Israel
State/province [34] 0 0
Ramat Gan
Country [35] 0 0
Italy
State/province [35] 0 0
Lombardia
Country [36] 0 0
Italy
State/province [36] 0 0
Veneto
Country [37] 0 0
Japan
State/province [37] 0 0
Hyogo
Country [38] 0 0
Japan
State/province [38] 0 0
Kanagawa
Country [39] 0 0
Japan
State/province [39] 0 0
Kitakyushu
Country [40] 0 0
Japan
State/province [40] 0 0
Nagoya
Country [41] 0 0
Japan
State/province [41] 0 0
Saitama
Country [42] 0 0
Japan
State/province [42] 0 0
Tokyo
Country [43] 0 0
Korea, Republic of
State/province [43] 0 0
Busan-gwangyeoksi
Country [44] 0 0
Korea, Republic of
State/province [44] 0 0
Daejeon-gwangyeoksi
Country [45] 0 0
Korea, Republic of
State/province [45] 0 0
Seoul-teukbyeolsi
Country [46] 0 0
Korea, Republic of
State/province [46] 0 0
Seoul
Country [47] 0 0
Malaysia
State/province [47] 0 0
Ampang
Country [48] 0 0
Malaysia
State/province [48] 0 0
Johor Bahru
Country [49] 0 0
Malaysia
State/province [49] 0 0
Kota Kinabalu
Country [50] 0 0
South Africa
State/province [50] 0 0
Port Elizabeth
Country [51] 0 0
Taiwan
State/province [51] 0 0
Changhua
Country [52] 0 0
Taiwan
State/province [52] 0 0
Taichung
Country [53] 0 0
Taiwan
State/province [53] 0 0
Taipei
Country [54] 0 0
Taiwan
State/province [54] 0 0
Taoyuan City
Country [55] 0 0
Turkey
State/province [55] 0 0
Adana
Country [56] 0 0
Turkey
State/province [56] 0 0
Akdeniz
Country [57] 0 0
Turkey
State/province [57] 0 0
Bornova
Country [58] 0 0
Turkey
State/province [58] 0 0
Gaziantep
Country [59] 0 0
Turkey
State/province [59] 0 0
Istanbul
Country [60] 0 0
Turkey
State/province [60] 0 0
Izmir
Country [61] 0 0
Turkey
State/province [61] 0 0
Kayseri
Country [62] 0 0
Turkey
State/province [62] 0 0
Samsun
Country [63] 0 0
Ukraine
State/province [63] 0 0
Kyiv
Country [64] 0 0
Ukraine
State/province [64] 0 0
Lviv
Country [65] 0 0
Ukraine
State/province [65] 0 0
Mykolaiv
Country [66] 0 0
United Kingdom
State/province [66] 0 0
London, City Of
Country [67] 0 0
United Kingdom
State/province [67] 0 0
Glasgow

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Genzyme, a Sanofi Company
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Clinical Sciences & Operations
Address 0 0
Sanofi
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
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: https://vivli.org
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT03754790