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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03417102




Registration number
NCT03417102
Ethics application status
Date submitted
25/01/2018
Date registered
31/01/2018
Date last updated
20/05/2020

Titles & IDs
Public title
A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors
Scientific 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
Secondary ID [1] 0 0
2016-001463-36
Secondary ID [2] 0 0
EFC14768
Universal Trial Number (UTN)
Trial acronym
ATLAS-INH
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hemophilia A 0 0
Hemophilia B 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
Treatment: Drugs - Bypassing agents

Experimental: Fitusiran - 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

Active Comparator: On demand bypassing agents - On-demand bypassing agents (BPAs) for treatment of breakthrough bleeding episodes. All patients will be treated for a total of 9 months.


Treatment: Drugs: fitusiran
solution for injection; by subcutaneous (SC) injection

Treatment: Drugs: Bypassing agents
solution for injection; by intravenous (IV) injection

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Annualized bleeding rate (ABR)
Timepoint [1] 0 0
8 months (efficacy period: day 29 to Month 9)
Secondary outcome [1] 0 0
Annualized bleeding rate (ABR)
Timepoint [1] 0 0
At 1 month (onset period) , 9 months (treatment period: 1 month onset period + efficacy period of 8 months)
Secondary outcome [2] 0 0
Annualized spontaneous bleeding rate
Timepoint [2] 0 0
8 months (efficacy period: day 29 to Month 9)
Secondary outcome [3] 0 0
Annualized joint bleeding rate
Timepoint [3] 0 0
8 months (efficacy period: day 29 to Month 9)
Secondary outcome [4] 0 0
Quality of Life (QOL) as measured by Haem-A-POL Questionnaire score on a scale of 1-100 with higher scores representing greater impairment
Timepoint [4] 0 0
9 months (treatment period: 1 month onset period + efficacy period of 8 months)
Secondary outcome [5] 0 0
Number of participants reported with treatment-emergent adverse events
Timepoint [5] 0 0
15 months (9 months treatment + 6 months follow up)

Eligibility
Key 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
Minimum age
12 Years
Maximum age
No limit
Gender
Males
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
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
Parallel
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
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Investigational Site Number 6101 - Camperdown
Recruitment hospital [2] 0 0
Investigational Site Number 6104 - Clayton
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment postcode(s) [2] 0 0
3168 - Clayton
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
Louisiana
Country [5] 0 0
United States of America
State/province [5] 0 0
Maryland
Country [6] 0 0
United States of America
State/province [6] 0 0
Nevada
Country [7] 0 0
United States of America
State/province [7] 0 0
Pennsylvania
Country [8] 0 0
Canada
State/province [8] 0 0
Montreal
Country [9] 0 0
China
State/province [9] 0 0
Beijing
Country [10] 0 0
China
State/province [10] 0 0
Guangzhou
Country [11] 0 0
China
State/province [11] 0 0
Hangzhou
Country [12] 0 0
China
State/province [12] 0 0
Shanghai
Country [13] 0 0
China
State/province [13] 0 0
Tianjin
Country [14] 0 0
France
State/province [14] 0 0
Lyon
Country [15] 0 0
France
State/province [15] 0 0
Rouen
Country [16] 0 0
Germany
State/province [16] 0 0
Frankfurt Am Main
Country [17] 0 0
Germany
State/province [17] 0 0
Leipzig
Country [18] 0 0
India
State/province [18] 0 0
Bangalore
Country [19] 0 0
India
State/province [19] 0 0
India
Country [20] 0 0
India
State/province [20] 0 0
Jaipur
Country [21] 0 0
India
State/province [21] 0 0
Lucknow
Country [22] 0 0
India
State/province [22] 0 0
Pune
Country [23] 0 0
India
State/province [23] 0 0
Vellore
Country [24] 0 0
Italy
State/province [24] 0 0
Florence
Country [25] 0 0
Italy
State/province [25] 0 0
Padua
Country [26] 0 0
Japan
State/province [26] 0 0
Japan
Country [27] 0 0
Japan
State/province [27] 0 0
Kita Kyushu-Shi
Country [28] 0 0
Korea, Republic of
State/province [28] 0 0
Daejeon
Country [29] 0 0
Korea, Republic of
State/province [29] 0 0
Seoul
Country [30] 0 0
Malaysia
State/province [30] 0 0
Kota Kinabalu
Country [31] 0 0
Malaysia
State/province [31] 0 0
Malaysia
Country [32] 0 0
South Africa
State/province [32] 0 0
Parktown
Country [33] 0 0
South Africa
State/province [33] 0 0
Polokwane
Country [34] 0 0
South Africa
State/province [34] 0 0
Port Elizabeth
Country [35] 0 0
Spain
State/province [35] 0 0
Madrid
Country [36] 0 0
Taiwan
State/province [36] 0 0
Changhua
Country [37] 0 0
Taiwan
State/province [37] 0 0
Taipei
Country [38] 0 0
Taiwan
State/province [38] 0 0
Taiwan
Country [39] 0 0
Turkey
State/province [39] 0 0
Adana
Country [40] 0 0
Turkey
State/province [40] 0 0
Akdeniz
Country [41] 0 0
Turkey
State/province [41] 0 0
Ankara
Country [42] 0 0
Turkey
State/province [42] 0 0
Istanbul
Country [43] 0 0
Turkey
State/province [43] 0 0
Izmir
Country [44] 0 0
Turkey
State/province [44] 0 0
Turkey
Country [45] 0 0
Ukraine
State/province [45] 0 0
Kyiv
Country [46] 0 0
Ukraine
State/province [46] 0 0
Lviv
Country [47] 0 0
United Kingdom
State/province [47] 0 0
London

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

Ethics approval
Ethics application status

Summary
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).
Trial website
https://clinicaltrials.gov/show/NCT03417102
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Clinical Sciences & Operations, MD
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

Summary results
Other publications