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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 (ATLAS-OLE)

Primary Purpose

Hemophilia

Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Fitusiran
Sponsored by
Genzyme, a Sanofi Company
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hemophilia

Eligibility Criteria

12 Years - undefined (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

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

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.

Sites / Locations

  • Investigational Site Number :8400009
  • Investigational Site Number :8400019
  • Investigational Site Number :8400016
  • Investigational Site Number :8400008
  • Investigational Site Number :8400002
  • Investigational Site Number :8400001
  • Investigational Site Number :8400011
  • Investigational Site Number :8400012
  • Investigational Site Number :8400007
  • Investigational Site Number :8400006
  • Investigational Site Number :0360001
  • Investigational Site Number :0360003
  • Investigational Site Number :0360002
  • Investigational Site Number :1240001
  • Investigational Site Number :1560004
  • Investigational Site Number :1560007
  • Investigational Site Number :1560014
  • Investigational Site Number :1560009
  • Investigational Site Number :1560002
  • Investigational Site Number :1560011
  • Investigational Site Number :1560005
  • Investigational Site Number :1560008
  • Investigational Site Number :1560012
  • Investigational Site Number :1560013
  • Investigational Site Number :1560010
  • Investigational Site Number :1560003
  • Investigational Site Number :1560006
  • Investigational Site Number :2080001
  • Investigational Site Number :2500002
  • Investigational Site Number :2500003
  • Investigational Site Number :2760001
  • Investigational Site Number :2760003
  • Investigational Site Number :3480002
  • Investigational Site Number :3560001
  • Investigational Site Number :3560005
  • Investigational Site Number :3560007
  • Investigational Site Number :3560002
  • Investigational Site Number :3560010
  • Investigational Site Number :3560004
  • Investigational Site Number :3720001
  • Investigational Site Number :3720002
  • Investigational Site Number :3760001
  • Investigational Site Number :3800002
  • Investigational Site Number :3800004
  • Investigational Site Number :3920002
  • Investigational Site Number :3920005
  • Investigational Site Number :3920003
  • Investigational Site Number :3920001
  • Investigational Site Number :3920004
  • Investigational Site Number :3920008
  • Investigational Site Number :4100001
  • Investigational Site Number :4100002
  • Investigational Site Number :4100003
  • Investigational Site Number :4100004
  • Investigational Site Number :4580003
  • Investigational Site Number :4580001
  • Investigational Site Number :4580002
  • Investigational Site Number :7100002
  • Investigational Site Number :1580002
  • Investigational Site Number :1580008
  • Investigational Site Number :1580004
  • Investigational Site Number :1580003
  • Investigational Site Number :1580001
  • Investigational Site Number :1580005
  • Investigational Site Number :1580007
  • Investigational Site Number :7920002
  • Investigational Site Number :7920004
  • Investigational Site Number :7920012
  • Investigational Site Number :7920006
  • Investigational Site Number :7920005
  • Investigational Site Number :7920003
  • Investigational Site Number :7920008
  • Investigational Site Number :7920007
  • Investigational Site Number :8040001
  • Investigational Site Number :8040003
  • Investigational Site Number :8040002
  • Investigational Site Number :8040004
  • Investigational Site Number :8260001
  • Investigational Site Number :8260004

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Fitusiran

Arm Description

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.

Outcomes

Primary Outcome Measures

Number of participants with treatment emergent adverse events (TEAEs)
The number of participants experiencing any TEAEs, serious TEAEs, discontinuation due to TEAEs and death will be reported

Secondary Outcome Measures

Annualized bleeding rate (ABR)
Annualized bleeding rate (ABR) in the treatment period
Annualized spontaneous bleeding rate
Annualized spontaneous bleeding rate in the treatment period
Annualized joint bleeding rate
Annualized joint bleeding rate in the treatment period
Changes in Haem-A-quality of life (QoL) score
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

Full Information

First Posted
November 25, 2018
Last Updated
December 12, 2022
Sponsor
Genzyme, a Sanofi Company
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1. Study Identification

Unique Protocol Identification Number
NCT03754790
Brief 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
Acronym
ATLAS-OLE
Official 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
Study Type
Interventional

2. Study Status

Record Verification Date
December 2022
Overall Recruitment Status
Active, not recruiting
Study Start Date
January 9, 2019 (Actual)
Primary Completion Date
December 18, 2026 (Anticipated)
Study Completion Date
December 18, 2026 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Genzyme, a Sanofi Company

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
Yes

5. Study Description

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
Detailed Description
The estimated total time on the study for a participant up to 90 months (including screening, treatment period and safety follow up period).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hemophilia

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
355 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Fitusiran
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
Fitusiran
Intervention Description
Pharmaceutical form:solution for injection Route of administration: subcutaneous
Primary Outcome Measure Information:
Title
Number of participants with treatment emergent adverse events (TEAEs)
Description
The number of participants experiencing any TEAEs, serious TEAEs, discontinuation due to TEAEs and death will be reported
Time Frame
from study baseline (day 1) up to maximum 88 months
Secondary Outcome Measure Information:
Title
Annualized bleeding rate (ABR)
Description
Annualized bleeding rate (ABR) in the treatment period
Time Frame
from first ever dose of fitusiran (day 1) up to maximum 78 months
Title
Annualized spontaneous bleeding rate
Description
Annualized spontaneous bleeding rate in the treatment period
Time Frame
from first ever dose of fitusiran (day 1) up to maximum 78 months
Title
Annualized joint bleeding rate
Description
Annualized joint bleeding rate in the treatment period
Time Frame
from first ever dose of fitusiran (day 1) up to maximum 78 months
Title
Changes in Haem-A-quality of life (QoL) score
Description
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
Time Frame
from first ever dose of fitusiran (day 1) up to maximum 78 months

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility 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 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Clinical Sciences & Operations
Organizational Affiliation
Sanofi
Official's Role
Study Director
Facility Information:
Facility Name
Investigational Site Number :8400009
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85016
Country
United States
Facility Name
Investigational Site Number :8400019
City
Los Angeles
State/Province
California
ZIP/Postal Code
90027
Country
United States
Facility Name
Investigational Site Number :8400016
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Facility Name
Investigational Site Number :8400008
City
Jacksonville
State/Province
Florida
ZIP/Postal Code
32207
Country
United States
Facility Name
Investigational Site Number :8400002
City
Tampa
State/Province
Florida
ZIP/Postal Code
33607
Country
United States
Facility Name
Investigational Site Number :8400001
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Facility Name
Investigational Site Number :8400011
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Facility Name
Investigational Site Number :8400012
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109
Country
United States
Facility Name
Investigational Site Number :8400007
City
Las Vegas
State/Province
Nevada
ZIP/Postal Code
89135
Country
United States
Facility Name
Investigational Site Number :8400006
City
Akron
State/Province
Ohio
ZIP/Postal Code
44308
Country
United States
Facility Name
Investigational Site Number :0360001
City
Camperdown
State/Province
New South Wales
ZIP/Postal Code
2050
Country
Australia
Facility Name
Investigational Site Number :0360003
City
Prahran
State/Province
Victoria
ZIP/Postal Code
3181
Country
Australia
Facility Name
Investigational Site Number :0360002
City
Murdoch
State/Province
Western Australia
ZIP/Postal Code
6961
Country
Australia
Facility Name
Investigational Site Number :1240001
City
Montreal
State/Province
Quebec
ZIP/Postal Code
H1T 2M4
Country
Canada
Facility Name
Investigational Site Number :1560004
City
Beijing
ZIP/Postal Code
100045
Country
China
Facility Name
Investigational Site Number :1560007
City
Beijing
ZIP/Postal Code
100730
Country
China
Facility Name
Investigational Site Number :1560014
City
Changsha
ZIP/Postal Code
410008
Country
China
Facility Name
Investigational Site Number :1560009
City
Chengdu
ZIP/Postal Code
610091
Country
China
Facility Name
Investigational Site Number :1560002
City
Guangzhou
ZIP/Postal Code
510515
Country
China
Facility Name
Investigational Site Number :1560011
City
Guiyang
ZIP/Postal Code
550004
Country
China
Facility Name
Investigational Site Number :1560005
City
Hangzhou
ZIP/Postal Code
89147
Country
China
Facility Name
Investigational Site Number :1560008
City
Jinan
ZIP/Postal Code
250013
Country
China
Facility Name
Investigational Site Number :1560012
City
Kunming
ZIP/Postal Code
650000
Country
China
Facility Name
Investigational Site Number :1560013
City
Lanzhou
ZIP/Postal Code
730000
Country
China
Facility Name
Investigational Site Number :1560010
City
Qingdao
ZIP/Postal Code
266555
Country
China
Facility Name
Investigational Site Number :1560003
City
Shanghai
ZIP/Postal Code
200025
Country
China
Facility Name
Investigational Site Number :1560006
City
Suzhou
ZIP/Postal Code
215006
Country
China
Facility Name
Investigational Site Number :2080001
City
Copenhagen
ZIP/Postal Code
2100
Country
Denmark
Facility Name
Investigational Site Number :2500002
City
Lyon
ZIP/Postal Code
69677
Country
France
Facility Name
Investigational Site Number :2500003
City
Paris
ZIP/Postal Code
75015
Country
France
Facility Name
Investigational Site Number :2760001
City
Berlin
ZIP/Postal Code
10249
Country
Germany
Facility Name
Investigational Site Number :2760003
City
Leipzig
ZIP/Postal Code
04103
Country
Germany
Facility Name
Investigational Site Number :3480002
City
Budapest
ZIP/Postal Code
1134
Country
Hungary
Facility Name
Investigational Site Number :3560001
City
Bangalore
ZIP/Postal Code
560034
Country
India
Facility Name
Investigational Site Number :3560005
City
Lucknow
ZIP/Postal Code
226003
Country
India
Facility Name
Investigational Site Number :3560007
City
Mumbai
ZIP/Postal Code
400 022
Country
India
Facility Name
Investigational Site Number :3560002
City
Pune
ZIP/Postal Code
411001
Country
India
Facility Name
Investigational Site Number :3560010
City
Pune
ZIP/Postal Code
411004
Country
India
Facility Name
Investigational Site Number :3560004
City
Vellore
ZIP/Postal Code
632004
Country
India
Facility Name
Investigational Site Number :3720001
City
Crumlin
State/Province
Dublin
ZIP/Postal Code
12
Country
Ireland
Facility Name
Investigational Site Number :3720002
City
Dublin
ZIP/Postal Code
DUBLIN 8
Country
Ireland
Facility Name
Investigational Site Number :3760001
City
Ramat Gan
ZIP/Postal Code
52662
Country
Israel
Facility Name
Investigational Site Number :3800002
City
Milano
State/Province
Lombardia
ZIP/Postal Code
20121
Country
Italy
Facility Name
Investigational Site Number :3800004
City
Padua
State/Province
Veneto
ZIP/Postal Code
35128
Country
Italy
Facility Name
Investigational Site Number :3920002
City
Nishinomiya
State/Province
Hyogo
ZIP/Postal Code
663-8501
Country
Japan
Facility Name
Investigational Site Number :3920005
City
Isehara
State/Province
Kanagawa
ZIP/Postal Code
2591193
Country
Japan
Facility Name
Investigational Site Number :3920003
City
Kitakyushu
ZIP/Postal Code
8078555
Country
Japan
Facility Name
Investigational Site Number :3920001
City
Nagoya
ZIP/Postal Code
4668560
Country
Japan
Facility Name
Investigational Site Number :3920004
City
Saitama
ZIP/Postal Code
330-8777
Country
Japan
Facility Name
Investigational Site Number :3920008
City
Tokyo
ZIP/Postal Code
1600023
Country
Japan
Facility Name
Investigational Site Number :4100001
City
Busan
State/Province
Busan-gwangyeoksi
ZIP/Postal Code
602-739
Country
Korea, Republic of
Facility Name
Investigational Site Number :4100002
City
Daejeon
State/Province
Daejeon-gwangyeoksi
ZIP/Postal Code
35233
Country
Korea, Republic of
Facility Name
Investigational Site Number :4100003
City
Seoul
State/Province
Seoul-teukbyeolsi
ZIP/Postal Code
03722
Country
Korea, Republic of
Facility Name
Investigational Site Number :4100004
City
Seoul
ZIP/Postal Code
3722
Country
Korea, Republic of
Facility Name
Investigational Site Number :4580003
City
Ampang
ZIP/Postal Code
68000
Country
Malaysia
Facility Name
Investigational Site Number :4580001
City
Johor Bahru
ZIP/Postal Code
80100
Country
Malaysia
Facility Name
Investigational Site Number :4580002
City
Kota Kinabalu
ZIP/Postal Code
88586
Country
Malaysia
Facility Name
Investigational Site Number :7100002
City
Port Elizabeth
ZIP/Postal Code
6001
Country
South Africa
Facility Name
Investigational Site Number :1580002
City
Changhua
ZIP/Postal Code
500
Country
Taiwan
Facility Name
Investigational Site Number :1580008
City
Taichung
ZIP/Postal Code
40201
Country
Taiwan
Facility Name
Investigational Site Number :1580004
City
Taichung
ZIP/Postal Code
40705
Country
Taiwan
Facility Name
Investigational Site Number :1580003
City
Taipei
ZIP/Postal Code
100
Country
Taiwan
Facility Name
Investigational Site Number :1580001
City
Taipei
ZIP/Postal Code
110
Country
Taiwan
Facility Name
Investigational Site Number :1580005
City
Taipei
ZIP/Postal Code
112
Country
Taiwan
Facility Name
Investigational Site Number :1580007
City
Taoyuan City
ZIP/Postal Code
33305
Country
Taiwan
Facility Name
Investigational Site Number :7920002
City
Adana
ZIP/Postal Code
01130
Country
Turkey
Facility Name
Investigational Site Number :7920004
City
Akdeniz
ZIP/Postal Code
07059
Country
Turkey
Facility Name
Investigational Site Number :7920012
City
Bornova
ZIP/Postal Code
35100
Country
Turkey
Facility Name
Investigational Site Number :7920006
City
Gaziantep
ZIP/Postal Code
27100
Country
Turkey
Facility Name
Investigational Site Number :7920005
City
Istanbul
ZIP/Postal Code
34093
Country
Turkey
Facility Name
Investigational Site Number :7920003
City
Izmir
ZIP/Postal Code
TR-35100
Country
Turkey
Facility Name
Investigational Site Number :7920008
City
Kayseri
ZIP/Postal Code
38039
Country
Turkey
Facility Name
Investigational Site Number :7920007
City
Samsun
ZIP/Postal Code
55200
Country
Turkey
Facility Name
Investigational Site Number :8040001
City
Kyiv
ZIP/Postal Code
04060
Country
Ukraine
Facility Name
Investigational Site Number :8040003
City
Kyiv
ZIP/Postal Code
1135
Country
Ukraine
Facility Name
Investigational Site Number :8040002
City
Lviv
ZIP/Postal Code
79044
Country
Ukraine
Facility Name
Investigational Site Number :8040004
City
Mykolaiv
ZIP/Postal Code
54058
Country
Ukraine
Facility Name
Investigational Site Number :8260001
City
London
State/Province
London, City Of
ZIP/Postal Code
SE1 9RT
Country
United Kingdom
Facility Name
Investigational Site Number :8260004
City
Glasgow
ZIP/Postal Code
G4 0SF
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing Plan Description
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

Learn more about this trial

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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