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HemophiliaPhase 3ATLAS-OLENCT03754790

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

A Phase 3 study testing Fitusiran in people with Hemophilia, run by Genzyme, a Sanofi Company at 79 sites in 20 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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… Read more about this study

Who is it for?
Ages 12 years and older, men only
Conditions the study is for
Hemophilia
What is being tested?
Fitusiran
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
281 participants
Where
79 sitesin 20 countries
Study dates
9 Jan 2019to 14 Sep 2026
Phase
Phase 3
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 12 years and older
  • Men only
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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
Show all 10Show fewer
  • 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.

Trial locations and contacts

79 sites in 20 countries.

79 sites in 20 countries

China13 sites

  • Investigational Site Number : 1560004

    Beijing, 100045

  • Investigational Site Number : 1560007

    Beijing, 100730

  • Investigational Site Number : 1560014

    Changsha, 410008

  • Investigational Site Number : 1560009

    Chengdu, 610091

  • Investigational Site Number : 1560002

    Guangzhou, 510515

  • Investigational Site Number : 1560011

    Guiyang, 550004

  • Investigational Site Number : 1560005

    Hangzhou, 89147

  • Investigational Site Number : 1560008

    Jinan, 250013

  • Investigational Site Number : 1560012

    Kunming, 650000

  • Investigational Site Number : 1560013

    Lanzhou, 730000

  • Investigational Site Number : 1560010

    Qingdao, 266555

  • Investigational Site Number : 1560003

    Shanghai, 200025

  • Investigational Site Number : 1560006

    Suzhou, 215006

Other trials in China

United States10 sites

  • Childrens Hospital Medical Center of Akron Site Number : 8400006

    Akron, Ohio, 44308

  • University of Michigan Hospital - 1500 E Medical Center Dr Site Number : 8400012

    Ann Arbor, Michigan, 48109-5000

  • ~Massachusetts General Hospital Site Number : 8400011

    Boston, Massachusetts, 02114

  • Rush University Medical Center -1725 W Harrison St Site Number : 8400001

    Chicago, Illinois, 60612-3863

  • Nemours Children's Clinic Site Number : 8400008

    Jacksonville, Florida, 32207

  • Alliance for Childhood Diseases Site Number : 8400007

    Las Vegas, Nevada, 89135-3011

  • Children's Hospital Los Angeles Site Number : 8400019

    Los Angeles, California, 90027

  • Center for Inherited Blood Disorders (CIBD) Site Number : 8400016

    Orange, California, 92868

  • Phoenix Childrens Hospital Site Number : 8400009

    Phoenix, Arizona, 85016

  • St Joseph's Children's Hospital of Tampa Site Number : 8400002

    Tampa, Florida, 33607

Other trials in United States

Turkey (Türkiye)8 sites

  • Investigational Site Number : 7920002

    Adana, 01130

  • Investigational Site Number : 7920004

    Akdeniz, 07059

  • Investigational Site Number : 7920012

    Bornova, 35100

  • Investigational Site Number : 7920006

    Gaziantep, 27100

  • Investigational Site Number : 7920005

    Istanbul, 34093

  • Investigational Site Number : 7920003

    Izmir, TR-35100

  • Investigational Site Number : 7920008

    Kayseri, 38039

  • Investigational Site Number : 7920007

    Samsun, 55200

Other trials in Turkey (Türkiye)

Taiwan7 sites

  • Investigational Site Number : 1580002

    Changhua, 500

  • Investigational Site Number : 1580008

    Taichung, 40201

  • Investigational Site Number : 1580004

    Taichung, 40705

  • Investigational Site Number : 1580003

    Taipei, 100

  • Investigational Site Number : 1580001

    Taipei, 110

  • Investigational Site Number : 1580005

    Taipei, 112

  • Investigational Site Number : 1580007

    Taoyuan City, 33305

Other trials in Taiwan

India6 sites

  • Investigational Site Number : 3560001

    Bangalore, 560034

  • Investigational Site Number : 3560005

    Lucknow, 226003

  • Investigational Site Number : 3560007

    Mumbai, 400 022

  • Investigational Site Number : 3560002

    Pune, 411001

  • Investigational Site Number : 3560010

    Pune, 411004

  • Investigational Site Number : 3560004

    Ranipet, 632517

Other trials in India

Japan6 sites

  • Investigational Site Number : 3920005

    Isehara, Kanagawa, 2591193

  • Investigational Site Number : 3920003

    Kitakyushu, 8078555

  • Investigational Site Number : 3920001

    Nagoya, 4668560

  • Investigational Site Number : 3920002

    Nishinomiya, Hyōgo, 663-8501

  • Investigational Site Number : 3920004

    Saitama, 330-8777

  • Investigational Site Number : 3920008

    Tokyo, 1600023

Other trials in Japan

South Korea4 sites

  • Investigational Site Number : 4100001

    Busan, Busan, 602-739

  • Investigational Site Number : 4100002

    Daejeon, Daejeon, 35233

  • Investigational Site Number : 4100003

    Seoul, Seoul-teukbyeolsi, 03722

  • Investigational Site Number : 4100004

    Seoul, 3722

Other trials in South Korea

Ukraine4 sites

  • Investigational Site Number : 8040001

    Kyiv, 04060

  • Investigational Site Number : 8040003

    Kyiv, 1135

  • Investigational Site Number : 8040002

    Lviv, 79044

  • Investigational Site Number : 8040004

    Mykolaiv, 54058

Other trials in Ukraine

Australia3 sites

  • Investigational Site Number : 0360001

    Camperdown, New South Wales, 2050

  • Investigational Site Number : 0360002

    Murdoch, Western Australia, 6961

  • Investigational Site Number : 0360003

    Prahran, Victoria, 3181

Other trials in Australia

Malaysia3 sites

  • Investigational Site Number : 4580003

    Ampang, 68000

  • Investigational Site Number : 4580001

    Johor Bahru, 80100

  • Investigational Site Number : 4580002

    Kota Kinabalu, 88586

Other trials in Malaysia

France2 sites

  • Investigational Site Number : 2500002

    Lyon, 69677

  • Investigational Site Number : 2500003

    Paris, 75015

Other trials in France

Germany2 sites

  • Investigational Site Number : 2760001

    Berlin, 10249

  • Investigational Site Number : 2760003

    Leipzig, 04103

Other trials in Germany

Ireland2 sites

  • Investigational Site Number : 3720001

    Crumlin, Dublin, 12

  • Investigational Site Number : 3720002

    Dublin, 8

Other trials in Ireland

Italy2 sites

  • Investigational Site Number : 3800002

    Milan, Lombardy, 20122

  • Investigational Site Number : 3800004

    Padua, Veneto, 35128

Other trials in Italy

United Kingdom2 sites

  • Investigational Site Number : 8260004

    Glasgow, G4 0SF

  • Investigational Site Number : 8260001

    London, London, City of, SE1 9RT

Other trials in United Kingdom

Canada1 site

  • Investigational Site Number : 1240001

    Montreal, Quebec, H1T 2M4

Other trials in Canada

Denmark1 site

  • Investigational Site Number : 2080001

    Copenhagen, 2100

Other trials in Denmark

Hungary1 site

  • Investigational Site Number : 3480002

    Budapest, 1134

Other trials in Hungary

Israel1 site

  • Investigational Site Number : 3760001

    Ramat Gan, 5262100

Other trials in Israel

South Africa1 site

  • Investigational Site Number : 7100002

    Port Elizabeth, 6001

Other trials in South Africa

About this study

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

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
Read the detailed description

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

Conditions
Hemophilia
Being tested
Fitusiran
Medical subject headings
Hemophilia A

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • FitusiranDrug

    Pharmaceutical form:solution for injection Route of administration: subcutaneous

Study groups in NCT03754790
Study groupRoleWhat they receive
FitusiranExperimentalParticipants 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.
Receives: Drug: Fitusiran

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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

    Measured: from study baseline (day 1) up to maximum 88 months

Secondary outcomes (4)

  1. Annualized bleeding rate (ABR)

    Annualized bleeding rate (ABR) in the treatment period

    Measured: from first ever dose of fitusiran (day 1) up to maximum 78 months

  2. Annualized spontaneous bleeding rate

    Annualized spontaneous bleeding rate in the treatment period

    Measured: from first ever dose of fitusiran (day 1) up to maximum 78 months

  3. Annualized joint bleeding rate

    Annualized joint bleeding rate in the treatment period

    Measured: from first ever dose of fitusiran (day 1) up to maximum 78 months

  4. 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) up to maximum 78 months

    Measured: from first ever dose of fitusiran (day 1) up to maximum 78 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted27 Nov 2018
  2. Study start9 Jan 2019
  3. Primary completion14 Sep 2026
  4. Study completion14 Sep 2026

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
281 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03754790
Sponsor's protocol ID
LTE15174
Other IDs
U1111-1210-0018, 2023-508884-59, 2018-002880-25, 2018, 2023
Acronym
ATLAS-OLE
Status last verified
Sep 2026
First submitted
25 Nov 2018
Last updated
30 Sep 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: 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

Publications

  • Young G, Kavakli K, Klamroth R, Matsushita T, Peyvandi F, Pipe SW, Rangarajan S, Shen MC, Srivastava A, Sun J, Tran H, You CW, Zulfikar B, Menapace LA, Zhang C, Shen Y, Puurunen M, Demissie M, Kenet G. Safety and efficacy of a fitusiran antithrombin-based dose regimen in people with hemophilia A or B: the ATLAS-OLE study. Blood. 2025 Jun 19;145(25):2966-2977. doi: 10.1182/blood.2024027008.

    PubMed 40053895

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Fitusiran in people with Hemophilia, run by Genzyme, a Sanofi Company at 79 sites in 20 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 30 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 79 sites in China, United States, Turkey (Türkiye), Taiwan, India and 15 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of participants with treatment emergent adverse events (TEAEs) (time frame: from study baseline (day 1) up to maximum 88 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03754790, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Sep 2026.

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