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

NCT03754790 | Active Not Recruiting Phase 3 DMC FDA Drug

• 4 other identifiers: LTE15174U1111-1210-00182023-508884-592018-002880-25
• Study Type: interventional
• Target: 281
• Locations: 20 countries
• Sites: 79

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

Conditions

Hemophilia

Outcome Measures

Primary Outcomes (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

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

Secondary Outcomes (4)

• Annualized bleeding rate (ABR)

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

• Annualized spontaneous bleeding rate

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

• Annualized joint bleeding rate

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

• Changes in Haem-A-quality of life (QoL) score

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

Study Arms (1)

Fitusiran

EXPERIMENTAL

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.

Drug: Fitusiran

Interventions

Fitusiran DRUG

Pharmaceutical form:solution for injection Route of administration: subcutaneous

Fitusiran

Eligibility Criteria

• Age: 12 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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
• 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.

Sponsors & Collaborators

• Genzyme, a Sanofi Company: lead

Study Sites (79)

Phoenix Childrens Hospital Site Number : 8400009

Phoenix, Arizona, 85016, United States

Location

Children's Hospital Los Angeles Site Number : 8400019

Los Angeles, California, 90027, United States

Location

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

Orange, California, 92868, United States

Location

Nemours Children's Clinic Site Number : 8400008

Jacksonville, Florida, 32207, United States

Location

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

Tampa, Florida, 33607, United States

Location

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

Chicago, Illinois, 60612-3863, United States

Location

~Massachusetts General Hospital Site Number : 8400011

Boston, Massachusetts, 02114, United States

Location

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

Ann Arbor, Michigan, 48109-5000, United States

Location

Alliance for Childhood Diseases Site Number : 8400007

Las Vegas, Nevada, 89135-3011, United States

Location

Childrens Hospital Medical Center of Akron Site Number : 8400006

Akron, Ohio, 44308, United States

Location

Investigational Site Number : 0360001

Camperdown, New South Wales, 2050, Australia

Location

Investigational Site Number : 0360003

Prahran, Victoria, 3181, Australia

Location

Investigational Site Number : 0360002

Murdoch, Western Australia, 6961, Australia

Location

Investigational Site Number : 1240001

Montreal, Quebec, H1T 2M4, Canada

Location

Investigational Site Number : 1560004

Beijing, 100045, China

Location

Investigational Site Number : 1560007

Beijing, 100730, China

Location

Investigational Site Number : 1560014

Changsha, 410008, China

Location

Investigational Site Number : 1560009

Chengdu, 610091, China

Location

Investigational Site Number : 1560002

Guangzhou, 510515, China

Location

Investigational Site Number : 1560011

Guiyang, 550004, China

Location

Investigational Site Number : 1560005

Hangzhou, 89147, China

Location

Investigational Site Number : 1560008

Jinan, 250013, China

Location

Investigational Site Number : 1560012

Kunming, 650000, China

Location

Investigational Site Number : 1560013

Lanzhou, 730000, China

Location

Investigational Site Number : 1560010

Qingdao, 266555, China

Location

Investigational Site Number : 1560003

Shanghai, 200025, China

Location

Investigational Site Number : 1560006

Suzhou, 215006, China

Location

Investigational Site Number : 2080001

Copenhagen, 2100, Denmark

Location

Investigational Site Number : 2500002

Lyon, 69677, France

Location

Investigational Site Number : 2500003

Paris, 75015, France

Location

Investigational Site Number : 2760001

Berlin, 10249, Germany

Location

Investigational Site Number : 2760003

Leipzig, 04103, Germany

Location

Investigational Site Number : 3480002

Budapest, 1134, Hungary

Location

Investigational Site Number : 3560001

Bangalore, 560034, India

Location

Investigational Site Number : 3560005

Lucknow, 226003, India

Location

Investigational Site Number : 3560007

Mumbai, 400 022, India

Location

Investigational Site Number : 3560002

Pune, 411001, India

Location

Investigational Site Number : 3560010

Pune, 411004, India

Location

Investigational Site Number : 3560004

Ranipet, 632517, India

Location

Investigational Site Number : 3720001

Crumlin, Dublin, 12, Ireland

Location

Investigational Site Number : 3720002

Dublin, 8, Ireland

Location

Investigational Site Number : 3760001

Ramat Gan, 5262100, Israel

Location

Investigational Site Number : 3800002

Milan, Lombardy, 20122, Italy

Location

Investigational Site Number : 3800004

Padua, Veneto, 35128, Italy

Location

Investigational Site Number : 3920002

Nishinomiya, Hyōgo, 663-8501, Japan

Location

Investigational Site Number : 3920005

Isehara, Kanagawa, 2591193, Japan

Location

Investigational Site Number : 3920003

Kitakyushu, 8078555, Japan

Location

Investigational Site Number : 3920001

Nagoya, 4668560, Japan

Location

Investigational Site Number : 3920004

Saitama, 330-8777, Japan

Location

Investigational Site Number : 3920008

Tokyo, 1600023, Japan

Location

Investigational Site Number : 4580003

Ampang, 68000, Malaysia

Location

Investigational Site Number : 4580001

Johor Bahru, 80100, Malaysia

Location

Investigational Site Number : 4580002

Kota Kinabalu, 88586, Malaysia

Location

Investigational Site Number : 7100002

Port Elizabeth, 6001, South Africa

Location

Investigational Site Number : 4100001

Busan, Busan, 602-739, South Korea

Location

Investigational Site Number : 4100002

Daejeon, Daejeon, 35233, South Korea

Location

Investigational Site Number : 4100003

Seoul, Seoul-teukbyeolsi, 03722, South Korea

Location

Investigational Site Number : 4100004

Seoul, 3722, South Korea

Location

Investigational Site Number : 1580002

Changhua, 500, Taiwan

Location

Investigational Site Number : 1580008

Taichung, 40201, Taiwan

Location

Investigational Site Number : 1580004

Taichung, 40705, Taiwan

Location

Investigational Site Number : 1580003

Taipei, 100, Taiwan

Location

Investigational Site Number : 1580001

Taipei, 110, Taiwan

Location

Investigational Site Number : 1580005

Taipei, 112, Taiwan

Location

Investigational Site Number : 1580007

Taoyuan, 33305, Taiwan

Location

Investigational Site Number : 7920002

Adana, 01130, Turkey (Türkiye)

Location

Investigational Site Number : 7920004

Akdeniz, 07059, Turkey (Türkiye)

Location

Investigational Site Number : 7920012

Bornova, 35100, Turkey (Türkiye)

Location

Investigational Site Number : 7920006

Gaziantep, 27100, Turkey (Türkiye)

Location

Investigational Site Number : 7920005

Istanbul, 34093, Turkey (Türkiye)

Location

Investigational Site Number : 7920003

Izmir, TR-35100, Turkey (Türkiye)

Location

Investigational Site Number : 7920008

Kayseri, 38039, Turkey (Türkiye)

Location

Investigational Site Number : 7920007

Samsun, 55200, Turkey (Türkiye)

Location

Investigational Site Number : 8040001

Kyiv, 04060, Ukraine

Location

Investigational Site Number : 8040003

Kyiv, 1135, Ukraine

Location

Investigational Site Number : 8040002

Lviv, 79044, Ukraine

Location

Investigational Site Number : 8040004

Mykolaiv, 54058, Ukraine

Location

Investigational Site Number : 8260001

London, London, City of, SE1 9RT, United Kingdom

Location

Investigational Site Number : 8260004

Glasgow, G4 0SF, United Kingdom

Location

Related Publications (1)

• 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.

  PMID: 40053895 DERIVED

Related Links

• LTE15174 Plain Language Results Summaries

MeSH Terms

Conditions

Hemophilia A

Interventions

fitusiran

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Clinical Sciences & Operations

  Sanofi

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 25, 2018
• First Posted: November 27, 2018
• Study Start: January 9, 2019
• Primary Completion (Estimated): November 5, 2026
• Study Completion (Estimated): November 5, 2026
• Last Updated: December 17, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will share

Locations

HU (1); MY (3); DK (1); IL (1); AU (3); ZA (1); CA (1); IE (2); TU (8); IN (6); US (10); DE (2); KR (4); UA (4); CN (13); TW (7); GB (2); IT (2); FR (2); JP (6)