Clinical Research Directory

Browse clinical research sites, groups, and studies.

Sites Groups Studies

Back to Studies

ACTIVE NOT RECRUITING

NCT04644575

PHASE3

Long-term Safety and Efficacy of Efanesoctocog Alfa (BIVV001) in Previously Treated Patients With Hemophilia A

Sponsor: Bioverativ, a Sanofi company

View on ClinicalTrials.gov

Summary

Primary Objective: \- To evaluate the long-term safety of BIVV001 in previously treated subjects with hemophilia A Secondary Objectives: * To evaluate the efficacy of BIVV001 as a prophylaxis treatment. * To evaluate the efficacy of BIVV001 in the treatment of bleeding episodes. * To evaluate BIVV001 consumption for prevention and treatment of bleeding episodes. * To evaluate the effect of BIVV001 prophylaxis on joint health outcomes. * To evaluate the effect of BIVV001 prophylaxis on Quality of Life (QoL) outcomes. * To evaluate the safety and tolerability of BIVV001 treatment. * To assess the PK of BIVV001 based on the one stage activated partial thromboplastin time (aPTT) and two-stage chromogenic FVIII activity assays (only applicable to Arm B). * To evaluate the efficacy of BIVV001 for perioperative management

Official title: A Phase 3 Open-label, Multicenter Study of the Long-term Safety and Efficacy of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein (rFVIIIFc-VWF-XTEN; BIVV001) in Previously Treated Patients With Severe Hemophilia A

Key Details

Gender

All

Age Range

Any - Any

Study Type

INTERVENTIONAL

Enrollment

261

Start Date

2021-02-23

Completion Date

2027-01-15

Last Updated

2025-08-01

Healthy Volunteers

Conditions

Hemophilia A

Interventions

DRUG

efanesoctocog alfa (BIVV001)

Pharmaceutical form:Solution for Injection Route of administration: Intravenous

Inclusion criteria : * For participants rolling over into Arm A * Participants who have completed the studies EFC16923, EFC16925, Arm B or Arm C of the current study, or any other potential BIVV001 study. * Male or Female * For participants new to BIVV001 (Arm B and C) * Participants who have severe hemophilia A, defined as \<1 IU/dL (\<1%) endogenous FVIII activity as documented either by central laboratory testing at screening or in historical medical records from a clinical laboratory demonstrating \<1% FVIII coagulant activity (FVIII:C) or a documented genotype known to produce severe hemophilia A. * Previous treatment for hemophilia A (prophylaxis or on-demand) with any recombinant and/or plasma-derived FVIII, or cryoprecipitate for at least 150 EDs or 50 EDs for participants aged \<6 years. * Platelet count ≥100 000 cells/μL at screening. * A participant known to be human immunodeficiency virus (HIV) antibody positive, either previously documented or identified from screening assessments, must have the following results prior to enrollment: CD4 lymphocyte count \>200 cells/mm³ and viral load of \<400 000 copies/mL * Male * Only for Arm B: Chinese participants * Only for Arm C: planned major surgery within 6 months after Day 1. Exclusion criteria: * For participants rolling over into Arm A * Positive inhibitor result, defined as ≥0.6 Bethesda units (BU)/mL. * Participation in another study. * For participants new to BIVV001 (Arm B and Arm C) * Any concurrent clinically significant liver disease that, in the opinion of the Investigator, would make the participant unsuitable for enrollment. This may include, but is not limited to cirrhosis, portal hypertension, and acute hepatitis. * Serious active bacterial, fungal, or viral infection (other than chronic hepatitis or HIV) present within 30 days of screening. * Other known coagulation disorder(s) in addition to hemophilia A. * History of hypersensitivity or anaphylaxis associated with any FVIII product. * History of a positive inhibitor (to FVIII) test defined as ≥0.6 BU/mL, or any value greater than or equal to the lower sensitivity cut-off for laboratories with cut-offs for inhibitor detection between 0.7 and 1.0 BU/mL, or clinical signs or symptoms of decreased response to FVIII administrations. Family history of inhibitors will not exclude the participant. * Positive inhibitor test (FVIII) result, defined as ≥0.6 BU/mL at screening. * Treatment with acetylsalicylic acid (ASA) or antiplatelet agents that are not nonsteroidal anti-inflammatory drugs (NSAIDs) within 2 weeks prior to screening. * Treatment with NSAIDs greater than the maximum dose specified in the regional prescribing information within 2 weeks prior to screening. * Systemic treatment within 12 weeks prior to Screening with chemotherapy and/or other immunosuppressive drugs (except for the treatment of hepatitis C virus \[HCV\] or HIV). * Emicizumab use within the 20 weeks prior to screening. * Major surgery within 8 weeks prior to screening. The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Locations (85)

Orthopaedic Institute for Children Site Number : 8400003

Los Angeles, California, United States

Children's Hospital Los Angeles Site Number : 8400009

Los Angeles, California, United States

University of California San Diego Site Number : 8400007

San Diego, California, United States

University of Florida Health Site Number : 8400008

Gainesville, Florida, United States

Children's Healthcare of Atlanta Site Number : 8400016

Atlanta, Georgia, United States

Rush University Medical Center Site Number : 8400010

Chicago, Illinois, United States

Children's Hospital Of Iowa Site Number : 8400011

Iowa City, Iowa, United States

University of Michigan Medical Center Site Number : 8400006

Ann Arbor, Michigan, United States

Michigan State University School Of Med Site Number : 8400002

East Lansing, Michigan, United States

Hemostasis and Thrombosis Center of Nevada Site Number : 8400001

Las Vegas, Nevada, United States

New York Presbyterian Hospital/Weill Cornell Medical Center Site Number : 8400017

New York, New York, United States

East Carolina University -2390 Hemby Ln Site Number : 8400015

Greenville, North Carolina, United States

Cincinnati Children's Hospital Medical Center Site Number : 8400012

Cincinnati, Ohio, United States

Children's Research Institute Site Number : 8400013

Columbus, Ohio, United States

Bloodworks Northwest Site Number : 8400005

Seattle, Washington, United States

Children's Hospital of Wisconsin Site Number : 8400014

Milwaukee, Wisconsin, United States

Investigational Site Number : 0320001

CABA, Buenos Aires F.D., Argentina

Investigational Site Number : 0320002

Godoy Cruz, Mendoza Province, Argentina

Investigational Site Number : 0320003

Buenos Aires, Argentina

Investigational Site Number : 0360004

Camperdown, New South Wales, Australia

Investigational Site Number : 0360001

Westmead, New South Wales, Australia

Investigational Site Number : 0360002

South Brisbane, Queensland, Australia

Investigational Site Number : 0360003

Murdoch, Western Australia, Australia

Investigational Site Number : 0560003

Woluwe-Saint-Lambert, Belgium

Hemocentro Campinas - UNICAMP Site Number : 0760001

Campinas, São Paulo, Brazil

Investigational Site Number : 1000171

Plovdiv, Bulgaria

Investigational Site Number : 1000172

Sofia, Bulgaria

Investigational Site Number : 1240005

Hamilton, Ontario, Canada

Investigational Site Number : 1240004

Hamilton, Ontario, Canada

Investigational Site Number : 1240002

Ottawa, Ontario, Canada

Investigational Site Number : 1240001

Toronto, Ontario, Canada

Investigational Site Number : 1560002

Beijing, China

Investigational Site Number : 1560006

Beijing, China

Investigational Site Number : 1560001

Guangzhou, China

Investigational Site Number : 1560003

Hangzhou, China

Investigational Site Number : 1560004

Hangzhou, China

Investigational Site Number : 1560005

Jinan, China

Investigational Site Number : 1560009

Kunming, China

Investigational Site Number : 1560010

Kunming, China

Investigational Site Number : 1560013

Lanzhou, China

Investigational Site Number : 1560007

Suzhou, China

Investigational Site Number : 2500005

Brest, France

Investigational Site Number : 2500004

Bron, France

Investigational Site Number : 2500001

Le Kremlin-Bicêtre, France

Investigational Site Number : 2500003

Lille, France

Investigational Site Number : 2500006

Marseille, France

Investigational Site Number : 2760304

Berlin, Germany

Investigational Site Number : 2760302

Bonn, Germany

Investigational Site Number : 2760001

Frankfurt am Main, Germany

Investigational Site Number : 2760002

München, Germany

Investigational Site Number : 3000001

Athens, Greece

Investigational Site Number : 3480002

Budapest, Hungary

Investigational Site Number : 3480004

Debrecen, Hungary

Investigational Site Number : 3480005

Pécs, Hungary

Investigational Site Number : 3720001

Dublin, Ireland

Investigational Site Number : 3800002

Naples, Campania, Italy

Investigational Site Number : 3800001

Milan, Italy

Investigational Site Number : 3800003

Vicenza, Italy

Investigational Site Number : 3920425

Nagoya, Aichi-ken, Japan

Investigational Site Number : 3920423

Kitakyushu-shi, Fukuoka, Japan

Investigational Site Number : 3920426

Kawasaki-shi, Kanagawa, Japan

Investigational Site Number : 3920422

Kashihara-Shi, Niigata, Japan

Investigational Site Number : 3920421

Shinjuku-ku, Tokyo, Japan

Investigational Site Number : 3920424

Suginami-ku, Tokyo, Japan

Investigational Site Number : 5280002

Amsterdam, Netherlands

Investigational Site Number : 5280001

Utrecht, Netherlands

Investigational Site Number : 4100603

Daegu, Daegu, South Korea

Investigational Site Number : 4100601

Seoul, Seoul-teukbyeolsi, South Korea

Investigational Site Number : 4100600

Seoul, Seoul-teukbyeolsi, South Korea

Investigational Site Number : 7240002

Esplugues de Llobregat, Catalunya [Cataluña], Spain

Investigational Site Number : 7240001

Madrid, Madrid, Comunidad de, Spain

Investigational Site Number : 7520001

Malmo, Sweden

Investigational Site Number : 7560001

Zurich, Switzerland

Investigational Site Number : 1580005

Changhua County, Taiwan

Investigational Site Number : 1580001

Taichung, Taiwan

Investigational Site Number : 1580003

Taichung, Taiwan

Investigational Site Number : 1580002

Taipei, Taiwan

Investigational Site Number : 1580004

Taipei, Taiwan

Investigational Site Number : 7920004

Antalya, Turkey (Türkiye)

Investigational Site Number : 7920001

Istanbul, Turkey (Türkiye)

Investigational Site Number : 7920003

Izmir, Turkey (Türkiye)

Investigational Site Number : 8260005

London, London, City of, United Kingdom

Investigational Site Number : 8260001

London, London, City of, United Kingdom

Investigational Site Number : 8260003

Birmingham, United Kingdom

Investigational Site Number : 8260004

Hampshire, United Kingdom