Safety, Efficacy and PK of BIVV001 in Pediatric Patients With Hemophilia A

NCT04759131 | Completed Phase 3 Results DMC FDA Drug

• 4 other identifiers: EFC162952020-000769-18U1111-1244-0558017464
• Study Type: interventional
• Target: 74
• Locations: 15 countries
• Sites: 40

Brief Summary

Primary Objective: \- To evaluate the safety of BIVV001 in previously treated pediatric participants 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 efficacy of BIVV001 for perioperative management.
• To evaluate the safety and tolerability of BIVV001 treatment.
• To assess the pharmacokinetics (PK) of BIVV001.

Conditions

Hemophilia A

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Neutralising Antibodies (Development of Inhibitors) Directed Against Factor VIII

  Inhibitor development was defined as an inhibitor result of greater than or equal to (\>=0.6) Bethesda units (BU/mL) that was confirmed by a second test result from a separate sample, drawn 2 to 4 weeks following the date when the original sample was drawn. Both tests must have been performed by the central laboratory using the Nijmegen modified Bethesda assay.

  Baseline up to Week 52

Secondary Outcomes (47)

• Annualized Bleeding Rate (ABR): For Treated Bleeds

  Baseline up to Week 52

• Sensitivity Analysis: Annualized Bleeding Rate: For Treated Bleeds

  Baseline up to Week 52

• Annualized Bleeding Rate for All Bleeding Episodes

  Baseline up to Week 52

• Sensitivity Analysis: Annualized Bleeding Rate for All Bleeding Episodes

  Baseline up to Week 52

• Annualized Bleeding Rate by Type of Bleed (Spontaneous, Traumatic and Unknown Type)

  Baseline up to Week 52

Study Arms (2)

BIVV001: Participants aged <6 Years

EXPERIMENTAL

Participants aged less than (\<) 6 years received BIVV001 at a dose of 50 international units per kilogram (IU/kg) intravenous (IV) injection once-weekly (QW) prophylaxis for 52 weeks.

Drug: efanesoctocog alfa (BIVV001)

BIVV001: Participants aged 6 to <12 Years

EXPERIMENTAL

Participants aged 6 to \<12 years received BIVV001 at a dose of 50 IU/kg IV injection QW prophylaxis for 52 weeks.

Drug: efanesoctocog alfa (BIVV001)

Interventions

efanesoctocog alfa (BIVV001) DRUG

Pharmaceutical form: solution for injection Route of administration: IV

BIVV001: Participants aged 6 to <12 Years; BIVV001: Participants aged <6 Years

Eligibility Criteria

• Age: Up to 11 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Participant must be younger than 12 years of age, at the time of signing the informed consent.
• Severe hemophilia A defined as \<1 international units per deciliter (IU/dL) (\<1 percent \[%\]) endogenous Factor VIII (FVIII) 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 exposure days (EDs) for participants aged 6 to \<12 years and above 50 EDs for participants aged \<6 years.
• Weight above or equal to 10 kg.

You may not qualify if:

• History of hypersensitivity or anaphylaxis associated with any FVIII product.
• History of a positive inhibitor (to FVIII) test defined as greater than or equal to (\>=) 0.6 Bethesda units (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 would not exclude the participant.
• Positive inhibitor test result, defined as \>=0.6 BU/mL at Screening.
• The above information was not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.

Sponsors & Collaborators

• Bioverativ, a Sanofi company: lead

Study Sites (40)

Children's Hospital Los Angeles-Site Number:8400006

Los Angeles, California, 90027, United States

Location

University of Florida-Site Number:8400009

Gainesville, Florida, 32610, United States

Location

Children's Healthcare of Atlanta-Site Number:8400019

Atlanta, Georgia, 30322, United States

Location

Rush University Medical Center-Site Number:8400001

Chicago, Illinois, 60612-3833, United States

Location

University of Iowa Hospitals and Clinics-Site Number:8400002

Iowa City, Iowa, 52242, United States

Location

NY Presbyterian - Weill Cornell Medical Center-Site Number:8400020

New York, New York, 10021, United States

Location

East Carolina University/Brody Medical Sciences Building-Site Number:8400015

Greenville, North Carolina, 27834, United States

Location

Cincinnati Children's Hospital Medical Center-Site Number:8400008

Cincinnati, Ohio, 45206, United States

Location

Childrens Hospital Of Columbus-Site Number:8400012

Columbus, Ohio, 43205, United States

Location

Children's Hospital Of Wisconsin-Site Number:8400005

Milwaukee, Wisconsin, 53226-0509, United States

Location

Investigational Site Number :0360001

Westmead, New South Wales, 2145, Australia

Location

Investigational Site Number :0360002

South Brisbane, Queensland, 4101, Australia

Location

Investigational Site Number :1240004

Hamilton, Ontario, L8N 3Z5, Canada

Location

Investigational Site Number :1240003

London, Ontario, N6A 4G5, Canada

Location

Investigational Site Number :1240002

Ottawa, Ontario, K1H 8L1, Canada

Location

Investigational Site Number :1240001

Toronto, Ontario, M5G 1X8, Canada

Location

Investigational Site Number :2500004

Bron, 69500, France

Location

Investigational Site Number :2500001

Le Kremlin-Bicêtre, 94275, France

Location

Investigational Site Number :2500003

Lille, 59037, France

Location

Investigational Site Number :2760001

Frankfurt am Main, 60590, Germany

Location

Investigational Site Number :2760002

München, 80337, Germany

Location

Investigational Site Number :3480005

Pécs, 7623, Hungary

Location

Investigational Site Number :3720001

Dublin, D12 N512, Ireland

Location

Investigational Site Number :3800001

Milan, 20121, Italy

Location

Investigational Site Number :3800002

Napoli, 80123, Italy

Location

Investigational Site Number :5280002

Amsterdam, 1105 AZ, Netherlands

Location

Investigational Site Number :5280001

Utrecht, 3584 CX, Netherlands

Location

Investigational Site Number :7240002

Esplugues de Llobregat, Catalunya [Cataluña], 08950, Spain

Location

Investigational Site Number :7240001

Madrid, Madrid, Comunidad de, 28046, Spain

Location

Investigational Site Number :7520001

Malmo, 20502, Sweden

Location

Investigational Site Number :7560001

Zurich, 8032, Switzerland

Location

Investigational Site Number :1580001

Taichung, 402, Taiwan

Location

Investigational Site Number :1580003

Taichung, 407, Taiwan

Location

Investigational Site Number :1580002

Taipei, 10002, Taiwan

Location

Investigational Site Number :1580004

Taipei, 11031, Taiwan

Location

Investigational Site Number :7920004

Antalya, 07059, Turkey (Türkiye)

Location

Investigational Site Number :7920001

Istanbul, 34390, Turkey (Türkiye)

Location

Investigational Site Number :7920003

Izmir, TR-35100, Turkey (Türkiye)

Location

Investigational Site Number :8260003

Birmingham, B4 6NH, United Kingdom

Location

Investigational Site Number :8260001

London, WC1N3JH, United Kingdom

Location

Related Publications (3)

• Malec L, Mathias M, Dunn AL, Nolan B, Wong N, Bystricka L, Khan U, Neill G, Santagostino E, Gunawardena S, Tarango C, Peyvandi F. Post hoc analysis of bleeding episodes and clinically relevant pharmacokinetic parameters among children <12 years old with severe hemophilia A receiving once-weekly efanesoctocog alfa prophylaxis in the XTEND-Kids phase 3 multinational trial. J Thromb Haemost. 2025 Nov;23(11):3481-3491. doi: 10.1016/j.jtha.2025.07.011. Epub 2025 Jul 21.

  PMID: 40701256 DERIVED

• Klamroth R, von Drygalski A, Hermans C, Park YS, Chan AKC, Kupesiz A, Alvarez-Roman MT, Malec L, Santagostino E, Neill G, Bystricka L, Dumont J, Abad-Franch L, Fetita LS, Khoo L. Perioperative Management With Efanesoctocog Alfa in Patients With Haemophilia A in the Phase 3 XTEND-1 and XTEND-Kids Studies. Haemophilia. 2025 May;31(3):391-400. doi: 10.1111/hae.70017. Epub 2025 Mar 18.

  PMID: 40099428 DERIVED

• Malec L, Peyvandi F, Chan AKC, Konigs C, Zulfikar B, Yuan H, Simpson M, Alvarez Roman MT, Carcao M, Staber JM, Dunn AL, Chou SC, d'Oiron R, Albisetti M, Demissie M, Santagostino E, Yarramaneni A, Wong N, Abad-Franch L, Gunawardena S, Fijnvandraat K; XTEND-Kids Trial Group. Efanesoctocog Alfa Prophylaxis for Children with Severe Hemophilia A. N Engl J Med. 2024 Jul 18;391(3):235-246. doi: 10.1056/NEJMoa2312611.

  PMID: 39018533 DERIVED

Related Links

• EFC16295 Plain Language Results Summary

MeSH Terms

Conditions

Hemophilia A

Interventions

BIVV001

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

Results Point of Contact

• Title: Trial Transparency Team
• Organization: Bioverativ Therapeutics Inc. (a Sanofi Company)

Contact-US@Sanofi.com

800-633-1610

Study Officials

• Clinical Sciences & Operations

  Sanofi

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 13, 2021
• First Posted: February 18, 2021
• Study Start: February 19, 2021
• Primary Completion: January 18, 2023
• Study Completion: January 18, 2023
• Last Updated: September 11, 2025
• Results First Posted: February 13, 2024

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will share

Locations

US (10); SE (1); TW (4); IE (1); ES (2); TU (3); FR (3); DE (2); IT (2); AU (2); CA (4); HU (1); NL (2); GB (2); CH (1)