Long-Term Safety and Efficacy of rFVIIIFc in the Prevention and Treatment of Bleeding Episodes in Previously Treated Participants With Hemophilia A

NCT01454739 | Completed Phase 3 Results DMC

• 2 other identifiers: 8HA01EXT2011-003072-37
• Study Type: interventional
• Target: 240
• Locations: 21 countries
• Sites: 77

Brief Summary

The primary objective of the study is to evaluate the long-term safety of recombinant human Factor VIII Fc fusion protein (rFVIIIFc) in participants with hemophilia A. The secondary objective of the study is to evaluate the efficacy of rFVIIIFc in the prevention and treatment of bleeding episodes in participants with hemophilia A.

Conditions

Hemophilia A

Keywords

rFVIIIFc; A-LONG Extension

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Any Positive Inhibitor Development

  An inhibitor test result greater than or equal to (\>=) 0.6 Bethesda units per milliliter (BU/mL), identified and confirmed by re-testing of a second sample obtained within 2 to 4 weeks, was considered positive. Both tests were to be performed using the Nijmegen-modified Bethesda Assay by the central laboratory. Data was summarized by treatment regimen for participants from 997HA301/997HA307/997HA309 combined and by age cohort (\<6 years and 6 to \<12 years old) and treatment regimen for participants from 8HA02PED per planned analysis. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.

  Approximately 5 years

Secondary Outcomes (6)

• Annualized Bleeding Rate (ABR)

  Approximately 5 years

• Annualized Spontaneous Joint Bleeding Episodes

  Approximately 5 years

• Total Number of Exposure Days (EDs)

  Approximately 5 years

• Annualized rFVIIIFc Consumption (International Units Per Kilogram [IU/kg])

  Approximately 5 years

• Physicians' Global Assessment of Participant's Response to rFVIIIFc Regimen Using a 4-Point Scale

  Approximately 5 years

Study Arms (2)

On-Demand

EXPERIMENTAL

The individual dose of rFVIIIFc to treat bleeding episodes will be based on participant's clinical condition, type and severity of the bleeding event, and if indicated, Factor VIII (FVIII) levels.

Drug: rFVIIIFc

Prophylaxis

EXPERIMENTAL

Tailored prophylaxis, Weekly prophylaxis or Personalized prophylaxis available.

Drug: rFVIIIFc

Interventions

rFVIIIFc DRUG

Administered as specified in the treatment arm.

Also known as: Eloctate, recombinant coagulation factor VIII Fc fusion protein, BIIB031, antihemophilic factor (recombinant) Fc fusion protein, efmoroctocog alfa

On-Demand; Prophylaxis

Eligibility Criteria

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

You may qualify if:

• Subjects who have completed previous rFVIIIFc studies (NCT01181128, NCT02083965, NCT01458106 and NCT02502149)
• Ability to understand purposes and risks of the study and to provide signed and dated informed consent (or assent, as applicable).

You may not qualify if:

• Confirmed positive high-titer inhibitor (≥5.00 BU/mL).

Sponsors & Collaborators

• Bioverativ Therapeutics Inc.: lead

Study Sites (78)

Research Site

Los Angeles, California, 90007, United States

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Los Angeles, California, 90027, United States

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Orange, California, 92868, United States

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Sacramento, California, 95817, United States

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San Diego, California, 92123, United States

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Washington D.C., District of Columbia, 20010, United States

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Indianapolis, Indiana, 46260, United States

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Iowa City, Iowa, 52242, United States

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New Orleans, Louisiana, 70112, United States

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Boston, Massachusetts, 02115, United States

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East Lansing, Michigan, 48823, United States

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St Louis, Missouri, 63104, United States

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Las Vegas, Nevada, 89109, United States

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Chapel Hill, North Carolina, 27599, United States

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Cincinnati, Ohio, 45229, United States

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Portland, Oregon, 97239, United States

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Philadelphia, Pennsylvania, 19104, United States

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Philadelphia, Pennsylvania, 19107, United States

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Pittsburgh, Pennsylvania, 15213, United States

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Houston, Texas, 77030, United States

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Salt Lake City, Utah, 84132, United States

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Seattle, Washington, 98104, United States

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Camperdown, New South Wales, 2050, Australia

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South Brisbane, Queensland, 4101, Australia

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Adelaide, South Australia, 5000, Australia

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Melbourne, Victoria, 3052, Australia

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Melbourne, Victoria, 3181, Australia

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Murdoch, Western Australia, 6150, Australia

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Subiaco, Western Australia, 6008, Australia

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Vienna, 1090, Austria

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Brussels, Brussels Capital, 1200, Belgium

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Campinas, São Paulo, 13083-878, Brazil

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Vancouver, British Columbia, V6Z 1Y6, Canada

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Toronto, Ontario, M5G 1X8, Canada

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Bron, Rhone, 69677, France

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Bonn, North Rhine-Westphalia, 53127, Germany

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Berlin, 10249, Germany

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Children Cancer Centre

Hong Kong, New Territories, Hong Kong

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Sir Yue Kong Pao Center for Cancer

Hong Kong, New Territories, Hong Kong

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Hong Kong, Hong Kong

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Bangalore, Karnataka, 560034, India

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Pune, Maharashtra, 411004, India

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New Delhi, National Capital Territory of Delhi, 110002, India

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Ludhiana, Punjab, 141008, India

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Vellore, Tamil Nadu, 632004, India

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Dublin, D12 N512, Ireland

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Ramat Gan, 52621, Israel

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Florence, 50134, Italy

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Milan, 20122, Italy

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Vicenza, 36100, Italy

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Nagoya, Aichi-ken, 466-8560, Japan

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Kitakyushu, Fukuoka, 807-8556, Japan

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Kawasaki, Kanagawa, 216-8511, Japan

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Kashihara-shi, Nara, 634-8522, Japan

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Shinjuku-ku, Tokyo-To, 160-0023, Japan

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Tokyo, Tokyo-To, 167-8515, Japan

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Groningen, 9713 GZ, Netherlands

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Auckland, 1023, New Zealand

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Christchurch, 8011, New Zealand

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Hamilton, 3200, New Zealand

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Palmerston North, 4410, New Zealand

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Wellington, 6021, New Zealand

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Lublin, 20-093, Poland

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Johannesburg, Gauteng, 2193, South Africa

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Cape Town, Western Cape, 7925, South Africa

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Barcelona, 8035, Spain

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Madrid, 28046, Spain

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Gothenburg, 41345, Sweden

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Zurich, 8091, Switzerland

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Cambridge, Cambridgeshire, CB2 0QQ, United Kingdom

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London, Greater London, E1 1BB, United Kingdom

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London, Greater London, SE1 7EH, United Kingdom

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London, Greater London, WC1N 3JH, United Kingdom

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Basingstoke, Hampshire, RG24 9NA, United Kingdom

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Hamstead, London, NW3 2QG, United Kingdom

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Glasgow, Strathclyde, G3 8SJ, United Kingdom

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Glasgow, Strathclyde, G4 0SF, United Kingdom

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London, SE1 7EH, United Kingdom

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Related Publications (3)

• Raheja P, Kragh N, Bystricka L, Eriksson D, Aroui K, Mezghani M, Barbier S, Linari S. Long-term efmoroctocog alfa prophylaxis improves perceived pain, mental, and physical health in patients with hemophilia A: post hoc analysis of phase III trials using patient-reported outcomes. Ther Adv Hematol. 2024 Jul 30;15:20406207241257917. doi: 10.1177/20406207241257917. eCollection 2024.

  PMID: 39091324 DERIVED

• Nolan B, Mahlangu J, Pabinger I, Young G, Konkle BA, Barnes C, Nogami K, Santagostino E, Pasi KJ, Khoo L, Winding B, Yuan H, Fruebis J, Rudin D, Oldenburg J. Recombinant factor VIII Fc fusion protein for the treatment of severe haemophilia A: Final results from the ASPIRE extension study. Haemophilia. 2020 May;26(3):494-502. doi: 10.1111/hae.13953. Epub 2020 Mar 30.

  PMID: 32227570 DERIVED

• Nolan B, Mahlangu J, Perry D, Young G, Liesner R, Konkle B, Rangarajan S, Brown S, Hanabusa H, Pasi KJ, Pabinger I, Jackson S, Cristiano LM, Li X, Pierce GF, Allen G. Long-term safety and efficacy of recombinant factor VIII Fc fusion protein (rFVIIIFc) in subjects with haemophilia A. Haemophilia. 2016 Jan;22(1):72-80. doi: 10.1111/hae.12766. Epub 2015 Jul 27.

  PMID: 26218032 DERIVED

MeSH Terms

Conditions

Hemophilia A

Interventions

factor VIII-Fc fusion protein

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: Bioverativ Study Medical Director
• Organization: Bioverativ Therapeutics Inc.

clinicaltrials@bioverativ.com

781-6631801

Study Officials

• Medical Director

  Bioverativ Therapeutics Inc.

  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: September 29, 2011
• First Posted: October 19, 2011
• Study Start: December 1, 2011
• Primary Completion: October 1, 2017
• Study Completion: October 1, 2017
• Last Updated: December 19, 2020
• Results First Posted: November 23, 2018

Record last verified: 2018-11

Locations

ES (2); DE (2); GB (9); CA (2); HK (3); SE (1); CH (1); IT (3); AU (7); ZA (2); IN (5); JP (6); NL (1); BE (1); US (22); BR (1); IE (1); IL (1); NZ (5); FR (1); PL (1)