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

NCT ID: NCT01454739

Last Updated: 2020-12-19

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

240 participants

Study Classification

INTERVENTIONAL

Study Start Date

2011-12-31

Study Completion Date

2017-10-31

Brief Summary

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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.

Detailed Description

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Participant will follow either a prophylaxis or on-demand regimen. The starting dose in this study will be determined by the clinical profile of the participant in the preceding studies A-LONG - 997HA301 (NCT01181128), pediatric study 8HA02PED (NCT01458106), 997HA307 (NCT02083965) and 997HA309 (NCT02502149).

Conditions

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Hemophilia A

Keywords

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rFVIIIFc A-LONG Extension

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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On-Demand

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.

Group Type EXPERIMENTAL

rFVIIIFc

Intervention Type DRUG

Administered as specified in the treatment arm.

Prophylaxis

Tailored prophylaxis, Weekly prophylaxis or Personalized prophylaxis available.

Group Type EXPERIMENTAL

rFVIIIFc

Intervention Type DRUG

Administered as specified in the treatment arm.

Interventions

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rFVIIIFc

Administered as specified in the treatment arm.

Intervention Type DRUG

Other Intervention Names

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Eloctate recombinant coagulation factor VIII Fc fusion protein BIIB031 antihemophilic factor (recombinant) Fc fusion protein efmoroctocog alfa

Eligibility Criteria

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Inclusion Criteria

* 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).

Exclusion Criteria

* Confirmed positive high-titer inhibitor (≥5.00 BU/mL).
Minimum Eligible Age

0 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Bioverativ Therapeutics Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Medical Director

Role: STUDY_DIRECTOR

Bioverativ Therapeutics Inc.

Locations

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Research Site

Los Angeles, California, United States

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Los Angeles, California, United States

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Orange, California, United States

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Sacramento, California, United States

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San Diego, California, United States

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

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Indianapolis, Indiana, United States

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Iowa City, Iowa, United States

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New Orleans, Louisiana, United States

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Boston, Massachusetts, United States

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East Lansing, Michigan, United States

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St Louis, Missouri, United States

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Las Vegas, Nevada, United States

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Chapel Hill, North Carolina, United States

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Cincinnati, Ohio, United States

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Portland, Oregon, United States

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Philadelphia, Pennsylvania, United States

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Philadelphia, Pennsylvania, United States

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Pittsburgh, Pennsylvania, United States

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Houston, Texas, United States

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Salt Lake City, Utah, United States

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Seattle, Washington, United States

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Camperdown, New South Wales, Australia

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South Brisbane, Queensland, Australia

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Adelaide, South Australia, Australia

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Melbourne, Victoria, Australia

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Melbourne, Victoria, Australia

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Murdoch, Western Australia, Australia

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Subiaco, Western Australia, Australia

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Vienna, , Austria

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Brussels, Brussels Capital, Belgium

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Campinas, São Paulo, Brazil

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Vancouver, British Columbia, Canada

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Toronto, Ontario, Canada

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Bron, Rhone, France

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Bonn, North Rhine-Westphalia, Germany

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Berlin, , 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, India

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Pune, Maharashtra, India

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New Delhi, National Capital Territory of Delhi, India

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Ludhiana, Punjab, India

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Vellore, Tamil Nadu, India

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Dublin, , Ireland

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Ramat Gan, , Israel

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Florence, , Italy

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Milan, , Italy

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Vicenza, , Italy

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Nagoya, Aichi-ken, Japan

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Kitakyushu, Fukuoka, Japan

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Kawasaki, Kanagawa, Japan

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Kashihara-shi, Nara, Japan

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Shinjuku-ku, Tokyo-To, Japan

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Tokyo, Tokyo-To, Japan

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Groningen, , Netherlands

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Auckland, , New Zealand

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Christchurch, , New Zealand

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Hamilton, , New Zealand

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Palmerston North, , New Zealand

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Wellington, , New Zealand

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Lublin, , Poland

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Johannesburg, Gauteng, South Africa

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Cape Town, Western Cape, South Africa

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Barcelona, , Spain

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Madrid, , Spain

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Gothenburg, , Sweden

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Zurich, , Switzerland

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Cambridge, Cambridgeshire, United Kingdom

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London, Greater London, United Kingdom

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London, Greater London, United Kingdom

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London, Greater London, United Kingdom

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Basingstoke, Hampshire, United Kingdom

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Hamstead, London, United Kingdom

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Glasgow, Strathclyde, United Kingdom

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Glasgow, Strathclyde, United Kingdom

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London, , United Kingdom

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Countries

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United States Australia Austria Belgium Brazil Canada France Germany Hong Kong India Ireland Israel Italy Japan Netherlands New Zealand Poland South Africa Spain Sweden Switzerland United Kingdom

References

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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.

Reference Type DERIVED
PMID: 39091324 (View on PubMed)

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.

Reference Type DERIVED
PMID: 32227570 (View on PubMed)

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.

Reference Type DERIVED
PMID: 26218032 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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2011-003072-37

Identifier Type: -

Identifier Source: secondary_id

8HA01EXT

Identifier Type: -

Identifier Source: org_study_id