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

NCT01425723 | Completed Phase 3 Results

• 2 other identifiers: 9HB01EXT2011-003075-11
• Study Type: interventional
• Target: 120
• Locations: 18 countries
• Sites: 49

Brief Summary

The primary objective of the study is to evaluate the long-term safety of rFIXFc in participants with hemophilia B. The secondary objective of this study is to evaluate the efficacy of rFIXFc in the prevention and treatment of bleeding episodes.

Conditions

Severe Hemophilia B

Keywords

B-LONG; B-YOND; B-LONG Extension; rFIXFc; Severe Hemophilia B

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), confirmed on 2 separate samples drawn 2 to 4 weeks apart, was considered positive. Both tests were to be performed by the central laboratory using the Nijmegen-modified Bethesda Assay. Data was summarized by treatment regimen for participants from Study 998HB102 and by age cohort (\<6 years and 6 to \<12 years old) and treatment regimen for participants from Study 9HB02PED 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 rFIXFc Consumption (International Units Per Kilogram [IU/kg])

  Approximately 5 years

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

  Approximately 5 years

Study Arms (2)

On-Demand

EXPERIMENTAL

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

Biological: rFIXFc

Prophylaxis

EXPERIMENTAL

Weekly prophylaxis, individualized prophylaxis or personalized prophylaxis available.

Biological: rFIXFc

Interventions

rFIXFc BIOLOGICAL

Administered as specified in the treatment arm.

Also known as: coagulation factor IX (recombinant) Fc fusion protein, Alprolix, BIIB029

On-Demand; Prophylaxis

Eligibility Criteria

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

You may qualify if:

• Subjects who have completed studies 998HB102 (NCT01027364) or 9HB02PED (NCT01440946) or other studies with rFIXFc
• Ability to understand the purposes \& risks of the study and provide signed and dated informed consent.

You may not qualify if:

• High-titer inhibitor (\>/=5.00 BU/mL)

Sponsors & Collaborators

• Bioverativ Therapeutics Inc.: lead

Study Sites (49)

Research Site

Phoenix, Arizona, 85016, United States

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Sacramento, California, 95817, United States

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Aurora, Colorado, 80045, United States

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Atlanta, Georgia, 30322, United States

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Honolulu, Hawaii, 96826, United States

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Indianapolis, Indiana, 46260, United States

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New Orleans, Louisiana, 70112, United States

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East Lansing, Michigan, 48823, United States

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Pittsburgh, Pennsylvania, 15213, United States

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Seattle, Washington, 98104, United States

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Adelaide, South Australia, 5000, Australia

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Parkville, Victoria, 3052, Australia

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Murdoch, Western Australia, 6150, Australia

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Perth, Western Australia, 6008, Australia

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Brussels, 1200, Belgium

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Leuven, 3000, Belgium

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Campinas, São Paulo, 13083-878, Brazil

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Toronto, Ontario, M5B 1W8, Canada

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Montreal, Quebec, H3T 1C5, Canada

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Beijing, Beijingshì, 100005, China

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Guangzhou, Guangdongsheng, 510515, China

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Shanghai, Shànghaishì, 200025, China

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Tianjing, Tianjinshì, 300020, China

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Marseille, Bouches-Du-Rhône, 13385, France

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Bonn, North Rhine-Westphalia, 53127, Germany

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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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Vellore, Tamil Nadu, 632004, India

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Dublin, D12 N512, Ireland

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Florence, 50134, Italy

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Milan, 20122, Italy

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Nagoya, Aichi-ken, 466-8550, Japan

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Kitakyushu, Fukuoka, 807-8555, 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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Utrecht, 3584 CX, Netherlands

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Lodz, 93-510, 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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Malmo, 20502, Sweden

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Stockholm, 17176, Sweden

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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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Basingstoke, Hampshire, RG24 9NA, United Kingdom

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Related Publications (3)

• Astermark J, Hermans C, Ezzalfani M, Sidhom A, Barbier S, Kragh N, Falk A, Eriksson D. Recombinant factor IX Fc prophylaxis reduces pain and increases levels of physical activity, with sustained, long-term improvements in patients with hemophilia B: post hoc analysis of phase III trials using patient-reported outcomes. Ther Adv Hematol. 2023 May 29;14:20406207231170701. doi: 10.1177/20406207231170701. eCollection 2023.

  PMID: 37283819 DERIVED

• Shapiro AD, Kulkarni R, Ragni MV, Chambost H, Mahlangu J, Oldenburg J, Nolan B, Ozelo MC, Foster MC, Willemze A, Barnowski C, Jain N, Winding B, Dumont J, Lethagen S, Barnes C, Pasi KJ. Post hoc longitudinal assessment of the efficacy and safety of recombinant factor IX Fc fusion protein in hemophilia B. Blood Adv. 2023 Jul 11;7(13):3049-3057. doi: 10.1182/bloodadvances.2022009230.

  PMID: 36848635 DERIVED

• Shapiro AD, Pasi KJ, Ozelo MC, Kulkarni R, Barnowski C, Winding B, Szamosi J, Lethagen S. Extending recombinant factor IX Fc fusion protein dosing interval to 14 or more days in patients with hemophilia B. Res Pract Thromb Haemost. 2018 Nov 29;3(1):109-113. doi: 10.1002/rth2.12163. eCollection 2019 Jan.

  PMID: 30656283 DERIVED

MeSH Terms

Conditions

Hemophilia B

Interventions

factor IX 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; Genetic Diseases, X-Linked

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: August 19, 2011
• First Posted: August 30, 2011
• Study Start: December 8, 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

IT (2); CN (4); BE (2); IN (3); SE (2); NL (1); US (10); IE (1); HK (2); ZA (2); BR (1); AU (4); JP (6); GB (4); CA (2); DE (1); FR (1); PL (1)