Study of Recombinant Coagulation Factor IX Fc Fusion Protein, BIIB029, in Previously Treated Pediatric Participants With Hemophilia B

NCT ID: NCT01440946

Last Updated: 2020-12-19

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 30 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2012-06-30
• Study Completion Date: 2014-11-30

Brief Summary

The primary objective of the study is to evaluate the safety of Recombinant Human Coagulation Factor IX Fc Fusion Protein (rFIXFc) in previously treated pediatric subjects with hemophilia B. Secondary objectives of this study in this study population are as follows: to evaluate the efficacy of rFIXFc for prevention and treatment of bleeding episodes; to evaluate and assess the pharmacokinetics (PK) of rFIXFc; to evaluate rFIXFc consumption for prevention and treatment of bleeding episodes

Detailed Description

At the Baseline visit (28 ± 7 days prior to Day 1), participants receive a single IV injection of prestudy FIX over 10 (±5) minutes in the clinic under medical supervision at a dose of 50 IU/kg. A washout period with no FIX treatment is required prior to administration of prestudy FIX and prior to rFIXFc. A PK assessment is done with prestudy FIX and also done with rFIXFc on Day 1. After completing the PK assessments, participants begin weekly prophylactic treatment with rFIXFc for approximately 50 weeks, to obtain 50 EDs. One ED is defined as a 24-hour period in which a participant received 1 or more doses of rFIXFc, with the time of the first injection of rFIXFc defined as the start of the ED.

Conditions

Hemophilia B

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

rFIXFc Prophylaxis

At Baseline and at Day 1, participants receive a single intravenous (IV) injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last PK sampling, the first prophylactic dose of approximately 50 to 60 IU/kg will be administered in clinic as an IV injection.

Dose could be increased or decreased in increments of 10 IU/kg; increases to a maximum of 100 IU/kg and frequency of administration to a maximum of twice weekly, were allowed as indicated.

Group Type: EXPERIMENTAL

rFIXFc

Intervention Type: DRUG

Vials of rFIXFc were combined as needed, based on the actual labeled potency to achieve the participant's calculated dose. Partial vial use was allowed, in order to achieve the calculated dose.

FIX

Intervention Type: DRUG

Vials of prestudy FIX (provided by the participants) were combined as needed, based on the nominal labeled potency (e.g., 250 IU, 500 IU, and 1000 IU), to achieve the participant's calculated dose.

Interventions

rFIXFc

Vials of rFIXFc were combined as needed, based on the actual labeled potency to achieve the participant's calculated dose. Partial vial use was allowed, in order to achieve the calculated dose.

Intervention Type: DRUG

FIX

Vials of prestudy FIX (provided by the participants) were combined as needed, based on the nominal labeled potency (e.g., 250 IU, 500 IU, and 1000 IU), to achieve the participant's calculated dose.

Intervention Type: DRUG

Other Intervention Names

BIIB029; recombinant coagulation factor IX Fc fusion protein; Alprolix®; Factor IX

Eligibility Criteria

Inclusion Criteria

• Severe hemophilia B defined as ≤ 2 IU/dl (≤ 2%) endogenous FIX
• Male < 12 years and weight ≥ 13 kg
• History of at least 50 documented prior exposure days to FIX
• No history of, or currently detectable, inhibitor

Exclusion Criteria

• Other coagulation disorders in addition to Hemophilia B
• History of anaphylaxis associated with any FIX or IV immunoglobulin administration

• Maximum Eligible Age: 11 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Swedish Orphan Biovitrum

INDUSTRY

Sponsor Role: collaborator

Bioverativ Therapeutics Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Medical Director

Role: STUDY_DIRECTOR

Bioverativ Therapeutics Inc.

Locations

Research Site

Phoenix, Arizona, United States

Research Site

Sacramento, California, United States

Research Site

Atlanta, Georgia, United States

Research Site

Honolulu, Hawaii, United States

Research Site

Indianapolis, Indiana, United States

Research Site

East Lansing, Michigan, United States

Research Site

Pittsburgh, Pennsylvania, United States

Research Site

Parkville, Victoria, Australia

Research Site

Subiaco, Western Australia, Australia

Research Site

Dublin, , Ireland

Research Site

Utrecht, , Netherlands

Research Site

Johannesburg, , South Africa

Research Site

Basingstoke, , United Kingdom

Research Site

Cambridge, , United Kingdom

Research Site

London, , United Kingdom

Countries

Canada; Hong Kong; United States; Australia; Ireland; Netherlands; South Africa; United Kingdom

References

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.

Reference Type: DERIVED

PMID: 36848635 (View on PubMed)

Fischer K, Kulkarni R, Nolan B, Mahlangu J, Rangarajan S, Gambino G, Diao L, Ramirez-Santiago A, Pierce GF, Allen G. Recombinant factor IX Fc fusion protein in children with haemophilia B (Kids B-LONG): results from a multicentre, non-randomised phase 3 study. Lancet Haematol. 2017 Feb;4(2):e75-e82. doi: 10.1016/S2352-3026(16)30193-4.

Reference Type: DERIVED

PMID: 28159192 (View on PubMed)

Other Identifiers

2011-003076-36

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

9HB02PED

Identifier Type: -

Identifier Source: org_study_id