Pivotal Study (Pharmacokinetics, Efficacy, Safety) of BAX 326 (rFIX) in Hemophilia B Patients

NCT ID: NCT01174446

Last Updated: 2021-05-20

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 86 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2010-07-29
• Study Completion Date: 2012-05-03

Brief Summary

The purpose of this pivotal Phase 1/3 study is to determine the pharmacokinetic (PK) parameters, the hemostatic efficacy, and the safety of BAX 326, a recombinant factor IX, in previously treated patients (PTPs) with severe and moderately severe hemophilia B.

Conditions

Hemophilia B

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

BAX 326

Recombinant factor IX (rFIX)

Group Type: EXPERIMENTAL

BAX 326

Intervention Type: BIOLOGICAL

• Study Part 1: Pharmacokinetic (PK) Crossover with BAX326 and BeneFIX
• Study Part 2: Open-label evaluation of prophylaxis and on-demand BAX326 only
• Study Part 3: Open-label repeat of PK evaluation (repeat Study Part 1) with BAX326 only and same study participants as Study Part 1

BeneFIX

Recombinant Factor IX (rFIX)

Group Type: ACTIVE_COMPARATOR

BeneFIX

Intervention Type: BIOLOGICAL

• Study Part 1: Pharmacokinetic (PK) Crossover with BAX326 and BeneFIX.
• BeneFIX only used in Part 1 of this study.
• Study Part 2 and 3 only utilized BAX326

Interventions

BAX 326

• Study Part 1: Pharmacokinetic (PK) Crossover with BAX326 and BeneFIX
• Study Part 2: Open-label evaluation of prophylaxis and on-demand BAX326 only
• Study Part 3: Open-label repeat of PK evaluation (repeat Study Part 1) with BAX326 only and same study participants as Study Part 1

Intervention Type: BIOLOGICAL

BeneFIX

• Study Part 1: Pharmacokinetic (PK) Crossover with BAX326 and BeneFIX.
• BeneFIX only used in Part 1 of this study.
• Study Part 2 and 3 only utilized BAX326

Intervention Type: BIOLOGICAL

Other Intervention Names

Recombinant factor IX (rFIX); RIXUBIS; Recombinant factor IX (rFIX)

Eligibility Criteria

Inclusion Criteria

• Participant is 12 to 65 years old at the time of screening
• Participant and/or legal representative has/have provided signed informed consent
• Participant has severe (factor IX (FIX) level < 1%) or moderately severe (FIX level 1-2%) hemophilia B (based on the one stage activated partial thromboplastin time (aPTT) assay), as tested at screening at the central laboratory
• Participant is previously treated with plasma-derived or recombinant FIX concentrate(s) for a minimum of 150 exposure days (EDs) (based on the participant's medical records); if a verifiable, documented history is unavailable, the participant can be enrolled if s/he has 100-150 EDs to any FIX product that are not fully documented and has participated in Study 050901 for at least 50 EDs to Immunine prior to enrollment (not valid for US and Japan).
• Participant has no evidence of a history of FIX inhibitors
• If the participant is to receive prophylactic treatment, the participant is willing to receive prophylactic treatment over a period of 6 months.
• If the participant is to receive on-demand treatment, the participant has ≥12 documented bleeding episodes requiring treatment within 12 months prior to enrollment and is willing to receive on-demand treatment for the duration of this study.

Exclusion Criteria

• The participant has a history of FIX inhibitors with a titer ≥0.6 Bethesda Units (BU) (as determined by the Nijmegen modification of the Bethesda assay or the assay employed in the respective local laboratory) at any time prior to screening
• The participant has a detectable FIX inhibitor at screening, with a titer ≥0.6 BU as determined by the Nijmegen modification of the Bethesda assay in the central laboratory
• The participant's weight is < 35 kg or > 120 kg
• The participant has a history of allergic reaction, eg, anaphylaxis, following exposure to FIX concentrate(s)
• The participant has a known hypersensitivity to hamster proteins or recombinant furin (rFurin)
• The participant has ongoing or recent evidence of a thrombotic disease, fibrinolysis or disseminated intravascular coagulation (DIC)

• Minimum Eligible Age: 12 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Baxalta now part of Shire

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Study Director

Role: STUDY_DIRECTOR

Takeda

Locations

Rosario, , Argentina

Brasília, , Brazil

São Paulo, , Brazil

Sofia, , Bulgaria

Santiago, , Chile

Bogotá, , Colombia

Cali, , Colombia

Prague, , Czechia

Hiroshima, , Japan

Nara, , Japan

Tochigi, , Japan

Tokyo, , Japan

Gdansk, , Poland

Krakow, , Poland

Lodz, , Poland

Warsaw, , Poland

Bucharest, , Romania

Timișoara, , Romania

Kirov, , Russia

Moscow, , Russia

Saint Petersburg, , Russia

Barcelona, , Spain

Malmo, , Sweden

Lviv, , Ukraine

London, , United Kingdom

Countries

Argentina; Brazil; Bulgaria; Chile; Colombia; Czechia; Japan; Poland; Romania; Russia; Spain; Sweden; Ukraine; United Kingdom

References

Windyga J, Lissitchkov T, Stasyshyn O, Mamonov V, Rusen L, Lamas JL, Oh MS, Chapman M, Fritsch S, Pavlova BG, Wong WY, Abbuehl BE. Pharmacokinetics, efficacy and safety of BAX326, a novel recombinant factor IX: a prospective, controlled, multicentre phase I/III study in previously treated patients with severe (FIX level <1%) or moderately severe (FIX level </=2%) haemophilia B. Haemophilia. 2014 Jan;20(1):15-24. doi: 10.1111/hae.12228. Epub 2013 Jul 9.

Reference Type: RESULT

PMID: 23834666 (View on PubMed)

Other Identifiers

2009-016720-31

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

250901

Identifier Type: -

Identifier Source: org_study_id