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

Information source: Baxalta US Inc.
ClinicalTrials.gov processed this data on August 20, 2015
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: Hemophilia B

Intervention: BAX 326 (Biological); BeneFIX (Biological)

Phase: Phase 3

Status: Completed

Sponsored by: Baxalta US Inc.

Official(s) and/or principal investigator(s):
Brigitt Abbuehl, MD, Study Director, Affiliation: Baxter Healthcare Corporation

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.

Clinical Details

Official title: Recombinant Factor IX (BAX 326): A Phase 1/3, Prospective, Controlled, Multicenter Study Evaluating Pharmacokinetics, Efficacy, Safety and Immunogenicity in Previously Treated Patients With Severe or Moderately Severe Hemophilia B

Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Treatment

Primary outcome: Study Part 1- Area Under the Plasma Concentration Versus Time Curve From 0 to 72 Hours Per Dose

Secondary outcome:

Study Parts 1 and 3: Area Under the Plasma Concentration/Time Curve From Time 0 to Infinity Per Dose (AUC0-∞/ Dose)

Study Parts 1 and 3: Mean Residence Time (MRT)

Study Parts 1 and 3: Clearance (CL)

Study Parts 1 and 3: Incremental Recovery at Cmax (IR at Cmax)

Incremental Recovery (IR) at 30 Minutes Over Time

Change in Incremental Recovery (IR) at 30 Minutes Over Time

Study Parts 1 and 3: Half Life (T 1/2)

Study Parts 1 and 3: Volume of Distribution at Steady State (Vss)

Study Part 2: Annualized Bleed Rate (ABR) During Treatment With BAX326

Bleeding Episodes Treated With 1, 2 or ≥3 Infusions of BAX326 by Bleeding Site and Cause

Hemostatic Efficacy at Resolution of All Bleeding Episodes (BEs) Treated With BAX326 by Bleeding Site and Cause

Total Weight-adjusted Dose Per Bleeding Episode (BEs) of All BEs Treated With BAX326 by Bleeding Site and Cause

Consumption of BAX326 Per Event Per Participant

Consumption of BAX326 Per Participant: Median Number of Infusions Per Month

Consumption of BAX326 Per Participant: Median Weight-adjusted Consumption Per Month

Number of Participants Who Developed Inhibitory Antibodies to Factor IX (FIX)

Occurrence of Total Binding Antibodies of Indeterminate Specificity (Within Assay Variability)

Occurrence of Treatment Related Total Binding Antibodies

Number of Participants Who Experienced Severe Allergic Reactions (e.g. Anaphylaxis)

Number of Participants Who Experienced Thrombotic Events

Number of Participants With Clinically Significant Changes in Laboratory Parameters: Clinical Chemistry

Number of Participants With Clinically Significant Changes in Laboratory Parameters: Hematology

Number of Participants With Clinically Significant Changes in Laboratory Parameters: Vital Signs

Number of Participants With Clinically Significant Changes in Laboratory Parameters: Thrombogenic Markers

Number of Adverse Events (AEs) After BAX326 Treatment

Number of Participants With Adverse Events (AEs) After BAX326 Treatment

EuroQoL (Quality of Life)-5 Dimensions (EQ-5D) Total Index Scores

EuroQoL (Quality of Life)-5 Dimensions Visual Analogue Scale (EQ-5D VAS) Scores

General Pain Assessment Through a Visual Analog Scale (VAS)

Short Form (36) Health Survey (SF-36): HRQoL 'Physical Component Score' (PCS)

SF-36: HRQoL 'Mental Health' (MH)

SF-36: HRQoL Physical Functioning' (PF)

SF-36: HRQoL Role-Physical (RP)

SF-36: HRQoL Role-Emotional

SF-36: HRQoL Bodily Pain

SF-36: HRQoL Mental Health

SF-36: HRQoL Vitality

SF-36: HRQoL Social Functioning

SF-36: HRQoL General Health

Pediatric Quality of Life Questionnaire (PedsQL) Physical Health Summary Score (Ages 12-16)

Pediatric Quality of Life Questionnaire (PedsQL) Psychosocial Health Summary Score (Ages 12-16)

Pediatric Quality of Life Questionnaire (PedsQL) Total Score (Ages 12-16)

Health-Related Quality of Life (HRQoL) Disease-specific: Haem-A-QoL

Health-Related Quality of Life (HRQoL) Disease-specific: Haemo-QoL - Participants On-Demand (Ages 12-16)

Health Resource Use - Number of Hospitalizations

Health Resource Use - Total Days of Hospital Stay

Health Resource Use - Emergency Room Visits

Health Resource Use - Unscheduled Doctor's Office Visits

Health Resource Use - Days Lost From Work or School

Eligibility

Minimum age: 12 Years. Maximum age: 65 Years. Gender(s): Both.

Criteria:

Main 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. Main 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)

Locations and Contacts

Rosario, Argentina

Brasilia, Brazil

Sao Paulo, Brazil

Sofia, Bulgaria

Santiago, Chile

Bogotá, Colombia

Cali, Colombia

Prague, Czech Republic

Hiroshima, Japan

Nara, Japan

Tochigi, Japan

Tokyo, Japan

Gdansk, Poland

Krakow, Poland

Lodz, Poland

Warsaw, Poland

Bucharest, Romania

Timisoara, Romania

Kirov, Russian Federation

Moscow, Russian Federation

St. Petersburg, Russian Federation

Barcelona, Spain

Malmö, Sweden

Lviv, Ukraine

London, United Kingdom

Additional Information

Starting date: July 2010
Last updated: June 26, 2015

Page last updated: August 20, 2015

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