BAX 326 Pediatric Study

NCT01488994 | Completed Phase 2 Results DMC

• 2 other identifiers: 2511012011-002437-19
• Study Type: interventional
• Target: 23
• Locations: 6 countries
• Sites: 11

Brief Summary

The purpose of this study is to assess BAX 326 pharmacokinetic parameters, to evaluate its hemostatic efficacy, safety, immunogenicity, and changes in health-related quality of life in pediatric patients.

Conditions

Hemophilia B

Outcome Measures

Primary Outcomes (1)

• Adverse Events (AEs) Possibly or Probably Related to BAX326

  Throughout study period (approximately 17 months)

Secondary Outcomes (29)

• Pharmacokinetics (PK): Total Area Under the Plasma Concentration Versus Time Curve From 0 to 72 Hours Post-infusion Per Dose (AUC 0-72h/Dose)

  Within 30 mins pre-infusion and 4 post-infusion timepoints

• Pharmacokinetics (PK): Total Area Under the Plasma Concentration Versus Time Curve From 0 to Infinity Post-infusion Per Dose (Total AUC/Dose)

  Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details.

• Pharmacokinetics (PK): Mean Residence Time (MRT)

  Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details.

• Pharmacokinetics (PK): Factor IX (FIX) Clearance (CL)

  Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details.

• Pharmacokinetics (PK): Incremental Recovery (IR)

  Within 30 mins pre-infusion and 30 mins post-infusion

Study Arms (2)

BAX326 < 6 years of age

EXPERIMENTAL

Biological: BAX326

BAX326 6 to <12 years of age

EXPERIMENTAL

Biological: BAX326

Interventions

BAX326 BIOLOGICAL

All participants underwent a pharmacokinetic evaluation with BAX326 (recombinant Factor IX) followed by twice weekly prophylactic treatment for 6 months or for at least 50 exposure days, whichever occurred last.

Also known as: BAX 326, RIXUBIS

BAX326 6 to <12 years of age; BAX326 < 6 years of age

Eligibility Criteria

• Age: Up to 12 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Participant and/or legal representative has/have voluntarily provided signed informed consent
• Participant has severe (FIX level \< 1%) or moderately severe (FIX level ≤ 2%) hemophilia B
• Participant is \< 12 years old at the time of screening
• Participant has no evidence of a history of FIX inhibitors (based on the participant's medical records)
• Participant is immunocompetent as evidenced by a CD4 count ≥ 200 cells/mm\^3

You may not qualify if:

• Participant has a detectable FIX inhibitor at screening, with a titer ≥ 0.6 Bethesda Unit (BU)
• Participant has a history of allergic reaction, e.g. anaphylaxis, following exposure to FIX concentrate(s)
• Participant has evidence of an ongoing or recent thrombotic disease
• Participant has an inherited or acquired hemostatic defect other than hemophilia B

Sponsors & Collaborators

• Baxalta now part of Shire: lead

Study Sites (11)

LNJP Maulana Azad Medical College & Associated Hospitals

New Delhi, 110002, India

Location

University Pediatric Hospital

Krakow, 30-663, Poland

Location

Stanislaw Popowski Provincial Specialist Pediatric Hospital

Olsztyn, 10-561, Poland

Location

Professor Tadeusz Sokolowski Independent Public Teaching Hospital of the Pomeranian Medical University in Szczecin

Szczecin, 71-252, Poland

Location

S.C. Sanador SRL

Bucharest, 11156, Romania

Location

Louis Turcanu Emergency Children's Hospital

Timișoara, 300011, Romania

Location

Pediatric Regional Clinical Hospital, Hematology Department

Krasnodar, 350007, Russia

Location

Republican Center for Hemophilia Treatment

Saint Petersburg, 195213, Russia

Location

Regional Clinical Hospital

Yekaterinburg, 620149, Russia

Location

State Institution "Institute of Blood Pathology and Transfusion Medicine of the Academy of Medical Sciences of Ukraine"

Lviv, 79044, Ukraine

Location

Manchester Children´s Hospital

Manchester, M13 9WL, United Kingdom

Location

Related Publications (1)

• Urasinski T, Stasyshyn O, Andreeva T, Rusen L, Perina FG, Oh MS, Chapman M, Pavlova BG, Valenta-Singer B, Abbuehl BE. Recombinant factor IX (BAX326) in previously treated paediatric patients with haemophilia B: a prospective clinical trial. Haemophilia. 2015 Mar;21(2):196-203. doi: 10.1111/hae.12548. Epub 2014 Dec 11.

  PMID: 25495591 RESULT

MeSH Terms

Conditions

Hemophilia B

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: Study Director
• Organization: Shire

ClinicalTransparency@shire.com

+1 866 842 5335

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 6, 2011
• First Posted: December 9, 2011
• Study Start: December 20, 2011
• Primary Completion: May 14, 2013
• Study Completion: May 14, 2013
• Last Updated: May 20, 2021
• Results First Posted: September 1, 2016

Record last verified: 2021-04

Locations

RO (2); RU (3); PL (3); UA (1); IN (1); GB (1)