BAX 326 Pediatric Study
This study is ongoing, but not recruiting participants.
Sponsor:
Baxter Healthcare Corporation
Collaborator:
Baxter Innovations GmbH
Information provided by (Responsible Party):
Baxter Healthcare Corporation
ClinicalTrials.gov Identifier:
NCT01488994
First received: December 6, 2011
Last updated: December 27, 2012
Last verified: December 2012
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Purpose
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.
| Condition | Intervention | Phase |
|---|---|---|
|
Hemophilia B |
Biological: Recombinant Factor IX |
Phase 2 Phase 3 |
| Study Type: | Interventional |
| Study Design: | Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | BAX 326 (Recombinant Factor IX): A Phase 2/3 Prospective, Uncontrolled, Multicenter Study Evaluating Pharmacokinetics, Efficacy, Safety, and Immunogenicity in Previously Treated Pediatric Patients With Severe (FIX Level < 1%) or Moderately Severe (FIX Level <= 2%) Hemophilia B |
Resource links provided by NLM:
Genetics Home Reference related topics:
hemophilia
Drug Information available for:
Factor IX
U.S. FDA Resources
Further study details as provided by Baxter Healthcare Corporation:
Primary Outcome Measures:
- Adverse events possibly or probably related to BAX 326 [ Time Frame: 7 months (per subject) ] [ Designated as safety issue: Yes ]
Secondary Outcome Measures:
- Area under the plasma concentration versus time curve from 0 to 72 hours post-infusion [ Time Frame: 7 post-infusion time points over a period up to 72 hours ] [ Designated as safety issue: No ]
- Annualized bleeding rate [ Time Frame: 26 (+/- 1) weeks ] [ Designated as safety issue: No ]
- Hemostatic efficacy of the acute management of bleeding episodes [ Time Frame: 26 (+/-1) weeks ] [ Designated as safety issue: No ]Efficacy assessments: Determination of factor IX level and pharmacokinetic parameters at certain time points, including incremental recovery; rating scale for treatment of bleeding episodes (excellent / good / fair / none)
| Estimated Enrollment: | 24 |
| Study Start Date: | December 2011 |
| Estimated Study Completion Date: | September 2013 |
| Estimated Primary Completion Date: | May 2013 (Final data collection date for primary outcome measure) |
Intervention Details:
-
Biological: Recombinant Factor IX
Prophylactic treatment: twice weekly. Subjects will will undergo a pharmacokinetic evaluation with recombinant Factor IX before starting the prophylactic treatment.
Other Name: BAX 326
Eligibility| Ages Eligible for Study: | up to 12 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
Main Inclusion Criteria:
- Subject and/or legal representative has/have voluntarily provided signed informed consent
- Subject has severe (FIX level < 1%) or moderately severe (FIX level <= 2%) hemophilia B
- Subject is < 12 years old at the time of screening
- Subject has no evidence of a history of FIX inhibitors (based on the subject's medical records)
- Subject is immunocompetent as evidenced by a CD4 count >= 200 cells/mm3
Main Exclusion Criteria:
- Subject has a detectable FIX inhibitor at screening, with a titer >= 0.6 BU
- Subject has a history of allergic reaction, e.g. anaphylaxis, following exposure to FIX concentrate(s)
- Subject has evidence of an ongoing or recent thrombotic disease
- Subject has an inherited or acquired hemostatic defect other than hemophilia B
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01488994
Locations
| Bulgaria | |
| Specialized Hospital For Active Treatment of Oncohematological Diseases in Children | |
| Sofia, Bulgaria, 1527 | |
| India | |
| LNJP Maulana Azad Medical College & Associated Hospitals | |
| New Delhi, India, 110002 | |
| Sahayadri Speciality Hospital | |
| Pune, India, 411004 | |
| Jehangir Hospital and Research Center | |
| Pune, India, 411001 | |
| Poland | |
| Antoni Jurasz University Hospital | |
| Bydgoszcz, Poland, 85-094 | |
| University Pediatric Hospital | |
| Cracow, Poland, 30-663 | |
| Maria Konopnicka Teaching Hospital, Medical University of Lodz | |
| Lodz, Poland, 91-738 | |
| Stanislaw Popowski Provincial Specialist Pediatric Hospital | |
| Olsztyn, Poland, 10-561 | |
| Professor Tadeusz Sokolowski Independent Public Teaching Hospital of the Pomeranian Medical University in Szczecin | |
| Szczecin, Poland, 71-252 | |
| Romania | |
| Prof. Dr. C.T. Nicolau National Institute for Transfusional Hematology | |
| Bucharest, Romania, 11156 | |
| Louis Turcanu Emergency Children's Hospital | |
| Timisoara, Romania, 300011 | |
| Russian Federation | |
| Regional Clinical Hospital | |
| Ekaterinburg, Russian Federation, 620149 | |
| Federal State Institution Kirov, Hematology and Blood Transfusion Research Institute under the Federal Medical and Biological Agency | |
| Kirov, Russian Federation, 610027 | |
| Pediatric Regional Clinical Hospital, Hematology Department | |
| Krasnodar, Russian Federation, 350007 | |
| Republican Center for Hemophilia Treatment | |
| St. Petersburg, Russian Federation, 195213 | |
| Ukraine | |
| State Institution "Institute of Blood Pathology and Transfusion Medicine of the Academy of Medical Sciences of Ukraine" | |
| Lviv, Ukraine, 79044 | |
| United Kingdom | |
| Manchester Children´s Hospital | |
| Manchester, United Kingdom, M13 9WL | |
Sponsors and Collaborators
Baxter Healthcare Corporation
Baxter Innovations GmbH
Investigators
| Study Director: | Brigitt Abbuehl, MD | Baxter Innovations GmbH |
More Information
No publications provided
| Responsible Party: | Baxter Healthcare Corporation |
| ClinicalTrials.gov Identifier: | NCT01488994 History of Changes |
| Other Study ID Numbers: | 251101, 2011-002437-19 |
| Study First Received: | December 6, 2011 |
| Last Updated: | December 27, 2012 |
| Health Authority: | Bulgaria: Bulgarian Drug Agency India: Drugs Controller General of India Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products Romania: National Medicines Agency Russia: FSI Scientific Center of Expertise of Medical Application Ukraine: State Pharmacological Center - Ministry of Health United Kingdom: Medicines and Healthcare Products Regulatory Agency |
Additional relevant MeSH terms:
|
Hemophilia B Hemophilia A Blood Coagulation Disorders, Inherited Blood Coagulation Disorders Hematologic Diseases |
Coagulation Protein Disorders Hemorrhagic Disorders Genetic Diseases, Inborn Genetic Diseases, X-Linked |
ClinicalTrials.gov processed this record on May 22, 2013