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Trial record 2 of 7 for:    IB1001

Study of Recombinant Factor IX Product, IB1001, in Previously Treated Pediatric Subjects With Hemophilia B

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ClinicalTrials.gov Identifier: NCT01271868
Recruitment Status : Terminated
First Posted : January 7, 2011
Results First Posted : April 11, 2019
Last Update Posted : April 11, 2019
Sponsor:
Information provided by (Responsible Party):
Aptevo Therapeutics ( Aptevo BioTherapeutics )

Brief Summary:
The Study's Primary Objective is to evaluate the pharmacokinetics, safety (acute effects associated with infusions, and inhibitor development) and efficacy (breakthrough bleeding and control of hemorrhaging during prophylaxis) of IB1001 in previously treated pediatric subjects with hemophilia B.

Condition or disease Intervention/treatment Phase
Hemophilia B Biological: IB1001 Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 9 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Study of Recombinant Factor IX Product, IB1001, in Previously Treated Pediatric Subjects With Hemophilia B
Actual Study Start Date : November 2010
Actual Primary Completion Date : December 2016
Actual Study Completion Date : December 2016


Arm Intervention/treatment
Experimental: IB1001 Biological: IB1001
Other Names:
  • Recombinant factor IX
  • IXINITY




Primary Outcome Measures :
  1. Number of Infusions Required for Bleed Control [ Time Frame: Treatment Study: at least 50 ED (approximately 6 months); actual mean subject duration: 39.7 ± 12.4 months ]

Secondary Outcome Measures :
  1. Area Under the Curve (0-inf) [ Time Frame: Pre-infusion to 72 hours following infusion ]

    Factor IX levels were assessed at the following time points:

    Pre-infusion, post-infusion at 15-30 min, 4-6 hours, 24-26 hours, 68-72 hours


  2. Terminal Half-life [ Time Frame: Pre-infusion to 72 hours following infusion ]

    Factor IX levels were assessed at the following time points:

    Pre-infusion, post-infusion at 15-30 min, 4-6 hours, 24-26 hours, 68-72 hours


  3. Concentration (Max) [ Time Frame: Pre-infusion to 72 hours following infusion ]

    Factor IX levels were assessed at the following time points:

    Pre-infusion, post-infusion at 15-30 min, 4-6 hours, 24-26 hours, 68-72 hours


  4. Incremental Recovery [ Time Frame: Pre-infusion to 72 hours following infusion ]

    Factor IX levels were assessed at the following time points:

    Pre-infusion, post-infusion at 15-30 min, 4-6 hours, 24-26 hours, 68-72 hours


  5. Clearance [ Time Frame: Pre-infusion to 72 hours following infusion ]

    Factor IX levels were assessed at the following time points:

    Pre-infusion, post-infusion at 15-30 min, 4-6 hours, 24-26 hours, 68-72 hours


  6. Volume of Distribution (Steady State) [ Time Frame: Pre-infusion to 72 hours following infusion ]

    Factor IX levels were assessed at the following time points:

    Pre-infusion, post-infusion at 15-30 min, 4-6 hours, 24-26 hours, 68-72 hours


  7. Annualized Bleed Rate [ Time Frame: Treatment Study: at least 50 ED (approximately 6 months); actual mean subject duration: 41.7+/- 11.7 Months ]
    Measure was assessed during the Treatment Study



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Ages Eligible for Study:   up to 12 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Subject's parent or legal guardian must give written Institutional Review Board (IRB)/Independent Ethics Committee (IEC)-approved informed consent and be willing to make the required study visits and follow instructions while enrolled in the study. For subjects ≥7 years of age, assent will be obtained if required by the institution. For subjects < 7 years of age, legal assent is not reasonable to obtain.
  2. Severe (factor IX activity ≤2 IU/dL) hemophilia B subjects currently on-demand therapy with a minimum of 2 bleeding episodes requiring factor IX therapy over the preceding 6 months or 4 bleeding episodes over the preceding 12 months; subjects on prophylaxis with a bleeding pattern similar to that above demonstrated prior to starting prophylaxis
  3. Immunocompetent (CD4 count >400/mm3) and not receiving immune modulating or chemotherapeutic agents
  4. Previously treated patients with a minimum of 50 exposure days to a factor IX preparation
  5. Platelet count at least 150,000/mm3
  6. Liver function: alanine transaminase [ALT] and aspartate transaminase [AST] ≤2 times the upper limit of the normal range
  7. Total bilirubin ≤1.5 times the upper limit of the normal range
  8. Renal function: serum creatinine ≤1.25 times the upper limit of the normal range
  9. Willingness to participate in the trial for approximately 6 months (50 exposures)
  10. Age ≤12 years
  11. Hemoglobin ≥7 g/dL at the time of the blood draw

Exclusion Criteria:

  1. History of factor IX inhibitor ≥0.6 Bethesda units (BU)
  2. Existence of another coagulation disorder
  3. Evidence of thrombotic disease, fibrinolysis, or disseminated intravascular coagulation (DIC)
  4. Use of an investigational drug within 30 days prior to study entry
  5. On medications that could impact hemostasis, such as aspirin
  6. History of poor compliance, a serious medical or social condition, or any other circumstance that, in the opinion of the investigator, would interfere with participation or compliance with the study protocol
  7. History of adverse reaction to either plasma-derived factor IX or recombinant factor IX that interfered with the subject's ability to treat bleeding episodes with a factor IX product

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01271868


Locations
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United States, Illinois
RUSH University Medical Center
Chicago, Illinois, United States, 60612
United States, Indiana
Indiana Hemophilia & Thrombosis Center
Indianapolis, Indiana, United States, 46260
United States, Texas
Gulf States Hemophilia & Thrombophilia Center
Houston, Texas, United States, 77030
India
AMRI Hospital, Institute of Haematology & Transfusion Medicine
Kolkata, India, 700073
Christian Medical College and Hospital
Ludhiana, India, 141008
Jehangir Clinical Development Centre Pvt. Ltd. Jehangir Hospital Premises
Pune, India, 411001
Sahyadri Speciality Hospital
Pune, India, 411004
Bhailal Amin General Hospital
Vadodara, India, 390003
Christian Medical College
Vellore, India, 632004
United Kingdom
Great Ormond Street Hospital for Children
London, United Kingdom, WC1N 3JH
Sponsors and Collaborators
Aptevo BioTherapeutics

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Responsible Party: Aptevo BioTherapeutics
ClinicalTrials.gov Identifier: NCT01271868     History of Changes
Other Study ID Numbers: IB1001-02
First Posted: January 7, 2011    Key Record Dates
Results First Posted: April 11, 2019
Last Update Posted: April 11, 2019
Last Verified: March 2019

Keywords provided by Aptevo Therapeutics ( Aptevo BioTherapeutics ):
Recombinant Factor IX

Additional relevant MeSH terms:
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Hemophilia A
Hemophilia B
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked