Skip to main content
OpenTrials
Completed

NCT Number: NCT00768287

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

Primary Objective:

To evaluate the safety (acute effects associated with infusions, and inhibitor development), pharmacokinetics (PK), and efficacy with respect to breakthrough bleeding during prophylaxis and with respect to control of hemorrhaging in both the prophylaxis and on demand groups of IB1001 in subjects with hemophilia B.

Key Secondary Objectives:

To evaluate the ability of IB1001 to provide coverage against bleeding under surgical circumstances; To evaluate the long-term safety and efficacy of IB1001

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Hemophilia B Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders

Age range

5 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

Centre Regional de Traitement de l 'Hemophilie, Nantes, Loire-Atlantique, France

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Patient must be willing to give written Institutional Review Board (IRB)/Independent Ethics Committee (IEC)-approved informed consent, make the required study visits, and follow instructions while enrolled in the study
  • Severe (factor IX activity ≤2 U/dL) hemophilia B subjects on demand therapy with a minimum of 3 bleeding episodes over the preceding 6 months or 6 bleeding episodes over the preceding 12 months; subjects on prophylaxis with a bleeding pattern as above demonstrated prior to starting prophylaxis
  • Immunocompetent (CD4 count >400/mm3) and not receiving immune modulating or chemotherapeutic agents
  • Previously treated patients with a minimum of 150 exposure days to a factor IX preparation
  • Platelet count at least 150,000/mm3
  • Liver function: alanine transaminase [ALT] and aspartate transaminase [AST] ≤2 times the upper limit of the normal range
  • Total bilirubin ≤1.5 times the upper limit of the normal range
  • Renal function: serum creatinine ≤1.25 times the upper limit of the normal range
  • Willingness to participate in the trial for up to 12-15 months
  • European Union (EU), Israel, and Canada: Age of at least 12 years and body weight of ≥40 kilograms to participate in any PK Study or the Surgical Sub-study [the Surgical Sub-study does not apply to the UK]; age of at least 12 years for the prophylaxis and on demand components of the Treatment Phase and Continuation Study

United States (US): Age of at least 12 years and body weight of ≥40 kilograms to participate in any PK Study or the Surgical Sub-study; age of at least 5 years for the prophylaxis and on demand components of the Treatment Phase and Continuation Study

  • Hemoglobin ≥7 g/dL at the time of the blood draw

Exclusion criteria

  • History of factor IX inhibitor ≥0.6 Bethesda units (BU)
  • Existence of another coagulation disorder
  • Evidence of thrombotic disease, fibrinolysis, or disseminated intravascular coagulation (DIC)
  • Use of an investigational drug within 30 days prior to study entry
  • On medications that could impact hemostasis, such as aspirin
  • 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
  • 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

Treatment and study plan

IB1001

Biological

Study Part 1: Randomized, double-blind, cross-over study with IB1001 and nonacog alfa; Study Part 2: Non-randomized, open-label evaluation of prophylaxis and on demand IB1001; Surgical Sub-study: Open-label evaluation of IB1001 during major surgery

Other names: Recombinant factor IX (rFIX), IXINITY

nonacog alfa

Biological

Study Part 1: Randomized, double-blind, cross-over study with IB1001 and nonacog alfa

Other names: Recombinant factor IX (rFIX), BeneFIX

Primary outcomes

  1. Degree of Hemorrhage Control by Treatment Regimen

    Time frame: Prophylaxis Group Duration of Treatment: 17.9 ± 9.6 months; On Demand Group Duration of Treatment: 15.9 ± 11.5 months

    Subject rating of bleed control within 6 hours of the time bleeding has stopped:

    • Excellent: a dramatic response with abrupt pain relief and clear reduction in joint or hemorrhage site size;
    • Good: pain relief or reduction in hemorrhage site size that may have required an additional infusion for resolution;
    • Fair: probable or slight beneficial response usually requiring one or more additional infusions for resolution;
    • Poor: no improvement or condition worsens.

Secondary outcomes

  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 30 min ± 5 min, 1 hour ± 5 min, 3 hours ± 30 min, 6 hours ± 1 hour, 9 hours ± 1 hour, 12 hours ± 2 hours, 24 hours ± 3 hours, 36 hours ± 3 hours, 48 hours ± 3 hours, 60 hours ± 3 hours, and 72 hours ± 3 hours.

  2. Area Under the Curve (0-72 hr)

    Time frame: Pre-infusion to 72 hours following infusion

    Factor IX levels were assessed at the following time points: Pre-infusion, post-infusion at 30 min ± 5 min, 1 hour ± 5 min, 3 hours ± 30 min, 6 hours ± 1 hour, 9 hours ± 1 hour, 12 hours ± 2 hours, 24 hours ± 3 hours, 36 hours ± 3 hours, 48 hours ± 3 hours, 60 hours ± 3 hours, and 72 hours ± 3 hours.

  3. 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 30 min ± 5 min, 1 hour ± 5 min, 3 hours ± 30 min, 6 hours ± 1 hour, 9 hours ± 1 hour, 12 hours ± 2 hours, 24 hours ± 3 hours, 36 hours ± 3 hours, 48 hours ± 3 hours, 60 hours ± 3 hours, and 72 hours ± 3 hours.

  4. 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 30 min ± 5 min, 1 hour ± 5 min, 3 hours ± 30 min, 6 hours ± 1 hour, 9 hours ± 1 hour, 12 hours ± 2 hours, 24 hours ± 3 hours, 36 hours ± 3 hours, 48 hours ± 3 hours, 60 hours ± 3 hours, and 72 hours ± 3 hours.

  5. 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 30 min ± 5 min, 1 hour ± 5 min, 3 hours ± 30 min, 6 hours ± 1 hour, 9 hours ± 1 hour, 12 hours ± 2 hours, 24 hours ± 3 hours, 36 hours ± 3 hours, 48 hours ± 3 hours, 60 hours ± 3 hours, and 72 hours ± 3 hours.

  6. Mean Residence Time

    Time frame: Pre-infusion to 72 hours following infusion

    Factor IX levels were assessed at the following time points: Pre-infusion, post-infusion at 30 min ± 5 min, 1 hour ± 5 min, 3 hours ± 30 min, 6 hours ± 1 hour, 9 hours ± 1 hour, 12 hours ± 2 hours, 24 hours ± 3 hours, 36 hours ± 3 hours, 48 hours ± 3 hours, 60 hours ± 3 hours, and 72 hours ± 3 hours.

  7. 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 30 min ± 5 min, 1 hour ± 5 min, 3 hours ± 30 min, 6 hours ± 1 hour, 9 hours ± 1 hour, 12 hours ± 2 hours, 24 hours ± 3 hours, 36 hours ± 3 hours, 48 hours ± 3 hours, 60 hours ± 3 hours, and 72 hours ± 3 hours.

  8. 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 30 min ± 5 min, 1 hour ± 5 min, 3 hours ± 30 min, 6 hours ± 1 hour, 9 hours ± 1 hour, 12 hours ± 2 hours, 24 hours ± 3 hours, 36 hours ± 3 hours, 48 hours ± 3 hours, 60 hours ± 3 hours, and 72 hours ± 3 hours.

  9. Annualized Bleed Rate

    Time frame: Prophylaxis Group Duration of Treatment: 17.9 ± 9.6 months; On Demand Group Duration of Treatment: 15.9 ± 11.5 months

    Measure was assessed during the Treatment Study

Other outcomes

  1. Blood Loss During Surgery

    Time frame: During the surgical procedure

    Surgeon assessment of blood loss during the procedure using the following descriptors: less than expected, expected, or more than expected. Measure was assessed during Surgical Substudy.

  2. Hemostasis Following Surgery

    Time frame: 12 and 24 hours after surgery

    Surgeon assessment of hemostasis at 12 and 24 hours after surgery using the following descriptors: superior, adequate, or poorly controlled. Measure was assessed during Surgical Substudy.

  3. Number of Surgeries Requiring Blood Transfusions

    Time frame: During the surgical procedure

    Measure was assessed during Surgical Substudy.

Sponsors and collaborators

Lead sponsor

Medexus Pharma, Inc.

Industry

Registry information

Official study title

Phase I/II/III Pharmacokinetic and Outcome Study of Recombinant Factor IX Product, IB1001, in Subjects With Hemophilia B

Important dates

Study start
2009
Primary completion
2013
Study completion
2016
First posted
Oct 8, 2008
Registry last updated
Apr 6, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.