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Completed

NCT Number: NCT01496274

A Safety and Efficacy Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Patients With Hemophilia B

This study will examine the safety, pharmacokinetics and efficacy of rIX-FP for the control and prevention of bleeding episodes in subjects who have previously received factor replacement therapy for hemophilia B.

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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

12 year–65 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

AKH Wien [Hämatologie, Hämostaseol, Vienna, Austria

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Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Male subjects, 12 to 65 years old
  • Severe hemophilia B (FIX activity of ≤ 2%)
  • Subjects who have received FIX products (plasma-derived and/or recombinant FIX) for > 150 exposure days (EDs)
  • No history of FIX inhibitor formation, no detectable inhibitors at Screening and no family history of inhibitors against FIX
  • Written informed consent for study participation
  • On-demand subjects only, who have experienced a minimum average of 2 non-trauma induced bleeding episodes requiring treatment with a FIX product during the previous 6 or 3 months

Exclusion criteria

  • Known hypersensitivity to any FIX product or hamster protein
  • Known congenital or acquired coagulation disorder other than congenital FIX deficiency
  • HIV positive subjects with a CD4 count < 200/mm3
  • Low platelet count, kidney or liver dysfunction
  • Recent life-threatening bleeding episode

Treatment and study plan

rIX-FP

Biological

Recombinant IX-FP (rIX-FP) is a fusion protein linking coagulation factor IX with albumin, and will be administered by intravenous administration

Primary outcomes

  1. Change in Frequency of Spontaneous Bleeding Events Between On-demand and Prophylaxis Treatments (Annualized)

    Time frame: Up to 26 weeks for on-demand regimen, and between 1 and 17 months for prophylaxis regimen.

    Subjects in the on-demand arm received on-demand dosing with rIX-FP for up to 26 weeks (on-demand regimen), and then received weekly prophylaxis with rIX-FP for the remainder of the study (prophylaxis regimen). The effectiveness of prophylaxis in comparison to on-demand therapy was investigated by comparing the same subject's annualized spontaneous bleeding rate (AsBR) during the on-demand regimen and during the prophylaxis regimen.

  2. Number of Subjects Developing Inhibitors Against Factor IX (FIX)

    Time frame: Up to 27.7 months (maximum)

    The number of participants developing inhibitors against factor IX (FIX) along with the 95% Clopper-Pearson confidence interval, are summarized for subjects with 50 or more exposure days (EDs) to rIX-FP, and for all participants in the study.

Secondary outcomes

  1. The Frequency of Related Adverse Events

    Time frame: For the duration of the study; median 20.27 months.

    The percentage of participants experiencing treatment-related adverse-events (TEAEs).

  2. Number of Subjects Developing Antibodies Against rIX-FP

    Time frame: For the duration of the study; median 20.27 months.

  3. Proportion of Bleeding Episodes Requiring One or ≤ Two Injections of rIX-FP to Achieve Hemostasis

    Time frame: For the duration of the study; median 20.27 months.

    Number of injections required to achieve hemostasis expressed as a percentage of the bleeding episodes requiring treatment.

  4. Investigator's Overall Clinical Assessment of Hemostatic Efficacy for Treatment of Bleeding Episodes, Based on a Four Point Ordinal Scales (Excellent, Good, Moderate, Poor/No Response)

    Time frame: For the duration of the study; median 20.27 months

    Number of bleeding episodes requiring treatment that resulted in hemostatic efficacy of excellent, good, moderate, poor/no response, according to the Investigator's clinical assessment of hemostatic efficacy, expressed as a percentage of the bleeding episodes requiring treatment.

  5. rIX-FP Consumed Per Month While Maintaining Assigned Prophylactic Treatment Interval During Routine Prophylaxis.

    Time frame: Median 269, 240, 386 and 316 days, respectively (see Description)

    Time frame: For Prophylaxis Arm 7-, 10- and 14-day regimens, median 269, 240 and 386 days respectively. For On-demand Arm, prophylaxis regimen, median 316 days.

  6. Incremental Recovery of rIX-FP

    Time frame: 336 hours

    Pharmacokinetic (PK) data are presented for a single 50 IU/kg dose of rIX-FP.

  7. Half-life (t1/2) of a Single Dose of rIX-FP

    Time frame: 336 hours

    PK data are presented for a single 50 IU/kg dose of rIX-FP.

  8. Area Under the Curve (AUC)

    Time frame: 336 hours

    AUC to the last sample with quantifiable drug concentration (AUClast) of a single dose of rIX-FP. PK data are presented for a single 50 IU/kg dose of rIX-FP.

  9. Clearance of a Single Dose of rIX-FP

    Time frame: 336 hours

    PK data are presented for a single 50 IU/kg dose of rIX-FP.

  10. Investigator's (or Surgeon's) Overall Clinical Assessment of Hemostatic Efficacy for Surgical Prophylaxis, Based on a Four Point Ordinal Scale (Excellent, Good, Moderate, Poor/No Response)

    Time frame: Up to 14 days after surgery

    Number of surgical events treated prophylactically with rIX-FP that resulted in hemostatic efficacy of excellent, good, moderate, poor/no response, according to the Investigator's (surgeon's) overall assessment of hemostatic efficacy for surgical prophylaxis.

  11. Annualized Spontaneous Bleeding Events Compared Between 7 Day Prophylactic and Extended Regimens

    Time frame: During treatment, between median 240 and 386 days per subject.

    Median number of spontaneous bleeds per year per subject comparing 7-, 10- and 14- day prophylactic regimens.

Sponsors and collaborators

Lead sponsor

CSL Behring

Industry

Registry information

Official study title

A Phase II/III Open-label, Multicenter, Safety and Efficacy Study of a Recombinant Coagulation Factor IX Albumin Fusion Protein (rIX-FP) in Subjects With Hemophilia B

Important dates

Study start
2012
Primary completion
2014
First posted
Dec 21, 2011
Registry last updated
May 9, 2016

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.

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