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

Browse trials for

• Hemophilia A 866
• Hemophilia B 271

Hemophilia A

866 studies on the registry are indexed under Hemophilia A; 137 are open to participants now.

This study's enrollment of 63 is above the median of 28 across 512 interventional studies indexed under Hemophilia A.

Lead sponsor

CSL Behring is the lead sponsor of 142 studies on the registry; 18 are open to participants now.

Of its 24 completed or terminated interventional studies of FDA-regulated products, 17 (71%) have results posted.

Study arms

• Experimental
  Prophylaxis

  Routine weekly prophylaxis and episodic treatment for bleeding episodes. An individualized dosing interval may be tested in sub-group subjects during the 2nd part of the trial. Subjects may participate in a surgical 'sub-study' in which rIX-FP may be administered prior to, during and after surgical intervention.

  Biological: rIX-FP

• Experimental
  On-demand

  Episodic treatment for bleeding episodes during the first 6 months then switch to routine weekly prophylaxis for a further 6 months Subjects may participate in a surgical 'sub-study' in which rIX-FP may be administered prior to, during and after surgical intervention.

  Biological: rIX-FP

Interventions

• BiologicalrIX-FP

  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)

   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.

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

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

   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.

   Time frame: Up to 27.7 months (maximum)

Secondary outcomes

1. The Frequency of Related Adverse Events

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

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

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

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

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

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)

   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.

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

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

   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.

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

6. Incremental Recovery of rIX-FP

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

   Time frame: 336 hours

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

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

   Time frame: 336 hours

8. Area Under the Curve (AUC)

   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.

   Time frame: 336 hours

9. Clearance of a Single Dose of rIX-FP

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

   Time frame: 336 hours

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)

    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.

    Time frame: Up to 14 days after surgery

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

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

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

Baseline characteristics

Publications

• Santagostino E, Martinowitz U, Lissitchkov T, Pan-Petesch B, Hanabusa H, Oldenburg J, Boggio L, Negrier C, Pabinger I, von Depka Prondzinski M, Altisent C, Castaman G, Yamamoto K, Alvarez-Roman MT, Voigt C, Blackman N, Jacobs I; PROLONG-9FP Investigators Study Group. Long-acting recombinant coagulation factor IX albumin fusion protein (rIX-FP) in hemophilia B: results of a phase 3 trial. Blood. 2016 Apr 7;127(14):1761-9. doi: 10.1182/blood-2015-09-669234. Epub 2016 Jan 11. PubMed 26755710 ↗