A Safety and Efficacy Extension Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Patients With Hemophilia B
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|ClinicalTrials.gov Identifier: NCT02053792|
Recruitment Status : Recruiting
First Posted : February 4, 2014
Last Update Posted : September 3, 2018
This study will examine the long-term safety and efficacy of rIX-FP for the control and prevention of bleeding episodes in children and adults with severe hemophilia B. The study will include subjects who have not previously been treated with Factor IX products, subjects who previously completed a CSL-sponsored rIX-FP lead-in study and subjects requiring major non-emergency surgery who have not previously completed a CSL-sponsored rIX-FP lead-in study.
A surgical prophylaxis substudy will examine the efficacy of rIX-FP in subjects with hemophilia B who are undergoing non-emergency major or minor surgery. An additional substudy will examine the safety and PK of subcutaneous (SC) administration of rIX-FP.
|Condition or disease||Intervention/treatment||Phase|
|Hemophilia B||Biological: rIX-FP||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||115 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase 3b Open-label, Multicenter, Safety and Efficacy Extension Study of a Recombinant Coagulation Factor IX Albumin Fusion Protein (rIX-FP) in Subjects With Hemophilia B|
|Actual Study Start Date :||February 6, 2014|
|Estimated Primary Completion Date :||March 30, 2020|
|Estimated Study Completion Date :||April 30, 2020|
Subjects will administer rIX-FP by intravenous infusion as routine prophylaxis, prevention, and on-demand treatment during a treatment period of approximately 3 years.
The routine prophylaxis treatment interval for previously treated patients may be changed at each scheduled 6-month follow-up assessment. On-demand treatment with rIX-FP will be used for all bleeding episodes requiring treatment. Subjects (other than those in France) may participate in a surgical 'substudy' in which rIX-FP may be administered before, during and after surgery. An additional substudy will examine the safety and PK of subcutaneous administration of rIX-FP.
For previously untreated patients, subjects will administer rIX-FP intravenously as weekly prophylaxis and/or on-demand treatment during the first 12 months, and as weekly routine prophylaxis thereafter.
The dose of rIX-FP administered will be based on the subject's previous rIX-FP use and/or pharmacokinetic data.
Recombinant Fusion Protein Linking Coagulation Factor IX with Albumin (rIX-FP)
- Main study: Total number of subjects who develop inhibitors against factor IX (FIX) [ Time Frame: Approximately 5 years ]
- Surgery substudy: Investigator's overall clinical assessment of hemostatic efficacy for surgical prophylaxis [ Time Frame: Immediately after surgery (0 hours) and at up to 3 timepoints thereafter up to 72 hours or discharge, whichever is earliest. ]Clinical assessment of hemostatic efficacy to be based on a four point ordinal scale (excellent, good, moderate, poor / none)
- Incremental recovery in previously untreated patients (PUPs) [ Time Frame: Approximately 30 minutes after infusion ]Incremental recovery expressed as IU/dL per IU/kg
- Main study: Comparison of annualized bleeding rate between different prophylaxis treatment intervals. [ Time Frame: Approximately 5 years ]Comparison of annualized bleeding rate by prophylaxis treatment interval for spontaneous treated bleeds and total treated bleeds.
- Main study: Comparison of annualized bleeding rate between 2 different prophylaxis treatment intervals and on-demand treatment. [ Time Frame: Approximately 5 years ]
For subjects from the lead-in study (CSL654_3001 [NCT01496274]), comparison of the annualized bleeding rate of spontaneous treated bleeds and total treated bleeds between routine prophylaxis treatment in this study (CSL654_3003) and:
- On-demand treatment during the lead-in study (CSL654_3001).
- Routine prophylaxis treatment with the different treatment interval used by the subject in the lead-in study (CSL654_3001).
- Main study: rIX-FP consumed per month per subject during routine prophylaxis treatment. [ Time Frame: Approximately 5 years ]
- Surgery substudy: The frequency of adverse events (AEs) related to rIX-FP [ Time Frame: 28 days after surgery (major surgery) or up to hospital discharge (minor surgery) ]
- Surgery substudy: Total number of subjects who develop inhibitors against FIX [ Time Frame: 28 days after surgery (major surgery) or up to hospital discharge (minor surgery) ]
- Surgery substudy: Total number of subjects who develop antibodies against rIX-FP. [ Time Frame: 28 days after surgery (major surgery) or up to hospital discharge (minor surgery) ]
- Surgery substudy: Predicted and intraoperative estimated blood loss. [ Time Frame: Predicted blood loss and intraoperative estimated blood loss to be determined before surgery and at the end of surgery, respectively ]
The predicted blood loss (mL) is based on blood loss expected from a non-hemophilic individual undergoing the same type / extent of procedure.
The estimated blood loss (mL) is the anesthesiologist's record of estimated blood loss during the procedure.
- Surgery substudy: Predicted and actual transfusion requirements. [ Time Frame: Predicted and actual transfusion requirements to be determined before and at the end of surgery, respectively ]
- Surgery substudy: Change in hemoglobin levels between baseline, intraoperatively and postoperatively (major surgery only). [ Time Frame: Before, during and up to 28 days after surgery ]
- Overall adverse events (AEs) and rIX-FP-related AEs [ Time Frame: Approximately 5 years ]The overall percentage of subjects with at least one AE, and the overall percentage of subjects with at least one rIX-FP-related AE
- Hemostatic response to rIX-FP treatment in PUPs [ Time Frame: Hemostatic response of rIX-FP treatment during the course of the study (up to 5 years) ]The investigator will rate the efficacy of the rIX-FP treatment based on a hemostatic efficacy four point rating scale of "excellent, good, moderate or poor/no response".
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): NCT02053792
|Contact: Clinical Trial Registration Coordinatorfirstname.lastname@example.org|
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|Study Director:||Program Director||CSL Behring|