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Efficacy and Safety of AlphaNine Versus BeneFIX in Patients With Severe Hereditary Haemophilia B

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ClinicalTrials.gov Identifier: NCT03091751
Recruitment Status : Completed
First Posted : March 27, 2017
Results First Posted : July 25, 2017
Last Update Posted : July 25, 2017
Sponsor:
Information provided by (Responsible Party):
Grifols Biologicals, LLC

Tracking Information
First Submitted Date  ICMJE March 16, 2017
First Posted Date  ICMJE March 27, 2017
Results First Submitted Date  ICMJE April 13, 2017
Results First Posted Date  ICMJE July 25, 2017
Last Update Posted Date July 25, 2017
Actual Study Start Date  ICMJE August 2005
Actual Primary Completion Date October 2009   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 26, 2017)
  • Mean Difference of Area Under the Curve (AUC): BeneFIX Compared to AlphaNine [ Time Frame: Baseline (prior to the infusion), and at 15 and 30 minutes, 1, 3, 6, 9, 24, 48, 72, and 74 hours following a single dose infusion of BeneFIX administered following a 7 to 15 day wash-out period. ]
    BeneFIX pharmacokinetic parameter of area under the curve (AUC 0-inf) was assessed and compared to the AlphaNine pharmacokinetic parameter (PK2 study).
  • Mean Difference of In Vivo Recovery: BeneFIX Compared to AlphaNine [ Time Frame: Baseline (prior to the infusion), and at 15 and 30 minutes, 1, 3, 6, 9, 24, 48, 72, and 74 hours following a single dose infusion of BeneFIX administered following a 7 to 15 day wash-out period. ]
    BeneFIX pharmacokinetic parameter of in vivo recovery was assessed and compared to the AlphaNine pharmacokinetic parameter (PK2 Study).
  • Mean Difference of Terminal Half-Life: BeneFIX Compared to AlphaNine [ Time Frame: Baseline (prior to the infusion), and at 15 and 30 minutes, 1, 3, 6, 9, 24, 48, 72, and 74 hours following a single dose infusion of BeneFIX administered following a 7 to 15 day wash-out period. ]
    BeneFIX pharmacokinetic parameter of terminal half-life was assessed and compared to the AlphaNine pharmacokinetic parameter (PK2 Study).
  • Mean Difference of Clearance: BeneFIX Compared to AlphaNine [ Time Frame: Baseline (prior to the infusion), and at 15 and 30 minutes, 1, 3, 6, 9, 24, 48, 72, and 74 hours following a single dose infusion of BeneFIX administered following a 7 to 15 day wash-out period. ]
    BeneFIX pharmacokinetic parameter of clearance was assessed and compared to the AlphaNine pharmacokinetic parameter (PK2 Study).
  • Mean Difference of Mean Residence Time (MRT): BeneFIX Compared to AlphaNine [ Time Frame: Baseline (prior to the infusion), and at 15 and 30 minutes, 1, 3, 6, 9, 24, 48, 72, and 74 hours following a single dose infusion of BeneFIX administered following a 7 to 15 day wash-out period. ]
    BeneFIX pharmacokinetic parameter of mean residence time (MRT 0-inf) was assessed and compared to the AlphaNine pharmacokinetic parameter (PK2 Study).
Original Primary Outcome Measures  ICMJE
 (submitted: March 21, 2017)
Factor IX Plasma Activity [ Time Frame: Baseline (prior to the infusion), and at 15 and 30 minutes, 1, 3, 6, 9, 24, 48, 50 (optional), 72 and 74 hours after the end of the infusion. ]
Change History Complete list of historical versions of study NCT03091751 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Efficacy and Safety of AlphaNine Versus BeneFIX in Patients With Severe Hereditary Haemophilia B
Official Title  ICMJE Efficacy and Safety of Factor IX (FIX) Contained in AlphaNine® and Its Pharmacokinetic Comparison With BeneFIX® in Patients With Severe Hereditary Haemophilia B
Brief Summary The goal of this non-randomized, multi-center study in subjects with severe hereditary haemophilia B was to determine and compare the pharmacokinetic and safety profiles of BeneFIX in subjects having had 2 prior pharmacokinetic assessments with AlphaNine.
Detailed Description

Two pharmacokinetic assessments (studies) were carried out in the same subjects during a previous clinical trial. The first pharmacokinetic study (PK1) was performed after a single dose of AlphaNine. The second pharmacokinetic study (PK2) was performed following 26 Weeks of AlphaNine treatment after PK1. To compare AlphaNine with BeneFIX, a third pharmacokinetic study (PK3) (current study) was performed after a single dose of BeneFIX administered following a 7- to 15-day wash-out period.

The main objective of the PK3 study was to assess the pharmacokinetic profile of BeneFIX and compare to the pharmacokinetic profile of AlphaNine from the PK2 study.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Hemophilia B
Intervention  ICMJE Drug: BeneFIX
BeneFIX is a recombinant FIX that contains nonacog alfa, reconstituted in solvent and administered as a single dose of 65-75 IU/kg.
Other Name: recombinant coagulation factor IX
Study Arms  ICMJE Experimental: BeneFIX
BeneFIX is a recombinant FIX provided in a vial containing 100 IU/mL lyophilized nonacog alfa accompanied with solvent for reconstitution and injection.
Intervention: Drug: BeneFIX
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: March 21, 2017)
13
Original Actual Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE October 2009
Actual Primary Completion Date October 2009   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Key Inclusion Criteria:

  • Participated in the previous study "Efficacy and safety of factor IX (FIX) contained in Alphananine in patients with severe hereditary haemophilia B":
  • Congenital deficiency in Factor IX (FIX)
  • FIX residual activity of ≤2% of normal
  • Had required FIX-containing products in the past and in clinical records that were collected data to assess a reliable estimation of at least 150 treatment exposure days to previous products
  • Was able to receive treatment for more than 10 days for a 6-month period

Key Exclusion Criteria:

  • Received a dose of FIX in the 7 days prior to the infusion
  • FIX inhibitor level of >0.5 Bethesda units (BU) or clinically relevant presence in the past (≥5 BU)
  • Active bleeding at the moment of infusion
  • Had a known allergic reaction to any BeneFIX component
  • Exhibited symptoms of any intercurrent infection (ie, fever, chills, nausea) at the time of the first infusion
  • Had any disease that might affect the distribution or metabolism of FIX and which could affect interpretation of the study (such as non-controlled diabetes mellitus)
  • Had non-controlled arterial hypertension
  • Had abnormal renal function (creatinine >1.5 mg/dL)
  • Had documented liver cirrhosis or any hepatic disorder with alanine aminotransferase (ALT) levels 2.5x upper limit of normal (ULN )
  • Prevision to be concomitantly treated with other FIX-containing products
  • Had conditions that might affect subject compliance (survival-limiting [in 2 year time] diseases, alcohol or other drug abuse, etc.)
  • Unable to provide a storage plasma sample before the first dose of BeneFIX
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 12 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Bulgaria
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03091751
Other Study ID Numbers  ICMJE IG-404-1
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Product Manufactured in and Exported from the U.S.: Yes
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Grifols Biologicals, LLC
Study Sponsor  ICMJE Grifols Biologicals, LLC
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Grifols Biologicals, LLC
Verification Date June 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP