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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 Inc.

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.

Condition or disease Intervention/treatment Phase
Hemophilia B Drug: BeneFIX Phase 2

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 : Interventional  (Clinical Trial)
Actual Enrollment : 13 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Efficacy and Safety of Factor IX (FIX) Contained in AlphaNine® and Its Pharmacokinetic Comparison With BeneFIX® in Patients With Severe Hereditary Haemophilia B
Actual Study Start Date : August 2005
Actual Primary Completion Date : October 2009
Actual Study Completion Date : October 2009


Arm Intervention/treatment
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.
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




Primary Outcome Measures :
  1. 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).

  2. 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).

  3. 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).

  4. 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).

  5. 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).



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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

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): NCT03091751


Locations
Bulgaria
Medical University Pleven
Pleven, Bulgaria, 5800
National Center of Haematology
Sofia, Bulgaria, 1000
Medical University, University Hospital "Sveta Marina",
Varna, Bulgaria, 9010
Sponsors and Collaborators
Grifols Biologicals Inc.

Responsible Party: Grifols Biologicals Inc.
ClinicalTrials.gov Identifier: NCT03091751     History of Changes
Other Study ID Numbers: IG-404-1
First Posted: March 27, 2017    Key Record Dates
Results First Posted: July 25, 2017
Last Update Posted: July 25, 2017
Last Verified: June 2017

Studies a U.S. FDA-regulated Drug Product: Yes
Product Manufactured in and Exported from the U.S.: Yes

Keywords provided by Grifols Biologicals Inc.:
Factor IX (FIX)

Additional relevant MeSH terms:
Hemophilia A
Hemophilia B
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked