Back to results

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

Primary Purpose

Hemophilia B

Status

Completed

Phase

Phase 2

Locations

Bulgaria

Study Type

Interventional

Intervention

BeneFIX

About this trial

This is an interventional treatment trial for Hemophilia B focused on measuring Factor IX (FIX)

Eligibility Criteria

12 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Medical University Pleven
National Center of Haematology
Medical University, University Hospital "Sveta Marina",

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

BeneFIX

Arm Description

BeneFIX is a recombinant FIX provided in a vial containing 100 IU/mL lyophilized nonacog alfa accompanied with solvent for reconstitution and injection.

Outcomes

Primary Outcome Measures

Mean Difference of Area Under the Curve (AUC): BeneFIX Compared to AlphaNine

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

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

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

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

BeneFIX pharmacokinetic parameter of mean residence time (MRT 0-inf) was assessed and compared to the AlphaNine pharmacokinetic parameter (PK2 Study).

Secondary Outcome Measures

Full Information

NCT ID

NCT03091751

First Posted

March 16, 2017

Last Updated

June 26, 2017

Sponsor

Grifols Biologicals, LLC

1. Study Identification

Unique Protocol Identification Number

NCT03091751

Brief Title

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

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

Study Type

Interventional

2. Study Status

Record Verification Date

June 2017

Overall Recruitment Status

Completed

Study Start Date

August 2005 (Actual)

Primary Completion Date

October 2009 (Actual)

Study Completion Date

October 2009 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Grifols Biologicals, LLC

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Product Manufactured in and Exported from the U.S.

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Hemophilia B

Keywords

Factor IX (FIX)

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

13 (Actual)

8. Arms, Groups, and Interventions

Arm Title

BeneFIX

Arm Type

Experimental

Arm Description

BeneFIX is a recombinant FIX provided in a vial containing 100 IU/mL lyophilized nonacog alfa accompanied with solvent for reconstitution and injection.

Intervention Type

Drug

Intervention Name(s)

BeneFIX

Other Intervention Name(s)

recombinant coagulation factor IX

Intervention Description

BeneFIX is a recombinant FIX that contains nonacog alfa, reconstituted in solvent and administered as a single dose of 65-75 IU/kg.

Primary Outcome Measure Information:

Title

Mean Difference of Area Under the Curve (AUC): BeneFIX Compared to AlphaNine

Description

BeneFIX pharmacokinetic parameter of area under the curve (AUC 0-inf) was assessed and compared to the AlphaNine pharmacokinetic parameter (PK2 study).

Time Frame