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

NCT ID: NCT03091751

Last Updated: 2017-07-25

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 13 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2005-08-31
• Study Completion Date: 2009-10-31

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.

Conditions

Hemophilia B

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

BeneFIX

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

Group Type: EXPERIMENTAL

BeneFIX

Intervention Type: DRUG

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

Interventions

BeneFIX

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

Intervention Type: DRUG

Other Intervention Names

recombinant coagulation factor IX

Eligibility Criteria

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

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

• Minimum Eligible Age: 12 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Grifols Biologicals, LLC

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Medical University Pleven

Pleven, , Bulgaria

National Center of Haematology

Sofia, , Bulgaria

Medical University, University Hospital "Sveta Marina",

Varna, , Bulgaria

Countries

Bulgaria

Other Identifiers

IG-404-1

Identifier Type: -

Identifier Source: org_study_id