A Safety and Efficacy Study of a Recombinant Factor IX in Patients With Severe Hemophilia B

Hemophilia B Clinical Trial

Official title:

A Phase I/II Open-label, Multicenter, Safety and Efficacy Study of a Recombinant Coagulation Factor IX Albumin Fusion Protein (rIX-FP) in Subjects With Hemophilia B

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01361126
• Other study ID #: CSL654_2004
• Secondary ID: 2010-023793-39
• Status: Completed
• Phase: Phase 1/Phase 2
• First received: May 25, 2011
• Last updated: November 5, 2015
• Start date: July 2011
• Est. completion date: July 2012

Study information

• Verified date: November 2015
• Source: CSL Behring
• Contact: n/a
• Is FDA regulated: No
• Health authority: Bulgaria: Bulgarian Drug AgencyIsrael: Ministry of Health
• Study type: Interventional

Clinical Trial Summary

This study will examine the safety and efficacy of a Recombinant Coagulation Factor IX Albumin Fusion Protein (rIX-FP) for the control and prevention of bleeding episodes in subjects who have previously received factor replacement therapy for hemophilia B. The study consists of a screening period, a pharmacokinetic (PK) period, followed by approximately a 5 month treatment period. Subjects will receive weekly routine prophylactic therapy and on-demand treatment for bleeding episodes. In addition, subjects who are not on routine factor replacement therapy prior to the study will receive only on-demand treatment for bleeding episodes.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 17
• Est. completion date: July 2012
• Est. primary completion date: June 2012
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 12 Years to 65 Years

Eligibility

Inclusion Criteria:

• Male subjects, 12 to 65 years old

• Severe hemophilia B (FIX activity of = 2%)

• Subjects who have received FIX products (plasma-derived and/or recombinant FIX) for > 150 exposure days (EDs)

• No history of FIX inhibitor formation, no detectable inhibitors at Screening and no family history of inhibitors against FIX

• Written informed consent for study participation obtained before undergoing any study specific procedures

Exclusion Criteria:

• Known hypersensitivity to any FIX product or hamster protein

• Known congenital or acquired coagulation disorder other than congenital FIX deficiency

• HIV positive subjects with a CD4 count < 200/mm3

• Low platelet count, abnormal kidney function, or liver disease

• On-demand subjects experiencing less than 12 or 6 non-trauma induced bleeding episodes requiring treatment with a FIX product during the previous 6 or 3 months, respectively

• Planned major surgical intervention during the study period

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Hemophilia A
• Hemophilia B

Intervention

Biological:
• Recombinant Coagulation Factor IX Albumin Fusion Protein
Study subjects will receive a single dose of 25IU/kg of rIX_FP for pharmacokinetic analysis. Subjects will then be treated for approximately 5 months. The treatment dose will be based on the subject's PK profile and the subject's bleeding phenotype.

Locations

Country	Name	City	State
Bulgaria	Study Site	Sofia
Israel	Study Site	Tel Aviv

Sponsors (1)

Lead Sponsor	Collaborator
CSL Behring

Countries where clinical trial is conducted

Bulgaria,  Israel, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Frequency of related adverse events		Approximately 20 weeks	Yes
Primary	Number of subjects developing inhibitors against factor IX (FIX)		Approximately 20 weeks	Yes
Primary	Number of subjects developing antibodies against rIX-FP		Approximately 20 weeks	Yes
Secondary	Area under the curve (AUC)	AUC to the last sample with quantifiable drug concentration (AUC0-t) of a single dose of rIX-FP	168 Hours	No
Secondary	Half-life (t1/2) of a single dose of rIX-FP		168 hours	No
Secondary	Incremental recovery of rIX-FP		30 minutes	No
Secondary	Clearance of a single dose of rIX-FP		168 hours	No
Secondary	Breakthrough bleeding events	Number of breakthrough bleeding events in subjects receiving prophylactic treatment regimen with rIX-FP	Week 9 to approximately Week 20	No