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

NCT01361126 | Completed Phase 1 Results DMC

• 2 other identifiers: CSL654_20042010-023793-39
• Study Type: interventional
• Target: 17
• Locations: 2 countries
• Sites: 2

Brief 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.

Conditions

Hemophilia B

Outcome Measures

Primary Outcomes (3)

• Number of Subjects With Treatment-related Adverse Events

  The causal relationship of each adverse event to rIX-FP was assessed by the Investigator.

  Approximately 20 weeks

• Number of Subjects With Inhibitors Against Factor IX (FIX)

  The presence of inhibitors against FIX was assessed by the central laboratory by a FIX potency assay. To quantify anti-FIX neutralizing antibodies, the Bethesda assay with the Nijmegen modification was used, and the results expressed as Bethesda Units per mL (BU/mL). A positive inhibitor test is \>=0.6 BU/mL.

  Baseline, Day 10 and Weeks 4, 12 and 20

• Number of Subjects Who Developed Antibodies to rIX-FP

  Antibodies against rIX-FP were detected using a direct binding enzyme-linked immunosorbent assay (ELISA).

  Pre-dose, Day 10 and Weeks 4, 12, and 20

Secondary Outcomes (5)

• Area Under the Curve to the Last Sample With Quantifiable Drug Concentration (AUC0-t) After a Single Dose of rIX-FP

  Pre-dose and up to 14 days after rIX-FP infusion.

• Half-life (t1/2) of a Single Dose of rIX-FP

  Pre-dose and up to 14 days after infusion

• Incremental Recovery of rIX-FP at 30 Minutes Following Infusion of rIX-FP

  30 minutes after infusion

• Clearance of a Single Dose of rIX-FP

  Pre-dose and up to 14 days after rIX-FP infusion

• Breakthrough Bleeding Events

  Week 9 to approximately Week 20

Study Arms (2)

On-demand

EXPERIMENTAL

The routine prophylactic therapy interval is targeted at every 7 days.

Biological: Recombinant Coagulation Factor IX Albumin Fusion Protein

Prophylactic

EXPERIMENTAL

On-demand subjects will receive rIX-FP only for the treatment of a bleeding episode.

Biological: Recombinant Coagulation Factor IX Albumin Fusion Protein

Interventions

Recombinant Coagulation Factor IX Albumin Fusion Protein BIOLOGICAL

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.

Also known as: CSL654

On-demand; Prophylactic

Eligibility Criteria

• Age: 12 Years - 65 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• CSL Behring: lead

Study Sites (2)

Study Site

Sofia, Bulgaria

Location

Study Site

Tel Aviv, Israel

Location

MeSH Terms

Conditions

Hemophilia B

Interventions

albutrepenonacog alfa

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked

Results Point of Contact

• Title: Clinical Trial Disclosure Manager
• Organization: CSL Behring

clinicaltrials@cslbehring.com

Use email contact

Study Officials

• Iris Jacobs, MD

  CSL Behring

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 25, 2011
• First Posted: May 26, 2011
• Study Start: July 1, 2011
• Primary Completion: June 1, 2012
• Study Completion: July 1, 2012
• Last Updated: May 9, 2016
• Results First Posted: May 9, 2016

Record last verified: 2016-04

Locations

IL (1); BU (1)