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Safety of 40K Pegylated Recombinant Factor IX in Non-Bleeding Patients With Haemophilia B

Primary Purpose

Congenital Bleeding Disorder, Haemophilia B

Status

Completed

Phase

Phase 1

Locations

International

Study Type

Interventional

Intervention

nonacog beta pegol

About this trial

This is an interventional treatment trial for Congenital Bleeding Disorder

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)MaleDoes not accept healthy volunteers

Inclusion Criteria:

Diagnosed with haemophilia B (baseline level of Factor IX less than or equal to 2%)
History of at least 150 exposure days to any Factor IX products
Body Mass Index (BMI) below 30.0 kg/m2 (inclusive)

Exclusion Criteria:

History of Factor IX inhibitors
Platelet count less than 50,000 platelets/microlitre (assessed by laboratory)
Kidney or liver dysfunction
Scheduled surgery requiring Factor IX replacement therapy, during the trial period

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Arm Label

25U/kg

50U/kg

100U/kg

Arm Description

Outcomes

Primary Outcome Measures

Frequency of Adverse Events (AEs), Serious Adverse Events (SAEs) and Medical Events of Special Interests (MESIs) reported during the trial period

Antibody formation against 40K PEG-rFIX and test for inhibitors (Bethesda)

Secondary Outcome Measures

AUC, CL, T½, Incremental recovery (first sample) from 0 to 48 hours after trial product administration

AUC, CL, T½, Incremental recovery (first sample) from 0 to 168 hours after trial product administration

Full Information

NCT ID

NCT00956345

First Posted

August 10, 2009

Last Updated

January 19, 2017

Sponsor

Novo Nordisk A/S

1. Study Identification

Unique Protocol Identification Number

NCT00956345

Brief Title

Safety of 40K Pegylated Recombinant Factor IX in Non-Bleeding Patients With Haemophilia B

Official Title

A Multi-Centre, Multi-National, Open-Label, Dose Escalation Trial Evaluating Safety and Pharmacokinetics of Ascending Intravenous Doses of 40K Pegylated Recombinant FIX in Non-Bleeding Patients With Haemophilia B.

Study Type

Interventional

2. Study Status

Record Verification Date

January 2017

Overall Recruitment Status

Completed

Study Start Date

August 2009 (undefined)

Primary Completion Date

July 2010 (Actual)

Study Completion Date

July 2010 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Novo Nordisk A/S

4. Oversight

Data Monitoring Committee

5. Study Description

Brief Summary

This trial is conducted in Europe, Japan and the United States of America (USA). The aim of this clinical trial is to investigate the safety and pharmacokinetics (the determination of the concentration of the administered medication in blood over time) of Pegylated Recombinant Factor IX (nonacog beta pegol) in Non-Bleeding Patients with Haemophilia B.

6. Conditions and Keywords

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

Congenital Bleeding Disorder, Haemophilia B

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

18 (Actual)

8. Arms, Groups, and Interventions

Arm Title

25U/kg

Arm Type

Experimental

Arm Title

50U/kg

Arm Type

Experimental

Arm Title

100U/kg

Arm Type

Experimental

Intervention Type

Drug

Intervention Name(s)

nonacog beta pegol

Other Intervention Name(s)

40K PEG-rFIX

Intervention Description

Cohort to receive a single dose of 25U/kg nonacog beta pegol administered intravenously (into the vein)

Intervention Type

Drug

Intervention Name(s)

nonacog beta pegol

Other Intervention Name(s)

40K PEG-rFIX

Intervention Description

Cohort to receive a single dose of 50U/kg nonacog beta pegol administered intravenously (into the vein)

Intervention Type

Drug

Intervention Name(s)

nonacog beta pegol

Other Intervention Name(s)

40K PEG-rFIX

Intervention Description

Cohort to receive a single dose of 100U/kg nonacog beta pegol administered intravenously (into the vein)

Primary Outcome Measure Information:

Title

Frequency of Adverse Events (AEs), Serious Adverse Events (SAEs) and Medical Events of Special Interests (MESIs) reported during the trial period

Time Frame

assessed up to five weeks after trial product administration

Title

Antibody formation against 40K PEG-rFIX and test for inhibitors (Bethesda)

Time Frame

assessed up to five weeks after trial product administration

Secondary Outcome Measure Information:

Title

AUC, CL, T½, Incremental recovery (first sample) from 0 to 48 hours after trial product administration

Time Frame

assessed up to five weeks after trial product administration

Title

AUC, CL, T½, Incremental recovery (first sample) from 0 to 168 hours after trial product administration

Time Frame

assessed up to five weeks after trial product administration

10. Eligibility

Sex

Male

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

65 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Diagnosed with haemophilia B (baseline level of Factor IX less than or equal to 2%) History of at least 150 exposure days to any Factor IX products Body Mass Index (BMI) below 30.0 kg/m2 (inclusive) Exclusion Criteria: History of Factor IX inhibitors Platelet count less than 50,000 platelets/microlitre (assessed by laboratory) Kidney or liver dysfunction Scheduled surgery requiring Factor IX replacement therapy, during the trial period

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Global Clinical Registry (GCR, 1452)

Organizational Affiliation

Novo Nordisk A/S

Official's Role

Study Director

Facility Information:

Facility Name

Novo Nordisk Investigational Site

City

Cincinnati

State/Province

Ohio

ZIP/Postal Code

45229

Country

United States

Facility Name

Novo Nordisk Investigational Site

City

Portland

State/Province

Oregon

ZIP/Postal Code

97239

Country

United States

Facility Name

Novo Nordisk Investigational Site

City

Richmond

State/Province

Virginia

ZIP/Postal Code

23219

Country

United States

Facility Name

Novo Nordisk Investigational Site

City

København

ZIP/Postal Code

2100

Country

Denmark

Facility Name

Novo Nordisk Investigational Site

City

Kremlin-Bicêtre

ZIP/Postal Code

94270

Country

France

Facility Name

Novo Nordisk Investigational Site

City

Lyon

ZIP/Postal Code

69003

Country

France

Facility Name

Novo Nordisk Investigational Site

City

Berlin

ZIP/Postal Code

10249

Country

Germany

Facility Name

Novo Nordisk Investigational Site

City

Bonn

ZIP/Postal Code

53127

Country

Germany

Facility Name

Novo Nordisk Investigational Site

City

Frankfurt/M.

ZIP/Postal Code

60590

Country

Germany

Facility Name

Novo Nordisk Investigational Site

City

Hannover

ZIP/Postal Code

30625

Country

Germany

Facility Name

Novo Nordisk Investigational Site

City

Kashihara-shi, Nara

ZIP/Postal Code

634 8522

Country

Japan

Facility Name

Novo Nordisk Investigational Site

City

Nagoya-shi, Aichi

ZIP/Postal Code

466 8560

Country

Japan

Facility Name

Novo Nordisk Investigational Site

City

Nishinomiya-shi

ZIP/Postal Code

663 8051

Country

Japan

Facility Name

Novo Nordisk Investigational Site

City

Madrid

ZIP/Postal Code

28046

Country

Spain

Facility Name

Novo Nordisk Investigational Site

City

Stockholm

ZIP/Postal Code

171 76

Country

Sweden

Facility Name

Novo Nordisk Investigational Site

City

London

ZIP/Postal Code

NW3 2QG

Country

United Kingdom

Facility Name

Novo Nordisk Investigational Site

City

London

ZIP/Postal Code

SE1 7EH

Country

United Kingdom

Facility Name

Novo Nordisk Investigational Site

City

Manchester

ZIP/Postal Code

M13 9WL

Country

United Kingdom

Facility Name

Novo Nordisk Investigational Site

City

Oxford

ZIP/Postal Code

OX3 7LJ

Country

United Kingdom

12. IPD Sharing Statement

Citations:

PubMed Identifier

21555744

Citation

Negrier C, Knobe K, Tiede A, Giangrande P, Moss J. Enhanced pharmacokinetic properties of a glycoPEGylated recombinant factor IX: a first human dose trial in patients with hemophilia B. Blood. 2011 Sep 8;118(10):2695-701. doi: 10.1182/blood-2011-02-335596. Epub 2011 May 9.

Results Reference

background

PubMed Identifier

22998153

Citation

Collins PW, Moss J, Knobe K, Groth A, Colberg T, Watson E. Population pharmacokinetic modeling for dose setting of nonacog beta pegol (N9-GP), a glycoPEGylated recombinant factor IX. J Thromb Haemost. 2012 Nov;10(11):2305-12. doi: 10.1111/jth.12000.

Results Reference

background

Links:

URL

http://novonordisk-trials.com

Description

Clinical Trials at Novo Nordisk

Learn more about this trial

Safety of 40K Pegylated Recombinant Factor IX in Non-Bleeding Patients With Haemophilia B

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Safety of 40K Pegylated Recombinant Factor IX in Non-Bleeding Patients With Haemophilia B

Congenital Bleeding Disorder, Haemophilia B

About this trial

Eligibility Criteria

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Arm 3

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

1. Study Identification

2. Study Status

3. Sponsor/Collaborators

4. Oversight

5. Study Description

6. Conditions and Keywords

7. Study Design

8. Arms, Groups, and Interventions

10. Eligibility

12. IPD Sharing Statement

Learn more about this trial