Congenital Bleeding DisorderPhase 1NCT00956345
Safety of 40K Pegylated Recombinant Factor IX in Non-Bleeding Patients With Haemophilia B
A Phase 1 study testing nonacog beta pegol in people with Congenital Bleeding Disorder and Haemophilia B, run by Novo Nordisk A/S at 19 sites in 8 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 65 years, men only
- Conditions the study is for
- Congenital Bleeding Disorder, Haemophilia B
- What is being tested?
- nonacog beta pegol
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 19 sitesin 8 countries
- Study dates
- Aug 2009to Jul 2010
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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)
Who cannot join
- 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
Trial locations and contacts
19 sites in 8 countries.
19 sites in 8 countries
Germany4 sites
Novo Nordisk Investigational Site
Berlin, 10249
Novo Nordisk Investigational Site
Bonn, 53127
Novo Nordisk Investigational Site
Frankfurt/M., 60590
Novo Nordisk Investigational Site
Hanover, 30625
United Kingdom4 sites
Novo Nordisk Investigational Site
London, NW3 2QG
Novo Nordisk Investigational Site
London, SE1 7EH
Novo Nordisk Investigational Site
Manchester, M13 9WL
Novo Nordisk Investigational Site
Oxford, OX3 7LJ
United States3 sites
Novo Nordisk Investigational Site
Cincinnati, Ohio, 45229
Novo Nordisk Investigational Site
Portland, Oregon, 97239
Novo Nordisk Investigational Site
Richmond, Virginia, 23219
Japan3 sites
Novo Nordisk Investigational Site
Kashihara-shi, Nara, 634 8522
Novo Nordisk Investigational Site
Nagoya-shi, Aichi, 466 8560
Novo Nordisk Investigational Site
Nishinomiya-shi, 663 8051
France2 sites
Novo Nordisk Investigational Site
Le Kremlin-Bicêtre, 94270
Novo Nordisk Investigational Site
Lyon, 69003
About this study
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.
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.
- Conditions
- Congenital Bleeding Disorder, Haemophilia B
- Being tested
- nonacog beta pegol
- Medical subject headings
- Hemophilia B
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
nonacog beta pegolDrug
Also called 40K PEG-rFIX
Cohort to receive a single dose of 25U/kg nonacog beta pegol administered intravenously (into the vein)
nonacog beta pegolDrug
Also called 40K PEG-rFIX
Cohort to receive a single dose of 50U/kg nonacog beta pegol administered intravenously (into the vein)
nonacog beta pegolDrug
Also called 40K PEG-rFIX
Cohort to receive a single dose of 100U/kg nonacog beta pegol administered intravenously (into the vein)
| Study group | Role | What they receive |
|---|---|---|
| 25U/kg | Experimental | Not described Receives: Drug: nonacog beta pegol |
| 50U/kg | Experimental | Not described Receives: Drug: nonacog beta pegol |
| 100U/kg | Experimental | Not described Receives: Drug: nonacog beta pegol |
What is being measured
The primary outcome is the main question the study is designed to answer.
Frequency of Adverse Events (AEs), Serious Adverse Events (SAEs) and Medical Events of Special Interests (MESIs) reported during the trial period
Measured: assessed up to five weeks after trial product administration
Antibody formation against 40K PEG-rFIX and test for inhibitors (Bethesda)
Measured: assessed up to five weeks after trial product administration
Secondary outcomes (2)
AUC, CL, T½, Incremental recovery (first sample) from 0 to 48 hours after trial product administration
Measured: assessed up to five weeks after trial product administration
AUC, CL, T½, Incremental recovery (first sample) from 0 to 168 hours after trial product administration
Measured: assessed up to five weeks after trial product administration
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Aug 2009 (estimated)
- Study startAug 2009
- Primary completionJul 2010
- Study completionJul 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Congenital Bleeding DisorderStudies of the same condition, open and under way.
- More trials run by Novo Nordisk A/SThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novo Nordisk A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Global Clinical Registry (GCR, 1452), Study director, Novo Nordisk A/S
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00956345
- Sponsor's protocol ID
- NN7999-3639
- Other IDs
- 2009-011085-28, 090857, 2009
- Status last verified
- Jan 2017
- First submitted
- 10 Aug 2009
- Last updated
- 20 Jan 2017
- Results posted
- No
- Data monitoring committee
- No
Publications
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.
PubMed 21555744Collins 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.
PubMed 22998153
Study at a glance
From the registry record, last updated by the sponsor on 20 Jan 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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.
- ClinicalTrials.gov ID
- NCT00956345
- Acronym / sponsor ID
- NN7999-3639
- Conditions
- Congenital Bleeding Disorder, Haemophilia B
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- Novo Nordisk A/SIndustry (pharma, biotech, devices)
- Study start
- Aug 2009
- Primary completion
- Jul 2010 (actual)
- Study completion
- Jul 2010 (actual)
- First posted
- 11 Aug 2009 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| 25U/kg | Experimental | Drug: nonacog beta pegol |
| 50U/kg | Experimental | Drug: nonacog beta pegol |
| 100U/kg | Experimental | Drug: nonacog beta pegol |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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)
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| nonacog beta pegolAlso: 40K PEG-rFIXCohort to receive a single dose of 25U/kg nonacog beta pegol administered intravenously (into the vein) | Drug | 25U/kg |
| nonacog beta pegolAlso: 40K PEG-rFIXCohort to receive a single dose of 50U/kg nonacog beta pegol administered intravenously (into the vein) | Drug | 50U/kg |
| nonacog beta pegolAlso: 40K PEG-rFIXCohort to receive a single dose of 100U/kg nonacog beta pegol administered intravenously (into the vein) | Drug | 100U/kg |
Primary outcome
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
Antibody formation against 40K PEG-rFIX and test for inhibitors (Bethesda)
Time frame: assessed up to five weeks after trial product administration
2 secondary outcomes
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
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Jan 2017.
- Background: PubMed 21555744Negrier 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.
- Background: PubMed 22998153Collins 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.
- Clinical Trials at Novo NordiskLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Global Clinical Registry (GCR, 1452) study director
Novo Nordisk A/S
19 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing nonacog beta pegol in people with Congenital Bleeding Disorder and Haemophilia B, run by Novo Nordisk A/S at 19 sites in 8 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 20 Jan 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 19 sites in Germany, United Kingdom, United States, Japan, France and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00956345, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Jan 2017.