Congenital Bleeding DisorderPhase 1pathfinder™1NCT01205724
Safety and Pharmacokinetics of NNC 0129-0000-1003 in Subjects With Haemophilia A
A Phase 1 study testing turoctocog alfa pegol in people with Congenital Bleeding Disorder and Haemophilia A, run by Novo Nordisk A/S at 18 sites in 7 countries.
Trial summary
This trial is conducted globally. 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 NNC 0129-0000-1003 in previously treated subjects with severe haemophilia A. 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 A
- What is being tested?
- turoctocog alfa pegol
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 27 participants
- Where
- 18 sitesin 7 countries
- Study dates
- Sep 2010to Apr 2011
- 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
- Haemophilia A
- Body Mass Index (BMI) below 35 kg/m2
- History of a minimum 150 exposure days (EDs) to FVIII products (prophylaxis/prevention/surgery/on-demand)
Who cannot join
- Any history of FVIII inhibitors
- Surgery planned to occur during the trial
- Platelet count less than 50,000 platelets/microlitre (assessed by laboratory)
- Congenital or acquired coagulation disorders other than haemophilia A
Trial locations and contacts
18 sites in 7 countries.
18 sites in 7 countries
United States5 sites
Novo Nordisk Investigational Site
Baltimore, Maryland, 21287
Novo Nordisk Investigational Site
Houston, Texas, 77030
Novo Nordisk Investigational Site
Iowa City, Iowa, 52242
Novo Nordisk Investigational Site
Lexington, Kentucky, 40536
Novo Nordisk Investigational Site
Philadelphia, Pennsylvania, 19104
Germany3 sites
Novo Nordisk Investigational Site
Frankfurt/M., 60590
Novo Nordisk Investigational Site
Giessen, 35392
Novo Nordisk Investigational Site
Hanover, 30625
Japan3 sites
Novo Nordisk Investigational Site
Kashihara-shi, Nara, 634 8522
Novo Nordisk Investigational Site
Nagoya-shi, Aichi, 466 8560
Novo Nordisk Investigational Site
Shinjuku-ku, Tokyo, 160 0023
United Kingdom3 sites
Novo Nordisk Investigational Site
London, NW3 2QG
Novo Nordisk Investigational Site
London, SE1 7EH
Novo Nordisk Investigational Site
Manchester, M13 9WL
Italy2 sites
Novo Nordisk Investigational Site
Florence, 50134
Novo Nordisk Investigational Site
Vicenza, 36100
Turkey (Türkiye)1 site
Novo Nordisk Investigational Site
Bornova-IZMIR, 35100
About this study
Official title: A Multi-National, Open-Label, Dose Escalation Trial, Evaluating Safety and Pharmacokinetics of Intravenous Doses of NNC 0129-0000-1003 in Patients With Haemophilia A
This trial is conducted globally. 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 NNC 0129-0000-1003 in previously treated subjects with severe haemophilia A.
- Conditions
- Congenital Bleeding Disorder, Haemophilia A
- Being tested
- turoctocog alfa pegol
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
turoctocog alfa pegolDrug
Also called NNC 0129-0000-1003
Single dose (low) administered intravenously (into the vein)
turoctocog alfa pegolDrug
Also called NNC 0129-0000-1003
Single dose (medium) administered intravenously (into the vein)
turoctocog alfa pegolDrug
Also called NNC 0129-0000-1003
Single dose (high) administered intravenously (into the vein)
| Study group | Role | What they receive |
|---|---|---|
| A | Experimental | Not described Receives: Drug: turoctocog alfa pegol |
| B | Experimental | Not described Receives: Drug: turoctocog alfa pegol |
| C | Experimental | Not described Receives: Drug: turoctocog alfa pegol |
What is being measured
The primary outcome is the main question the study is designed to answer.
Frequency of adverse events (AEs) reported after administration of trial product
Measured: up to four weeks after trial product administration
Secondary outcomes (2)
Area under the curve (AUC), total clearance (CL), terminal half-life (T½), incremental recovery (first sample)
Measured: from 0 to 168 hours after trial product administration
Safety assessment including physical examination, vital signs, electrocardiogram (ECG) and clinical laboratory assessments
Measured: up to four 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 posted20 Sep 2010 (estimated)
- Study startSep 2010
- Primary completionApr 2011
- Study completionApr 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)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
- 27 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
- NCT01205724
- Sponsor's protocol ID
- NN7088-3776
- Other IDs
- 2010-018520-68, U1111-1116-2043, JapicCTI-101293
- Acronym
- pathfinder™1
- Status last verified
- Feb 2017
- First submitted
- 17 Sep 2010
- Last updated
- 10 Feb 2017
- Results posted
- No
- Data monitoring committee
- No
Publications
Tiede A, Brand B, Fischer R, Kavakli K, Lentz SR, Matsushita T, Rea C, Knobe K, Viuff D. Enhancing the pharmacokinetic properties of recombinant factor VIII: first-in-human trial of glycoPEGylated recombinant factor VIII in patients with hemophilia A. J Thromb Haemost. 2013 Apr;11(4):670-8. doi: 10.1111/jth.12161.
PubMed 23398640
Study at a glance
From the registry record, last updated by the sponsor on 10 Feb 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-National, Open-Label, Dose Escalation Trial, Evaluating Safety and Pharmacokinetics of Intravenous Doses of NNC 0129-0000-1003 in Patients With Haemophilia A
- ClinicalTrials.gov ID
- NCT01205724
- Acronym / sponsor ID
- pathfinder™1 / NN7088-3776
- Conditions
- Congenital Bleeding Disorder, Haemophilia A
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 27 participants (actual)
- Lead sponsor
- Novo Nordisk A/SIndustry (pharma, biotech, devices)
- Study start
- Sep 2010
- Primary completion
- Apr 2011 (actual)
- Study completion
- Apr 2011 (actual)
- First posted
- 20 Sep 2010 (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 globally. 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 NNC 0129-0000-1003 in previously treated subjects with severe haemophilia A.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| A | Experimental | Drug: turoctocog alfa pegol |
| B | Experimental | Drug: turoctocog alfa pegol |
| C | Experimental | Drug: turoctocog alfa 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)
- Haemophilia A
- Body Mass Index (BMI) below 35 kg/m2
- History of a minimum 150 exposure days (EDs) to FVIII products (prophylaxis/prevention/surgery/on-demand)
Key exclusion criteria (4 listed)
- Any history of FVIII inhibitors
- Surgery planned to occur during the trial
- Platelet count less than 50,000 platelets/microlitre (assessed by laboratory)
- Congenital or acquired coagulation disorders other than haemophilia A
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 |
|---|---|---|
| turoctocog alfa pegolAlso: NNC 0129-0000-1003Single dose (low) administered intravenously (into the vein) | Drug | A |
| turoctocog alfa pegolAlso: NNC 0129-0000-1003Single dose (medium) administered intravenously (into the vein) | Drug | B |
| turoctocog alfa pegolAlso: NNC 0129-0000-1003Single dose (high) administered intravenously (into the vein) | Drug | C |
Primary outcome
Frequency of adverse events (AEs) reported after administration of trial product
Time frame: up to four weeks after trial product administration
2 secondary outcomes
Area under the curve (AUC), total clearance (CL), terminal half-life (T½), incremental recovery (first sample)
Time frame: from 0 to 168 hours after trial product administration
Safety assessment including physical examination, vital signs, electrocardiogram (ECG) and clinical laboratory assessments
Time frame: up to four 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 10 Feb 2017.
- Results publication: PubMed 23398640Tiede A, Brand B, Fischer R, Kavakli K, Lentz SR, Matsushita T, Rea C, Knobe K, Viuff D. Enhancing the pharmacokinetic properties of recombinant factor VIII: first-in-human trial of glycoPEGylated recombinant factor VIII in patients with hemophilia A. J Thromb Haemost. 2013 Apr;11(4):670-8. doi: 10.1111/jth.12161.
- 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
18 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing turoctocog alfa pegol in people with Congenital Bleeding Disorder and Haemophilia A, run by Novo Nordisk A/S at 18 sites in 7 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 10 Feb 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 18 sites in United States, Germany, Japan, United Kingdom, Italy and 2 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) reported after administration of trial product (time frame: up to four weeks after trial product administration). 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 NCT01205724, which also lists 1 linked publication.
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 10 Feb 2017.