Congenital Bleeding DisorderPhase 2NCT00951405
Safety and Efficacy of 3 Different Doses of Long Acting Factor VII in Haemophilia A or B Patients With Inhibitors
A Phase 2 study testing activated recombinant human factor VII, long acting in people with Congenital Bleeding Disorder and Haemophilia A With Inhibitors, run by Novo Nordisk A/S at 25 sites in 11 countries.
Trial summary
This trial is conducted in Asia, Europe, Japan and North America. The aim of this clinical trial is to investigate the safety and the efficacy of a prophylactic treatment option with long acting coagulation factor VII (LA-rFVIIa) for haemophilia patients with inhibitors. Read more about this study
- Who is it for?
- Ages 12 to 65 years, men only
- Conditions the study is for
- Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
- What is being tested?
- activated recombinant human factor VII, long acting
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 23 participants
- Where
- 25 sitesin 11 countries
- Study dates
- 1 Sep 2009to 29 Mar 2011
- Phase
- Phase 2
- 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 12 to 65 years
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male haemophilia A or B patients with inhibitors
- Willing to undergo a bleeding preventive regimen of 3 months' duration and a total trial length of approximately 8 months
- Historical or ongoing high titre inhibitor (more than or equal to 5 BU) based on either medical records, laboratory report reviews, patient and/or care provider interviews
- At least 2 bleeding episodes requiring bypassing haemostatic-drug-based treatment within the last month or 12 bleeding episodes within the last 6 months prior to observation period
- Body weight between 30 and 100 kg (both inclusive)
Who cannot join
- Body Mass Index (BMI) above 30 kg/m2
- Immune tolerance induction therapy within the last month prior to entering observation phase period
- Known active pseudo tumours
- Platelet count less than 50,000 platelets/microL (based on local laboratory value at screening visit)
- Congenital or acquired coagulation disorders other than haemophilia A or B
- Surgery within one month prior to the observation period. Catheter, stents and dental extractions do not count as surgeries, i.e. they will not exclude the patient. Port insertion is classified as surgery
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- Scheduled major and/or orthopaedic surgery, during the trial period until Follow up visit. Catheter, stents and dental extractions do not count as surgeries and will not exclude the patient. Port insertion is classified as surgery
- Advanced atherosclerotic disease (i.e. known history of ischemic heart disease, or ischemic stroke)
- Any clinical signs or known history of thromboembolic events incl. known deep vein thrombosis (DVT)
- Known or clinically suspected allergy to activated recombinant human factor VII (NovoSeven®/NovoSeven RT®/Niastase®)
- Prothrombin Time (PT) prolongation (30% above normal limits, or more than 5 seconds compared to control or International Normalised Range (INR) more than 1.7 as defined by local laboratory ranges at screening visit
- Severe liver disease (ALAT more than 4 times of the upper limit of normal reference range) (as defined by local laboratory ranges) within a year of enrolment or at the screening
- Clinical signs of renal dysfunction (dialysis) and/or creatinine levels more than or equal to 20% above upper normal limit (according to local laboratory range at the screening visit)
- Dosing of any investigational drug within the last 30 days prior to the present trial
- Any disease or condition which, according to the investigator's judgement, could imply a potential hazard to the subject, interfere with the trial participation or trial outcome
- HIV positive patients who either have low CD4+ lymphocyte count ( 200/microL or less based on medical records within 6 months or laboratory screening at screening visit), or who are HCV-PCR positive (based on medical records), or who both have low CD4+ lymphocyte count (200/microL or less) and are HCV-PCR positive. If HCV-PCR testing is not locally available, a HIV positive patient who is HCV antibody positive cannot be included
- Need to use other PEGylated pharmaceutical drug during the trial period
Trial locations and contacts
25 sites in 11 countries.
25 sites in 11 countries
United States7 sites
Novo Nordisk Investigational Site
Boston, Massachusetts, 02115
Novo Nordisk Investigational Site
Hershey, Pennsylvania, 17033
Novo Nordisk Investigational Site
Little Rock, Arkansas, 72202
Novo Nordisk Investigational Site
Los Angeles, California, 90027
Novo Nordisk Investigational Site
Orange, California, 92868
Novo Nordisk Investigational Site
Portland, Oregon, 97239
Novo Nordisk Investigational Site
Tampa, Florida, 33607
Japan4 sites
Novo Nordisk Investigational Site
Kashihara-shi, Nara, 634 8522
Novo Nordisk Investigational Site
Kitakyusyu, Fukuoka, 807 8555
Novo Nordisk Investigational Site
Nishinomiya-shi, 663 8051
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
Oxford, OX3 7LJ
France2 sites
Novo Nordisk Investigational Site
Le Kremlin-Bicêtre, 94270
Novo Nordisk Investigational Site
Paris, 75015
South Africa2 sites
Novo Nordisk Investigational Site
Durban, KwaZulu-Natal, 4013
Novo Nordisk Investigational Site
Parktown Johannesburg, Gauteng, 2193
Turkey (Türkiye)2 sites
Novo Nordisk Investigational Site
Ankara, 06500
Novo Nordisk Investigational Site
Istanbul, 34098
Serbia and Montenegro1 site
Novo Nordisk Investigational Site
Belgrade, 11000
About this study
Official title: An Exploratory Multi-Centre, Multi-National, Randomised, Double Blinded, Parallel Arm Trial Evaluating Safety, Pharmacokinetics and Dose-finding of Prophylactic Administration of Long Acting rFVIIa (LA-rFVIIa) in Haemophilia A or B Patients With Inhibitors
This trial is conducted in Asia, Europe, Japan and North America. The aim of this clinical trial is to investigate the safety and the efficacy of a prophylactic treatment option with long acting coagulation factor VII (LA-rFVIIa) for haemophilia patients with inhibitors.
- Conditions
- Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
- Being tested
- activated recombinant human factor VII, long acting
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
activated recombinant human factor VII, long actingDrug
Also called NN7128, LA-rFVIIa
After an observation period of 3 months, every 2nd day intravenous (i.v.) injection with 25 microgrammes/kg activated recombinant human factor VII, long acting, for 3 months
activated recombinant human factor VII, long actingDrug
Also called NN7128, LA-rFVIIa
After an observation period of 3 months, every 2nd day intravenous (i.v.) injection with 100 microgrammes/kg activated recombinant human factor VII, long acting, for 3 months
activated recombinant human factor VII, long actingDrug
Also called NN7128, LA-rFVIIa
After an observation period of 3 months, every 2nd day intravenous (i.v.) injection with 200 microgrammes/kg activated recombinant human factor VII, long acting, for 3 months
| Study group | Role | What they receive |
|---|---|---|
| A | Experimental | Not described Receives: Drug: activated recombinant human factor VII, long acting |
| B | Experimental | Not described Receives: Drug: activated recombinant human factor VII, long acting |
| C | Experimental | Not described Receives: Drug: activated recombinant human factor VII, long acting |
What is being measured
The primary outcome is the main question the study is designed to answer.
Thrombogenecity
Measured: at all scheduled visits (1 - 9)
Immunogenecity: Neutralising Antibody Development
Measured: at all scheduled visits (1 - 9)
Secondary outcomes (2)
AUC(0-48h) and AUC: Area under the FVIIa activity-time profile in the given time period, which is a measure of total blood exposure
Measured: at visit 2 and visit 7 until 48 hours after trial product administration
Annualized bleeding rates
Measured: During observation period; from visit 1 until visit 2 and treatment period; from visit 2 until visit 7. In total a period of 6 to 8 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Aug 2009 (estimated)
- Study start1 Sep 2009
- Primary completion29 Mar 2011
- Study completion29 Mar 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 2
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 23 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
- NCT00951405
- Sponsor's protocol ID
- NN7128-1907
- Other IDs
- 2008-006424-54, JapicCTI-090860, U1111-1111-8584, 2008
- Status last verified
- Sep 2018
- First submitted
- 3 Aug 2009
- Last updated
- 5 Sep 2018
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Ljung R, Karim FA, Saxena K, Suzuki T, Arkhammar P, Rosholm A, Giangrande P; Pioneer1 Investigators. 40K glycoPEGylated, recombinant FVIIa: 3-month, double-blind, randomized trial of safety, pharmacokinetics and preliminary efficacy in hemophilia patients with inhibitors. J Thromb Haemost. 2013 Jul;11(7):1260-8. doi: 10.1111/jth.12237.
PubMed 23578227
Study at a glance
From the registry record, last updated by the sponsor on 5 Sep 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Exploratory Multi-Centre, Multi-National, Randomised, Double Blinded, Parallel Arm Trial Evaluating Safety, Pharmacokinetics and Dose-finding of Prophylactic Administration of Long Acting rFVIIa (LA-rFVIIa) in Haemophilia A or B Patients With Inhibitors
- ClinicalTrials.gov ID
- NCT00951405
- Acronym / sponsor ID
- NN7128-1907
- Conditions
- Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 23 participants (actual)
- Lead sponsor
- Novo Nordisk A/SIndustry (pharma, biotech, devices)
- Study start
- 1 Sep 2009 (actual)
- Primary completion
- 29 Mar 2011 (actual)
- Study completion
- 29 Mar 2011 (actual)
- First posted
- 4 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 Asia, Europe, Japan and North America. The aim of this clinical trial is to investigate the safety and the efficacy of a prophylactic treatment option with long acting coagulation factor VII (LA-rFVIIa) for haemophilia patients with inhibitors.
Study design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| A | Experimental | Drug: activated recombinant human factor VII, long acting |
| B | Experimental | Drug: activated recombinant human factor VII, long acting |
| C | Experimental | Drug: activated recombinant human factor VII, long acting |
Who may qualify
- Ages
- 12 to 65 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Male haemophilia A or B patients with inhibitors
- Willing to undergo a bleeding preventive regimen of 3 months' duration and a total trial length of approximately 8 months
- Historical or ongoing high titre inhibitor (more than or equal to 5 BU) based on either medical records, laboratory report reviews, patient and/or care provider interviews
- At least 2 bleeding episodes requiring bypassing haemostatic-drug-based treatment within the last month or 12 bleeding episodes within the last 6 months prior to observation period
- Body weight between 30 and 100 kg (both inclusive)
Key exclusion criteria (17 listed)
- Body Mass Index (BMI) above 30 kg/m2
- Immune tolerance induction therapy within the last month prior to entering observation phase period
- Known active pseudo tumours
- Platelet count less than 50,000 platelets/microL (based on local laboratory value at screening visit)
- Congenital or acquired coagulation disorders other than haemophilia A or B
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 |
|---|---|---|
| activated recombinant human factor VII, long actingAlso: NN7128, LA-rFVIIaAfter an observation period of 3 months, every 2nd day intravenous (i.v.) injection with 25 microgrammes/kg activated recombinant human factor VII, long acting, for 3 months | Drug | A |
| activated recombinant human factor VII, long actingAlso: NN7128, LA-rFVIIaAfter an observation period of 3 months, every 2nd day intravenous (i.v.) injection with 100 microgrammes/kg activated recombinant human factor VII, long acting, for 3 months | Drug | B |
| activated recombinant human factor VII, long actingAlso: NN7128, LA-rFVIIaAfter an observation period of 3 months, every 2nd day intravenous (i.v.) injection with 200 microgrammes/kg activated recombinant human factor VII, long acting, for 3 months | Drug | C |
Primary outcome
Thrombogenecity
Time frame: at all scheduled visits (1 - 9)
Immunogenecity: Neutralising Antibody Development
Time frame: at all scheduled visits (1 - 9)
2 secondary outcomes
AUC(0-48h) and AUC: Area under the FVIIa activity-time profile in the given time period, which is a measure of total blood exposure
Time frame: at visit 2 and visit 7 until 48 hours after trial product administration
Annualized bleeding rates
Time frame: During observation period; from visit 1 until visit 2 and treatment period; from visit 2 until visit 7. In total a period of 6 to 8 months
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 5 Sep 2018.
- Derived from this study: PubMed 23578227Ljung R, Karim FA, Saxena K, Suzuki T, Arkhammar P, Rosholm A, Giangrande P; Pioneer1 Investigators. 40K glycoPEGylated, recombinant FVIIa: 3-month, double-blind, randomized trial of safety, pharmacokinetics and preliminary efficacy in hemophilia patients with inhibitors. J Thromb Haemost. 2013 Jul;11(7):1260-8. doi: 10.1111/jth.12237.
- 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
25 sites in 11 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing activated recombinant human factor VII, long acting in people with Congenital Bleeding Disorder and Haemophilia A With Inhibitors, run by Novo Nordisk A/S at 25 sites in 11 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 5 inclusion and 17 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 5 Sep 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 25 sites in United States, Japan, United Kingdom, France, South Africa and 6 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
Thrombogenecity (time frame: at all scheduled visits (1 - 9)). 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 NCT00951405, 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 5 Sep 2018.