Congenital Bleeding DisorderPhase 1NCT01561924
Prediction of the Efficacy of Activated Recombinant Human Factor VII in Adult Congenital Haemophilia A or B Patients With Inhibitors by Use of Thromboelastography
A Phase 1 study testing eptacog alfa (activated) in people with Congenital Bleeding Disorder and Haemophilia A With Inhibitors, run by Novo Nordisk A/S at 7 sites in 4 countries.
Trial summary
This trial is conducted in Europe and the United States of America (USA). The aim of this trial is to evaluate the basal and spiked TEG® (Thromboelastography) or ROTEM® (Thromboelastometry) profiles of frequently bleeding haemophilia subjects with inhibitors in a non-bleeding state. Read more about this study
- Who is it for?
- Ages 16 years and older, men only
- Conditions the study is for
- Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
- What is being tested?
- eptacog alfa (activated)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 17 participants
- Where
- 7 sitesin 4 countries
- Study dates
- Nov 2005to May 2006
- 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 16 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Confirmed diagnosis of congenital haemophilia A or B with a FVIII:C clot activity or FIX:C clot activity, respectively, less than 5%
- Presently use of activated recombinant human factor VII (NovoSeven®) as haemostatic agent for preventive treatment or treatment of bleeding episodes
- A documented historical or present record for the presence of inhibitors to factor VIII or IX, respectively
- A documented history of 2 or more joint bleeding episodes during the preceding 12 months
- Subjects must be in a non-bleeding state (i.e. no clinical manifestations of active bleeds)
Who cannot join
- Subjects who have received any haemostatic treatment for a bleeding episode within the last 7 days prior to this trial
- Immune tolerance therapy within the last 30 days prior to this trial
- Clinically relevant coagulation disorders other than congenital haemophilia A or B
- Thrombocytopenia (platelet count below 60,000 platelets/mcl)
- Prophylactic haemostatic treatment within 3 days prior to this trial
Trial locations and contacts
7 sites in 4 countries.
7 sites in 4 countries
United States4 sites
Novo Nordisk Investigational Site
Berkeley, California, 94704
Novo Nordisk Investigational Site
Iowa City, Iowa, 52242
Novo Nordisk Investigational Site
Los Angeles, California, 90027
Novo Nordisk Investigational Site
Philadelphia, Pennsylvania, 19104
About this study
Official title: An Exploratory Non-interventional Study of Ex-vivo Spiking Followed by Thromboelastography as a Method for Predicting the Efficacy of Recombinant Activated Human FVII (rFVIIa, NovoSeven®) in Adult Congenital Haemophilia A or B Patients With Inhibitors
This trial is conducted in Europe and the United States of America (USA). The aim of this trial is to evaluate the basal and spiked TEG® (Thromboelastography) or ROTEM® (Thromboelastometry) profiles of frequently bleeding haemophilia subjects with inhibitors in a non-bleeding state.
Read the detailed description
The TEG parameters are: R time (Reaction Time), K time (K Time (arbitrary measurement)), a (a angle), MA (Maximum Amplitude) and LY30 (Lysis 30 min after MA) while the ROTEM parameters are: CT (Clotting Time), CFT (Clot Formation Time), a (a angle), MCF (Maximum Clot Firmness) and LI60 (Lysis index 60 min after CT)
- Conditions
- Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
- Being tested
- eptacog alfa (activated)
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
eptacog alfa (activated)Drug
Also called activated recombinant human factor VII
Subjects will be called to attend the clinic in a non-bleeding state. Blood samples will be drawn for thromboelastogram profiling and will be in-vitro spiked with different doses of activatated recombinant human factor VII.
| Study group | Role | What they receive |
|---|---|---|
| Ex vivo | Experimental | Not described Receives: Drug: eptacog alfa (activated) |
What is being measured
The primary outcome is the main question the study is designed to answer.
TEG parameters obtained at baseline and with activated recombinant human factor VII
ROTEM parameters obtained at baseline and with activated recombinant human factor VII
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Mar 2012 (estimated)
- Study startNov 2005
- Primary completionMay 2006
- Study completionMay 2006
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- Diagnosis
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 17 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
- NCT01561924
- Sponsor's protocol ID
- F7HAEM-1675
- Status last verified
- Nov 2016
- First submitted
- 21 Mar 2012
- Last updated
- 23 Nov 2016
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Nov 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Exploratory Non-interventional Study of Ex-vivo Spiking Followed by Thromboelastography as a Method for Predicting the Efficacy of Recombinant Activated Human FVII (rFVIIa, NovoSeven®) in Adult Congenital Haemophilia A or B Patients With Inhibitors
- ClinicalTrials.gov ID
- NCT01561924
- Acronym / sponsor ID
- F7HAEM-1675
- Conditions
- Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 17 participants (actual)
- Lead sponsor
- Novo Nordisk A/SIndustry (pharma, biotech, devices)
- Study start
- Nov 2005
- Primary completion
- May 2006 (actual)
- Study completion
- May 2006 (actual)
- First posted
- 23 Mar 2012 (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 and the United States of America (USA). The aim of this trial is to evaluate the basal and spiked TEG® (Thromboelastography) or ROTEM® (Thromboelastometry) profiles of frequently bleeding haemophilia subjects with inhibitors in a non-bleeding state.
Detailed description
The TEG parameters are: R time (Reaction Time), K time (K Time (arbitrary measurement)), a (a angle), MA (Maximum Amplitude) and LY30 (Lysis 30 min after MA) while the ROTEM parameters are: CT (Clotting Time), CFT (Clot Formation Time), a (a angle), MCF (Maximum Clot Firmness) and LI60 (Lysis index 60 min after CT)
Study design
- Primary purpose
- Diagnosis
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Ex vivo | Experimental | Drug: eptacog alfa (activated) |
Who may qualify
- Ages
- 16 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Confirmed diagnosis of congenital haemophilia A or B with a FVIII:C clot activity or FIX:C clot activity, respectively, less than 5%
- Presently use of activated recombinant human factor VII (NovoSeven®) as haemostatic agent for preventive treatment or treatment of bleeding episodes
- A documented historical or present record for the presence of inhibitors to factor VIII or IX, respectively
- A documented history of 2 or more joint bleeding episodes during the preceding 12 months
- Subjects must be in a non-bleeding state (i.e. no clinical manifestations of active bleeds)
Key exclusion criteria (5 listed)
- Subjects who have received any haemostatic treatment for a bleeding episode within the last 7 days prior to this trial
- Immune tolerance therapy within the last 30 days prior to this trial
- Clinically relevant coagulation disorders other than congenital haemophilia A or B
- Thrombocytopenia (platelet count below 60,000 platelets/mcl)
- Prophylactic haemostatic treatment within 3 days prior to this trial
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 |
|---|---|---|
| eptacog alfa (activated)Also: activated recombinant human factor VIISubjects will be called to attend the clinic in a non-bleeding state. Blood samples will be drawn for thromboelastogram profiling and will be in-vitro spiked with different doses of activatated recombinant human factor VII. | Drug | Ex vivo |
Primary outcome
TEG parameters obtained at baseline and with activated recombinant human factor VII
ROTEM parameters obtained at baseline and with activated recombinant human factor VII
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 23 Nov 2016.
- 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
7 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing eptacog alfa (activated) in people with Congenital Bleeding Disorder and Haemophilia A With Inhibitors, run by Novo Nordisk A/S at 7 sites in 4 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 5 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 23 Nov 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United States, Denmark, France, United Kingdom. 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?
TEG parameters obtained at baseline and with activated recombinant human factor VII. 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?
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 23 Nov 2016.