Congenital Bleeding DisorderPhase 3adept™2NCT01392547
Efficacy and Safety of NNC 0078-0000-0007 in Patients With Congenital Haemophilia and Inhibitors
A Phase 3 study testing vatreptacog alfa (activated) and eptacog alfa (activated) in people with Congenital Bleeding Disorder and Haemophilia A With Inhibitors, run by Novo Nordisk A/S at 32 sites in 19 countries.
Trial summary
This trial is conducted globally. The purpose of this trial is to confirm the efficacy and safety of NNC 0078-0000-0007 in patients with congenital haemophilia and inhibitors. Read more about this study
- Who is it for?
- Ages 12 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?
- vatreptacog alfa (activated) and eptacog alfa (activated)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 72 participants
- Where
- 32 sitesin 19 countries
- Study dates
- Jul 2011to Aug 2012
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male patient with clinical diagnosis of congenital haemophilia A or B and inhibitors to coagulation factors VIII or IX
- Minimum of five bleeds requiring haemostatic drug treatment within the previous 12 months at trial entry
Who cannot join
- Previous participation in this trial defined as withdrawal after administration of trial product
- Patient has received an investigational medicinal product within 30 days prior to this trial
- Congenital or acquired coagulation disorders other than haemophilia A or B
- Any clinical signs or known history of arterial thrombotic events or of deep venous thrombosis or pulmonary embolism (as defined by available medical records)
- Platelet count of less than 50,000 platelets/mcL (at the screening visit)
- ALAT (alanine-transaminase) of more than 3 times the upper normal limit (according to laboratory reference ranges)
Show all 9Show fewer
- Factor VIII/IX Immune Tolerance Induction regimen planned to occur during the trial
- Ongoing bleeding prophylaxis regimens or planned bleeding prophylaxis to occur during the trial
- HIV (Human Immunodeficiency Virus) positive with current CD4+ count of less than 200/mcL (defined by medical records)
Trial locations and contacts
32 sites in 19 countries.
32 sites in 19 countries
United States14 sites
Novo Nordisk Clinical Trial Call Center
Atlanta, Georgia, 30322
Novo Nordisk Clinical Trial Call Center
Augusta, Georgia, 30912
Novo Nordisk Clinical Trial Call Center
Aurora, Colorado, 80045
Novo Nordisk Clinical Trial Call Center
Boston, Massachusetts, 02115
Novo Nordisk Clinical Trial Call Center
Brooklyn, New York, 11219
Novo Nordisk Clinical Trial Call Center
Detroit, Michigan, 48202-2608
Novo Nordisk Clinical Trial Call Center
Iowa City, Iowa, 52242
Novo Nordisk Clinical Trial Call Center
Los Angeles, California, 90007
Novo Nordisk Clinical Trial Call Center
Los Angeles, California, 90027
Novo Nordisk Clinical Trial Call Center
Orange, California, 92868
Novo Nordisk Clinical Trial Call Center
Portland, Oregon, 97239
Novo Nordisk Clinical Trial Call Center
Richmond, Virginia, 23219
Novo Nordisk Clinical Trial Call Center
Tampa, Florida, 33607
Novo Nordisk Clinical Trial Call Center
Tucson, Arizona, 85724-0001
About this study
Official title: Efficacy and Safety of NNC 0078-0000-0007 in Treatment of Acute Bleeding Episodes in Patients With Congenital Haemophilia and Inhibitors
This trial is conducted globally. The purpose of this trial is to confirm the efficacy and safety of NNC 0078-0000-0007 in patients with congenital haemophilia and inhibitors.
Read the detailed description
Scheduled dose visit in a non-bleeding state. Single dose of NNC 0078-0000-0007 (vatreptocog alfa (activated)) every 3 months.
- Conditions
- Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
- Being tested
- vatreptacog alfa (activated), eptacog alfa (activated)
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
vatreptacog alfa (activated)Drug
1-3 doses per bleeding episode
eptacog alfa (activated)Drug
1-3 doses per bleeding episode
| Study group | Role | What they receive |
|---|---|---|
| rFVIIa | Experimental | Not described Receives: Drug: eptacog alfa (activated) |
| vatreptocog alfa | Experimental | Not described Receives: Drug: vatreptacog alfa (activated) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Effective Bleeding Control Defined as no Additional Haemostatic Medication (Other Than Trial Product) Given
Measured: Within 12 hours of first trial product administration
Secondary outcomes (4)
Effective and Sustained Bleeding Control
Measured: Up to 48 hours after first trial product administration
Number of Doses of Trial Product Given for Each Acute Bleed
Measured: Up to 6 hours after first trial product administration
Number of Adverse Events
Any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment.
Measured: Adverse events were captured from the time of consent to 1 month (+14 days) after last administration of trial product.
Immunogenicity (Inhibitor Development)
Immunogenicity was tested by formation of neutralising antibodies towards vatreptacog alfa and/or FVII. Radioimmunoassay using [125I]-labelled vatreptacog alfa or rFVIIa was used to screen plasma samples for development of anti-drug antibodies
Measured: Adverse events were captured from the time of consent to the end of trial visit 1 month (+14 days) after last administration of trial product.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jul 2011 (estimated)
- Study startJul 2011
- Primary completionAug 2012
- Study completionAug 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 72 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
- NCT01392547
- Sponsor's protocol ID
- NN1731-3562
- Other IDs
- 2010-023803-92, U1111-1118-2228, JapicCTI-111595
- Acronym
- adept™2
- Status last verified
- Feb 2015
- First submitted
- 8 Jul 2011
- Last updated
- 15 May 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 May 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of NNC 0078-0000-0007 in Treatment of Acute Bleeding Episodes in Patients With Congenital Haemophilia and Inhibitors
- ClinicalTrials.gov ID
- NCT01392547
- Acronym / sponsor ID
- adept™2 / NN1731-3562
- Conditions
- Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 72 participants (actual)
- Lead sponsor
- Novo Nordisk A/SIndustry (pharma, biotech, devices)
- Study start
- Jul 2011
- Primary completion
- Aug 2012 (actual)
- Study completion
- Aug 2012 (actual)
- First posted
- 12 Jul 2011 (estimated)
- Results
- Posted 6 Dec 2013
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 purpose of this trial is to confirm the efficacy and safety of NNC 0078-0000-0007 in patients with congenital haemophilia and inhibitors.
Detailed description
Scheduled dose visit in a non-bleeding state. Single dose of NNC 0078-0000-0007 (vatreptocog alfa (activated)) every 3 months.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| rFVIIa | Experimental | Drug: eptacog alfa (activated) |
| vatreptocog alfa | Experimental | Drug: vatreptacog alfa (activated) |
Who may qualify
- Ages
- 12 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Male patient with clinical diagnosis of congenital haemophilia A or B and inhibitors to coagulation factors VIII or IX
- Minimum of five bleeds requiring haemostatic drug treatment within the previous 12 months at trial entry
Key exclusion criteria (9 listed)
- Previous participation in this trial defined as withdrawal after administration of trial product
- Patient has received an investigational medicinal product within 30 days prior to this trial
- Congenital or acquired coagulation disorders other than haemophilia A or B
- Any clinical signs or known history of arterial thrombotic events or of deep venous thrombosis or pulmonary embolism (as defined by available medical records)
- Platelet count of less than 50,000 platelets/mcL (at the screening visit)
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 |
|---|---|---|
| vatreptacog alfa (activated)1-3 doses per bleeding episode | Drug | vatreptocog alfa |
| eptacog alfa (activated)1-3 doses per bleeding episode | Drug | rFVIIa |
Primary outcome
Effective Bleeding Control Defined as no Additional Haemostatic Medication (Other Than Trial Product) Given
Time frame: Within 12 hours of first trial product administration
4 secondary outcomes
Effective and Sustained Bleeding Control
Time frame: Up to 48 hours after first trial product administration
Number of Doses of Trial Product Given for Each Acute Bleed
Time frame: Up to 6 hours after first trial product administration
Number of Adverse Events
Time frame: Adverse events were captured from the time of consent to 1 month (+14 days) after last administration of trial product.
Immunogenicity (Inhibitor Development)
Time frame: Adverse events were captured from the time of consent to the end of trial visit 1 month (+14 days) after last administration of trial product.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 May 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- 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
32 sites in 19 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing vatreptacog alfa (activated) and eptacog alfa (activated) in people with Congenital Bleeding Disorder and Haemophilia A With Inhibitors, run by Novo Nordisk A/S at 32 sites in 19 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 2 inclusion and 9 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 15 May 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 32 sites in United States, Austria, Brazil, Croatia, Greece and 14 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 6 Dec 2013). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Effective Bleeding Control Defined as no Additional Haemostatic Medication (Other Than Trial Product) Given (time frame: Within 12 hours of first trial product administration). 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 15 May 2017.