Congenital Coagulation Factor VII DeficiencyPhase 1NCT02470871
Study of the Pharmacokinetics and Safety of Recombinant Factor VIIa Fusion Protein (rVIIa-FP, CSL689) in Patients With Congenital Factor VII Deficiency
A Phase 1 study testing Eptacog alfa (activated) or pdFVII and CSL689 in people with Congenital Coagulation Factor VII Deficiency, run by CSL Behring at 2 sites in 2 countries.
Trial summary
The purpose of this study is to investigate the pharmacokinetics (PK) and safety of rVIIa-FP (CSL689) in a total of 10 to 16 male or female adults with inherited coagulation factor VII (FVII) deficiency. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Congenital Coagulation Factor VII Deficiency
- What is being tested?
- Eptacog alfa (activated) or pdFVII and CSL689
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 2 sitesin 2 countries
- Study dates
- Jul 2015to Oct 2016
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Proven congenital FVII deficiency.
- Age ≥ 18 years.
- FVII level < 2% of normal levels.
- Minimum of 50 previous exposure days to pdFVII (including prothrombin complex concentrates [PCCs]) or rFVIIa.
Who cannot join
- History of, or risk factors for, thromboembolic events, including known deep vein thrombosis.
- Inhibitor to FVII or rFVIIa, current or historic.
- Known or suspected hypersensitivity to hamster protein, to CSL689, or to any excipient of CSL689.
- Known or suspected allergy to rFVIIa or hamster protein.
- Major surgery within 1 month before screening.
- Advanced atherosclerotic disease (ie, known history of ischemic heart disease, or ischemic stroke).
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- Human immunodeficiency virus (HIV)-positive subjects with cluster of differentiation 4 (CD4)+ lymphocyte count of < 200/µL at screening.
- Use of an investigational agent within 30 days before the study.
- Use of concomitant therapy not permitted during the study (ie, other platelet inhibitors, desmopressin, fibrinolysis inhibitors, except if used as local treatment [eg, for oral bleeds])
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
About this study
Official title: Multi-center, Randomized, Open-label, Parallel-Arm, Single-dose, Pharmacokinetic Study of rVIIa-FP (CSL689) in Subjects With Congenital Factor VII Deficiency
The purpose of this study is to investigate the pharmacokinetics (PK) and safety of rVIIa-FP (CSL689) in a total of 10 to 16 male or female adults with inherited coagulation factor VII (FVII) deficiency. Subjects will receive a single dose of their routine FVII replacement product (ie, either recombinant activated coagulation FVII [rFVIIa, eptacog alfa (activated)] or plasma-derived FVII [pdFVII]) as a comparator, and will then be randomly assigned to a single low dose or a single high dose of the study product CSL689 (8 subjects per CSL689 dose level). Serial blood samples for PK analysis will be taken up to 24 hours after the eptacog alfa (activated) or pdFVII injection, and up to 48 hours after the CSL689 injection. Subject safety will be routinely monitored throughout the study.
- Being tested
- Eptacog alfa (activated) or pdFVII, CSL689
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Eptacog alfa (activated) or pdFVIIBiological
Comparator Drug 1: Recombinant activated FVII (rFVIIa). Subjects with eptacog alfa (activated) as their routine FVII replacement therapy will receive a single dose of eptacog alfa (activated) in the study.
Comparator Drug 2: Plasma-derived FVII (pdFVII). Subjects with pdFVII as their routine FVII replacement therapy will receive a single injection of pdFVII in the study.
CSL689Biological
Experimental Drug: Recombinant fusion protein, linking activated FVII with albumin (rVIIa-FP). Subjects will receive a single dose of CSL689 at either a low dose (Arm 1) or a high dose (Arm 2)
| Study group | Role | What they receive |
|---|---|---|
| Low-dose CSL689 | Experimental | Single dose of subject's routine FVII replacement therapy (either eptacog alfa [activated] [ie, comparator drug 1] or pdFVII [ie, comparator drug 2]), followed by a single dose of CSL689 at the low dose Receives: Biological: Eptacog alfa (activated) or pdFVII; Biological: CSL689 |
| High-dose CSL689 | Experimental | Single dose of subject's routine FVII replacement therapy (either eptacog alfa [activated] [ie, comparator drug 1] or pdFVII [ie, comparator drug 2]), followed by a single dose of CSL689 at the high dose. Receives: Biological: Eptacog alfa (activated) or pdFVII; Biological: CSL689 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Terminal half-life of plasma FVIIa activity
Measured: Up to 48 hours after CSL689 injection
Maximum observed plasma FVIIa activity
Measured: Before injection and at up to 9 time points until 48 hours after injection
Area under the curve (AUC0-t)
Area under plasma FVIIa activity versus time curve from time 0 to last sample with quantifiable activity
Measured: Before injection and at up to 9 time points until 48 hours after injection
Secondary outcomes (7)
Total clearance
Total clearance of plasma FVIIa activity
Measured: Before injection and at up to 9 time points until 48 hours after injection
Volume of distribution of the terminal phase
Measured: Before injection and at up to 9 time points until 48 hours after injection
AUC(0-inf)
Area under plasma FVIIa activity versus time curve from time 0 extrapolated to infinity
Measured: Before injection and at up to 9 time points until 48 hours after injection
Incremental recovery
Incremental recovery of plasma FVIIa activity
Measured: Before injection and at up to 9 time points until 48 hours after injection
Time of occurrence of maximum observed plasma FVIIa activity
Measured: Before injection and at up to 9 time points until 48 hours after injection
Number of subjects with antibodies against Chinese hamster ovary protein and FVII
Measured: Up to 30 days after CSL689 injection
Number of subjects with inhibitors against FVII
Measured: Up to 30 days after CSL689 injection
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jun 2015 (estimated)
- Study startJul 2015
- Primary completionJul 2016
- Study completionOct 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Congenital Coagulation Factor VII DeficiencyStudies of the same condition, open and under way.
- More trials run by CSL BehringThe 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
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- CSL Behring
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Alex Veldman, Study director, CSL Behring
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02470871
- Sponsor's protocol ID
- CSL689_1002
- Other IDs
- 2014-002982-32, 2014
- Status last verified
- Nov 2016
- First submitted
- 10 Jun 2015
- Last updated
- 26 Apr 2017
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Apr 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multi-center, Randomized, Open-label, Parallel-Arm, Single-dose, Pharmacokinetic Study of rVIIa-FP (CSL689) in Subjects With Congenital Factor VII Deficiency
- ClinicalTrials.gov ID
- NCT02470871
- Acronym / sponsor ID
- CSL689_1002
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- CSL BehringIndustry (pharma, biotech, devices)
- Study start
- Jul 2015
- Primary completion
- Jul 2016 (actual)
- Study completion
- Oct 2016 (actual)
- First posted
- 12 Jun 2015 (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.
The purpose of this study is to investigate the pharmacokinetics (PK) and safety of rVIIa-FP (CSL689) in a total of 10 to 16 male or female adults with inherited coagulation factor VII (FVII) deficiency. Subjects will receive a single dose of their routine FVII replacement product (ie, either recombinant activated coagulation FVII [rFVIIa, eptacog alfa (activated)] or plasma-derived FVII [pdFVII]) as a comparator, and will then be randomly assigned to a single low dose or a single high dose of the study product CSL689 (8 subjects per CSL689 dose level). Serial blood samples for PK analysis will be taken up to 24 hours after the eptacog alfa (activated) or pdFVII injection, and up to 48 hours after the CSL689 injection. Subject safety will be routinely monitored throughout the study.
Study design
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Low-dose CSL689 | Experimental | Biological: Eptacog alfa (activated) or pdFVII, Biological: CSL689Single dose of subject's routine FVII replacement therapy (either eptacog alfa [activated] [ie, comparator drug 1] or pdFVII [ie, comparator drug 2]), followed by a single dose of CSL689 at the low dose |
| High-dose CSL689 | Experimental | Biological: Eptacog alfa (activated) or pdFVII, Biological: CSL689Single dose of subject's routine FVII replacement therapy (either eptacog alfa [activated] [ie, comparator drug 1] or pdFVII [ie, comparator drug 2]), followed by a single dose of CSL689 at the high dose. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Proven congenital FVII deficiency.
- Age ≥ 18 years.
- FVII level < 2% of normal levels.
- Minimum of 50 previous exposure days to pdFVII (including prothrombin complex concentrates [PCCs]) or rFVIIa.
Key exclusion criteria (9 listed)
- History of, or risk factors for, thromboembolic events, including known deep vein thrombosis.
- Inhibitor to FVII or rFVIIa, current or historic.
- Known or suspected hypersensitivity to hamster protein, to CSL689, or to any excipient of CSL689.
- Known or suspected allergy to rFVIIa or hamster protein.
- Major surgery within 1 month before screening.
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) or pdFVIIComparator Drug 1: Recombinant activated FVII (rFVIIa). Subjects with eptacog alfa (activated) as their routine FVII replacement therapy will receive a single dose of eptacog alfa (activated) in the study. Comparator Drug 2: Plasma-derived FVII (pdFVII). Subjects with pdFVII as their routine FVII replacement therapy will receive a single injection of pdFVII in the study. | Biological | High-dose CSL689, Low-dose CSL689 |
| CSL689Experimental Drug: Recombinant fusion protein, linking activated FVII with albumin (rVIIa-FP). Subjects will receive a single dose of CSL689 at either a low dose (Arm 1) or a high dose (Arm 2) | Biological | High-dose CSL689, Low-dose CSL689 |
Primary outcome
Terminal half-life of plasma FVIIa activity
Time frame: Up to 48 hours after CSL689 injection
Maximum observed plasma FVIIa activity
Time frame: Before injection and at up to 9 time points until 48 hours after injection
Area under the curve (AUC0-t)
Time frame: Before injection and at up to 9 time points until 48 hours after injection
Area under plasma FVIIa activity versus time curve from time 0 to last sample with quantifiable activity
7 secondary outcomes
Total clearance
Time frame: Before injection and at up to 9 time points until 48 hours after injection
Volume of distribution of the terminal phase
Time frame: Before injection and at up to 9 time points until 48 hours after injection
AUC(0-inf)
Time frame: Before injection and at up to 9 time points until 48 hours after injection
Incremental recovery
Time frame: Before injection and at up to 9 time points until 48 hours after injection
Time of occurrence of maximum observed plasma FVIIa activity
Time frame: Before injection and at up to 9 time points until 48 hours after injection
Number of subjects with antibodies against Chinese hamster ovary protein and FVII
Time frame: Up to 30 days after CSL689 injection
Number of subjects with inhibitors against FVII
Time frame: Up to 30 days after CSL689 injection
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 26 Apr 2017.
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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.
Alex Veldman study director
CSL Behring
2 sites in 2 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) or pdFVII and CSL689 in people with Congenital Coagulation Factor VII Deficiency, run by CSL Behring at 2 sites in 2 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 4 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 26 Apr 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Netherlands, Norway. 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?
Terminal half-life of plasma FVIIa activity (time frame: Up to 48 hours after CSL689 injection). The study lists 3 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 26 Apr 2017.