HemophiliaPhase 1rhFVIIaNCT01708564
A Phase I Safety, Pharmacokinetics and Pharmacodynamics Study of Recombinant Factor VIIa in Adult Patients With Hemophilia A or B
A Phase 1 study testing rhFVIIa in people with Hemophilia, run by rEVO Biologics at 3 sites in 2 countries.
Trial summary
This study will assess the pharmacokinetics and pharmacodynamics of rhFVIIa at three dose levels. The results will help identify the most optimal doses to take forward to the Phase 2/3 studies where bleedings in hemophilia patients with inhibitors will be treated with rhFVIIa. Read more about this study
- Who is it for?
- Ages 18 to 75 years, men only
- Conditions the study is for
- Hemophilia
- What is being tested?
- rhFVIIa
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 3 sitesin 2 countries
- Study dates
- Oct 2012to Jun 2013
- 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 75 years
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- be male with a diagnosis of moderate or severe congenital hemophilia A and/or B (with or without inhibitors)
- be 18 years or older, up to and including 75 years of age
- be capable of understanding and willing to comply with the conditions of the protocol
- have read, understood and provided written informed consent
Who cannot join
- have any coagulation disorder other than hemophilia A or B
- have a body weight >105 kg (231 lb)
- be immuno-suppressed (i.e., the patient should not receive systemic immunosuppressive medication <30 days prior to enrollment, CD4 counts at screening should be >200/µl)
- have a known allergy or hypersensitivity to rabbits
- have platelet count <100,000/mL
- have had within one month prior to first administration of the study drug in this study a major surgical procedure (e.g. orthopedic, abdominal)
Show all 14Show fewer
- have an active, ongoing bleeding for which the patient is being treated, or treatment for a bleeding was stopped within 24 hours of the time of study drug administration
- have received a Factor VII or FVIIa containing product (either plasma derived or recombinant) within 72 hours prior to any study drug administration
- have received an investigational drug within 30 days of the first study drug administration, or is expected to receive such drug during participation in this study
- have a clinically relevant hepatic (hepatic enzymes >3 times the upper limit of normal) and/or renal impairment (creatinine >2 times the upper limit of normal)
- have a history of arterial and/or venous thromboembolic events (such as myocardial infarction, ischemic strokes, transient ischemic attacks, deep venous thrombosis or pulmonary embolism) within 2 years prior to first dose of study drug, have an arterial stent in place or have clinically significant atherosclerotic disease (e.g., angina pectoris, peripheral vascular disease)
- use any anticoagulant for arterial/venous obstructions and/or atrial fibrillation within 7 days prior to first study drug administration
- have an active malignancy (those with non-melanoma skin cancer are allowed)
- have any life-threatening disease or other disease or condition which, according to the investigator's judgment, could imply a potential hazard to the patient, interfere with the trial participation or trial outcome
Trial locations and contacts
3 sites in 2 countries.
3 sites in 2 countries
United States2 sites
RUSH Hemophilia & Thrombophilia Center
Chicago, Illinois, 60612
UC Davis Health System Internal Medicine: Hematology & Oncology
Sacramento, California, 95817
About this study
Official title: A Phase 1b, Dose Escalation Study to Assess the Safety, Pharmacokinetics and Pharmacodynamics of Coagulation Factor VIIa (Recombinant) in Congenital Hemophilia A or B Patients
This study will assess the pharmacokinetics and pharmacodynamics of rhFVIIa at three dose levels. The results will help identify the most optimal doses to take forward to the Phase 2/3 studies where bleedings in hemophilia patients with inhibitors will be treated with rhFVIIa.
- Conditions
- Hemophilia
- Being tested
- rhFVIIa
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
rhFVIIaBiological
Also called Coagulation Factor VIIa (Recombinant)
Patients will be administered low, intermediate and high doses of rhFVIIa
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | 10 patients administered a single low dose of rhFVIIa Receives: Biological: rhFVIIa |
| Cohort 2 | Experimental | 10 patients administered a single intermediate dose of rhFVIIa Receives: Biological: rhFVIIa |
| Cohort 3 | Experimental | 10 patients administered a single high dose of rhFVIIa Receives: Biological: rhFVIIa |
What is being measured
The primary outcome is the main question the study is designed to answer.
Factor VIIa concentration in patient plasma as measured by FVIIa PK and PD assays
Measured: Up to 36 hours of dosing
Secondary outcomes (1)
Incidence of patients with treatment emergent adverse events
Measured: Up to 28 days after dosing
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Oct 2012 (estimated)
- Study startOct 2012
- Primary completionJun 2013
- Study completionJun 2013
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 HemophiliaStudies of the same condition, open and under way.
- 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
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- rEVO Biologics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Johan Frieling, MD,PhD, Study director, rEVO Biologics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01708564
- Sponsor's protocol ID
- GTC-FVIIa-005-11
- Other IDs
- 2012-002285-13
- Acronym
- rhFVIIa
- Status last verified
- Jul 2013
- First submitted
- 15 Oct 2012
- Last updated
- 30 Jul 2013
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Carcao M, Hermans C, Giermasz A, Kessler C, Miesbach W, Quon D, Windyga J, Mahlangu J. Safety and Use of Eptacog Beta 225 microg/kg in Patients With Haemophilia A or B With Inhibitors. Haemophilia. 2025 Sep;31(5):957-965. doi: 10.1111/hae.70083. Epub 2025 Jul 17.
PubMed 40674256
Study at a glance
From the registry record, last updated by the sponsor on 30 Jul 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1b, Dose Escalation Study to Assess the Safety, Pharmacokinetics and Pharmacodynamics of Coagulation Factor VIIa (Recombinant) in Congenital Hemophilia A or B Patients
- ClinicalTrials.gov ID
- NCT01708564
- Acronym / sponsor ID
- rhFVIIa / GTC-FVIIa-005-11
- Conditions
- Hemophilia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- rEVO BiologicsIndustry (pharma, biotech, devices)
- Study start
- Oct 2012
- Primary completion
- Jun 2013 (actual)
- Study completion
- Jun 2013 (actual)
- First posted
- 17 Oct 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 study will assess the pharmacokinetics and pharmacodynamics of rhFVIIa at three dose levels. The results will help identify the most optimal doses to take forward to the Phase 2/3 studies where bleedings in hemophilia patients with inhibitors will be treated with rhFVIIa.
Study design
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Biological: rhFVIIa10 patients administered a single low dose of rhFVIIa |
| Cohort 2 | Experimental | Biological: rhFVIIa10 patients administered a single intermediate dose of rhFVIIa |
| Cohort 3 | Experimental | Biological: rhFVIIa10 patients administered a single high dose of rhFVIIa |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- be male with a diagnosis of moderate or severe congenital hemophilia A and/or B (with or without inhibitors)
- be 18 years or older, up to and including 75 years of age
- be capable of understanding and willing to comply with the conditions of the protocol
- have read, understood and provided written informed consent
Key exclusion criteria (14 listed)
- have any coagulation disorder other than hemophilia A or B
- have a body weight >105 kg (231 lb)
- be immuno-suppressed (i.e., the patient should not receive systemic immunosuppressive medication <30 days prior to enrollment, CD4 counts at screening should be >200/µl)
- have a known allergy or hypersensitivity to rabbits
- have platelet count <100,000/mL
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 |
|---|---|---|
| rhFVIIaAlso: Coagulation Factor VIIa (Recombinant)Patients will be administered low, intermediate and high doses of rhFVIIa | Biological | Cohort 1, Cohort 2, Cohort 3 |
Primary outcome
Factor VIIa concentration in patient plasma as measured by FVIIa PK and PD assays
Time frame: Up to 36 hours of dosing
1 secondary outcome
Incidence of patients with treatment emergent adverse events
Time frame: Up to 28 days after dosing
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 30 Jul 2013.
- Derived from this study: PubMed 40674256Carcao M, Hermans C, Giermasz A, Kessler C, Miesbach W, Quon D, Windyga J, Mahlangu J. Safety and Use of Eptacog Beta 225 microg/kg in Patients With Haemophilia A or B With Inhibitors. Haemophilia. 2025 Sep;31(5):957-965. doi: 10.1111/hae.70083. Epub 2025 Jul 17.
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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.
Johan Frieling, MD,PhD study director
rEVO Biologics
3 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 rhFVIIa in people with Hemophilia, run by rEVO Biologics at 3 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 14 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 30 Jul 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States, Netherlands. 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?
Factor VIIa concentration in patient plasma as measured by FVIIa PK and PD assays (time frame: Up to 36 hours of dosing). 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 NCT01708564, 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 30 Jul 2013.