Brief Title
A Phase I Safety, Pharmacokinetics and Pharmacodynamics Study of Recombinant Factor VIIa in Adult Patients With Hemophilia A or B
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
Brief 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.
Study Phase
Phase 1
Study Type
Interventional
Primary Outcome
Factor VIIa concentration in patient plasma as measured by FVIIa PK and PD assays
Secondary Outcome
Incidence of patients with treatment emergent adverse events
Condition
Hemophilia
Intervention
rhFVIIa
Study Arms / Comparison Groups
Cohort 1
Description: 10 patients administered a single low dose of rhFVIIa
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Biological
Estimated Enrollment
15
Start Date
October 2012
Completion Date
June 2013
Primary Completion Date
June 2013
Eligibility Criteria
Inclusion Criteria:
1. be male with a diagnosis of moderate or severe congenital hemophilia A and/or B (with
or without inhibitors)
2. be 18 years or older, up to and including 75 years of age
3. be capable of understanding and willing to comply with the conditions of the protocol
4. have read, understood and provided written informed consent
Exclusion Criteria:
1. have any coagulation disorder other than hemophilia A or B
2. have a body weight >105 kg (231 lb)
3. 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)
4. have a known allergy or hypersensitivity to rabbits
5. have platelet count <100,000/mL
6. have had within one month prior to first administration of the study drug in this
study a major surgical procedure (e.g. orthopedic, abdominal)
7. 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
8. have received a Factor VII or FVIIa containing product (either plasma derived or
recombinant) within 72 hours prior to any study drug administration
9. 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
10. 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)
11. 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)
12. use any anticoagulant for arterial/venous obstructions and/or atrial fibrillation
within 7 days prior to first study drug administration
13. have an active malignancy (those with non-melanoma skin cancer are allowed)
14. 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
Gender
Male
Ages
18 Years - 75 Years
Accepts Healthy Volunteers
No
Contacts
Johan Frieling, MD,PhD, ,
Location Countries
Netherlands
Location Countries
Netherlands
Administrative Informations
NCT ID
NCT01708564
Organization ID
GTC-FVIIa-005-11
Secondary IDs
2012-002285-13
Responsible Party
Sponsor
Study Sponsor
rEVO Biologics
Study Sponsor
Johan Frieling, MD,PhD, Study Director, rEVO Biologics
Verification Date
July 2013