Clinical trial NCT01708564: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Hemophilia
Lead sponsor: rEVO Biologics
Sponsor class: INDUSTRY
First posted: 2012-10-17
Enrollment: 15
Sex: MALE
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: BASIC_SCIENCE

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.


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

Primary outcomes:
measure: Factor VIIa concentration in patient plasma as measured by FVIIa PK and PD assays
timeFrame: Up to 36 hours of dosing
Secondary outcomes:
measure: Incidence of patients with treatment emergent adverse events
timeFrame: Up to 28 days after dosing
Other outcomes:
Locations:
city: Sacramento
country: United States
facility: UC Davis Health System Internal Medicine: Hematology & Oncology
state: California
city: Chicago
country: United States
facility: RUSH Hemophilia & Thrombophilia Center
state: Illinois
city: Leiden
country: Netherlands
facility: Centre for Human Drug Research
Study officials:
affiliation: rEVO Biologics
name: Johan Frieling, MD,PhD
role: STUDY_DIRECTOR
References: