A Phase I Safety, Pharmacokinetics and Pharmacodynamics Study of Recombinant Factor VIIa in Adult Patients With Hemophilia A or B (rhFVIIa)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
rEVO Biologics
ClinicalTrials.gov Identifier:
NCT01708564
First received: October 15, 2012
Last updated: July 29, 2013
Last verified: July 2013
  Purpose

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.


Condition Intervention Phase
Hemophilia
Biological: rhFVIIa
Phase 1

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Basic Science
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

Resource links provided by NLM:


Further study details as provided by rEVO Biologics:

Primary Outcome Measures:
  • Factor VIIa concentration in patient plasma as measured by FVIIa PK and PD assays [ Time Frame: Up to 36 hours of dosing ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Incidence of patients with treatment emergent adverse events [ Time Frame: Up to 28 days after dosing ] [ Designated as safety issue: Yes ]

Enrollment: 15
Study Start Date: October 2012
Study Completion Date: June 2013
Primary Completion Date: June 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Cohort 1
10 patients administered a single low dose of rhFVIIa
Biological: rhFVIIa
Patients will be administered low, intermediate and high doses of rhFVIIa
Other Name: Coagulation Factor VIIa (Recombinant)
Experimental: Cohort 2
10 patients administered a single intermediate dose of rhFVIIa
Biological: rhFVIIa
Patients will be administered low, intermediate and high doses of rhFVIIa
Other Name: Coagulation Factor VIIa (Recombinant)
Experimental: Cohort 3
10 patients administered a single high dose of rhFVIIa
Biological: rhFVIIa
Patients will be administered low, intermediate and high doses of rhFVIIa
Other Name: Coagulation Factor VIIa (Recombinant)

  Eligibility

Ages Eligible for Study:   18 Years to 75 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01708564

Locations
United States, California
UC Davis Health System Internal Medicine: Hematology & Oncology
Sacramento, California, United States, 95817
United States, Illinois
RUSH Hemophilia & Thrombophilia Center
Chicago, Illinois, United States, 60612
Netherlands
Centre for Human Drug Research
Leiden, Netherlands
Sponsors and Collaborators
rEVO Biologics
Investigators
Study Director: Johan Frieling, MD,PhD rEVO Biologics
  More Information

No publications provided

Responsible Party: rEVO Biologics
ClinicalTrials.gov Identifier: NCT01708564     History of Changes
Other Study ID Numbers: GTC-FVIIa-005-11, 2012-002285-13
Study First Received: October 15, 2012
Last Updated: July 29, 2013
Health Authority: United States: Food and Drug Administration
Netherlands: The Central Committee on Research Involving Human Subjects (CCMO)

Additional relevant MeSH terms:
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on October 16, 2014