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

NCT01708564 | Completed Phase 1 DMC

• 2 other identifiers: GTC-FVIIa-005-112012-002285-13
• Study Type: interventional
• Target: 15
• Locations: 2 countries
• Sites: 3

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.

Conditions

Hemophilia

Outcome Measures

Primary Outcomes (1)

• Factor VIIa concentration in patient plasma as measured by FVIIa PK and PD assays

  Up to 36 hours of dosing

Secondary Outcomes (1)

• Incidence of patients with treatment emergent adverse events

  Up to 28 days after dosing

Study Arms (3)

Cohort 1

EXPERIMENTAL

10 patients administered a single low dose of rhFVIIa

Biological: rhFVIIa

Cohort 2

EXPERIMENTAL

10 patients administered a single intermediate dose of rhFVIIa

Biological: rhFVIIa

Cohort 3

EXPERIMENTAL

10 patients administered a single high dose of rhFVIIa

Biological: rhFVIIa

Interventions

rhFVIIa BIOLOGICAL

Patients will be administered low, intermediate and high doses of rhFVIIa

Also known as: Coagulation Factor VIIa (Recombinant)

Cohort 1; Cohort 2; Cohort 3

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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)
• 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

Sponsors & Collaborators

• rEVO Biologics: lead

Study Sites (3)

UC Davis Health System Internal Medicine: Hematology & Oncology

Sacramento, California, 95817, United States

Location

RUSH Hemophilia & Thrombophilia Center

Chicago, Illinois, 60612, United States

Location

Centre for Human Drug Research

Leiden, Netherlands

Location

Related Publications (1)

• 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.

  PMID: 40674256 DERIVED

MeSH Terms

Conditions

Hemophilia A

Interventions

Factor VII

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Enzyme Precursors; Enzymes and Coenzymes; Blood Coagulation Factors; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Protein Precursors; Biological Factors

Study Officials

• Johan Frieling, MD,PhD

  rEVO Biologics

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: BASIC SCIENCE
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 15, 2012
• First Posted: October 17, 2012
• Study Start: October 1, 2012
• Primary Completion: June 1, 2013
• Study Completion: June 1, 2013
• Last Updated: July 30, 2013

Record last verified: 2013-07

Locations

US (2); NL (1)