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

Hemophilia Clinical Trial

— rhFVIIa  

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01708564
• Other study ID #: GTC-FVIIa-005-11
• Secondary ID: 2012-002285-13
• Status: Completed
• Phase: Phase 1
• First received: October 15, 2012
• Last updated: July 29, 2013
• Start date: October 2012
• Est. completion date: June 2013

Study information

• Verified date: July 2013
• Source: rEVO Biologics
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationNetherlands: The Central Committee on Research Involving Human Subjects (CCMO)
• Study type: Interventional

Clinical 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 15
• Est. completion date: June 2013
• Est. primary completion date: June 2013
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 18 Years to 75 Years

Eligibility

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

Study Design

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

Related Conditions & MeSH terms

• Hemophilia
• Hemophilia A

Intervention

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

Locations

Country	Name	City	State
Netherlands	Centre for Human Drug Research	Leiden
United States	RUSH Hemophilia & Thrombophilia Center	Chicago	Illinois
United States	UC Davis Health System Internal Medicine: Hematology & Oncology	Sacramento	California

Sponsors (1)

Lead Sponsor	Collaborator
rEVO Biologics

Countries where clinical trial is conducted

United States,  Netherlands, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Factor VIIa concentration in patient plasma as measured by FVIIa PK and PD assays		Up to 36 hours of dosing	No
Secondary	Incidence of patients with treatment emergent adverse events		Up to 28 days after dosing	Yes