A Study of a Long-Acting r-Factor 7a (Factor VIIa) in Adult Men With Hemophilia A or B

Hemophilia A Clinical Trial

Official title:

A Phase 1/2a, Open-Label, Multicenter, Dose Escalation Study to Assess the Safety, Pharmacokinetics (PK) and Pharmacodynamics (PD) Profile of a Long Acting Recombinant FVIIa (MOD-5014) in Adult Men With Hemophilia A or B

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02418793
• Other study ID #: CP-5-001
• Status: Completed
• Phase: Phase 1
• Start date: May 2015
• Est. completion date: September 2018

Study information

• Verified date: May 2021
• Source: OPKO Health, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The purpose of the current Phase 1/2a single dose, dose-escalating study is to evaluate the acute safety, pharmacokinetics (PK) and pharmacodynamics (PD) properties of MOD-5014 in adult subjects with moderate/severe congenital hemophilia A or B. This will be a single-dose, open label, dose-escalating study. Each dose cohort will be concluded by a safety review, following which escalation to the next dose cohort will be approved.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 27
• Est. completion date: September 2018
• Est. primary completion date: September 2018
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 18 Years to 65 Years

Eligibility

Inclusion Criteria:

• Diagnosis of moderate or severe congenital Hemophilia A or B with or without inhibitors

Exclusion Criteria:

• Diagnosis of any coagulation disorder other than Hemophilia A or B

• Receipt of any immunomodulatory therapy within 3 months prior to screening, with the exception of Hepatitis C or HIV therapy

• Have had, within one month prior to study drug administration, a major surgical procedure (e.g. orthopedic, abdominal) or have an elective surgery planned within the study period

• Use of any anticoagulant for arterial/venous obstructions and/or atrial fibrillation within 7 days prior to first study drug administration

• Malignancy within past 5 years (excluding non-melanoma skin cancer)

Related Conditions & MeSH terms

• Hemophilia A
• Hemophilia B

Intervention

Drug:
• MOD-5014
Long Acting Factor VIIa

Locations

Country	Name	City	State
United States	Rush University Medical Center	Chicago	Illinois
United States	UT Southwestern Medical Center	Dallas	Texas
United States	City of Hope	Duarte	California
United States	Penn State Milton S. Hershey Medical Center	Hershey	Pennsylvania
United States	The Gulf States Hemophilia and Thrombophilia Center	Houston	Texas
United States	Indiana Hemophilia & Thrombosis Center	Indianapolis	Indiana
United States	Children's Hospital Los Angeles	Los Angeles	California
United States	Orthopaedic Hemophilia Treatment Center	Los Angeles	California
United States	Bleeding & Clotting Disorders Institute	Peoria	Illinois
United States	UPMC Presbyterian Shadyside	Pittsburgh	Pennsylvania
United States	University of California Davis Medical Center	Sacramento	California

Sponsors (1)

Lead Sponsor	Collaborator
OPKO Health, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Pharmacodynamic Profile (evaluate the pharmacodynamic response (FVIIa clotting activity, coagulation parameters) of single IV administration of escalating MOD-5014 doses)	To evaluate the pharmacodynamic response (FVIIa clotting activity, coagulation parameters) of single IV administration of escalating MOD-5014 doses in hemophilic subjects with and without inhibitors.	14 days
Primary	Composite safety and tolerability parameters as measured by adverse events, electrocardiograms (ECG), laboratory results, vital signs and injection site reactions	To assess the acute safety and tolerability of single intravenous (IV) administration of escalating MOD-5014 doses in hemophilic subjects with and without inhibitors.	30 days
Secondary	Pharmacokinetic Profile	To evaluate the pharmacokinetic profile (MOD-5014 serum levels; Area Under the Curve (AUC); Cmax; Tmax; T1/2) of single IV administration of escalating MOD-5014 doses in hemophilic subjects with and without inhibitors.	14 days