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

May 18, 2021 updated by: OPKO Health, Inc.

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

27

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • Duarte, California, United States
        • City of Hope
      • Los Angeles, California, United States, 90027
        • Children's Hospital Los Angeles
      • Los Angeles, California, United States
        • Orthopaedic Hemophilia Treatment Center
      • Sacramento, California, United States
        • University of California Davis Medical Center
    • Illinois
      • Chicago, Illinois, United States
        • Rush University Medical Center
      • Peoria, Illinois, United States
        • Bleeding & Clotting Disorders Institute
    • Indiana
      • Indianapolis, Indiana, United States
        • Indiana Hemophilia & Thrombosis center
    • Pennsylvania
      • Hershey, Pennsylvania, United States
        • Penn State Milton S. Hershey Medical Center
      • Pittsburgh, Pennsylvania, United States
        • UPMC Presbyterian Shadyside
    • Texas
      • Dallas, Texas, United States
        • UT Southwestern Medical Center
      • Houston, Texas, United States
        • The Gulf States Hemophilia and Thrombophilia Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 65 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

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)

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Dose Cohort 1
Lowest MOD-5014 dose tested in the study
Drug: MOD-5014
Long Acting Factor VIIa
Experimental: Dose Cohort 2
MOD-5014 Dose cohort 2
Drug: MOD-5014
Long Acting Factor VIIa
Experimental: Dose Cohort 3
MOD-5014 Dose cohort 3
Drug: MOD-5014
Long Acting Factor VIIa
Experimental: Dose Cohort 4
MOD-5014 Dose cohort 4
Drug: MOD-5014
Long Acting Factor VIIa
Experimental: Dose Cohort 5
MOD-5014 Dose cohort 5
Drug: MOD-5014
Long Acting Factor VIIa
Experimental: Dose Cohort 6
Highest MOD-5014 dose tested in the study
Drug: MOD-5014
Long Acting Factor VIIa

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Composite safety and tolerability parameters as measured by adverse events, electrocardiograms (ECG), laboratory results, vital signs and injection site reactions
Time Frame: 30 days
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 Outcome Measures

Outcome Measure
Measure Description
Time Frame
Pharmacokinetic Profile
Time Frame: 14 days
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

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
Pharmacodynamic Profile (evaluate the pharmacodynamic response (FVIIa clotting activity, coagulation parameters) of single IV administration of escalating MOD-5014 doses)
Time Frame: 14 days
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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

OPKO Health, Inc.

Investigators

  • Study Director: Guy Ben-Bashat, OPKO Health, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

May 1, 2015

Primary Completion (Actual)

September 1, 2018

Study Completion (Actual)

September 1, 2018

Study Registration Dates

First Submitted

March 31, 2015

First Submitted That Met QC Criteria

April 15, 2015

First Posted (Estimate)

April 16, 2015

Study Record Updates

Last Update Posted (Actual)

May 20, 2021

Last Update Submitted That Met QC Criteria

May 18, 2021

Last Verified

May 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CP-5-001

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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