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

Primary Purpose

Hemophilia A, Hemophilia B

Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
MOD-5014
Sponsored by
OPKO Health, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hemophilia A focused on measuring Hemophilia, Factor VII, Factor VIIa, Inhibitors, Long Acting

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)MaleDoes not accept healthy volunteers

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)

Sites / Locations

  • City of Hope
  • Children's Hospital Los Angeles
  • Orthopaedic Hemophilia Treatment Center
  • University of California Davis Medical Center
  • Rush University Medical Center
  • Bleeding & Clotting Disorders Institute
  • Indiana Hemophilia & Thrombosis Center
  • Penn State Milton S. Hershey Medical Center
  • UPMC Presbyterian Shadyside
  • UT Southwestern Medical Center
  • The Gulf States Hemophilia and Thrombophilia Center

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm 5

Arm 6

Arm Type

Experimental

Experimental

Experimental

Experimental

Experimental

Experimental

Arm Label

Dose Cohort 1

Dose Cohort 2

Dose Cohort 3

Dose Cohort 4

Dose Cohort 5

Dose Cohort 6

Arm Description

Lowest MOD-5014 dose tested in the study

MOD-5014 Dose cohort 2

MOD-5014 Dose cohort 3

MOD-5014 Dose cohort 4

MOD-5014 Dose cohort 5

Highest MOD-5014 dose tested in the study

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

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.

Full Information

First Posted
March 31, 2015
Last Updated
May 18, 2021
Sponsor
OPKO Health, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT02418793
Brief Title
A Study of a Long-Acting r-Factor 7a (Factor VIIa) in Adult Men With Hemophilia A or B
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
Study Type
Interventional

2. Study Status

Record Verification Date
May 2021
Overall Recruitment Status
Completed
Study Start Date
May 2015 (undefined)
Primary Completion Date
September 2018 (Actual)
Study Completion Date
September 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
OPKO Health, Inc.

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hemophilia A, Hemophilia B
Keywords
Hemophilia, Factor VII, Factor VIIa, Inhibitors, Long Acting

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
27 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Dose Cohort 1
Arm Type
Experimental
Arm Description
Lowest MOD-5014 dose tested in the study
Arm Title
Dose Cohort 2
Arm Type
Experimental
Arm Description
MOD-5014 Dose cohort 2
Arm Title
Dose Cohort 3
Arm Type
Experimental
Arm Description
MOD-5014 Dose cohort 3
Arm Title
Dose Cohort 4
Arm Type
Experimental
Arm Description
MOD-5014 Dose cohort 4
Arm Title
Dose Cohort 5
Arm Type
Experimental
Arm Description
MOD-5014 Dose cohort 5
Arm Title
Dose Cohort 6
Arm Type
Experimental
Arm Description
Highest MOD-5014 dose tested in the study
Intervention Type
Drug
Intervention Name(s)
MOD-5014
Intervention Description
Long Acting Factor VIIa
Primary Outcome Measure Information:
Title
Composite safety and tolerability parameters as measured by adverse events, electrocardiograms (ECG), laboratory results, vital signs and injection site reactions
Description
To assess the acute safety and tolerability of single intravenous (IV) administration of escalating MOD-5014 doses in hemophilic subjects with and without inhibitors.
Time Frame
30 days
Secondary Outcome Measure Information:
Title
Pharmacokinetic Profile
Description
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.
Time Frame
14 days
Other Pre-specified Outcome Measures:
Title
Pharmacodynamic Profile (evaluate the pharmacodynamic response (FVIIa clotting activity, coagulation parameters) of single IV administration of escalating MOD-5014 doses)
Description
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.
Time Frame
14 days

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Guy Ben-Bashat
Organizational Affiliation
OPKO Health, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
City of Hope
City
Duarte
State/Province
California
Country
United States
Facility Name
Children's Hospital Los Angeles
City
Los Angeles
State/Province
California
ZIP/Postal Code
90027
Country
United States
Facility Name
Orthopaedic Hemophilia Treatment Center
City
Los Angeles
State/Province
California
Country
United States
Facility Name
University of California Davis Medical Center
City
Sacramento
State/Province
California
Country
United States
Facility Name
Rush University Medical Center
City
Chicago
State/Province
Illinois
Country
United States
Facility Name
Bleeding & Clotting Disorders Institute
City
Peoria
State/Province
Illinois
Country
United States
Facility Name
Indiana Hemophilia & Thrombosis Center
City
Indianapolis
State/Province
Indiana
Country
United States
Facility Name
Penn State Milton S. Hershey Medical Center
City
Hershey
State/Province
Pennsylvania
Country
United States
Facility Name
UPMC Presbyterian Shadyside
City
Pittsburgh
State/Province
Pennsylvania
Country
United States
Facility Name
UT Southwestern Medical Center
City
Dallas
State/Province
Texas
Country
United States
Facility Name
The Gulf States Hemophilia and Thrombophilia Center
City
Houston
State/Province
Texas
Country
United States

12. IPD Sharing Statement

Learn more about this trial

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

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