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

NCT ID: NCT02418793

Last Updated: 2021-05-20

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

27 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-05-31

Study Completion Date

2018-09-30

Brief Summary

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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.

Detailed Description

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Conditions

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Hemophilia A Hemophilia B

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Dose Cohort 1

Lowest MOD-5014 dose tested in the study

Group Type EXPERIMENTAL

MOD-5014

Intervention Type DRUG

Long Acting Factor VIIa

Dose Cohort 2

MOD-5014 Dose cohort 2

Group Type EXPERIMENTAL

MOD-5014

Intervention Type DRUG

Long Acting Factor VIIa

Dose Cohort 3

MOD-5014 Dose cohort 3

Group Type EXPERIMENTAL

MOD-5014

Intervention Type DRUG

Long Acting Factor VIIa

Dose Cohort 4

MOD-5014 Dose cohort 4

Group Type EXPERIMENTAL

MOD-5014

Intervention Type DRUG

Long Acting Factor VIIa

Dose Cohort 5

MOD-5014 Dose cohort 5

Group Type EXPERIMENTAL

MOD-5014

Intervention Type DRUG

Long Acting Factor VIIa

Dose Cohort 6

Highest MOD-5014 dose tested in the study

Group Type EXPERIMENTAL

MOD-5014

Intervention Type DRUG

Long Acting Factor VIIa

Interventions

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MOD-5014

Long Acting Factor VIIa

Intervention Type DRUG

Eligibility Criteria

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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)
Minimum Eligible Age

18 Years

Maximum Eligible Age

65 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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OPKO Health, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Guy Ben-Bashat

Role: STUDY_DIRECTOR

OPKO Health, Inc.

Locations

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City of Hope

Duarte, California, United States

Site Status

Children's Hospital Los Angeles

Los Angeles, California, United States

Site Status

Orthopaedic Hemophilia Treatment Center

Los Angeles, California, United States

Site Status

University of California Davis Medical Center

Sacramento, California, United States

Site Status

Rush University Medical Center

Chicago, Illinois, United States

Site Status

Bleeding & Clotting Disorders Institute

Peoria, Illinois, United States

Site Status

Indiana Hemophilia & Thrombosis Center

Indianapolis, Indiana, United States

Site Status

Penn State Milton S. Hershey Medical Center

Hershey, Pennsylvania, United States

Site Status

UPMC Presbyterian Shadyside

Pittsburgh, Pennsylvania, United States

Site Status

UT Southwestern Medical Center

Dallas, Texas, United States

Site Status

The Gulf States Hemophilia and Thrombophilia Center

Houston, Texas, United States

Site Status

Countries

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United States

Other Identifiers

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CP-5-001

Identifier Type: -

Identifier Source: org_study_id

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