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

Phase 1

Completed

• Conditions: Hemophilia A; Hemophilia B
• Interventions: Drug: MOD-5014

• Registration Number: NCT02418793
• Lead Sponsor: OPKO Health, Inc.

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2015-03-31
• Study First Submitted QC: 2015-04-15
• Study First Posted: 2015-04-16
• Study Start: 2015-05
• Primary Completion: 2018-09
• Study Completion: 2018-09
• Status Verified: 2021-05
• Last Update Submitted: 2021-05-18
• Last Update Posted: 2021-05-20

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 27

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Dose Cohort 4	MOD-5014	MOD-5014 Dose cohort 4
Dose Cohort 1	MOD-5014	Lowest MOD-5014 dose tested in the study
Dose Cohort 2	MOD-5014	MOD-5014 Dose cohort 2
Dose Cohort 3	MOD-5014	MOD-5014 Dose cohort 3
Dose Cohort 5	MOD-5014	MOD-5014 Dose cohort 5
Dose Cohort 6	MOD-5014	Highest MOD-5014 dose tested in the study

Primary Outcome Measures

Name	Time	Method
Composite safety and tolerability parameters as measured by adverse events, electrocardiograms (ECG), laboratory results, vital signs and injection site reactions	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.

Secondary Outcome Measures

Name	Time	Method
Pharmacokinetic Profile	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.

Trial Locations

Locations (11)

Penn State Milton S. Hershey Medical Center; 🇺🇸 Hershey, Pennsylvania, United States

City of Hope; 🇺🇸 Duarte, California, United States

Orthopaedic Hemophilia Treatment Center; 🇺🇸 Los Angeles, California, United States

Rush University Medical Center; 🇺🇸 Chicago, Illinois, United States

Indiana Hemophilia & Thrombosis Center; 🇺🇸 Indianapolis, Indiana, United States

The Gulf States Hemophilia and Thrombophilia Center; 🇺🇸 Houston, Texas, United States

Children's Hospital Los Angeles; 🇺🇸 Los Angeles, California, United States

University of California Davis Medical Center; 🇺🇸 Sacramento, California, United States

UPMC Presbyterian Shadyside; 🇺🇸 Pittsburgh, Pennsylvania, United States

Bleeding & Clotting Disorders Institute; 🇺🇸 Peoria, Illinois, United States

UT Southwestern Medical Center; 🇺🇸 Dallas, Texas, United States