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

Study Overview
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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.

Official Title
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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

Conditions
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Hemophilia A, Hemophilia B

Intervention / Treatment
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* Drug: MOD-5014


Participation Criteria
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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)

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 65 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

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


Arms and Interventions

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

What is the study measuring?
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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 | 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 | 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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Hemophilia, Factor VII, Factor VIIa, Inhibitors, Long Acting