Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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.
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Dose Cohort 1 | Experimental | Lowest MOD-5014 dose tested in the study |
|
| Dose Cohort 2 | Experimental | MOD-5014 Dose cohort 2 |
|
| Dose Cohort 3 | Experimental | MOD-5014 Dose cohort 3 |
|
| Dose Cohort 4 | Experimental | MOD-5014 Dose cohort 4 |
|
| Dose Cohort 5 | Experimental | MOD-5014 Dose cohort 5 |
|
| Dose Cohort 6 | Experimental | Highest MOD-5014 dose tested in the study |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| MOD-5014 | Drug | Long Acting Factor VIIa |
|
| 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 |
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Pharmacodynamic Profile (evaluate the pharmacodynamic response (FVIIa clotting activity, coagulation parameters) of single IV administration of escalating MOD-5014 doses) | 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 |
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Guy Ben-Bashat | OPKO Health, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| City of Hope | Duarte | California | United States | |||
| Children's Hospital Los Angeles |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Los Angeles |
| California |
| 90027 |
| United States |
| Orthopaedic Hemophilia Treatment Center | Los Angeles | California | United States |
| University of California Davis Medical Center | Sacramento | California | United States |
| Rush University Medical Center | Chicago | Illinois | United States |
| Bleeding & Clotting Disorders Institute | Peoria | Illinois | United States |
| Indiana Hemophilia & Thrombosis Center | Indianapolis | Indiana | United States |
| Penn State Milton S. Hershey Medical Center | Hershey | Pennsylvania | United States |
| UPMC Presbyterian Shadyside | Pittsburgh | Pennsylvania | United States |
| UT Southwestern Medical Center | Dallas | Texas | United States |
| The Gulf States Hemophilia and Thrombophilia Center | Houston | Texas | United States |
| ID | Term |
|---|---|
| D006467 | Hemophilia A |
| D002836 | Hemophilia B |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D040181 | Genetic Diseases, X-Linked |
Not provided
Not provided