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

OPKO Health

Status and phase: Completed; Phase 1

Conditions: Hemophilia B; Hemophilia A

Treatments: Drug: MOD-5014

Study type: Interventional

Funder types: Industry

Identifiers

NCT02418793

CP-5-001

Details and patient eligibility

About

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.

Enrollment: 27 patients

Sex: Male

Ages: 18 to 65 years old

Volunteers: No 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)

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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27 participants in 6 patient groups

Dose Cohort 1

Experimental group

Description:

Lowest MOD-5014 dose tested in the study

Treatment:

Drug: MOD-5014

Dose Cohort 2

Experimental group

Description:

MOD-5014 Dose cohort 2

Treatment:

Drug: MOD-5014

Dose Cohort 3

Experimental group

Description:

MOD-5014 Dose cohort 3

Treatment:

Drug: MOD-5014

Dose Cohort 4

Experimental group

Description:

MOD-5014 Dose cohort 4

Treatment:

Drug: MOD-5014

Dose Cohort 5

Experimental group

Description:

MOD-5014 Dose cohort 5

Treatment:

Drug: MOD-5014

Dose Cohort 6

Experimental group

Description:

Highest MOD-5014 dose tested in the study

Treatment:

Drug: MOD-5014