NCT02919800

A Single-dose, Dose-escalation Study of a Long-acting MOD-5014 in Healthy Adult Male

A Phase 1,Randomized, Single-blind, Placebo-controlled, Single Dose, Dose-escalated Study to Assess the Safety, Pharmacokinetic and Pharmacodynamic Profile of Subcutaneous Administration of a Long-acting Recombinant Factor VIIa in Healthy Adult Males

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 32 (actual)
• Sponsor: OPKO Health, Inc. · Industry
• Sex: Male
• Age: 18 Years – 50 Years
• Healthy volunteers: Accepted

Summary

A Phase 1, randomized, single-blind, placebo-controlled, single dose, dose-escalation study to assess the safety, pharmacokinetic and pharmacodynamic profile of subcutaneous administration of a long-acting recombinant factor VIIa (MOD-5014) in healthy adult males.

Detailed description

This will be a single-dose, randomized, single-blind, placebo-controlled, dose-escalating study. The study will include four escalating dose groups, with eight subjects in each dose group. Subjects will be randomized in 3:1 ratio to receive a single SC injection of MOD-5014 (n=6) or a placebo (n=2), and will be followed up for 30 days. The initial MOD-5014 dose group will receive 100 µg/kg followed by single doses of 200, 400 and 600 µg/kg administered to subsequent subject cohorts. The decision to proceed to the higher dose level will be made by a Data Safety Monitoring Board (DSMB) after review of relevant safety data (including adverse events, clinical laboratory and vital signs), collected up to and including 7 days after the last subject of the previous dose group has been dosed. Common Terminology Criteria for Adverse Events (CTCAE) guidelines will be used to determine maximum tolerated dose (MTD) and dose limiting toxicity (DLT). Dose escalation will be permitted if the prior dose is well tolerated, and there are no safety or tolerability concerns raised by the investigator, sponsor, medical monitor or DSMB over 7 days post-dosing.

Conditions

• Hemophilia A or B With Inhibitors

Interventions

Type	Name	Description
BIOLOGICAL	MOD-5014	MOD-5014, a long-acting modified recombinant Factor VIIa
OTHER	MOD-5014 Placebo	Placebo solution for SC injection containing the same inactive ingredients used in the active drug product at matching volumes

Timeline

• Start date: 2017-01-22
• Primary completion: 2018-02-21
• Completion: 2018-02-21
• First posted: 2016-09-29
• Last updated: 2019-09-30

Locations

1 site across 1 country: Israel