Safety, Tolerability, Pharmacokinetics of ELX-02 in Healthy Adult Volunteers

Summary

This is the first study in humans of ELX-02, an advanced synthetic aminoglycoside optimized as a translational read-through drug (TRID) for the treatment of genetic conditions caused by nonsense. mutations. This is a classical Phase 1a study designed as a randomized, double-blinded, placebo-controlled, single dose escalation to evaluate the safety, tolerability and pharmacokinetics of ELX-02 in healthy adult volunteers.

Timeline

Start

2016-07

Primary completion

2017-02

Completion

2017-05

Drugs

Indications