Safety, Tolerability, Pharmacokinetics of ELX-02 in Healthy Adult Volunteers
Status:
Terminated
Trial end date:
2017-05-01
Target enrollment:
Participant gender:
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.