clinical trial · NCT03292302
Phase 1 Study of ELX-02 in Healthy Adults
Genetic DiseaseNonsense MutationPlaceboELX-02
brief summary
Phase 1 Single Ascending Dose Study in Normal Healthy Volunteers
started
Sep 26, 2017
primary completion
Dec 15, 2017
completion
Dec 15, 2017
last updated
Aug 22, 2023
detailed description
This is a 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.
official title
A Phase 1a, Randomized, Double-blinded, Placebo-Controlled, Single Dose Escalation Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ELX-02 in Healthy Adult Volunteers
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol