NCT03309605

Phase 1 Study of ELX-02 in Healthy Adult Subjects

A Phase 1, Randomized, Double-Blinded, Placebo-Controlled, Third Party Open, Multiple Dose Escalation, Single Center Study to Evaluate the Safety, Tolerability and Pharmacokinetics of Subcutaneously Administered ELX-02 in Independent Consecutive Cohorts of Healthy Subjects

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 62 (actual)
• Sponsor: Eloxx Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 18 Years – 55 Years
• Healthy volunteers: Accepted

Summary

Phase 1 Multiple Ascending Dose Study in Normal Healthy Volunteers

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 1b study designed as a randomized, double-blinded, placebo-controlled, multiple dose escalation to evaluate the safety, tolerability, and pharmacokinetics of ELX-02 in healthy adult volunteers.

Conditions

• Genetic Disease
• Nonsense Mutation

Interventions

Type	Name	Description
DRUG	ELX-02	ELX-02 is a synthetic, designer eukaryotic ribosomal specific glycoside (ERSG) optimized as a translational read-through drug
DRUG	Placebo	Placebo

Timeline

• Start date: 2017-10-11
• Primary completion: 2019-06-17
• Completion: 2019-07-17
• First posted: 2017-10-13
• Last updated: 2021-03-16
• Results posted: 2021-03-16

Locations

1 site across 1 country: Belgium