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

Phase 1

Terminated

• Conditions: Nonsense Mutations; Genetic Diseases
• Interventions: Drug: Placebo; Drug: ELX-02

• Registration Number: NCT02807961
• Lead Sponsor: Eloxx Pharmaceuticals, Inc.

Brief 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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2016-06-10
• Study First Submitted QC: 2016-06-20
• Study First Posted: 2016-06-21
• Study Start: 2016-07
• Primary Completion: 2017-02
• Study Completion: 2017-05
• Status Verified: 2017-08
• Last Update Submitted: 2019-10-19
• Last Update Posted: 2019-10-22

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 42

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	Placebo comparator arm
Active treatment	ELX-02	ELX-02, active comparator

Primary Outcome Measures

Name	Time	Method
Adverse Events	0-10 days	Incidence and characteristics of adverse events occurring following single doses of ELX-02
Pharmacokinetics	0-10 days	The following PK parameters will be calculated based on ELX-02 plasma concentrations: Cmax, Tmax, AUC24h, AUCinf, AUC144h, t½, mean residence time (MRT), volume of distribution (Vd), clearance (CL), absolute bioavailability (F) and an estimation of ELX-02 dose linearity of PK parameters. The following parameters will be calculated based on ELX-02 urine concentrations: urinary mass excretion of ELX-02 (in mass and %dose) and cumulated urinary excretion (in % dose) for each collection interval and total renal clearance. The following parameters will be calculated from the individual urine drug concentrations: Ae, Ae0-12h, Ae12-24h, Ae24-48h, Ae0-48h, fe.