Phase 1 Study of ELX-02 in Healthy Adults

Phase 1

Completed

• Conditions: Nonsense Mutation; Genetic Disease
• Interventions: Drug: Placebo; Drug: ELX-02

• Registration Number: NCT03292302
• Lead Sponsor: Eloxx Pharmaceuticals, Inc.

Brief Summary

Phase 1 Single 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 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.

Study Timeline

• Study First Submitted: 2017-08-30
• Study First Submitted QC: 2017-09-20
• Study First Posted: 2017-09-25
• Study Start: 2017-09-26
• Primary Completion: 2017-12-15
• Study Completion: 2017-12-15
• Status Verified: 2019-10
• Last Update Submitted: 2023-08-17
• Last Update Posted: 2023-08-22

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 18

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo Comparator Arm	Placebo	Placebo
Active treatment	ELX-02	ELX-02

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: Mean residence time (MRT)	0-10 days	MRT will be determined for ELX-02 and an estimation of ELX-02 dose proportionality of PK parameters.
Pharmacokinetics: Area under the plasma concentration-time curve calculated from time of administration to time 48h (AUC48h)	0-10 days	AUC48h will be determined for ELX-02 and an estimation of ELX-02 dose proportionality of PK parameters.
Pharmacokinetics: Area under the concentration-time curve from time 0 extrapolated to infinity (AUCinf)	0-10 days	AUCinf will be determined for ELX-02 and an estimation of ELX-02 dose proportionality of PK parameters.
Pharmacokinetics: Volume of distribution (Vd/F)	0-10 days	Vd/F will be determined for ELX-02 and an estimation of ELX-02 dose proportionality of PK parameters.
Pharmacokinetics: Elimination half-life (t1/2)	0-10 days	t1/2 will be determined for ELX-02 and an estimation of ELX-02 dose proportionality of PK parameters.
Pharmacokinetics: Clearance (CL/F)	0-10 days	CL/F will be determined for ELX-02 and an estimation of ELX-02 dose proportionality of PK parameters.
Pharmacokinetics: Area under the plasma concentration-time curve calculated from time of administration to time 24h (AUC24h)	0-10 days	AUC24h will be determined for ELX-02 and an estimation of ELX-02 dose proportionality of PK parameters.
Pharmacokinetics: Maximum plasma concentration (Cmax)	0-10 days	Cmax will be determined for ELX-02 and an estimation of ELX-02 dose proportionality of PK parameters.
Pharmacokinetics: Time at which Cmax occurs (tmax)	0-10 days	tmax will be determined for ELX-02 and an estimation of ELX-02 dose proportionality of PK parameters.

Trial Locations

Locations (1)

SGS Life Sciences, Clinical Pharmacology Unit; 🇧🇪 Antwerp, Belgium