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
Overview
- Phase
- Phase 1
- Intervention
- Placebo
- Conditions
- Genetic Disease
- Sponsor
- Eloxx Pharmaceuticals, Inc.
- Enrollment
- 18
- Locations
- 1
- Primary Endpoint
- Adverse Events
- Status
- Completed
- Last Updated
- 2 years ago
Overview
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.
Investigators
Eligibility Criteria
Inclusion Criteria
- Not provided
Exclusion Criteria
- Not provided
Arms & Interventions
Placebo Comparator Arm
Placebo
Intervention: Placebo
Active treatment
ELX-02
Intervention: ELX-02
Outcomes
Primary Outcomes
Adverse Events
Time Frame: 0-10 days
Incidence and characteristics of adverse events occurring following single doses of ELX-02
Pharmacokinetics: Mean residence time (MRT)
Time Frame: 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)
Time Frame: 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)
Time Frame: 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)
Time Frame: 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)
Time Frame: 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)
Time Frame: 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)
Time Frame: 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)
Time Frame: 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)
Time Frame: 0-10 days
tmax will be determined for ELX-02 and an estimation of ELX-02 dose proportionality of PK parameters.