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
Trial Snapshot
- Phase
- Phase 1
- Status
- Terminated
- Sponsor
- Eloxx Pharmaceuticals, Inc.
- Enrollment
- 42
- Primary Endpoint
- Adverse Events
Study Overview
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.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Eligibility Criteria
- Ages
- 18 Years to 45 Years (Adult)
- Sex
- All
- Accepts Healthy Volunteers
- Yes
Inclusion Criteria
- Not provided
Exclusion Criteria
- Not provided
Arms & Interventions
Placebo
Placebo comparator arm
Intervention: Placebo (Drug)
Active treatment
ELX-02, active comparator
Intervention: ELX-02 (Drug)
Outcomes
Primary Outcomes
Adverse Events
Time Frame: 0-10 days
Incidence and characteristics of adverse events occurring following single doses of ELX-02
Pharmacokinetics
Time Frame: 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.
Secondary Outcomes
No secondary outcomes reported