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
Overview
- Phase
- Phase 1
- Intervention
- Placebo
- Conditions
- Genetic Disease
- Sponsor
- Eloxx Pharmaceuticals, Inc.
- Enrollment
- 62
- Locations
- 1
- Primary Endpoint
- Pharmacokinetic Parameter - Plasma AUC0-inf
- Status
- Completed
- Last Updated
- 5 years ago
Overview
Brief 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.
Investigators
Eligibility Criteria
Inclusion Criteria
- Not provided
Exclusion Criteria
- Not provided
Arms & Interventions
Placebo Comparator Arm
Placebo
Intervention: Placebo
ELX-02
ELX-02
Intervention: ELX-02
Outcomes
Primary Outcomes
Pharmacokinetic Parameter - Plasma AUC0-inf
Time Frame: Day 29: pre-dose, 15 min, 30 min, 45 min, 1h, 3h, 6h, 12h, 24h, 36h, 48h, 72h post dose
Day 29 Area under the curve (AUC0-inf) of ELX-02 plasma concentration following the subcutaneous (SC) dose on Day 29 extrapolated to infinity
Pharmacokinetic Parameter Plasma - Tmax
Time Frame: Day 29: pre-dose, 15 min, 30 min, 45 min, 1h, 3h, 6h, 12h, 24h, 36h, 48h, 72h post-dose
Day 29 Time to maximum concentration (Tmax) of ELX-02 plasma concentrations following the subcutaneous (SC) dose on Day 29
Urine Pharmacokinetics Parameter - CLR24h
Time Frame: 24 h
Renal clearance on Day 29 (CLR=Ae24h/plasmaAUC24h)
Urine Pharmacokinetics Parameter - Fe 12h on Day 29
Time Frame: 12 h on Day 29
Percent of dose excreted (Fe) in urine on Day 29
Pharmacokinetic Parameter - Plasma Tmax
Time Frame: Day 1: pre-dose, 15 min, 30 min, 45 min, 1h, 3h, 6h, 12h, 24h, 36h, 48h, 72h post-dose
Day 1 Time to maximum concentration (Tmax) of ELX-02 plasma concentrations following the subcutaneous (SC) dose on Day 1
Pharmacokinetic Parameters - Plasma Cmax
Time Frame: Day 29: pre-dose, 15 min, 30 min, 45 min, 1h, 3h, 6h, 12h, 24h, 36h, 48h, 72h post-dose
Day 29 Peak Plasma Concentration (Cmax) of ELX-02 following the subcutaneous (SC) dose on Day 29 to 72 hours post-dose
Pharmacokinetic Parameter - Plasma Rac(AUC24h)
Time Frame: Day 1 and Day 24 hr
Accumulation ratio, calculated as AUC24h Day29/AUC24h Day 1
Pharmacokinetic Parameter - Plasma RAC(Cmax)
Time Frame: Day 1 and Day 29
Accumulation ratio, calculated as Cmax Day29/Cmax Day 1
Urine Pharmacokinetics Parameter - Ae72h
Time Frame: Day 29: pre-dose and during 0-12h, 12-24h, 24-48h, and 48-72h post-dose
Day 29 Cumulative amount of unchanged drug excreted into urine (Ae72h) of ELX-02 following the subcutaneous (SC) dose on Day 29
Urine Pharmacokinetic Parameter - Rmax
Time Frame: Day 29: pre-dose and during 0-12h, 12-24h, 24-48h, and 48-72h post-dose
Day 29 Maximum rate of urinary extraction (Rmax) of ELX-02 in each collection time interval following the subcutaneous (SC) dose on Day 29
Urine Pharmacokinetics Parameter - CLR24h on Day 1
Time Frame: 24 hours
Renal clearance on Day 1 (CLR=Ae24h/plasmaAUC24h)
Pharmacokinetic Parameters - Plasma AUC0-24
Time Frame: Day 1: pre-dose, 15 min, 30 min, 45 min, 1h, 3h, 6h, 12h, 24h, post-dose
Day 1 Area under the curve (AUC0-24) of ELX-02 plasma concentration following the subcutaneous (SC) dose on Day 1 to 24 hours post-ose
Pharmacokinetic Parameters- Plasma AUC0-24
Time Frame: Day 29: pre-dose, 15 min, 30 min, 45 min, 1h, 3h, 6h, 12h, 24h, post-dose
Day 29 Area under the curve (AUC0-24) of ELX-02 plasma concentration following the subcutaneous (SC) dose on Day 29 to 24 hours post-dose
Urine Pharmacokinetics Parameter - Fe12h Day 1
Time Frame: 12 hours
Percent of dose excreted (Fe) in urine on Day 1
Secondary Outcomes
- Number of Patients Experiencing at Least One Treatment-Emergent Adverse Events (TEAEs)(Day 1-29)