Phase 1 Study of ELX-02 in Healthy Adult Subjects
- Registration Number
- NCT03309605
- Lead Sponsor
- Eloxx Pharmaceuticals, Inc.
- 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 62
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Placebo Comparator Arm Placebo Placebo ELX-02 ELX-02 ELX-02
- Primary Outcome Measures
Name Time Method Pharmacokinetic Parameter - Plasma AUC0-inf 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 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 24 h Renal clearance on Day 29 (CLR=Ae24h/plasmaAUC24h)
Urine Pharmacokinetics Parameter - Fe 12h on Day 29 12 h on Day 29 Percent of dose excreted (Fe) in urine on Day 29
Pharmacokinetic Parameter - Plasma Tmax 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 Parameter - Plasma Rac(AUC24h) Day 1 and Day 24 hr Accumulation ratio, calculated as AUC24h Day29/AUC24h Day 1
Pharmacokinetic Parameter - Plasma RAC(Cmax) Day 1 and Day 29 Accumulation ratio, calculated as Cmax Day29/Cmax Day 1
Pharmacokinetic Parameters - Plasma Cmax 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
Urine Pharmacokinetics Parameter - Ae72h 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 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 24 hours Renal clearance on Day 1 (CLR=Ae24h/plasmaAUC24h)
Pharmacokinetic Parameters - Plasma AUC0-24 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 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 12 hours Percent of dose excreted (Fe) in urine on Day 1
- Secondary Outcome Measures
Name Time Method Number of Patients Experiencing at Least One Treatment-Emergent Adverse Events (TEAEs) Day 1-29 TEAEs are undesirable events not present prior to medical treatment, or an already present event that worsens either in intensity or frequency following the study treatment
Trial Locations
- Locations (1)
SGS Life Sciences, Clinical Pharmacology Unit
🇧🇪Antwerp, Belgium