Phase 1 - Pharmacokinetics in Patients with Impaired Renal Function
The study is a two-center, Phase 1, open-label, single-dose, one-period, four-parallel-group, PK study in subjects with various severities of renal dysfunction and healthy volunteers. Subjects will be categorized in 4 groups: Group 1: subjects with mild renal impairment Group 2: subjects with moderate renal impairment Group 3: subjects with severe renal impairment Group 4 (control group): subjects with normal renal function The mild (group 1) and moderate (group 2) patients with renal disease will be dosed first, in a parallel fashion. At this point, interim PK analyses will be performed and a safety committee composed of Sponsor and Contract Research Organization (CRO) members will jointly review the PK data before dosing the patients with severe renal disease (group 3). Control subjects (group 4) will be recruited after the recruitment of groups 1 to 3.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
24
ELX-02 is a synthetic, designer eukaryotic ribosomal specific glycoside (ERSG) optimized as a translational read-through drug. All groups will get the same treatment.
Inventiv Health Clinical -Research Pharmacy Unit
Miami, Florida, United States
University of Miami
Miami, Florida, United States
Pharmacokinetic Parameters- Plasma AUC0-24
Area under the curve (AUC0-24) of ELX-02 plasma concentration following a single subcutaneous (SC) dose
Time frame: 0.25, 0.5, 0.75, 1, 2, 4, 6, 12, and 24 hours after dosing
Pharmacokinetic Parameters- Plasma Cmax
Peak Plasma Concentration (Cmax) of ELX-02 following a single subcutaneous (SC) dose in subjects with normal renal function, mild, moderate, or severe renal impairment
Time frame: 0.25, 0.5, 0.75, 1, 2, 4, 6, 12, 24, 36, 48, 72, and 168 (Day 8) hours after dosing
AUC0-inf
Area under the curve (AUC0-inf) of ELX-02 plasma concentration following a single subcutaneous (SC) dose
Time frame: 0.25, 0.5, 0.75, 1, 2, 4, 6, 12, 24, 36, 48, 72, and 168 (Day 8) hours after dosing
Pharmacokinetic Parameters - Plasma Tmax
Time to maximum concentration (Tmax) of ELX-02 plasma concentration following a single subcutaneous (SC) dose
Time frame: 0.25, 0.5, 0.75, 1, 2, 4, 6, 12, and 24 hours after dosing.
Urine Pharmacokinetics Parameter - Ae0-t
Cumulative amount of unchanged drug excreted into urine (Ae0-t) of ELX-02 following a single subcutaneous (SC) dose
Time frame: Pre-dose (first void in the morning of Day 1), 0-3, 3-6, 6-9, 9-12, 12-18, 18-24, 24-36, 36-48, and 48-72 hours post-dose
Urine Pharmacokinetic Parameter - Rmax
Maximum rate of urinary extraction (Rmax) of ELX-02 following a single subcutaneous (SC) dose
Time frame: Pre-dose (first void in the morning of Day 1), 0-3, 3-6, 6-9, 9-12, 12-18, 18-24, 24-36, 36-48, and 48-72 hours post-dose
Number of Patients Reporting Treatment-Emergent Adverse Events (TEAEs) [Safety]
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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
Time frame: 1-8 days