Primary Objectives * To assess the safety and tolerability of RGLS8429 * To assess the impact of RGLS8429 on ADPKD biomarkers Secondary Objectives * To assess the impact of RGLS8429 on height-adjusted total kidney volume (htTKV) * To characterize the pharmacokinetic (PK) properties of RGLS8429 * To assess the impact of RGLS8429 on renal function
This is a randomized, double-blind, placebo-controlled multiple ascending dose and an open-label fixed-dose Phase 1b study consisting of two parts, Part A and Part B. In Part A, multiple ascending doses of RGLS8429 or placebo will be administered via subcutaneous injection to subjects with ADPKD to evaluate the safety, tolerability, pharmacodynamics and pharmacokinetics of RGLS8429. In Part B, a fixed-dose of RGLS8429 will be administered via subcutaneous injection to subjects with ADPKD to evaluate the safety, tolerability, pharmacodynamics and pharmacokinetics of RGLS8429.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
68
Centricity Research Phoenix Multispecialty
Mesa, Arizona, United States
Safety and tolerability of RGLS4829: Incidence of AEs
Incidence of adverse events over time
Time frame: Baseline to Day 113
Impact of RGLS8429 on ADPKD biomarkers
Change from baseline in PC1, PC2, NGAL, and KIM-1 urine biomarkers
Time frame: Baseline to Day 113
Impact of RGLS8429 on height-adjusted total kidney volume (htTKV)
Change from baseline in htTKV
Time frame: Baseline to Day 113
Pharmacokinetic properties of RGLS8429: Cmax
Maximum observed concentration (Cmax) of RGLS8429
Time frame: Baseline to Day 113
Pharmacokinetic properties of RGLS8429: Tmax
Time to maximum observed concentration (Tmax) of RGLS8429
Time frame: Baseline to Day 113
Pharmacokinetic properties of RGLS8429: t½
Half-life of RGLS8429 (t½)
Time frame: Baseline to Day 113
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