Clinical trial NCT06932146: A Phase 2a Study of LLX-424 in Patients With a History of Kidney Stones
Official title: A Phase 2a Randomized, Double-blind, Placebo-controlled Study of the Safety, Tolerability, Pharmacokinetics (PK), and Pharmacodynamic (PD) Effects of LLX-424 in Patients With a History of Kidney Stones
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Kidney Stones
Keywords: oxalate
glycolate oxidase
Lead sponsor: Lilac Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2025-04-17
Enrollment: 51
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This is a Phase 2a study of glycolate oxidase inhibitor LLX-424 in patients with a history of kidney stones. Eligible subjects with a history of kidney stones will be randomized to receive study drug (LLX-424 or placebo) for 8 weeks.

Detailed description: This is a Phase 2a, randomized, double-blind, placebo-controlled study designed to assess the safety, tolerability, PK, and PD effects of LLX-424 in patients with a history of kidney stones.

Following completion of screening procedures, on Study Day 1, eligible subjects will be randomized in a 2:1 ratio to receive either LLX-424 or matching placebo, respectively.

An outpatient safety Follow-up Visit will be scheduled on Day 63 or 7 days after the last dose (end of study visit).

Eligibility criteria: Inclusion Criteria:

* History of kidney stones documented in medical records
* 24-hour urine oxalate excretion
* Body mass index 18.5 to 38 kg/m2 inclusive
* Estimated glomerular filtration rate ≥ 60 mL/min/1.73m2

Exclusion Criteria:

* History or medical record evidence of kidney stones that are not calcium oxalate
* Unstable kidney function
* Spot urine protein to creatinine ratio ≥ 1 mg/mg at screening
* History of kidney transplantation
* Hemoglobin A1c \> 9.5% at screening
* History of enteric hyperoxaluria, intestinal malabsorption, bariatric surgery, intestinal surgery, inflammatory bowel disease
* History or evidence of cirrhosis

Primary outcomes:
measure: Treatment-emergent adverse events (TEAEs)
timeFrame: From the first dose and for up to 9 weeks
measure: Percentage of subjects with study drug-related severe laboratory-based AEs
timeFrame: From the first dose and for up to 9 weeks
measure: QT interval on ECG
timeFrame: From the first dose and for up to 9 weeks
Secondary outcomes:
measure: Mean change from baseline in 24-hour urine oxalate excretion rate
timeFrame: From pre-dose to Study Day 56
measure: Peak plasma concentration (Cmax) of LLX-424
timeFrame: From pre-dose to Study Day 56
measure: Area under the curve (AUC) of plasma LLX-424 concentration versus time after dosage
timeFrame: From pre-dose to Study Day 56
measure: Peak plasma concentration (Cmax) of glycolate
timeFrame: From pre-dose to Study Day 56
measure: Area under the curve (AUC) of plasma glycolate concentration versus time after dosage
timeFrame: From pre-dose to Study Day 56
Other outcomes:
Locations:
city: Saint Paul
country: United States
facility: Clinical Research Site #2
state: Minnesota
city: Springfield
country: United States
facility: Clinical Research Site #3
state: Missouri
city: San Antonio
country: United States
facility: Clinical Research Site #1
state: Texas
Study officials:
References: