A Phase 2a Study of LLX-424 in Patients With a History of Kidney Stones

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

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.

Study Overview

• Status: Completed
• Conditions: Kidney Stones
• Intervention / Treatment: Drug: LLX-424

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).

• Study Type: Interventional
• Enrollment (Actual): 51
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Minnesota
    • Saint Paul, Minnesota, United States, 55114
      • Clinical Research Site #2
  • Missouri
    • Springfield, Missouri, United States, 65802
      • Clinical Research Site #3
  • Texas
    • San Antonio, Texas, United States, 78217
      • Clinical Research Site #1

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: LLX-424; Tablets for oral administration	Drug: LLX-424; Tablets for oral administration; Other Names: glycolate oxidase inhibitor
Placebo Comparator: Placebo; Tablets for oral administration	Drug: LLX-424; Tablets for oral administration; Other Names: glycolate oxidase inhibitor

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Treatment-emergent adverse events (TEAEs)	Percentage of subjects with TEAEs	From the first dose and for up to 9 weeks
Percentage of subjects with study drug-related severe laboratory-based AEs	Laboratory-based adverse events	From the first dose and for up to 9 weeks
QT interval on ECG	Percentage of subjects with an increase from baseline in the QTcF by > 60 milliseconds	From the first dose and for up to 9 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Mean change from baseline in 24-hour urine oxalate excretion rate	Comparison between LLX-424 group and the placebo group	From pre-dose to Study Day 56
Peak plasma concentration (Cmax) of LLX-424	Cmax of plasma LLX-424	From pre-dose to Study Day 56
Area under the curve (AUC) of plasma LLX-424 concentration versus time after dosage	AUC of plasma LLX-424	From pre-dose to Study Day 56
Peak plasma concentration (Cmax) of glycolate	Cmax of plasma glycolate	From pre-dose to Study Day 56
Area under the curve (AUC) of plasma glycolate concentration versus time after dosage	AUC of plasma glycolate	From pre-dose to Study Day 56

Collaborators and Investigators

• Sponsor: Lilac Therapeutics, Inc.

Publications and helpful links

Helpful Links

• Sponsor website

Study record dates

Study Major Dates

• Study Start (Actual): March 23, 2025
• Primary Completion (Actual): September 30, 2025
• Study Completion (Actual): September 30, 2025

Study Registration Dates

• First Submitted: April 2, 2025
• First Submitted That Met QC Criteria: April 9, 2025
• First Posted (Actual): April 17, 2025

Study Record Updates

• Last Update Posted (Estimated): October 29, 2025
• Last Update Submitted That Met QC Criteria: October 27, 2025
• Last Verified: October 1, 2025

More Information

Terms related to this study

• Keywords: oxalate; glycolate oxidase
• Additional Relevant MeSH Terms: Urogenital Diseases; Male Urogenital Diseases; Calculi; Pathological Conditions, Anatomical; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urolithiasis; Urinary Calculi; Nephrolithiasis; Pathological Conditions, Signs and Symptoms; Kidney Calculi; Dermatologic Agents; Keratolytic Agents; Glycolic acid
• Other Study ID Numbers: LLX-002

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No