Part 1 - A Clinical Trial in Patients With Frequently Relapsing and Steroid-Dependent Nephrotic Syndrome

A Phase 2 Clinical Trial Investigating the Safety and Efficacy of ADX-629 in Subjects With Frequently Relapsing and Steroid-Dependent Nephrotic Syndrome

A Clinical Trial Investigating the Safety and Efficacy of ADX-629 in Subjects with Frequently Relapsing and Steroid-Dependent Nephrotic Syndrome

ADX-629-MCD-001 is a multi-center, two-part, Phase 2 clinical trial designed to evaluate the safety and efficacy of ADX-629 in Subjects with frequently relapsing and steroid-dependent nephrotic syndrome. Study drug will be administered orally every 12 hours (twice daily) to eligible subjects for 90 days. The study will enroll approximately 35 children and adults with nephrotic syndrome who have had a nephrotic relapse in the 6 months prior to screening.

The clinical trial is divided into 2 parts: Part 1 (open-label) and Part 2 (double-blinded, randomized).

Part 1: Approximately 5 eligible subjects will receive open-label ADX-629 (250 mg) twice daily for 90 days.

Part 2: Approximately 30 eligible subjects will be randomized in a 2:1 ratio to receive either ADX-629 (250 mg) or matching placebo twice daily for 90 days.

Patients who are interested in participating will be provided detailed information about the study including description of study assessments/procedures, possible side-effects, alternative treatments, and potential benefits.

Inclusion Criteria:

• Adults ≥18 years of age
• History of nephrotic syndrome, characterized by heavy proteinuria and hypoalbuminemia
• Recent nephrotic relapse in the 6 months prior to screening
• Estimated glomerular filtration rate (eGFR) of ≥45 during screening
• Not pregnant, breastfeeding, or lactating and agree to use a highly effective method of acceptable contraceptive for the trial duration, if applicable

Exclusion Criteria:

• Subjects with collapsing FSGS focal segmental glomerulosclerosis (FSGS)
• Known secondary cause of nephrotic syndrome (e.g., diabetic nephropathy, systemic lupus erythematous, sickle cell anemia, malignancy, drug-induced, malaria)
• History of kidney transplantation or other solid organ transplantation
• History of any unstable chronic diseases/conditions, clinically significant abnormalities, or findings, that in the opinion of the Investigator, could compromise subject safety or affect the conduct of the trial