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

Phase 2

Withdrawn

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

Detailed Description: 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.

Recruitment & Eligibility

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

Arm && Interventions

Group	Intervention	Description
ADX-629	ADX-629	-

Primary Outcome Measures

Name	Time	Method
Adverse Event (AE) Query	From Day 1 to Day 90	Incidence and severity of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)

Secondary Outcome Measures

Name	Time	Method
Relapse Frequency	From Day 1 to Day 90	Proportion of subjects with one or more relapse(s), as defined by treatment with corticosteroid therapy

Locations (11): University of Colorado
🇺🇸
Aurora, Colorado, United States
Emory University - Pediatric Nephrology
🇺🇸
Atlanta, Georgia, United States
Nevada Kidney Disease Hypertension Center (NKDHC)
🇺🇸
Las Vegas, Nevada, United States
East Carolina University - Nephrology
🇺🇸
Greenville, North Carolina, United States
The Ohio State University (OSU) Wexner Medical Center
🇺🇸
Columbus, Ohio, United States
Amicis Research Center - Northridge
🇺🇸
Northridge, California, United States
South Florida Research Institute
🇺🇸
Lauderdale Lakes, Florida, United States
Northwest Louisiana Nephrology
🇺🇸
Shreveport, Louisiana, United States
ClinCept, LLC
🇺🇸
Bremen, Georgia, United States
Southern Utah Kidney and Hypertension Center
🇺🇸
Saint George, Utah, United States
University of Toledo Medical Center
🇺🇸
Toledo, Ohio, United States