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

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

Detailed Description
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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.

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

Conditions
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Nephrotic Syndrome

Intervention / Treatment
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* Drug: ADX-629


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: ADX-629<br> | Drug: ADX-629<br>* ADX-629 (250 milligrams) administered twice daily<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Adverse Event (AE) Query | Incidence and severity of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) | From Day 1 to Day 90 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Relapse Frequency | Proportion of subjects with one or more relapse(s), as defined by treatment with corticosteroid therapy | From Day 1 to Day 90 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Idiopathic nephrotic syndrome, Focal segmental glomerulosclerosis (FSGS), Minimal change disease, Frequently relapsing nephrotic syndrome, ADX-629, Reactive aldehyde species (RASP), Chronic kidney disease