Nephrotic Syndrome
Conditions
Keywords
Idiopathic nephrotic syndrome, Focal segmental glomerulosclerosis (FSGS), Minimal change disease, Frequently relapsing nephrotic syndrome, ADX-629, Reactive aldehyde species (RASP), Chronic kidney disease
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.
Interventions
DRUGADX-629
ADX-629 (250 milligrams) administered twice daily
Sponsors
Aldeyra Therapeutics, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| Relapse Frequency | From Day 1 to Day 90 | Proportion of subjects with one or more relapse(s), as defined by treatment with corticosteroid therapy |
Countries
United States
Outcome results
None listed