Efficacy and safety of DMX-200 in patients with focal segmental glomerulosclerosis

Recruitment & Eligibility

Status: Recruiting

Sex: All

Target Recruitment: 326

Inclusion Criteria

Primary FSGS, genetic FSGS, or FSGS of undetermined cause (FSGS-UC). NOTE: Primary FSGS and FSGS-UC must be biopsy-proven. The kidney biopsy could have been obtained at any time and should be based on light microscopy with supportive findings on either electron microscopy or immunofluorescence. No biopsy is required where there is a documented genetic mutation of a podocyte protein associated with FSGS. All patients should demonstrate a clinical history and disease course consistent with FSGS, OLE: The patient received blinded IP throughout the duration of the double-blind period up to the Week 104 (EOT – DB) visit (ie, did not permanently discontinue the IP)., Must be either receiving an ARB at the maximal tolerated dose and = 50% of the maximum recommended dose per the product label prior to Screening, or willing to transition to this treatment, If taking corticosteroids, the dosage must be stable for =4 weeks prior to Screening and during Stabilization, and patients must have no plan to change their treatment regimen during study, If taking aldosterone inhibitors, mineralocorticoid receptor antagonists, direct renin inhibitors, sodium-glucose co-transporter-2 inhibitors, or endothelin receptor antagonists (including dual antagonists), the dose and regimen must be stable for =12 weeks prior to Screening and during Stabilization and patients must have no plan to change their treatment regimen during the study, Urine protein/creatinine ratio (PCR) >1.5 g/g (>169.5 mg/mmol) or 24-hour total protein >1.5 g/day based on 24-hour urine collection during Screening, Estimated glomerular filtration rate (eGFR) =25 mL/min/1.73 m2 at Screening using the CKD Epidemiology Collaboration (CKD-EPI) Creatinine Equation (2009) for patients =18 years of age or the Modified Schwartz formula for patients <18 years of age, Seated blood pressure =160/100 mm Hg (mean of 3 values) (patients =18 years of age) or between the 5th and 95th percentile for age, sex, and height (patients <18 years of age) at Screening, Body weight =35 kg (all patients) AND a body mass index (BMI) =40 kg/m2 (patients =18 years of age) or between the 5th and 98th percentile for age and sex (patients <18 years of age) at Screening. For patients with moderate or severe edema, BMI will be calculated based on remission or premorbid weight measured within 3 months prior to Screening, if available. If not available, BMI will be calculated based on the estimated dry weight, based on the Investigator’s clinical judgment, OLE: Patients who have completed participation in the double-blind period, including the Week 104 visit, and who may derive benefit from (continued) treatment with DMX-200, and/or continued follow-up, as assessed by the Investigator, in consultation and agreement with the Medical Monitor.

Exclusion Criteria

Has FSGS secondary to another condition, OLE: Any safety concerns identified during the double-blind period which, in the Investigator’s opinion, may interfere with the patient’s continued participation during the OLE period., History of type 1 diabetes mellitus, or uncontrolled type 2 diabetes mellitus (defined as glycated hemoglobin >8%), History of lymphoma, leukemia, or any active malignancy within the past 2 years (except for basal cell or squamous cell carcinomas of the skin or cervical carcinoma in situ that have been resected and with no evidence of metastatic disease), Active clinically significant hepatobiliary disease, Documented history of heart failure (New York Heart Association Class III/IV) or a major adverse cardiac event within 12 weeks prior to Screening, Serum potassium levels >5.5 mmol/L at Screening, Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) >2 × upper limit of normal at Screening, Treatment with immunosuppressant biological drugs, calcineurin inhibitors, cyclophosphamide, azathioprine, or mycophenolate mofetil within 12 weeks prior to Screening, OLE: The patient has met the criteria for permanent IP discontinuation as defined in Section 7.1 or study discontinuation as defined in Section 7.2 at Week 104, or prior to the first open-label dose of DMX 200 at Week 108.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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