Focal segmental glomerulosclerosis MedDRA version: 21.1 Level: PT Classification code 10067757 Term: Focal segmental glomerulosclerosis System Organ Class: 10038359 - Renal and urinary disorders MedDRA version: 21.1 Level: LLT Classification code 10016832 Term: Focal & segmental glomerulosclerosis System Organ Class: 10038359 - Renal and urinary disorders
Conditions
Interventions
Product Name: Repagermanium
Product Code: DMX-200
Pharmaceutical Form: Capsule
INN or Proposed INN: Repagermanium
Current Sponsor code: DMX-200
Concentration unit: mg milligram(s)
Concentration type:
Sponsors
Dimerix Bioscience Pty Ltd
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Key Inclusion Criteria: - A diagnosis of FSGS confirmed by kidney biopsy or documentation of a genetic mutation in a podocyte protein associated with FSGS. NOTE: The biopsy can have been obtained at any time. Diagnosis of FSGS should be based on light microscopy with supportive findings on either electron microscopy or immunofluorescence analysis and the clinical history and disease course consistent with primary FSGS, genetic FSGS, or FSGS of undetermined cause. - 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, or sodium-glucose co-transporter-2 inhibitors, the dose and regimen must be stable for =26 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) (see Appendix 3 for calculation details). - Seated blood pressure =160/100 mm Hg (mean of 3 values) at Screening. - Body mass index =40 kg/m2 at Screening. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 263 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 23
Exclusion criteria
Exclusion criteria: Key Exclusion Criteria: - Has FSGS secondary to another condition. - 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 26 weeks prior to Screening.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the efficacy of DMX-200 in terms of urine PCR and eGFR slope in patients with FSGS who are receiving an ARB;Secondary Objective: - To evaluate the safety and tolerability of treatment with DMX-200 in patients with FSGS who are receiving an ARB - To evaluate the effect of DMX-200 on kidney function parameters including proteinuria in patients with FSGS who are receiving an ARB;Primary end point(s): 1.Percent change in urine PCR (based on 24-hour urine collection) following treatment with DMX-200 compared with placebo 2.Slope of eGFR following treatment with DMX-200 compared with placebo ;Timepoint(s) of evaluation of this end point: 1. from Baseline to Week 35 2. from Baseline to Week 104 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Incidence and severity of AEs following treatment with DMX-200 compared with placebo 2. Incidence of clinically significant changes in the safety profile of patients treated with DMX-200 compared with placebo, as measured by changes from baseline in clinical laboratory evaluations (hematology, coagulation, clinical chemistry, and urinalysis), ECGs, vital signs, and physical examinations 3. Proportion of responders and non-responders following treatment with DMX-200 compared with placebo, defined as: - Complete response: 24-hour urine PCR reduction to <0.3 g/g [<33.9 mg/mmol] - Modified partial remission (FPRE): 24-hour urine PCR reduction =40% from Baseline and <1.5 g/g [<169.5 mg/mmol] - No response (failure to meet any response criteria) 4. Proportion of patients on treatment with DMX 200 compared with placebo that meet a composite endpoint of worsening in kidney function, as defined by the onset of kidney failure (initiation of chronic dialysis, kidney transplantation, or a sustained eGFR of <15 mL/min/1.73 m2), a 30% decline in eGFR from Baseline, or death from kidney or cardiovascular causes ;Timepoint(s) of evaluation of this end point: 1 to 4. Throughout study duration | — |
Countries
Argentina, Australia, Brazil, Denmark, France, Hong Kong, New Zealand, Spain, Taiwan, United Kingdom, United States
Contacts
Public ContactClinical Trials Information
Dimerix Bioscience Pty Ltd
Outcome results
None listed