A Study to Understand the Genetics and Clinical Course of Focal Segmental Glomerulosclerosis (FSGS), Treatment-Resistant Minimal Change Disease (TR-MCD), and Diabetic Nephropathy (DN)
- Conditions
- Glomerulosclerosis, Focal SegmentalDiabetic NephropathiesMinimal Change Disease
- Interventions
- Other: Diabetic Nephropathy (DN)Other: FSGS/TR-MCD
- Registration Number
- NCT04235621
- Lead Sponsor
- Goldfinch Bio, Inc.
- Brief Summary
This is a study with 2 parts. Part 1 comprises a visit to collect biological samples necessary for the molecular characterization of chronic kidney disease. Part 2 comprises an observational period of 5 visits over a period up to 8 weeks. During Part 2, baseline tests will be conducted, and urine will be collected approximately every 2 weeks for 8 weeks. Patients may participate in Part 1, Part 2, or both, and will be followed for up to 1 year consisting of data collection from the patient's medical records and home collection of urine samples every 4 months.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 20
For FSGS/TR-MCD patients :
- Competent and willing to provide informed consent and adhere to all study assessments and restrictions.
- Male or female ≥ 18 years of age with FSGS or TR-MCD at the time of providing written informed consent.
- Diagnosis of FSGS or TR-MCD, based on either biopsy or genetic testing.
- Urinary protein to creatinine ratio (UPCR) ≥ 1.0 g/g.
- Estimated glomerular filtration rate (eGFR) ≥ 45 mL/min/1.73 m2.
For DN patients:
- Competent and willing to provide informed consent and adhere to all study assessments and restrictions.
- Male or female ≥ 18 years of age with DN at the time of providing written informed consent.
- Diagnosis of type 2 diabetes
- Urinary albumin to creatinine ratio (UACR) ≥ 150 mg/g.
- Estimated glomerular filtration rate (eGFR) ≥ 45 mL/min/1.73 m2.
For FSGS/TR-MCD patients:
- Evidence of another kidney disease or kidney disease secondary to an infectious process.
- History of human immunodeficiency virus (HIV), hepatitis B, or hepatitis C. Patients whose results are compatible with prior immunization or treatment may be included.
- Body mass index (BMI) > 42 kg/m2.
- Significant history or evidence of clinically significant disorder, condition, current illness, or disease that, in the opinion of the Investigator, would pose a risk to patient safety or interfere with the study evaluation, procedures, or completion (eg, severe cardiac disease, cardiac conduction defect, or severe or chronic hepatobiliary disease).
- History of malignancy not in remission within the last 5 years other than adequately treated basal cell or squamous cell skin cancer or cervical carcinoma in situ.
- History of any organ or bone marrow transplant, including kidney grafts.
- History of alcoholism or drug/chemical abuse within 12 months.
- Preplanned surgery or procedures that would interfere with the conduct of the study.
For DN patients:
- Evidence of another kidney disease or kidney disease secondary to an infectious process.
- History of HIV, hepatitis B, or hepatitis C. Patients whose results are compatible with prior immunization or treatment may be included.
- BMI > 42 kg/m2.
- Significant history or evidence of clinically significant disorder, condition, current illness, or disease that, in the opinion of the Investigator, would pose a risk to patient safety or interfere with the study evaluation, procedures, or completion (eg, severe cardiac disease, cardiac conduction defect, or severe or chronic hepatobiliary disease).
- History of malignancy not in remission within the last 5 years other than adequately treated basal cell or squamous cell skin cancer or cervical carcinoma in situ.
- History of any organ or bone marrow transplant, including kidney grafts.
- History of alcoholism or drug/chemical abuse within 12 months.
- Preplanned surgery or procedures that would interfere with the conduct of the study.
- Renal disease that requires immunosuppressive therapy (currently, or in the past).
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Diabetic Nephropathy (DN) Diabetic Nephropathy (DN) Patients with DN FSGS/TR-MCD FSGS/TR-MCD Patients with FSGS/TR-MCD
- Primary Outcome Measures
Name Time Method Change in Urine Biomarker: Urea Approximately 1 year Gene expression profile and phenotype of inducible pluripotent stem cell (iPSC)-generated organoids Baseline/Biomarker collection visit Generation of iPSC from whole blood sample
Change from Baseline Patient-reported Assessment of FSGS Symptoms Approximately 8 weeks FSGS/TR-MCD patients will assess disease symptomatology utilizing the FSGS Symptom Diary and FSGS Symptom Impact Questionnaire
Change in Urine Biomarker: Synaptopodin Approximately 1 year Number of patients with genetic variants predicted to be associated with chronic kidney disease and functional consequence Baseline/Biomarker collection visit DNA analysis of blood sample
Change from Baseline Patient-reported Assessment of Health Status Approximately 8 weeks Patients will assess health status using the 36-Item Short Form Health Survey (SF-36)
Change in Urine Protein-to-Creatinine Ratio (UPCR) Approximately 1 year Estimated Glomerular Filtration Rate (eGFR) Baseline/Biomarker collection visit Change in Urine Biomarker: Rac1 Approximately 1 year Change in Urine Biomarker: Other Exploratory Approximately 1 year Change from Baseline Patient-reported Assessment of Fatigue Approximately 8 weeks Patients will assess the symptom of fatigue utilizing the Modified Fatigue Impact Scale
Incidence of Untoward Medical Occurrences Approximately 1 year Incidence of untoward medical occurrences that result in death; are life threatening; require inpatient hospitalization or prolongation of existing hospitalization; result in persistent or significant disability/incapacity; results in a congenital anomaly/birth defect; or result in an important medical event.
Change in Urine Biomarker: Podocin Approximately 1 year Change in Serum/Plasma Biomarker: Other Exploratory Approximately 8 weeks Change from Baseline Clinician-reported Assessment of Edema Approximately 8 weeks Clinicians will assess edema in FSGS/TR-MCD patients using a standardized measurement of edema in FSGS/TR-MCD patients
Change in Urine Albumin-to-Creatinine Ratio (UACR) Approximately 1 year Change in Estimated Glomerular Filtration Rate (eGFR) Approximately 8 weeks Change in Urine Biomarker: Nephrin Approximately 1 year % of Patients with Change in Treatment Approximately 1 year Change in treatment as indicated by patient medical record
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (18)
St. Clair Nephrology
🇺🇸Roseville, Michigan, United States
DaVita Clinical Research
🇺🇸Minneapolis, Minnesota, United States
DaVita Pelican Point Dialysis
🇺🇸Las Vegas, Nevada, United States
Southeast Renal Research Institute
🇺🇸Chattanooga, Tennessee, United States
South Texas Renal Care Group (Downtown)
🇺🇸San Antonio, Texas, United States
San Antonio Kidney Disease Center Physicians Group (Wurzbach Road)
🇺🇸San Antonio, Texas, United States
San Antonio Kidney Disease Center Physicians Group (Carnoustie Drive)
🇺🇸San Antonio, Texas, United States
South Texas Renal Care Group (Westover Hills)
🇺🇸San Antonio, Texas, United States
Akron Nephrology Associates, Inc.
🇺🇸Akron, Ohio, United States
Glendale Adventist Medical Center
🇺🇸Glendale, California, United States
Arlington Nephrology, PC
🇺🇸Arlington, Texas, United States
Renal and Transplant Associates of New England, PC
🇺🇸Springfield, Massachusetts, United States
High Desert Nephrology
🇺🇸Gallup, New Mexico, United States
Aventiv Research - Phoenix
🇺🇸Mesa, Arizona, United States
DaVita Mojave Sage Dialysis
🇺🇸Victorville, California, United States
Colorado Kidney Care
🇺🇸Denver, Colorado, United States
Clinical Research Consultants
🇺🇸Kansas City, Missouri, United States
North Texas Kidney Disease Association
🇺🇸Lewisville, Texas, United States