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)

A Study to Characterize the Genetic, Biomarker, and Clinical Profile of Patients With Focal Segmental Glomerulosclerosis (FSGS), Treatment-Resistant Minimal Change Disease (TR-MCD), and Diabetic Nephropathy (DN)

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.

Study Overview

• Status: Terminated
• Conditions: Diabetic Nephropathies; Glomerulosclerosis, Focal Segmental; Minimal Change Disease
• Intervention / Treatment: Other: FSGS/TR-MCD; Other: Diabetic Nephropathy (DN)

• Study Type: Observational
• Enrollment (Actual): 20

Contacts and Locations

Study Locations

• United States
  • Arizona
    • Mesa, Arizona, United States, 85210-6041
      • Aventiv Research - Phoenix
  • California
    • Glendale, California, United States, 91206
      • Glendale Adventist Medical Center
    • Victorville, California, United States, 92395-8322
      • DaVita Mojave Sage Dialysis
  • Colorado
    • Denver, Colorado, United States, 80230-7200
      • Colorado Kidney Care
  • Massachusetts
    • Springfield, Massachusetts, United States, 01107-1369
      • Renal and Transplant Associates of New England, PC
  • Michigan
    • Roseville, Michigan, United States, 48066
      • St. Clair Nephrology
  • Minnesota
    • Minneapolis, Minnesota, United States, 55404-1212
      • Davita Clinical Research
  • Missouri
    • Kansas City, Missouri, United States, 64111
      • Clinical Research Consultants
  • Nevada
    • Las Vegas, Nevada, United States, 89129-6201
      • DaVita Pelican Point Dialysis
  • New Mexico
    • Gallup, New Mexico, United States, 87301-5655
      • High Desert Nephrology
  • Ohio
    • Akron, Ohio, United States, 44302-1715
      • Akron Nephrology Associates, Inc.
  • Tennessee
    • Chattanooga, Tennessee, United States, 37404-2743
      • Southeast Renal Research Institute
  • Texas
    • Arlington, Texas, United States, 76015
      • Arlington Nephrology, PC
    • Lewisville, Texas, United States, 75057-6039
      • North Texas Kidney Disease Association
    • San Antonio, Texas, United States, 78207
      • South Texas Renal Care Group (Downtown)
    • San Antonio, Texas, United States, 78229-3809
      • San Antonio Kidney Disease Center Physicians Group (Wurzbach Road)
    • San Antonio, Texas, United States, 78251-1230
      • South Texas Renal Care Group (Westover Hills)
    • San Antonio, Texas, United States, 78258-4800
      • San Antonio Kidney Disease Center Physicians Group (Carnoustie Drive)

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Patients will be drawn from participating investigators' clinics

Description

Inclusion Criteria:

For FSGS/TR-MCD patients :

1. Competent and willing to provide informed consent and adhere to all study assessments and restrictions.
2. Male or female ≥ 18 years of age with FSGS or TR-MCD at the time of providing written informed consent.
3. Diagnosis of FSGS or TR-MCD, based on either biopsy or genetic testing.
4. Urinary protein to creatinine ratio (UPCR) ≥ 1.0 g/g.
5. Estimated glomerular filtration rate (eGFR) ≥ 45 mL/min/1.73 m2.

For DN patients:

1. Competent and willing to provide informed consent and adhere to all study assessments and restrictions.
2. Male or female ≥ 18 years of age with DN at the time of providing written informed consent.
3. Diagnosis of type 2 diabetes
4. Urinary albumin to creatinine ratio (UACR) ≥ 150 mg/g.
5. Estimated glomerular filtration rate (eGFR) ≥ 45 mL/min/1.73 m2.

Exclusion Criteria:

For FSGS/TR-MCD patients:

1. Evidence of another kidney disease or kidney disease secondary to an infectious process.
2. History of human immunodeficiency virus (HIV), hepatitis B, or hepatitis C. Patients whose results are compatible with prior immunization or treatment may be included.
3. Body mass index (BMI) > 42 kg/m2.
4. 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).
5. 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.
6. History of any organ or bone marrow transplant, including kidney grafts.
7. History of alcoholism or drug/chemical abuse within 12 months.
8. Preplanned surgery or procedures that would interfere with the conduct of the study.

For DN patients:

1. Evidence of another kidney disease or kidney disease secondary to an infectious process.
2. History of HIV, hepatitis B, or hepatitis C. Patients whose results are compatible with prior immunization or treatment may be included.
3. BMI > 42 kg/m2.
4. 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).
5. 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.
6. History of any organ or bone marrow transplant, including kidney grafts.
7. History of alcoholism or drug/chemical abuse within 12 months.
8. Preplanned surgery or procedures that would interfere with the conduct of the study.
9. Renal disease that requires immunosuppressive therapy (currently, or in the past).

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
FSGS/TR-MCD; Patients with FSGS/TR-MCD	Other: FSGS/TR-MCD; This is a non-interventional study
Diabetic Nephropathy (DN); Patients with DN	Other: Diabetic Nephropathy (DN); This is a non-interventional study

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in Urine Protein-to-Creatinine Ratio (UPCR)		Approximately 1 year
Change in Urine Albumin-to-Creatinine Ratio (UACR)		Approximately 1 year
Estimated Glomerular Filtration Rate (eGFR)		Baseline/Biomarker collection visit
Change in Estimated Glomerular Filtration Rate (eGFR)		Approximately 8 weeks
Change in Urine Biomarker: Nephrin		Approximately 1 year
Change in Urine Biomarker: Podocin		Approximately 1 year
Change in Urine Biomarker: Rac1		Approximately 1 year
Change in Urine Biomarker: Synaptopodin		Approximately 1 year
Change in Urine Biomarker: Urea		Approximately 1 year
Change in Urine Biomarker: Other Exploratory		Approximately 1 year
Change in Serum/Plasma Biomarker: Other Exploratory		Approximately 8 weeks
Number of patients with genetic variants predicted to be associated with chronic kidney disease and functional consequence	DNA analysis of blood sample	Baseline/Biomarker collection visit
Gene expression profile and phenotype of inducible pluripotent stem cell (iPSC)-generated organoids	Generation of iPSC from whole blood sample	Baseline/Biomarker collection visit
Change from Baseline Patient-reported Assessment of FSGS Symptoms	FSGS/TR-MCD patients will assess disease symptomatology utilizing the FSGS Symptom Diary and FSGS Symptom Impact Questionnaire	Approximately 8 weeks
Change from Baseline Patient-reported Assessment of Health Status	Patients will assess health status using the 36-Item Short Form Health Survey (SF-36)	Approximately 8 weeks
Change from Baseline Patient-reported Assessment of Fatigue	Patients will assess the symptom of fatigue utilizing the Modified Fatigue Impact Scale	Approximately 8 weeks
Change from Baseline Clinician-reported Assessment of Edema	Clinicians will assess edema in FSGS/TR-MCD patients using a standardized measurement of edema in FSGS/TR-MCD patients	Approximately 8 weeks
Incidence of Untoward Medical Occurrences	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.	Approximately 1 year
% of Patients with Change in Treatment	Change in treatment as indicated by patient medical record	Approximately 1 year

Collaborators and Investigators

• Sponsor: Goldfinch Bio, Inc.

Study record dates

Study Major Dates

• Study Start (ACTUAL): December 20, 2019
• Primary Completion (ACTUAL): May 27, 2020
• Study Completion (ACTUAL): May 27, 2020

Study Registration Dates

• First Submitted: December 31, 2019
• First Submitted That Met QC Criteria: January 16, 2020
• First Posted (ACTUAL): January 22, 2020

Study Record Updates

• Last Update Posted (ACTUAL): July 20, 2021
• Last Update Submitted That Met QC Criteria: July 17, 2021
• Last Verified: July 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Urologic Diseases; Endocrine System Diseases; Diabetes Complications; Diabetes Mellitus; Nephritis; Glomerulonephritis; Nephrosis; Kidney Diseases; Diabetic Nephropathies; Glomerulosclerosis, Focal Segmental; Nephrosis, Lipoid
• Other Study ID Numbers: GFB-887-501

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Patient reported outcome (and other relevant data) will be shared with other researchers.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No