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)

NCT04235621 | Terminated

• 1 other identifier: GFB-887-501
• Study Type: observational
• Target: 20
• Locations: 1 country
• Sites: 18

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.

Conditions

Glomerulosclerosis, Focal Segmental; Minimal Change Disease; Diabetic Nephropathies

Outcome Measures

Primary Outcomes (19)

• 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

Study Arms (2)

FSGS/TR-MCD

Patients with FSGS/TR-MCD

Other: FSGS/TR-MCD

Diabetic Nephropathy (DN)

Patients with DN

Other: Diabetic Nephropathy (DN)

Interventions

FSGS/TR-MCD OTHER

This is a non-interventional study

FSGS/TR-MCD

Diabetic Nephropathy (DN) OTHER

This is a non-interventional study

Diabetic Nephropathy (DN)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients will be drawn from participating investigators' clinics

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Goldfinch Bio, Inc.: lead

Study Sites (18)

Aventiv Research - Phoenix

Mesa, Arizona, 85210-6041, United States

Location

Glendale Adventist Medical Center

Glendale, California, 91206, United States

Location

DaVita Mojave Sage Dialysis

Victorville, California, 92395-8322, United States

Location

Colorado Kidney Care

Denver, Colorado, 80230-7200, United States

Location

Renal and Transplant Associates of New England, PC

Springfield, Massachusetts, 01107-1369, United States

Location

St. Clair Nephrology

Roseville, Michigan, 48066, United States

Location

DaVita Clinical Research

Minneapolis, Minnesota, 55404-1212, United States

Location

Clinical Research Consultants

Kansas City, Missouri, 64111, United States

Location

DaVita Pelican Point Dialysis

Las Vegas, Nevada, 89129-6201, United States

Location

High Desert Nephrology

Gallup, New Mexico, 87301-5655, United States

Location

Akron Nephrology Associates, Inc.

Akron, Ohio, 44302-1715, United States

Location

Southeast Renal Research Institute

Chattanooga, Tennessee, 37404-2743, United States

Location

Arlington Nephrology, PC

Arlington, Texas, 76015, United States

Location

North Texas Kidney Disease Association

Lewisville, Texas, 75057-6039, United States

Location

South Texas Renal Care Group (Downtown)

San Antonio, Texas, 78207, United States

Location

San Antonio Kidney Disease Center Physicians Group (Wurzbach Road)

San Antonio, Texas, 78229-3809, United States

Location

South Texas Renal Care Group (Westover Hills)

San Antonio, Texas, 78251-1230, United States

Location

San Antonio Kidney Disease Center Physicians Group (Carnoustie Drive)

San Antonio, Texas, 78258-4800, United States

Location

Biospecimen

Retention: SAMPLES WITH DNA

whole blood, urine

MeSH Terms

Conditions

Glomerulosclerosis, Focal Segmental; Nephrosis, Lipoid; Diabetic Nephropathies

Condition Hierarchy (Ancestors)

Glomerulonephritis; Nephritis; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Nephrosis; Diabetes Complications; Diabetes Mellitus; Endocrine System Diseases

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 31, 2019
• First Posted: January 22, 2020
• Study Start: December 20, 2019
• Primary Completion: May 27, 2020
• Study Completion: May 27, 2020
• Last Updated: July 20, 2021

Record last verified: 2021-07

Data Sharing

• IPD Sharing: Will share

Locations

US (18)