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)

Diabetic Nephropathies Clinical Trial

Official title:

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)

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT04235621
• Other study ID #: GFB-887-501
• Status: Terminated
• Start date: December 20, 2019
• Est. completion date: May 27, 2020

Study information

• Verified date: July 2021
• Source: Goldfinch Bio, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 20
• Est. completion date: May 27, 2020
• Est. primary completion date: May 27, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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).

Related Conditions & MeSH terms

• Diabetic Nephropathies
• Glomerulosclerosis, Focal Segmental
• Kidney Diseases
• Minimal Change Disease
• Nephrosis, Lipoid

Intervention

Other:
• FSGS/TR-MCD
This is a non-interventional study
• Diabetic Nephropathy (DN)
This is a non-interventional study

Locations

Country	Name	City	State
United States	Akron Nephrology Associates, Inc.	Akron	Ohio
United States	Arlington Nephrology, PC	Arlington	Texas
United States	Southeast Renal Research Institute	Chattanooga	Tennessee
United States	Colorado Kidney Care	Denver	Colorado
United States	High Desert Nephrology	Gallup	New Mexico
United States	Glendale Adventist Medical Center	Glendale	California
United States	Clinical Research Consultants	Kansas City	Missouri
United States	DaVita Pelican Point Dialysis	Las Vegas	Nevada
United States	North Texas Kidney Disease Association	Lewisville	Texas
United States	Aventiv Research - Phoenix	Mesa	Arizona
United States	DaVita Clinical Research	Minneapolis	Minnesota
United States	St. Clair Nephrology	Roseville	Michigan
United States	San Antonio Kidney Disease Center Physicians Group (Carnoustie Drive)	San Antonio	Texas
United States	San Antonio Kidney Disease Center Physicians Group (Wurzbach Road)	San Antonio	Texas
United States	South Texas Renal Care Group (Downtown)	San Antonio	Texas
United States	South Texas Renal Care Group (Westover Hills)	San Antonio	Texas
United States	Renal and Transplant Associates of New England, PC	Springfield	Massachusetts
United States	DaVita Mojave Sage Dialysis	Victorville	California

Sponsors (1)

Lead Sponsor	Collaborator
Goldfinch Bio, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in Urine Protein-to-Creatinine Ratio (UPCR)		Approximately 1 year
Primary	Change in Urine Albumin-to-Creatinine Ratio (UACR)		Approximately 1 year
Primary	Estimated Glomerular Filtration Rate (eGFR)		Baseline/Biomarker collection visit
Primary	Change in Estimated Glomerular Filtration Rate (eGFR)		Approximately 8 weeks
Primary	Change in Urine Biomarker: Nephrin		Approximately 1 year
Primary	Change in Urine Biomarker: Podocin		Approximately 1 year
Primary	Change in Urine Biomarker: Rac1		Approximately 1 year
Primary	Change in Urine Biomarker: Synaptopodin		Approximately 1 year
Primary	Change in Urine Biomarker: Urea		Approximately 1 year
Primary	Change in Urine Biomarker: Other Exploratory		Approximately 1 year
Primary	Change in Serum/Plasma Biomarker: Other Exploratory		Approximately 8 weeks
Primary	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
Primary	Gene expression profile and phenotype of inducible pluripotent stem cell (iPSC)-generated organoids	Generation of iPSC from whole blood sample	Baseline/Biomarker collection visit
Primary	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
Primary	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
Primary	Change from Baseline Patient-reported Assessment of Fatigue	Patients will assess the symptom of fatigue utilizing the Modified Fatigue Impact Scale	Approximately 8 weeks
Primary	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
Primary	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
Primary	% of Patients with Change in Treatment	Change in treatment as indicated by patient medical record	Approximately 1 year