Clinical trial NCT04235621: 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)
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)
Study type: OBSERVATIONAL
Overall status: TERMINATED
Conditions: Glomerulosclerosis, Focal Segmental
Minimal Change Disease
Diabetic Nephropathies
Lead sponsor: Goldfinch Bio, Inc.
Sponsor class: INDUSTRY
First posted: 2020-01-22
Enrollment: 20
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients will be drawn from participating investigators' clinics

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.


Eligibility criteria: 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).

Primary outcomes:
measure: Change in Urine Protein-to-Creatinine Ratio (UPCR)
timeFrame: Approximately 1 year
measure: Change in Urine Albumin-to-Creatinine Ratio (UACR)
timeFrame: Approximately 1 year
measure: Estimated Glomerular Filtration Rate (eGFR)
timeFrame: Baseline/Biomarker collection visit
measure: Change in Estimated Glomerular Filtration Rate (eGFR)
timeFrame: Approximately 8 weeks
measure: Change in Urine Biomarker: Nephrin
timeFrame: Approximately 1 year
measure: Change in Urine Biomarker: Podocin
timeFrame: Approximately 1 year
measure: Change in Urine Biomarker: Rac1
timeFrame: Approximately 1 year
measure: Change in Urine Biomarker: Synaptopodin
timeFrame: Approximately 1 year
measure: Change in Urine Biomarker: Urea
timeFrame: Approximately 1 year
measure: Change in Urine Biomarker: Other Exploratory
timeFrame: Approximately 1 year
measure: Change in Serum/Plasma Biomarker: Other Exploratory
timeFrame: Approximately 8 weeks
measure: Number of patients with genetic variants predicted to be associated with chronic kidney disease and functional consequence
timeFrame: Baseline/Biomarker collection visit
measure: Gene expression profile and phenotype of inducible pluripotent stem cell (iPSC)-generated organoids
timeFrame: Baseline/Biomarker collection visit
measure: Change from Baseline Patient-reported Assessment of FSGS Symptoms
timeFrame: Approximately 8 weeks
measure: Change from Baseline Patient-reported Assessment of Health Status
timeFrame: Approximately 8 weeks
measure: Change from Baseline Patient-reported Assessment of Fatigue
timeFrame: Approximately 8 weeks
measure: Change from Baseline Clinician-reported Assessment of Edema
timeFrame: Approximately 8 weeks
measure: Incidence of Untoward Medical Occurrences
timeFrame: Approximately 1 year
measure: % of Patients with Change in Treatment
timeFrame: Approximately 1 year
Secondary outcomes:
Other outcomes:
Locations:
city: Mesa
country: United States
facility: Aventiv Research - Phoenix
state: Arizona
city: Glendale
country: United States
facility: Glendale Adventist Medical Center
state: California
city: Victorville
country: United States
facility: DaVita Mojave Sage Dialysis
state: California
city: Denver
country: United States
facility: Colorado Kidney Care
state: Colorado
city: Springfield
country: United States
facility: Renal and Transplant Associates of New England, PC
state: Massachusetts
city: Roseville
country: United States
facility: St. Clair Nephrology
state: Michigan
city: Minneapolis
country: United States
facility: DaVita Clinical Research
state: Minnesota
city: Kansas City
country: United States
facility: Clinical Research Consultants
state: Missouri
city: Las Vegas
country: United States
facility: DaVita Pelican Point Dialysis
state: Nevada
city: Gallup
country: United States
facility: High Desert Nephrology
state: New Mexico
city: Akron
country: United States
facility: Akron Nephrology Associates, Inc.
state: Ohio
city: Chattanooga
country: United States
facility: Southeast Renal Research Institute
state: Tennessee
city: Arlington
country: United States
facility: Arlington Nephrology, PC
state: Texas
city: Lewisville
country: United States
facility: North Texas Kidney Disease Association
state: Texas
city: San Antonio
country: United States
facility: South Texas Renal Care Group (Downtown)
state: Texas
city: San Antonio
country: United States
facility: San Antonio Kidney Disease Center Physicians Group (Wurzbach Road)
state: Texas
city: San Antonio
country: United States
facility: South Texas Renal Care Group (Westover Hills)
state: Texas
city: San Antonio
country: United States
facility: San Antonio Kidney Disease Center Physicians Group (Carnoustie Drive)
state: Texas
Study officials:
References: