Glomerulosclerosis, Focal SegmentalNCT04235621
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)
An observational study of Glomerulosclerosis, Focal Segmental and Minimal Change Disease, run by Goldfinch Bio, Inc. at 18 sites in United States.
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Glomerulosclerosis, Focal Segmental, Minimal Change Disease, Diabetic Nephropathies
- What is being tested?
- FSGS/TR-MCD and Diabetic Nephropathy (DN)
- How many people
- 20 participants
- Where
- 18 sitesUnited States
- Study dates
- 20 Dec 2019to 27 May 2020
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Patients will be drawn from participating investigators' clinics
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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.
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- 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).
Trial locations and contacts
18 sites in 1 country.
18 sites in 1 country
United States18 sites
Akron Nephrology Associates, Inc.
Akron, Ohio, 44302-1715
Arlington Nephrology, PC
Arlington, Texas, 76015
Southeast Renal Research Institute
Chattanooga, Tennessee, 37404-2743
Colorado Kidney Care
Denver, Colorado, 80230-7200
High Desert Nephrology
Gallup, New Mexico, 87301-5655
Glendale Adventist Medical Center
Glendale, California, 91206
Clinical Research Consultants
Kansas City, Missouri, 64111
DaVita Pelican Point Dialysis
Las Vegas, Nevada, 89129-6201
North Texas Kidney Disease Association
Lewisville, Texas, 75057-6039
Aventiv Research - Phoenix
Mesa, Arizona, 85210-6041
DaVita Clinical Research
Minneapolis, Minnesota, 55404-1212
St. Clair Nephrology
Roseville, Michigan, 48066
South Texas Renal Care Group (Downtown)
San Antonio, Texas, 78207
San Antonio Kidney Disease Center Physicians Group (Wurzbach Road)
San Antonio, Texas, 78229-3809
South Texas Renal Care Group (Westover Hills)
San Antonio, Texas, 78251-1230
San Antonio Kidney Disease Center Physicians Group (Carnoustie Drive)
San Antonio, Texas, 78258-4800
Renal and Transplant Associates of New England, PC
Springfield, Massachusetts, 01107-1369
DaVita Mojave Sage Dialysis
Victorville, California, 92395-8322
About this study
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)
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.
- Being tested
- FSGS/TR-MCD, Diabetic Nephropathy (DN)
- Medical subject headings
- Glomerulosclerosis, Focal Segmental, Nephrosis, Lipoid, Diabetic Nephropathies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FSGS/TR-MCDOther
This is a non-interventional study
Diabetic Nephropathy (DN)Other
This is a non-interventional study
| Study group | Role | What they receive |
|---|---|---|
| FSGS/TR-MCD | Patients with FSGS/TR-MCD Receives: Other: FSGS/TR-MCD | |
| Diabetic Nephropathy (DN) | Patients with DN Receives: Other: Diabetic Nephropathy (DN) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Urine Protein-to-Creatinine Ratio (UPCR)
Measured: Approximately 1 year
Change in Urine Albumin-to-Creatinine Ratio (UACR)
Measured: Approximately 1 year
Estimated Glomerular Filtration Rate (eGFR)
Measured: Baseline/Biomarker collection visit
Change in Estimated Glomerular Filtration Rate (eGFR)
Measured: Approximately 8 weeks
Change in Urine Biomarker: Nephrin
Measured: Approximately 1 year
Change in Urine Biomarker: Podocin
Measured: Approximately 1 year
Change in Urine Biomarker: Rac1
Measured: Approximately 1 year
Change in Urine Biomarker: Synaptopodin
Measured: Approximately 1 year
Change in Urine Biomarker: Urea
Measured: Approximately 1 year
Change in Urine Biomarker: Other Exploratory
Measured: Approximately 1 year
Change in Serum/Plasma Biomarker: Other Exploratory
Measured: 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
Measured: Baseline/Biomarker collection visit
Gene expression profile and phenotype of inducible pluripotent stem cell (iPSC)-generated organoids
Generation of iPSC from whole blood sample
Measured: 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
Measured: 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)
Measured: Approximately 8 weeks
Change from Baseline Patient-reported Assessment of Fatigue
Patients will assess the symptom of fatigue utilizing the Modified Fatigue Impact Scale
Measured: 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
Measured: 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.
Measured: Approximately 1 year
% of Patients with Change in Treatment
Change in treatment as indicated by patient medical record
Measured: Approximately 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jan 2020
- Study start20 Dec 2019
- Primary completion27 May 2020
- Study completion27 May 2020
Why it stopped: Sponsor decision for administrative reasons
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Glomerulosclerosis, Focal SegmentalStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Goldfinch Bio, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04235621
- Sponsor's protocol ID
- GFB-887-501
- Status last verified
- Jul 2021
- First submitted
- 31 Dec 2019
- Last updated
- 20 Jul 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Patient reported outcome (and other relevant data) will be shared with other researchers.
Study at a glance
From the registry record, last updated by the sponsor on 20 Jul 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT04235621
- Acronym / sponsor ID
- GFB-887-501
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Sponsor decision for administrative reasons
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Goldfinch Bio, Inc.Industry (pharma, biotech, devices)
- Study start
- 20 Dec 2019 (actual)
- Primary completion
- 27 May 2020 (actual)
- Study completion
- 27 May 2020 (actual)
- First posted
- 22 Jan 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| FSGS/TR-MCD | – | Other: FSGS/TR-MCDPatients with FSGS/TR-MCD |
| Diabetic Nephropathy (DN) | – | Other: Diabetic Nephropathy (DN)Patients with DN |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients will be drawn from participating investigators' clinics
Key inclusion criteria (12 listed)
- 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.
Key exclusion criteria (19 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| FSGS/TR-MCDThis is a non-interventional study | Other | FSGS/TR-MCD |
| Diabetic Nephropathy (DN)This is a non-interventional study | Other | Diabetic Nephropathy (DN) |
Primary outcome
Change in Urine Protein-to-Creatinine Ratio (UPCR)
Time frame: Approximately 1 year
Change in Urine Albumin-to-Creatinine Ratio (UACR)
Time frame: Approximately 1 year
Estimated Glomerular Filtration Rate (eGFR)
Time frame: Baseline/Biomarker collection visit
Change in Estimated Glomerular Filtration Rate (eGFR)
Time frame: Approximately 8 weeks
Change in Urine Biomarker: Nephrin
Time frame: Approximately 1 year
Change in Urine Biomarker: Podocin
Time frame: Approximately 1 year
Change in Urine Biomarker: Rac1
Time frame: Approximately 1 year
Change in Urine Biomarker: Synaptopodin
Time frame: Approximately 1 year
Change in Urine Biomarker: Urea
Time frame: Approximately 1 year
Change in Urine Biomarker: Other Exploratory
Time frame: Approximately 1 year
Change in Serum/Plasma Biomarker: Other Exploratory
Time frame: Approximately 8 weeks
Number of patients with genetic variants predicted to be associated with chronic kidney disease and functional consequence
Time frame: Baseline/Biomarker collection visit
DNA analysis of blood sample
Gene expression profile and phenotype of inducible pluripotent stem cell (iPSC)-generated organoids
Time frame: Baseline/Biomarker collection visit
Generation of iPSC from whole blood sample
Change from Baseline Patient-reported Assessment of FSGS Symptoms
Time frame: Approximately 8 weeks
FSGS/TR-MCD patients will assess disease symptomatology utilizing the FSGS Symptom Diary and FSGS Symptom Impact Questionnaire
Change from Baseline Patient-reported Assessment of Health Status
Time frame: Approximately 8 weeks
Patients will assess health status using the 36-Item Short Form Health Survey (SF-36)
Change from Baseline Patient-reported Assessment of Fatigue
Time frame: Approximately 8 weeks
Patients will assess the symptom of fatigue utilizing the Modified Fatigue Impact Scale
Change from Baseline Clinician-reported Assessment of Edema
Time frame: Approximately 8 weeks
Clinicians will assess edema in FSGS/TR-MCD patients using a standardized measurement of edema in FSGS/TR-MCD patients
Incidence of Untoward Medical Occurrences
Time frame: 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.
% of Patients with Change in Treatment
Time frame: Approximately 1 year
Change in treatment as indicated by patient medical record
Results and safety
The study was stopped early (reason given: Sponsor decision for administrative reasons). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Jul 2021.
- Individual participant dataSharing planned: Yes. Patient reported outcome (and other relevant data) will be shared with other researchers.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
18 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Glomerulosclerosis, Focal Segmental and Minimal Change Disease, run by Goldfinch Bio, Inc. at 18 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 20 Jul 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 18 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was stopped early (reason given: Sponsor decision for administrative reasons). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Urine Protein-to-Creatinine Ratio (UPCR) (time frame: Approximately 1 year). The study lists 19 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Jul 2021.