Ignite Creation Date:
2025-12-25 @ 3:25 AM
Ignite Modification Date:
2025-12-26 @ 2:05 AM
Study NCT ID:
NCT06065605
Status:
UNKNOWN
Last Update Posted:
2023-10-04
First Post:
2023-09-11
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Assess Urine Biomarkers to Predict Nephropathy in Fabry Disease
Sponsor:
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Organization:
Study Overview
Official Title:
Pilot Study to Assess Clinical and Pivotal Biomarkers in the Urine to Predict the Progression of Nephropathy in Fabry Disease
Status:
UNKNOWN
Status Verified Date:
2023-09
Last Known Status:
RECRUITING
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The purpose of this research is to collect biological samples (urine) to develop assays for immune biomarkers to possibly in the future be able to screen subjects with Fabry disease and be able to understand better progression of nephropathy in Fabry disease and predict nephropathy in Fabry disease.
Detailed Description:
This is a study to assess the markers related to autophagy, apoptosis, pyroptosis, and inflammatory markers related to NFkB, TNF-alpha, and TGF-β1 pathways in the urine. Urinary biomarkers will then be compared to the standard measures of kidney function and proteinuria: GFR, cystatin-C, B2M, bikunin, NGAL. Gb3 and Lyso-Gb3, urine microalbumin, and urine protein-to-creatinine (UPCR) ratio. Investigators will also analyze the role of therapy, especially for the inflammatory responses in participants on stable enzyme replacement therapy (ERT) with that of patients naïve to therapy.
There will be a total of 25 biomarkers that will be assessed during the study. Biomarkers of inflammation
1. Il-4
2. Il-6
3. IL-8
4. Il-10
5. Il-12
6. Il-18
7. MCP1
8. TGF-β1
9. IFN-γ
10. TNF-α
11. IL-1β
12. RANTES
13. BAFF
14. APRIL
15. PAI-1 Biomarkers of kidney function and proteinuria
16. B2M
17. Bikunin
18. NGAL
19. Osteopontin
20. Clusterin
21. Creatinine Acute kidney injury
22. KIM-1
23. YKL-40
24. EGF
25. CK-18 M30
There will be a total of 25 biomarkers that will be assessed during the study. Biomarkers of inflammation
1. Il-4
2. Il-6
3. IL-8
4. Il-10
5. Il-12
6. Il-18
7. MCP1
8. TGF-β1
9. IFN-γ
10. TNF-α
11. IL-1β
12. RANTES
13. BAFF
14. APRIL
15. PAI-1 Biomarkers of kidney function and proteinuria
16. B2M
17. Bikunin
18. NGAL
19. Osteopontin
20. Clusterin
21. Creatinine Acute kidney injury
22. KIM-1
23. YKL-40
24. EGF
25. CK-18 M30
Study Oversight
Has Oversight DMC:
False
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: