Ignite Creation Date:
2024-05-05 @ 4:37 PM
Last Modification Date:
2024-10-26 @ 9:22 AM
Study NCT ID:
NCT00286156
Status:
COMPLETED
Last Update Posted:
2015-04-06
First Post:
2006-02-01
Brief Title:
Pilot Study of Rapamycin as Treatment for Autosomal Dominant Polycystic Kidney Disease ADPKD
Sponsor:
The Cleveland Clinic
Organization:
The Cleveland Clinic
Study Overview
Official Title:
Pilot Study of Rapamycin as Treatment for Autosomal Dominant Polycystic Kidney Disease
Status:
COMPLETED
Status Verified Date:
2014-03
Last Known Status:
None
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:
This study is a prospective randomized open-label pilot clinical trial designed to compare the effects of an agent that has antiproliferative 12 antiangiogenesis 3and tumor-progression blocking capabilities 4 namely rapamycin Rapamune in the treatment of autosomal-dominant polycystic kidney disease ADPKD
Up to this time only generic renal disease treatments for ADPKD have been in use such as the treatment of hypertension urinary tract infections renal stones renal call carcinomas and replacement therapy with dialysis andor renal transplantation The fundamental aberrations in ADPKD are proliferation of cyst-forming tubuloepithelial cells secretion of cytokine-rich fluid into those cysts and progressive cyst expansion and release of inflammatory mediators that injure surrounding normal renal tissue Consequently therapy directed specifically at blocking the proliferation of tubuloepithelial cells and their tendency to malignant transformation as well as impeding their blood supply should have obvious merit
General Procedures
In Group I participants will have an iothalamate glomerular filtration rate GFR equal to or greater than 60 mlmin173 m2 and in Group II participants will have a GFR less than 25-59 mlmin173 m2 Both males and females with ADPKD who volunteer and qualify will be randomly and prospectively assigned to treatment with rapamycin at either a high or low trough blood level or to standard care each 13 of enrolled patients for one year The two treatment groups will receive rapamycin doses aimed at maintaining the 20- to 24-hour trough blood levels at either 2 to 5 ngmL low-dose or greater than 5 to 8 ngmL high-dose These trough levels are in the lower range of levels used when treating renal transplant recipients in whom trough levels are typically maintained between 5 and 15 ngmL
Up to this time only generic renal disease treatments for ADPKD have been in use such as the treatment of hypertension urinary tract infections renal stones renal call carcinomas and replacement therapy with dialysis andor renal transplantation The fundamental aberrations in ADPKD are proliferation of cyst-forming tubuloepithelial cells secretion of cytokine-rich fluid into those cysts and progressive cyst expansion and release of inflammatory mediators that injure surrounding normal renal tissue Consequently therapy directed specifically at blocking the proliferation of tubuloepithelial cells and their tendency to malignant transformation as well as impeding their blood supply should have obvious merit
General Procedures
In Group I participants will have an iothalamate glomerular filtration rate GFR equal to or greater than 60 mlmin173 m2 and in Group II participants will have a GFR less than 25-59 mlmin173 m2 Both males and females with ADPKD who volunteer and qualify will be randomly and prospectively assigned to treatment with rapamycin at either a high or low trough blood level or to standard care each 13 of enrolled patients for one year The two treatment groups will receive rapamycin doses aimed at maintaining the 20- to 24-hour trough blood levels at either 2 to 5 ngmL low-dose or greater than 5 to 8 ngmL high-dose These trough levels are in the lower range of levels used when treating renal transplant recipients in whom trough levels are typically maintained between 5 and 15 ngmL
Detailed Description:
This study is a prospective randomizedopen label pilot clinical trial designed to compare the effects of an agent that has antiproliferative 12 antiangiogenesis 3and tumor-progression blocking capabilities 4 namely rapamycin Rapamune in the treatment of autosomal-dominant polycystic kidney disease ADPKD
Up to this time only generic renal disease treatments for ADPKD have been in use such as the treatment of hypertension urinary tract infections renal stones renal call carcinomas and replacement therapy with dialysis andor renal transplantation The fundamental aberrations in ADPKD are proliferation of cyst-forming tubuloepithelial cells secretion of cytokine-rich fluid into those cysts and progressive cyst expansion and release of inflammatory mediators that injure surrounding normal renal tissue Consequently therapy directed specifically at blocking the proliferation of tubuloepithelial cells and their tendency to malignant transformation as well as impeding their blood supply should have obvious merit
General Procedures
In Group I participants will have an iothalamate glomerular filtration rate GFR equal to or greater than 60 mlmin173 m2 and in Group II participants will have a GFR less than 25-59 mlmin173 m2 Both males and females with ADPKD who volunteer and qualify will be randomly and prospectively assigned to treatment with rapamycin at either a high or low trough blood level or to standard care each 13 of enrolled patients for one year The two treatment groups will receive rapamycin doses aimed at maintaining the 20- to 24-hour trough blood levels at either 2 to 5 ngmL low-dose or greater than 5 to 8 ngmL high-dose These trough levels are in the lower range of levels used when treating renal transplant recipients in whom trough levels are typically maintained between 5 and 15 ngmL
Up to this time only generic renal disease treatments for ADPKD have been in use such as the treatment of hypertension urinary tract infections renal stones renal call carcinomas and replacement therapy with dialysis andor renal transplantation The fundamental aberrations in ADPKD are proliferation of cyst-forming tubuloepithelial cells secretion of cytokine-rich fluid into those cysts and progressive cyst expansion and release of inflammatory mediators that injure surrounding normal renal tissue Consequently therapy directed specifically at blocking the proliferation of tubuloepithelial cells and their tendency to malignant transformation as well as impeding their blood supply should have obvious merit
General Procedures
In Group I participants will have an iothalamate glomerular filtration rate GFR equal to or greater than 60 mlmin173 m2 and in Group II participants will have a GFR less than 25-59 mlmin173 m2 Both males and females with ADPKD who volunteer and qualify will be randomly and prospectively assigned to treatment with rapamycin at either a high or low trough blood level or to standard care each 13 of enrolled patients for one year The two treatment groups will receive rapamycin doses aimed at maintaining the 20- to 24-hour trough blood levels at either 2 to 5 ngmL low-dose or greater than 5 to 8 ngmL high-dose These trough levels are in the lower range of levels used when treating renal transplant recipients in whom trough levels are typically maintained between 5 and 15 ngmL
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None