Effect of Galactose on Permeblity Factor in Patients With FSGS and CKD Stage 5
Primary Purpose
Focal Segmental Glomerulosclerosis
Status
Withdrawn
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Galactose
Sponsored by
About this trial
This is an interventional treatment trial for Focal Segmental Glomerulosclerosis focused on measuring FSGS, Permeability factor, Galactose, Primary FSGS, resistant to immunosuppressive medication
Eligibility Criteria
Inclusion Criteria:
- Primary FSGS
- CKD Stage 5
- Resistance to steroids and another immunosuppressive medication
Exclusion Criteria:
- Secondary FSGS
Sites / Locations
- Medical College of Wisconsin
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
1
Arm Description
Galactose
Outcomes
Primary Outcome Measures
Reduction in FSGS permeability factor
Secondary Outcome Measures
Full Information
NCT ID
NCT00816504
First Posted
December 31, 2008
Last Updated
September 3, 2015
Sponsor
Northwell Health
Collaborators
Medical College of Wisconsin
1. Study Identification
Unique Protocol Identification Number
NCT00816504
Brief Title
Effect of Galactose on Permeblity Factor in Patients With FSGS and CKD Stage 5
Official Title
Effect of Galactose on Permeability Factor in Patients With Focal Segmental Glomerulosclerosis (FSGS)and Chronic Kidney Disease Stage 5
Study Type
Interventional
2. Study Status
Record Verification Date
September 2015
Overall Recruitment Status
Withdrawn
Why Stopped
Under additional IRB Review
Study Start Date
December 2008 (undefined)
Primary Completion Date
December 2009 (Actual)
Study Completion Date
December 2009 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Northwell Health
Collaborators
Medical College of Wisconsin
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
This study is a proof-of-concept clinical study designed to test the hypothesis that oral administration of galactose can lower the level of a circulating factor that increases glomerular permeability to albumin in patients with resistant FSGS.
Detailed Description
Patients with resistant FSGS have persistent proteinuria and a high risk of progression to end stage kidney disease (ESKD). A circulating factor that increases glomerular permeability to albumin (Palb) has been detected in over 50% of these patients. While the molecular identity of the factor has not been fully established, in vitro studies and limited clinical experience suggest that galactose can reduce the level of the FSGS permeability factor.
This study is a pilot study to determine if oral administration of galactose can lower the circulating level of the FSGS permeability factor.
Five patients with biopsy proven primary FSGS - native or transplant kidney, receiving plasmapheresis - who are resistant to steroids and one other immunosuppressive agents will be studied.
The only eligibility factor is presence of the FSGS permeability factor.
The experimental intervention is administration of galactose, orally, 0.2 g/kg body weight/dose twice daily. the duration of treatment will be 28 days,.
Patients will be seen on days 0, 14, and 28 of treatment. They will be seen at week 8, 16 and 24 after discontinuation of the galactose.
Physical examination and routine laboratory tests (SMAC, CBC, urine protein:creatinine ratio in an early morning sample) will be done at each visit. The FSGS permeability factor will be assayed at days 0 and 28 of treatment and 6 months after discontinuation of the galactose. The permeability factor will be tested in the laboratory of Virginia Savin MD (Medical college of Wisconsin) using previously described methods.
All other treatments will be unchanged during the 28 day oral galactose Treatment Period.
The study will be analyzed based on the number of patients in whom the FSGS permeability factor is lowered to normal levels.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Focal Segmental Glomerulosclerosis
Keywords
FSGS, Permeability factor, Galactose, Primary FSGS, resistant to immunosuppressive medication
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
0 (Actual)
8. Arms, Groups, and Interventions
Arm Title
1
Arm Type
Experimental
Arm Description
Galactose
Intervention Type
Drug
Intervention Name(s)
Galactose
Intervention Description
Oral galactose, 0.2 g/kg/dose twice daily for 28 days
Primary Outcome Measure Information:
Title
Reduction in FSGS permeability factor
Time Frame
28 days
10. Eligibility
Sex
All
Minimum Age & Unit of Time
2 Years
Maximum Age & Unit of Time
60 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Primary FSGS
CKD Stage 5
Resistance to steroids and another immunosuppressive medication
Exclusion Criteria:
Secondary FSGS
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Howard Trachtman, MD
Organizational Affiliation
Schneider Children's Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Medical College of Wisconsin
City
Milwaukee
State/Province
Wisconsin
ZIP/Postal Code
53226
Country
United States
12. IPD Sharing Statement
Citations:
PubMed Identifier
15384010
Citation
Trachtman H, Greenbaum LA, McCarthy ET, Sharma M, Gauthier BG, Frank R, Warady B, Savin VJ. Glomerular permeability activity: prevalence and prognostic value in pediatric patients with idiopathic nephrotic syndrome. Am J Kidney Dis. 2004 Oct;44(4):604-10.
Results Reference
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Effect of Galactose on Permeblity Factor in Patients With FSGS and CKD Stage 5
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