Effect of Galactose on Permeblity Factor in Patients With FSGS and CKD Stage 5 - Full Text View

Sponsors and Collaborators:	North Shore Long Island Jewish Health System Medical College of Wisconsin, Milwaukee, WI Columbia University, College of Physicians & Surgeons, New York, NY University of North Carolina at Chapel Hill, NC
Information provided by:	North Shore Long Island Jewish Health System
ClinicalTrials.gov Identifier:	NCT00816504

Purpose

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.

Condition	Intervention	Phase
Focal Segmental Glomerulosclerosis	Drug: Galactose	Phase I

Drug Information available for: Galactose

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Open Label, Uncontrolled, Single Group Assignment, Efficacy Study
Official Title:	Effect of Galactose on Permeability Factor in Patients With Focal Segmental Glomerulosclerosis (FSGS)and Chronic Kidney Disease Stage 5

Further study details as provided by North Shore Long Island Jewish Health System:

Primary Outcome Measures:

Reduction in FSGS permeability factor [ Time Frame: 28 days ] [ Designated as safety issue: No ]

Estimated Enrollment:	5
Study Start Date:	December 2008
Estimated Study Completion Date:	December 2009
Estimated Primary Completion Date:	December 2009 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
1: Experimental Galactose	Drug: Galactose Oral galactose, 0.2 g/kg/dose twice daily for 28 days

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.

Eligibility

Ages Eligible for Study:	2 Years to 60 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Primary FSGS
CKD Stage 5
Resistance to steroids and another immunosuppressive medication

Exclusion Criteria:

Secondary FSGS

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00816504

Contacts

Contact: Howard Trachtman, MD	718-470-3423	trachtma@lij.edu
Contact: Suzanne Vento, RN	718-470-3499	svento@lij.edu

Locations

United States, New York
College of Physicians & Surgeons	Recruiting
New York, New York, United States, 10032
Contact: Gerald Appel, MD 212-305-3273 nephroman@msn.com
Principal Investigator: Gerald Appel, MD
United States, North Carolina
University of North Carolina	Recruiting
Chapel Hill, North Carolina, United States, 27599-7155
Contact: Debbie Gipson, MD 919-966-2561 ext 235 debbie_gipson@unc.med.edu
Principal Investigator: Debbie Gipson, MD
United States, Wisconsin
Medical College of Wisconsin	Recruiting
Milwaukee, Wisconsin, United States, 53226
Contact: Virginia Savin, MD 414-805-9050 vsavin@mcw.edu
Principal Investigator: Virginia Savin, MD

Sponsors and Collaborators

North Shore Long Island Jewish Health System

Medical College of Wisconsin, Milwaukee, WI

Columbia University, College of Physicians & Surgeons, New York, NY

University of North Carolina at Chapel Hill, NC

Investigators

Principal Investigator:

Howard Trachtman, MD

Schneider Children's Hospital

More Information

Provides important information about FSGS This link exits the ClinicalTrials.gov site

Publications:

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.

Responsible Party:	Schneider Children's Hospital ( Howard Trachtman )
Study ID Numbers:	08-124, NIDDK70341
Study First Received:	December 31, 2008
Last Updated:	December 31, 2008
ClinicalTrials.gov Identifier:	NCT00816504
Health Authority:	United States: Food and Drug Administration

Keywords provided by North Shore Long Island Jewish Health System:

FSGS
Permeability factor
Galactose
Primary FSGS, resistant to immunosuppressive medication

Study placed in the following topic categories:

Glomerulosclerosis, Focal Segmental
Glomerulonephritis
Urologic Diseases
Renal Insufficiency, Chronic

Nephritis
Kidney Failure, Chronic
Kidney Diseases
Focal segmental glomerulosclerosis

ClinicalTrials.gov processed this record on January 13, 2009