Oral Galactose in Children With Steroid Resistant Nephrotic Syndrome
Focal Segmental Glomerulosclerosis (FSGS) is a devastating kidney disease which is difficult to treat and carries a poor prognosis, with 50% of affected children progressing to end stage renal disease (ESRD). The purpose of this study is to investigate oral galactose as a benign treatment for FSGS in children. The investigators hypothesize that galactose, a simple milk sugar thought to bind to the protein factor (FSPF) that causes FSGS thereby inactivating it and stopping it from damaging the kidney, resulting in a reduction in glomerular permeability to albumin and decrease in proteinuria in children with nephrotic syndrome secondary to FSGS.
Focal Segmental Glomerulosclerosis
Steroid Resistant Nephrotic Syndrome
|Study Design:||Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Effect of Oral Galactose on the Level of Focal Sclerosis Permeability Factor and Proteinuria in Children With Steroid Resistant Nephrotic Syndrome: A Pilot Study|
- Focal Segmental Glomerulosclerosis Permeability Factor (FSPF) [ Time Frame: 16 weeks ] [ Designated as safety issue: No ]FSPF is reported in relation to its induction of glomerular albumin permeability (Palb) of isolated glomeruli on a range from 0 to 1, with 0 indicative of normal glomeruli and 1 indicative of injury to the permeability barrier. Results will be considered clinically significant if the following criteria is met in response to oral galactose therapy at week 16: Reduction in FSPF to <0.5 Palb or decrease in FSPF by > 0.3 Palb.
- Number of Participants Achieving Complete or Partial Remission at 16 Weeks [ Time Frame: 16 weeks ] [ Designated as safety issue: No ]Results will be considered clinically significant if the following criteria is met in response to oral galactose therapy at week 16. Complete remission is defined as (Urine Protein:Creatinine ratio [UPC] <0.2 g/g). Partial remission is defined as UPC 0.2-2 g/g.
|Study Start Date:||October 2009|
|Study Completion Date:||March 2013|
|Primary Completion Date:||March 2013 (Final data collection date for primary outcome measure)|
Oral galactose will be given at a dose of 0.2gm/kg/dose twice a day (BID) to a maximum of 15 gm BID for a period of 16 weeks.
Oral galactose will be initiated at a dose of 0.2gm/kg/dose twice daily to a maximum of 15 gm BID for a period of 4 months. The prescribed dose of D-galactose powder will be dispensed to subjects in packets, mixed with 4 ounces of water, and consumed orally.
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Please refer to this study by its ClinicalTrials.gov identifier: NCT01113385
|United States, District of Columbia|
|Children's National Medical Center|
|Washington DC, District of Columbia, United States, 20010|
|Principal Investigator:||Asha Moudgil, MD||Children's Research Institute|
|Study Director:||Kristen Sgambat, MS, RD||Children's Research Institute|