Oral Galactose in Children With Steroid Resistant Nephrotic Syndrome

Completed · Not applicable

Conditions studied: Focal Segmental Glomerulosclerosis, Steroid Resistant Nephrotic Syndrome

In brief

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.

Key facts

Study ID

NCT01113385

Run by

Children's National Research Institute

People needed

7

Starts

2009-10-01

Expected to finish

2013-03-01

Last updated by the study team

2014-09-15

Who can join

Age: 2 and older, up to 21. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 2-21 years old
• Biopsy proven FSGS or minimal change with steroid resistance
• Presence of FSPF (defined as permeability activity >0.5)
• Presence of nephrotic range proteinuria (urine protein: creatinine ratio >2) at the time of enrollment.
• Persistent nephrotic range proteinuria despite being on stable immunosuppressive medications (cyclosporine, tacrolimus or mycophenolate mofetil) for at least 12 weeks and/or persistent nephrotic range proteinuria despite being on stable dose of angiotensin converting enzyme (ACE) inhibitors or angiotensin receptor blockers (ARBs) for 12 weeks.
• Stable serum creatinine (change of less than 0.3 mg/dl) in the prior 3 months.
• Schwartz estimated (e) glomerular filtration rate (GFR) >60ml/min/1.73m2

You may not qualify if…

• Secondary FSGS
• Onset of nephrotic syndrome in infancy.
• Presence of acute renal failure (as defined by acute kidney injury criteria) at the time of enrollment. These children can be enrolled 1 month after resolution of acute renal failure (ARF).
• Decreasing renal function (persistent increase in serum creatinine of greater than 0.3 mg/dl over baseline in the prior 3 months).
• Use of another investigational drug
• Pregnant or unable to comply with contraceptive measures in females of child bearing age
• eGFR < 60 ml/min per 1.73 m2
• Children with Galactosemia
• Children with type 1 or 2 diabetes

Where it is running

• Children's National Medical Center — Washington D.C., District of Columbia, United States

Full record on ClinicalTrials.gov

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