Oral Galactose in Children With Steroid Resistant Nephrotic Syndrome

• Status: Completed
• Trial end date: 2013-03-01
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: N/A
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Children's National Research Institute; Children's Research Institute
• Collaborator: National Kidney Foundation

Criteria

Inclusion Criteria:

1. 2-21 years old

2. Biopsy proven FSGS or minimal change with steroid resistance

3. Presence of FSPF (defined as permeability activity >0.5)

4. Presence of nephrotic range proteinuria (urine protein: creatinine ratio >2) at the time of enrollment.

5. 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.

6. Stable serum creatinine (change of less than 0.3 mg/dl) in the prior 3 months.

7. Schwartz estimated (e) glomerular filtration rate (GFR) >60ml/min/1.73m2

Exclusion Criteria:

1. Secondary FSGS

2. Onset of nephrotic syndrome in infancy.

3. 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).

4. Decreasing renal function (persistent increase in serum creatinine of greater than 0.3 mg/dl over baseline in the prior 3 months).

5. Use of another investigational drug

6. Pregnant or unable to comply with contraceptive measures in females of child bearing age

7. eGFR < 60 ml/min per 1.73 m2

8. Children with Galactosemia

9. Children with type 1 or 2 diabetes