N/A
N=7
Oral Galactose in Children With Steroid Resistant Nephrotic Syndrome
Focal Segmental Glomerulosclerosis · Steroid Resistant Nephrotic Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT01113385 ↗Enrolled (actual)
7
Serious AEs
0.0%
Results posted
Sep 2014
Primary outcome: Primary: Focal Segmental Glomerulosclerosis Permeability Factor (FSPF) — 0.69; 0.35 Palb — p=0.009
Study Design & Population
- Study type
- Interventional
- Phase
- N/A
- Interventions
- D-Galactose (Drug)
- Age
- Pediatric, Adult · 2+ yrs
- Sex
- All
- Sponsor
- Children's National Research Institute
- Primary completion
- Mar 2013
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Focal Segmental Glomerulosclerosis Permeability Factor (FSPF) |
0.69; 0.35 | 0.009 sig |
| SECONDARY Number of Participants Achieving Complete or Partial Remission at 16 Weeks |
— | — |
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.
Eligibility Criteria
Inclusion Criteria
- 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
Exclusion Criteria
- 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
Data sourced from ClinicalTrials.gov (NCT01113385). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.