Oral Galactose in Children With Steroid Resistant Nephrotic Syndrome

This study has been completed.
Sponsor:
Collaborator:
National Kidney Foundation
Information provided by (Responsible Party):
Asha Moudgil, Children's Research Institute
ClinicalTrials.gov Identifier:
NCT01113385
First received: April 28, 2010
Last updated: September 8, 2014
Last verified: September 2014
  Purpose

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.


Condition Intervention
Focal Segmental Glomerulosclerosis
Steroid Resistant Nephrotic Syndrome
Drug: D-Galactose

Study Type: Interventional
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

Resource links provided by NLM:


Further study details as provided by Children's Research Institute:

Primary Outcome Measures:
  • 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.


Secondary Outcome Measures:
  • 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.


Enrollment: 7
Study Start Date: October 2009
Study Completion Date: March 2013
Primary Completion Date: March 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Galactose
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.
Drug: D-Galactose
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.
Other Names:
  • D-galactose
  • Galactose

  Show Detailed Description

  Eligibility

Ages Eligible for Study:   2 Years to 21 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01113385

Locations
United States, District of Columbia
Children's National Medical Center
Washington DC, District of Columbia, United States, 20010
Sponsors and Collaborators
Children's Research Institute
National Kidney Foundation
Investigators
Principal Investigator: Asha Moudgil, MD Children's Research Institute
Study Director: Kristen Sgambat, MS, RD Children's Research Institute
  More Information

Publications:

Responsible Party: Asha Moudgil, Professor of Pediatrics, Children's Research Institute
ClinicalTrials.gov Identifier: NCT01113385     History of Changes
Other Study ID Numbers: 4657
Study First Received: April 28, 2010
Results First Received: July 21, 2014
Last Updated: September 8, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Children's Research Institute:
Nephrotic syndrome
Focal Segmental glomerulosclerosis
FSGS
Minimal change
Steroid resistant
Pediatric
Proteinuria
Galactose
FSPF

Additional relevant MeSH terms:
Syndrome
Nephrotic Syndrome
Glomerulosclerosis, Focal Segmental
Disease
Pathologic Processes
Nephrosis
Kidney Diseases
Urologic Diseases
Glomerulonephritis
Nephritis

ClinicalTrials.gov processed this record on September 18, 2014