Text Size

Pilot Study of Mycophenolate Mofetil in Congenital Uropathies

This study has been completed.
Sponsor:
Collaborators:
Hoffmann-La Roche
University of Medicine and Dentistry New Jersey
New York Presbyterian Hospital
Information provided by:
North Shore Long Island Jewish Health System
ClinicalTrials.gov Identifier:
NCT00193635
First received: September 9, 2005
Last updated: October 17, 2007
Last verified: October 2007
History of Changes
  Purpose

Congenital or hereditary structural anomalies of the genitourinary tract account for approximately half of all cases of end stage renal disease in the pediatric population. Despite optimal medical management, when the GFR falls below 50 ml/min/1.73 M2, nearly 40% of affected children will require dialysis or a renal transplant within 2 years. At present, there is no specific treatment for patients with congenital uropathies that can retard the progressive loss of kidney function and forestall the need for renal replacement therapy.

There is evidence in experimental animals and in patients with chronic renal failure (CRF) that immunoeffector mechanisms are activated within the renal parenchyma. Infiltration of the kidney by macrophages, monocytes, and lymphocytes, activation of renal tubular epithelial cells, and release of pro-inflammatory cytokines result in fibrosis and irreversible organ damage.

Mycophenolate mofetil (MMF) is a new immunosuppressive agent that is used to prevent acute rejection in kidney transplant recipients. It attenuates renal damage in the remnant kidney model of CRF in which there is no primary immunological injury. Therefore, this pilot study is designed to test the hypothesis that immunosuppressive treatment with MMF in children with structural causes of CRF will be safely tolerated and that this therapy will retard progressive decline in renal function.

Patients with congenital uropathy, 3-16 years of age and with a GFR less than 50 ml/ml/1.73 M2, will be treated with MMF for 24 months. The two primary endpoints are: (1) safety and tolerance of the drug; and (2) need for dialysis or kidney transplantation. It is anticipated that the MMF will be free of significant toxicity and that administration of the drug will reduce the frequency of progression to end stage renal disease from 38% to 19%. Patients will be followed at 3-month intervals and they will undergo serial assessment of proteinuria, estimated GFR and iothalamate clearance, urinary cytokine excretion, urine flow cytometry, and immunologic testing.

The significance of this pilot study is that it may provide evidence in support of a randomized, double-blind, placebo-controlled trial of immunological treatment of congenital structural causes of CRF in children


Condition Intervention Phase
Congenital Urological Abnormalities
 Drug: mycophenolate mofetil
 Phase 1

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Pilot Study of Mycophenolate Mofetil in Congenital Uropathies

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by North Shore Long Island Jewish Health System:

Primary Outcome Measures:
  • Reduction in GFR [ Time Frame: 24 month treatment period ]

Secondary Outcome Measures:
  • Safety and tolerance of MMF [ Time Frame: 24 month treatment period ]

Enrollment: 12
Study Start Date: March 2002
Study Completion Date: August 2007
Arms Assigned Interventions
Active Comparator: 1
oral MMF
 Drug: mycophenolate mofetil
oral drug administration
Other Name: Cellcept

  Show Detailed Description

  Eligibility

Ages Eligible for Study:   3 Years to 16 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Age 3-16 GFR <50 ml/min/1.73 m2 Congenital abnormality of urinary tract

Exclusion Criteria:

  • Known hepatic, hematologic, GII, infectious disease Sensitivity to MMF Glomerular disease
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00193635

Locations
United States, New Jersey
Robert Wood Johnson Medical center
New Brunswick, New Jersey, United States, 08903-0019
United States, New York
Schneider Children's Hospital
New Hyde Park, New York, United States, 11040
Cornell Weill Medical Center
New York, New York, United States, 10021-4873
Sponsors and Collaborators
North Shore Long Island Jewish Health System
Hoffmann-La Roche
University of Medicine and Dentistry New Jersey
New York Presbyterian Hospital
Investigators
Principal Investigator: Howard Trachtman, MD Schneider Children's Hospital of North Shore-LIJ Health System
  More Information

No publications provided by North Shore Long Island Jewish Health System

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

ClinicalTrials.gov Identifier: NCT00193635     History of Changes
Other Study ID Numbers: CEL214, CEL214
Study First Received: September 9, 2005
Last Updated: October 17, 2007
Health Authority: United States: Food and Drug Administration

Keywords provided by North Shore Long Island Jewish Health System:
Congenital uropathies
Mycophenolate mofetil
GFR
Iothalamate clearance
 Urine cytometry
Children
Congenital urological abnormalities which represent stuctural defects in the genitourinary system

Additional relevant MeSH terms:
Congenital Abnormalities
Urologic Diseases
Mycophenolate mofetil
Mycophenolic Acid
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
 Pharmacologic Actions
Antibiotics, Antineoplastic
Antineoplastic Agents
Therapeutic Uses
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action

ClinicalTrials.gov processed this record on May 22, 2013