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Pilot Study of Mycophenolate Mofetil in Congenital Uropathies

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00193635
Recruitment Status : Completed
First Posted : September 19, 2005
Last Update Posted : October 19, 2007
Hoffmann-La Roche
University of Medicine and Dentistry of New Jersey
New York Presbyterian Hospital
Information provided by:
Northwell Health

Brief Summary:

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 or disease Intervention/treatment Phase
Congenital Urological Abnormalities Drug: mycophenolate mofetil Phase 1

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 12 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Pilot Study of Mycophenolate Mofetil in Congenital Uropathies
Study Start Date : March 2002
Actual Study Completion Date : August 2007

Arm Intervention/treatment
Active Comparator: 1
oral MMF
Drug: mycophenolate mofetil
oral drug administration
Other Name: Cellcept

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

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

Information from the National Library of Medicine

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Ages Eligible for Study:   3 Years to 16 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00193635

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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
Northwell Health
Hoffmann-La Roche
University of Medicine and Dentistry of New Jersey
New York Presbyterian Hospital
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Principal Investigator: Howard Trachtman, MD Schneider Children's Hospital of North Shore-LIJ Health System
Publications automatically indexed to this study by Identifier (NCT Number):
Layout table for additonal information Identifier: NCT00193635    
Other Study ID Numbers: CEL214
First Posted: September 19, 2005    Key Record Dates
Last Update Posted: October 19, 2007
Last Verified: October 2007
Keywords provided by Northwell Health:
Congenital uropathies
Mycophenolate mofetil
Iothalamate clearance
Urine cytometry
Congenital urological abnormalities which represent stuctural defects in the genitourinary system
Additional relevant MeSH terms:
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Urologic Diseases
Congenital Abnormalities
Mycophenolic Acid
Antibiotics, Antineoplastic
Antineoplastic Agents
Antibiotics, Antitubercular
Antitubercular Agents
Anti-Bacterial Agents
Anti-Infective Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action