Pilot Study of Atorvastatin in Children With Chronic Hyperlipidemia Secondary to Nephrotic Syndrome

Phase 2TerminatedNCT00004466

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)5 enrolled

Overview

OBJECTIVES: I. Determine the effect of atorvastatin on the plasma levels of lipids, Lp(a), and apoproteins for treating hyperlipidemia in children with nephrotic syndrome in whom proteinuria and hyperlipidemia persist after other appropriate measures to treat their primary disease have been exhausted. II. Determine the safety and tolerability of atorvastatin in these patients. III. Provide preliminary data for a future investigation into the potential effect that lowering cholesterol levels may have on the rate of progression of renal insufficiency in such patients.

PROTOCOL OUTLINE: This is a randomized, double blind, placebo controlled, multicenter study. After 3 months of low cholesterol diet, patients are randomized to receive atorvastatin tablets daily (arm I) or placebo tablets daily (arm II) for 3 months. Arm I patients receive increasing doses of atorvastatin every 4 weeks until individual maximum tolerated doses (MTDs) are determined. After 3 months of treatment, all patients are given atorvastatin in a 6-9 month open label extended evaluation. Arm I patients receive atorvastatin for an additional 6 months and arm II patients receive atorvastatin for 9 months with increasing doses of atorvastatin every 4 weeks for the first 3 months until MTDs are determined. Patients are followed every 6-8 weeks for one year.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Nephrotic Syndrome

Eligibility

Sex: ALLMin age: 6 YearsMax age: 17 Years

Medical Language ↔ Plain English

PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Chronic hyperlipidemia with treatment-resistant nephrotic syndrome; Must have received at least 2 months of therapy with steroids on a daily or alternate basis Primary nephropathy described as minimal change nephrotic syndrome, mesangioproliferative glomerulonephritis, IgM neuropathy, and focal segmental glomerulosclerosis Glomerular filtration rate at least 30 mL/min LDL cholesterol at least 160 mg/dL --Prior/Concurrent Therapy-- No concurrent medications affecting or interacting with lipids or atorvastatin, with the exception of angiotensin converting enzyme inhibitors at discretion of referring physician, including: lipid-lowering medications, beta blockers, thiazides, fish oils, cyclosporine, Cytoxan, azathioprine, chlorambucil, and erythromycin Concurrent prednisone and other corticosteroids allowed on a continual basis at a dose of no greater than 1 mg/kg every other day (maximum dose, no greater than 40 mg every other day); Concurrent acute courses of steroids no greater than 1 week for other unrelated conditions (e.g., asthma) also allowed --Patient Characteristics-- Hepatic: ALT or AST less than 2 times normal Renal: See Disease Characteristics; Creatine phosphokinase less than 3 times normal Other: No history of familial hypercholesterolemia; No systemic disease such as systemic lupus, Schoenlein-Henoch purpura, Hodgkin's disease, polyarteritis nodosum, sickle cell disease, or HIV; Not pregnant; Effective contraception required of all adolescent patients

Pilot Study of Atorvastatin in Children With Chronic Hyperlipidemia Secondary to Nephrotic Syndrome

Overview

Conditions

Interventions

Eligibility

Locations (1)

Outcomes

Primary Outcomes