Phase II Study of High-Dose Cyclophosphamide in Patients With Refractory Pemphigus

Johns Hopkins University35 enrolled

Overview

OBJECTIVES: I. Determine the response rate and 1-year event-free survival of patients with refractory pemphigus treated with high-dose cyclophosphamide.

PROTOCOL OUTLINE: Patients receive cyclophosphamide IV on days 1-4 and filgrastim (G-CSF) beginning on day 10 and continuing until blood counts recover. Patients are followed monthly for 6 months, every 2 months for 6 months, every 4 months for a year, and then annually thereafter.

Study Type

INTERVENTIONAL

Purpose

TREATMENT

Enrollment

Conditions

Pemphigus

Interventions

cyclophosphamideDRUG

filgrastimDRUG

Eligibility

Sex: ALLMin age: 18 YearsMax age: 70 Years

Medical Language ↔ Plain English

PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Definitive diagnosis of pemphigus, including all of the following: Typical clinical lesions Histologically proven by skin or mucous membrane biopsy Pemphigus IgG autoantibodies in skin or mucous membrane by direct immunofluorescence (IF) Circulating pemphigus IgG autoantibodies in serum by indirect IF Dependence on high-dose corticosteroids Persistent disease activity despite treatment with mycophenolate mofetil or azathioprine, or inability to tolerate these drugs (for paraneoplastic pemphigus, persistent disease activity despite treatment with cyclosporine) --Prior/Concurrent Therapy-- No concurrent cytotoxic therapy --Patient Characteristics-- Performance status: Karnofsky 20-100% Renal: Creatinine no greater than 2.5 mg/dL Cardiovascular: LVEF at least 40% Pulmonary: FVC, FEV1, or DLCO at least 50% predicted Other: Not pregnant Fertile patients must use effective contraception during and for 9 months after study Not preterminal or moribund

Locations (1)

Johns Hopkins University School of Medicine

Baltimore, Maryland, United States

Data from ClinicalTrials.gov

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