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

OBJECTIVES:

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

Completed

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Key information

Conditions

Pemphigus Autoimmune Diseases Communicable Diseases Disease Attributes Immune System Diseases Infections Pathologic Processes Pathological Conditions, Signs and Symptoms Rare Diseases Skin Diseases Skin Diseases, Vesiculobullous Skin and Connective Tissue Diseases

Age range

18 year–70 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Johns Hopkins University School of Medicine

Baltimore, Maryland, 21205, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Cyclophosphamide

Drug

Filgrastim

Drug

Sponsors and collaborators

Lead sponsor

Johns Hopkins University

Other

Registry information

Important dates

Study start: 1999
Primary completion: 2007
First posted: Feb 2, 2001
Registry last updated: Oct 2, 2008

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed