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

NCT ID: NCT00010413

Last Updated: 2008-10-02

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

35 participants

Study Classification

INTERVENTIONAL

Study Start Date

1999-04-30

Brief Summary

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OBJECTIVES:

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

Detailed Description

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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.

Conditions

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Pemphigus

Keywords

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immunologic disorders and infectious disorders pemphigus rare disease

Study Design

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Primary Study Purpose

TREATMENT

Interventions

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cyclophosphamide

Intervention Type DRUG

filgrastim

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

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
Minimum Eligible Age

18 Years

Maximum Eligible Age

70 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Johns Hopkins University

OTHER

Sponsor Role lead

Principal Investigators

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Grant J. Anhalt

Role: STUDY_CHAIR

Johns Hopkins University

Locations

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Johns Hopkins University School of Medicine

Baltimore, Maryland, United States

Site Status

Countries

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United States

Other Identifiers

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JHOC-J9912

Identifier Type: -

Identifier Source: secondary_id

JHOC-99022610

Identifier Type: -

Identifier Source: secondary_id

199/15674

Identifier Type: -

Identifier Source: org_study_id