NCT00010413

Brief Summary

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

Trial Health

80
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
35

participants targeted

Target at P25-P50 for phase_2

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

April 1, 1999

Completed
1.8 years until next milestone

First Submitted

Initial submission to the registry

February 2, 2001

Completed
Same day until next milestone

First Posted

Study publicly available on registry

February 2, 2001

Completed
6.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 1, 2007

Completed
Last Updated

October 2, 2008

Status Verified

October 1, 2008

Enrollment Period

8.3 years

First QC Date

February 2, 2001

Last Update Submit

October 1, 2008

Conditions

Pemphigus

Keywords

immunologic disorders and infectious disorderspemphigusrare disease

Interventions

cyclophosphamideDRUG
filgrastimDRUG

Eligibility Criteria

Age18 Years - 70 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
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

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

Study Sites (1)

Johns Hopkins University School of Medicine

Baltimore, Maryland, 21205, United States

Location

MeSH Terms

Conditions

PemphigusImmune System DiseasesCommunicable DiseasesRare Diseases

Interventions

CyclophosphamideFilgrastim

Condition Hierarchy (Ancestors)

Skin Diseases, VesiculobullousSkin DiseasesSkin and Connective Tissue DiseasesAutoimmune DiseasesInfectionsDisease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Phosphoramide MustardsNitrogen Mustard CompoundsMustard CompoundsHydrocarbons, HalogenatedHydrocarbonsOrganic ChemicalsPhosphoramidesOrganophosphorus CompoundsGranulocyte Colony-Stimulating FactorColony-Stimulating FactorsGlycoproteinsGlycoconjugatesCarbohydratesHematopoietic Cell Growth FactorsCytokinesIntercellular Signaling Peptides and ProteinsPeptidesAmino Acids, Peptides, and ProteinsProteinsBiological Factors

Study Officials

  • Grant J. Anhalt

    Johns Hopkins University

    STUDY CHAIR

Study Design

Study Type
interventional
Phase
phase 2
Purpose
TREATMENT
Sponsor Type
OTHER

Study Record Dates

First Submitted

February 2, 2001

First Posted

February 2, 2001

Study Start

April 1, 1999

Primary Completion

August 1, 2007

Last Updated

October 2, 2008

Record last verified: 2008-10

Locations

US(1)