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

Sponsor:	Johns Hopkins University
Information provided by:	Office of Rare Diseases (ORD)
ClinicalTrials.gov Identifier:	NCT00010413

Purpose

OBJECTIVES:

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

Condition	Intervention	Phase
Pemphigus	Drug: cyclophosphamide Drug: filgrastim	Phase II

Study Type:	Interventional
Study Design:	Primary Purpose: Treatment

Resource links provided by NLM:

MedlinePlus related topics: Pemphigus

Drug Information available for: Cyclophosphamide Filgrastim Lenograstim Granulocyte colony-stimulating factor

U.S. FDA Resources

Further study details as provided by Office of Rare Diseases (ORD):

Estimated Enrollment:	35
Study Start Date:	April 1999
Primary Completion Date:	August 2007 (Final data collection date for primary outcome measure)

Detailed Description:

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.

Eligibility

Ages Eligible for Study:	18 Years to 70 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00010413

Locations

United States, Maryland
Johns Hopkins University School of Medicine
Baltimore, Maryland, United States, 21205

Sponsors and Collaborators

Johns Hopkins University

Investigators

Study Chair:

Grant J. Anhalt

Johns Hopkins University

More Information

No publications provided

ClinicalTrials.gov Identifier:	NCT00010413 History of Changes
Other Study ID Numbers:	199/15674, JHOC-J9912, JHOC-99022610
Study First Received:	February 2, 2001
Last Updated:	October 1, 2008
Health Authority:	Unspecified

Keywords provided by Office of Rare Diseases (ORD):

immunologic disorders and infectious disorders
pemphigus
rare disease

Additional relevant MeSH terms:

Pemphigus
Skin Diseases, Vesiculobullous
Skin Diseases
Autoimmune Diseases
Immune System Diseases
Cyclophosphamide
Lenograstim
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs

Pharmacologic Actions
Antirheumatic Agents
Therapeutic Uses
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists
Adjuvants, Immunologic

ClinicalTrials.gov processed this record on February 12, 2012