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

This study has been completed.
Sponsor:
Information provided by:
Office of Rare Diseases (ORD)
ClinicalTrials.gov Identifier:
NCT00010413
First received: February 2, 2001
Last updated: October 1, 2008
Last verified: October 2008
  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 2

Study Type: Interventional
Study Design: Primary Purpose: Treatment

Resource links provided by NLM:


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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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
Autoimmune Diseases
Immune System Diseases
Skin Diseases
Skin Diseases, Vesiculobullous
Cyclophosphamide
Alkylating Agents
Antineoplastic Agents
Antineoplastic Agents, Alkylating
Antirheumatic Agents
Immunologic Factors
Immunosuppressive Agents
Molecular Mechanisms of Pharmacological Action
Myeloablative Agonists
Pharmacologic Actions
Physiological Effects of Drugs
Therapeutic Uses

ClinicalTrials.gov processed this record on October 20, 2014