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Sponsored by: |
Johns Hopkins University |
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Information provided by: | Office of Rare Diseases (ORD) |
ClinicalTrials.gov Identifier: | NCT00010413 |
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: | Treatment |
Estimated Enrollment: | 35 |
Study Start Date: | April 1999 |
Primary Completion Date: | August 2007 (Final data collection date for primary outcome measure) |
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.
Ages Eligible for Study: | 18 Years to 70 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
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
United States, Maryland | |
Johns Hopkins University School of Medicine | |
Baltimore, Maryland, United States, 21205 |
Study Chair: | Grant J. Anhalt | Johns Hopkins University |
Study ID Numbers: | 199/15674, JHOC-J9912, JHOC-99022610 |
Study First Received: | February 2, 2001 |
Last Updated: | October 1, 2008 |
ClinicalTrials.gov Identifier: | NCT00010413 History of Changes |
Health Authority: | Unspecified |
immunologic disorders and infectious disorders pemphigus rare disease |
Autoimmune Diseases Immunologic Factors Skin Diseases, Vesiculobullous Skin Diseases Rare Diseases Antineoplastic Agents, Alkylating |
Cyclophosphamide Antirheumatic Agents Alkylating Agents Immunosuppressive Agents Pemphigus |
Autoimmune Diseases Molecular Mechanisms of Pharmacological Action Skin Diseases, Vesiculobullous Skin Diseases Immune System Diseases Immunologic Factors Antineoplastic Agents Physiological Effects of Drugs Cyclophosphamide |
Immunosuppressive Agents Pharmacologic Actions Pemphigus Therapeutic Uses Myeloablative Agonists Antineoplastic Agents, Alkylating Antirheumatic Agents Alkylating Agents |