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High-dose Cyclophosphamide for Moderate to Severe Refractory Chronic Inflammatory Demyelinating Polyneuropathy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01236456
Recruitment Status : Withdrawn (I am relocating to Johns Hopkins Medical Center)
First Posted : November 8, 2010
Last Update Posted : August 12, 2011
Information provided by:
Stony Brook University

Brief Summary:
The primary endpoint of this study is to determine what percentage of patients receiving high-dose Cyclophosphamide may experience a halt in the worsening of their disease or experience improvement of their disease and for how long the benefit may last.

Condition or disease Intervention/treatment Phase
Chronic Inflammatory Demyelinating Polyneuropathy Drug: Cyclophosphamide Phase 2

Detailed Description:

Chronic inflammatory demyelinating polyneuropathy (CIDP) is a common and under-recognized peripheral neuropathy that is thought to be immune-mediated. Randomized, placebo controlled clinical trials in CIDP demonstrate benefit from treatment with corticosteroids, plasmapheresis, and IV Ig. However, not all patients respond to these therapies. IV cyclophosphamide, cyclosporine, interferons, total lymphoid irradiation, and mycophenolate mofetil have been proposed as appropriate therapies for refractory patients.

Patients with CIDP often respond to immune-modulating treatment. However, the high rate of relapse and treatment-related side effects result in poor long-term outcomes for many patients. CIDP is assumed to be an autoimmune disease, but the pathogenesis is poorly understood. T cell infiltrates are predominantly CD8, suggesting a T cell mediated process. There is not, however, restricted T cell receptor Vbeta utilization seen in sural nerve biopsies. Immunoglobulin and complement deposits noted on the myelin sheaths support an antibody-mediated process. Antibodies to the P0 myelin protein are seen in a minority of patients. High-dose cyclophosphamide is believed to eradicate both B and T lymphocytes. This therapy does not damage hematopoietic stem cells, which allows for rapid white cell recovery without stem cell rescue.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 25 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase II Trial of High-dose Cyclophosphamide for Moderate to Severe Refractory Chronic Inflammatory Demyelinating Polyneuropathy
Study Start Date : October 2003
Estimated Study Completion Date : November 2006

Primary Outcome Measures :
  1. The primary endpoint of this study is to evaluate the response rate of CIDP patients as determined by functional score, change in Summated compound motor action potential and strength, after high-dose cyclophosphamide therapy.

Secondary Outcome Measures :
  1. The secondary endpoint of this study is to determine remission duration.

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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Diagnosis of CIDP according to the American Academy of Neurology clinical and electrophysiologic criteria
  • Age >18 but < 75 years
  • Modified Rankin Scale score of >3 after two standard treatment regimens
  • Patient must have a left ventricular ejection fraction of >45%
  • Serum Creatinine <3mg/dL
  • Willingness to participate in a clinical trial

Exclusion Criteria:

  • Patients who are preterminal or moribund
  • Patients with active malignancies
  • Patients with chromosomal abnormalities or peripheral blood counts suggestive of myelodysplastic syndrome
  • Patients with active bacterial or fungal infections requiring oral or intravenous antimicrobials are not eligible until resolution of the infection
  • Pregnant women and breast-feeding women

Publications of Results:
Layout table for additonal information Identifier: NCT01236456     History of Changes
Other Study ID Numbers: 65865
First Posted: November 8, 2010    Key Record Dates
Last Update Posted: August 12, 2011
Last Verified: August 2011
Keywords provided by Stony Brook University:
chronic inflammatory demyelinating polyneuropathy
Additional relevant MeSH terms:
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Polyradiculoneuropathy, Chronic Inflammatory Demyelinating
Peripheral Nervous System Diseases
Neuromuscular Diseases
Nervous System Diseases
Autoimmune Diseases of the Nervous System
Demyelinating Diseases
Autoimmune Diseases
Immune System Diseases
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists