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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Recruitment Status : Withdrawn (I am relocating to Johns Hopkins Medical Center)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

Tracking Information
First Submitted Date  ICMJE November 5, 2010
First Posted Date  ICMJE November 8, 2010
Last Update Posted Date August 12, 2011
Study Start Date  ICMJE October 2003
Primary Completion Date Not Provided
Current Primary Outcome Measures  ICMJE
 (submitted: November 5, 2010)
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.
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT01236456 on Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: November 5, 2010)
The secondary endpoint of this study is to determine remission duration.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
Descriptive Information
Brief Title  ICMJE High-dose Cyclophosphamide for Moderate to Severe Refractory Chronic Inflammatory Demyelinating Polyneuropathy
Official Title  ICMJE A Phase II Trial of High-dose Cyclophosphamide for Moderate to Severe Refractory Chronic Inflammatory Demyelinating Polyneuropathy
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.
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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Chronic Inflammatory Demyelinating Polyneuropathy
Intervention  ICMJE Drug: Cyclophosphamide
Study Arms  ICMJE Not Provided
Publications *

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status  ICMJE Withdrawn
Estimated Enrollment  ICMJE
 (submitted: November 5, 2010)
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE November 2006
Primary Completion Date Not Provided
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 75 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Not Provided
Removed Location Countries United States
Administrative Information
NCT Number  ICMJE NCT01236456
Other Study ID Numbers  ICMJE 65865
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Not Provided
Study Sponsor  ICMJE Stony Brook University
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Stony Brook University
Verification Date August 2011

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP