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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

WITHDRAWN

Clinical Phase

PHASE2

Study Classification

INTERVENTIONAL

Study Start Date

2003-10-31

Study Completion Date

2006-11-30

Brief Summary

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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

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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.

Conditions

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Chronic Inflammatory Demyelinating Polyneuropathy

Keywords

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chronic inflammatory demyelinating polyneuropathy cyclophosphamide autoimmune

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Interventions

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Cyclophosphamide

Intervention Type DRUG

Eligibility Criteria

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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

Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Countries

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United States

References

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Gladstone DE, Prestrud AA, Brannagan TH 3rd. High-dose cyclophosphamide results in long-term disease remission with restoration of a normal quality of life in patients with severe refractory chronic inflammatory demyelinating polyneuropathy. J Peripher Nerv Syst. 2005 Mar;10(1):11-6. doi: 10.1111/j.1085-9489.2005.10104.x.

Reference Type RESULT

PMID: 15703014 (View on PubMed)

Brannagan TH 3rd, Pradhan A, Heiman-Patterson T, Winkelman AC, Styler MJ, Topolsky DL, Crilley PA, Schwartzman RJ, Brodsky I, Gladstone DE. High-dose cyclophosphamide without stem-cell rescue for refractory CIDP. Neurology. 2002 Jun 25;58(12):1856-8. doi: 10.1212/wnl.58.12.1856.