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

November 2, 2021 updated by: Stony Brook University

A Phase II Trial of High-dose Cyclophosphamide for Moderate to Severe Refractory Chronic Inflammatory Demyelinating Polyneuropathy

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.

Study Overview

Status

Withdrawn

Conditions

Intervention / Treatment

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

Interventional

Phase

  • Phase 2

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 75 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
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

Outcome Measure
The secondary endpoint of this study is to determine remission duration.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Stony Brook University

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

October 1, 2003

Study Completion (Anticipated)

November 1, 2006

Study Registration Dates

First Submitted

November 5, 2010

First Submitted That Met QC Criteria

November 5, 2010

First Posted (Estimate)

November 8, 2010

Study Record Updates

Last Update Posted (Actual)

November 9, 2021

Last Update Submitted That Met QC Criteria

November 2, 2021

Last Verified

November 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 65865

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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