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

Sponsor
Stony Brook University (Other)
Overall Status
Withdrawn
CT.gov ID
NCT01236456
Collaborator
(none)
0
Enrollment
37
Duration (Months)

Study Details

Study Description

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 DiseaseIntervention/TreatmentPhase
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.

Study Design

Study Type:
Interventional
Actual Enrollment :
0 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 :
Oct 1, 2003
Anticipated Study Completion Date :
Nov 1, 2006

Outcome Measures

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. []

Eligibility Criteria

Criteria

Ages Eligible for Study:
18 Years to 75 Years
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

Contacts and Locations

Locations

No locations specified.

Sponsors and Collaborators

  • Stony Brook University

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
, ,
ClinicalTrials.gov Identifier:
NCT01236456
Other Study ID Numbers:
  • 65865
First Posted:
Nov 8, 2010
Last Update Posted:
Nov 9, 2021
Last Verified:
Nov 1, 2021

Study Results

No Results Posted as of Nov 9, 2021