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

This study has been withdrawn prior to enrollment.
(I am relocating to Johns Hopkins Medical Center)
Sponsor:
Information provided by:
Stony Brook University
ClinicalTrials.gov Identifier:
NCT01236456
First received: November 5, 2010
Last updated: August 11, 2011
Last verified: August 2011
  Purpose

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 Intervention Phase
Chronic Inflammatory Demyelinating Polyneuropathy
Drug: Cyclophosphamide
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase II Trial of High-dose Cyclophosphamide for Moderate to Severe Refractory Chronic Inflammatory Demyelinating Polyneuropathy

Resource links provided by NLM:


Further study details as provided by Stony Brook University:

Primary Outcome Measures:
  • 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:
  • The secondary endpoint of this study is to determine remission duration.

Estimated Enrollment: 25
Study Start Date: October 2003
Estimated Study Completion Date: November 2006
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.

  Eligibility

Ages Eligible for Study:   18 Years to 75 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

No Contacts or Locations Provided
  More Information

Publications:
ClinicalTrials.gov Identifier: NCT01236456     History of Changes
Other Study ID Numbers: 65865
Study First Received: November 5, 2010
Last Updated: August 11, 2011
Health Authority: United States: Food and Drug Administration

Keywords provided by Stony Brook University:
chronic inflammatory demyelinating polyneuropathy
cyclophosphamide
autoimmune

Additional relevant MeSH terms:
Polyneuropathies
Polyradiculoneuropathy, Chronic Inflammatory Demyelinating
Demyelinating Diseases
Peripheral Nervous System Diseases
Neuromuscular Diseases
Nervous System Diseases
Polyradiculoneuropathy
Autoimmune Diseases of the Nervous System
Autoimmune Diseases
Immune System Diseases
Cyclophosphamide
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Pharmacologic Actions
Antirheumatic Agents
Therapeutic Uses
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists

ClinicalTrials.gov processed this record on July 26, 2014