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

Related Clinical Trial
A Safety Study of GAMMAGARD LIQUID (GGL) in Participants With Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) CIDP07 Rozanolixizumab Post Trial Access Program (the PTA) Study to Evaluate Safety and Efficacy of Different PANZYGA Dose Regimens in Pediatric Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) Patients Immunoadsorption Versus Immunoglobulins for Treatment of Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) Hizentra® in Inflammatory Neuropathies – pHeNIx Study Proof-of-concept Study for BIVV020 in Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) Subcutaneous Immunoglobulin in De-novo CIDP (SIDEC) InertiaLocoGraphy as a Biomarker of Immunoglobulin Therapy Efficacy in Chronic Inflammatory Demyelinating Polyradiculoneuropathy Rituximab in Chronic Inflammatory Demyelinating Polyneuropathy: A Phase II Study Randomized Open-label Trial to Compare Efficacy and Tolerance of Corticosteroids and IVIg Study to Evaluate Safety and Efficacy of Three Different Dosages of NewGam in Patients With Chronic Inflammatory Demyelinating Poly (Radiculo) Neuropathy A Registered Cohort Study of Immune-Mediated Neuropathies Interest of Mycophenolate for CIDP Weaning Efficacy and Safety Study of I10E in Treatment of Patients With CIDP Transcriptome Analysis of the Peripheral Blood in CIDP Phase III Efficacy, Safety, and Tolerability Study of HYQVIA/HyQvia and GAMMAGARD LIQUID/KIOVIG in CIDP Safety and Efficacy Study of Three Different Dosages of NewGam in Patients With CIDP Efficacy and Safety Study of I10E in the Maintenance Treatment of Patients With CIDP: Extension of PRISM Study I10E-1302 Evaluating the Effectiveness of Telemonitoring System in the Management of Patients With CIDP Subcutaneous Immunoglobulin for CIDP Long-Term Tolerability and Safety of HYQVIA/HyQvia in CIDP The Evaluation of Efficacy and Safety of Rituximab in Refractory CIDP Patients With IgG4 Autoantibodies Single vs. Multiple Privigen Dose Regimens in Pediatric CIDP Hematopoietic Stem Cell Transplantation in Chronic Inflammatory Demyelinating Polyneuropathy Lipoic Acid to Treat Chronic Inflammatory Demyelinating Polyneuropathy MRI in Diagnosing and Monitoring CIDP High-dose Cyclophosphamide for Moderate to Severe Refractory Chronic Inflammatory Demyelinating Polyneuropathy Biomark Study: Predict Intravenous Immunoglobulin Responders in Chronic Inflammatory Demyelinating Polyradiculoneuropathy A Study to Assess the Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod in Adults With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP, an Autoimmune Disorder That Affects the Peripheral Nerves) Phase III Clinical Trial of NPB-01maintenance Therapy in Patients With Chronic Inflammatory Demyelinating Polyneuropathy. IVIg Treatment-Related Fluctuations in CIDP Patients Using Daily Grip Strength Measurements A Study to Assess the Long-term Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod in Adults With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP, an Autoimmune Disorder That Affects the Peripheral Nerves) Safety and Efficacy of Avonex in Subjects With Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) Evaluate Efficacy and Safety of Fingolimod 0.5 mg Orally Once Daily Versus Placebo in Chronic Inflammatory Demyelinating Polyradiculoneuropathy Patients. Prevalence of Decreased Corneal Sensation in Patients With Chronic Inflammatory Demyelinating Polyneuropathy Panzyga in CIDP Administered at Different Infusion Rates Study of Efficacy and Safety of Privigen in Subjects With Chronic Inflammatory Demyelinating Polyneuropathy Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) and Treatment With Subcutaneous Immunoglobulin (IgPro20) A Study to Assess Long-term Safety, Tolerability and Efficacy of Rozanolixizumab in Subjects With Chronic Inflammatory Demyelinating Polyradiculoneuropathy A Study to Assess the Efficacy, Safety and Tolerability of Rozanolixizumab in Subjects With Chronic Inflammatory Demyelinating Polyradiculoneuropathy

Brief Title

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

Official Title

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 Phase

Phase 2

Study Type

Interventional


Primary Outcome

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

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

Condition

Chronic Inflammatory Demyelinating Polyneuropathy

Intervention

Cyclophosphamide


Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

0

Start Date

October 2003

Completion Date

November 2006


Eligibility 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
      

Gender

All

Ages

18 Years - 75 Years

Accepts Healthy Volunteers

No

Contacts

, , 



Administrative Informations


NCT ID

NCT01236456

Organization ID

65865



Study Sponsor

Stony Brook University


Study Sponsor

, , 


Verification Date

November 2021