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Hematopoietic Stem Cell Transplantation in Chronic Inflammatory Demyelinating Polyneuropathy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00278629
Recruitment Status : Completed
First Posted : January 18, 2006
Results First Posted : July 23, 2020
Last Update Posted : July 23, 2020
Information provided by (Responsible Party):
Richard Burt, MD, Northwestern University

Brief Summary:
Chronic inflammatory demyelinating polyneuropathy is disease believed to be due to immune cells, cells which normally protect the body, but are now attacking the nerves in the body. As a result, the affected nerves fail to respond, or respond only weakly, to stimuli causing numbing, tingling, pain, and progressive muscle weakness.The likelihood of progression of the disease is high. This study is designed to examine whether treating patients with high dose cyclophosphamide (a drug which reduces the function of the immune system) and ATG (a protein that kills the immune cells that are thought to be causing disease), followed by return of the previously collected blood stem cells will stop the progression of CIDP. Stem cells are undeveloped cells that have the capacity to grow into mature blood cells, which normally circulate in the blood stream. The purpose of the high dose cyclophosphamide and ATG is to destroy the cells in the immune system. The purpose of the stem cell infusion is to evaluate whether this treatment will produce a normal immune system that will no longer attack the body.

Condition or disease Intervention/treatment Phase
Chronic Inflammatory Demyelinating Polyneuropathy Biological: hematopoietic stem cell transplantation Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 80 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Non-myeloablative Autologous Hematopoietic Stem Cell Transplantation in Patients With Chronic Inflammatory Demyelinating Polyneuropathy: A Phase II Trial
Actual Study Start Date : February 21, 2005
Actual Primary Completion Date : January 14, 2017
Actual Study Completion Date : November 4, 2019

Arm Intervention/treatment
Experimental: Hematopoietic Stem Cell Transplantation in CIDP
Autologous hematopoietic stem cell transplantation will be performed after conditioning regimen of cyclophosphamide 200 mg/kg/intravenously(IV), rATG(thymoglobulin) 5.5 mg/kg/IV and rituximab 1000mg/IV.
Biological: hematopoietic stem cell transplantation
Autologous hematopoietic stem cell transplantation

Primary Outcome Measures :
  1. Number of Participants With Survival [ Time Frame: up to 5 years ]
    Survival of participants who survived the treatment and up to 5 years post treatment.

Secondary Outcome Measures :
  1. Disease Improvement - Medication Free Remission [ Time Frame: 6 months, 1, 2, 3, 4 and 5 years post transplant ]
    Post transplant immune medication-free remission with stable or improving neurological exam

  2. Disease Improvement - Ambulatory Assistance [ Time Frame: pre transplant, 6 months, 1, 2, 3, 4 and 5 years post transplant ]
    Disease Improvement - Requires No Assistance to Ambulate

  3. Change in Disability and Strength Functional Scales [ Time Frame: Pre Transplant, 6 months, 1, 2, 3, 4 and 5 years post transplant ]
    Change in the Medical Research Council (MRC) Scale: range is 0 (quadriplegic) - 60 (normal).

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion criteria:

  • Definite CIDP according to the European Federation of Neurological Societies/Peripheral Nerve Society (EFNS/PNS) criteria


  • Clinically typical or atypical CIDP


  • Failure to tolerate or respond to, or an incomplete response to, or relapse after at least 3 months of conventional treatment consisting of corticosteroids (equivalent dosage of prednisone 1.0/mg/day to 0.75mg/kg/day to start with adequate tapering trials of no less than 0.5mg/kg/day), and/or either intravenous immunoglobulin (IVIg) or plasmapheresis or cytoxan or rituxan
  • Failure to respond to therapy is defined by:

    1. Persistent muscle weakness Grade 3/5 or worse (MRC) in at least one muscle or grade 4/5 in at least two muscle groups OR
    2. Persistent dysphagia documented by either aspiration or insufficient clearing on videofluoroscopic examination.


    3. Persistent incapacitating sensory loss (e.g. gait ataxia, falls > 1/month)


    4. If patients are on IVIG or plasmapheresis, neurologic condition is documented to deteriorate (for example, new or increase finger tip paresthesias or increased leg heaviness) upon stopping IVIG (or plasmapheresis)@
  • Monoclonal gammopathy of undetermined significance (MUGS) (in which the pathogenesis of are thought to be the same as CIDP) will be allowed provided bone marrow aspirate and biopsy rules out multiple myeloma.
  • Other immune mediated or suspected immune mediated neuropathies such as multifocal motor neuropathy or anti-myelin-associated glycoprotein (anti-MAG) neuropathy may be treated but will be analyzed and reported separately.

Exclusion Criteria:

  • Any evidence of hereditary cause for neuropathy that is known or likely hereditary demyelination neuropathy because of family history, foot deformity, mutilation of hands or feet, retinitis pigmentosa, ichthyosis, or liability to pressure palsies.
  • Diphtheria, drug, or toxin exposure likely to be cause of neuropathy
  • Conditions in which the pathogenesis of the neuropathy may be different from CIDP such as: Lyme disease (Borrelia burgdorferi infection), POEMS syndrome, Osteosclerotic myeloma, malignancies such as Waldenstrom macroglobulinemia, and Castleman's)
  • Multiple myeloma
  • HIV positive
  • Insulin dependent Diabetes mellitus
  • Chronic active hepatitis
  • Age > 65 years old or < 18 years old
  • Significant end organ damage such as (not caused by CIDP):

    1. Left ventricular ejection fraction (LVEF) <40% or deterioration of LVEF during exercise test on multigated acquisition scan (MUGA) or echocardiogram.
    2. Untreated life-threatening arrhythmia.
    3. Active ischemic heart disease or heart failure or myocardial infarction within the last 6 months
    4. Diffusing capacity of lung for carbon monoxide (DLCO) <40% or forced expiratory volume at one second (FEV1) / forced expiratory volume (FEV) < 50%
    5. Serum creatinine >2.0.
    6. Liver cirrhosis, transaminases > 2 x of normal limits or bilirubin >2.0 unless due to Gilbert disease.
  • Prior history of malignancy except localized basal cell or squamous skin cancer or other localized cancer considered cured only by surgery
  • Positive pregnancy test, inability or unwillingness to pursue effective means of birth control, or failure to willingly accept or comprehend irreversible sterility as a side effect of therapy.
  • Psychiatric illness or mental deficiency making compliance with treatment or informed consent impossible.
  • Inability to give informed consent.
  • Major hematological abnormalities such as platelet count less than 100,000/ul or absolute neutrophil count (ANC) less than 1000/ul.
  • Failure to collect at least 2.0 x 106 cluster of differentiation 34 (CD34+) cells by apheresis and, if necessary, bone marrow harvest is a contraindication to treatment, i.e., receiving the conditioning regimen.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00278629

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United States, Illinois
Northwestern University, Feinberg School of Medicine
Chicago, Illinois, United States, 60611
Sponsors and Collaborators
Northwestern University
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Principal Investigator: Richard Burt, MD Northwestern University
Additional Information:
Publications of Results:
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Responsible Party: Richard Burt, MD, MD, Northwestern University Identifier: NCT00278629    
Other Study ID Numbers: NU FDA CIDP.AUTO2003
First Posted: January 18, 2006    Key Record Dates
Results First Posted: July 23, 2020
Last Update Posted: July 23, 2020
Last Verified: June 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Additional relevant MeSH terms:
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Polyradiculoneuropathy, Chronic Inflammatory Demyelinating
Peripheral Nervous System Diseases
Neuromuscular Diseases
Nervous System Diseases
Autoimmune Diseases of the Nervous System
Demyelinating Diseases
Autoimmune Diseases
Immune System Diseases