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Hematopoietic Stem Cell Support in Chronic Inflammatory Demyelinating Polyneuropathy
This study is currently recruiting participants.
Verified by Northwestern University, November 2008
Sponsored by: Northwestern University
Information provided by: Northwestern University
ClinicalTrials.gov Identifier: NCT00278629
  Purpose

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 your 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 Intervention Phase
Polyneuropathy
Procedure: hematopoietic stem cell transplantation
Phase I

Drug Information available for: Cyclophosphamide
U.S. FDA Resources
Study Type: Interventional
Study Design: Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study
Official Title: High Dose Cyclophosphamide & ATG With Hematopoietic Stem Cell Support in Patients With Chronic Inflammatory Demyelinating Polyneuropathy: A Phase I Trial

Further study details as provided by Northwestern University:

Primary Outcome Measures:
  • Survival;Disease improvement;Time to disease progression [ Time Frame: 5 years after transplant ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 10
Study Start Date: March 2005
Estimated Study Completion Date: March 2011
Estimated Primary Completion Date: March 2010 (Final data collection date for primary outcome measure)
Intervention Details:
    Procedure: hematopoietic stem cell transplantation
    Autologous hematopoietic stem cell transplantation
  Eligibility

Ages Eligible for Study:   up to 65 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Age 65 years old or less at the time of pretransplant evaluation.
  2. Definite or probable CIDP according to the criteria of the Ad Hoc Subcommittee of the American Academy of Neurology AIDS Task Force (SEE APPENDIX).
  3. Failure to respond to, or an incomplete response to, or relapse to at least 3 months of conventional treatment consisting of corticosteroids (equivalent dosage of prednisone 1.0/mg/day to start), and IVIG or plasmapheresis and patients also must have failed one or more of the following: cyclophosphamide, tacrolimus, azathioprine, cyclosporin A, methotrexate, mycophenolate mofetil, TNF inhibitors (i.e. etanercept) or any other immunosuppressive drugs or immune modulators.
  4. Failure to therapy defined by (one of the following) (not caused by unrelated conditions):

    1. Persistent muscle weakness Grade 3/5 or worse (MRC) in at least one muscle in two limbs (e.g. both deltoids, both hip flexors) or one limb.
    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)

Exclusion Criteria:

  • 1. Any evidence of another cause for neuropathy.

    2. Nerve pathology (if available) not consistent with CIDP.

    3. Poor performance (PS) status (ECOG >2) at the time of entry, unless decline of PS is due to the disease itself.

    4. Significant end organ damage such as (not caused by CIDP):

    1. LVEF <40% or deterioration of LVEF during exercise test on MUGA or echocardiogram.
    2. Untreated life-threatening arrhythmia.
    3. Active ischemic heart disease or heart failure.
    4. DLCO <40% or FEV1/FEV < 50%.
    5. Serum creatinine >2.5 or creatinine clearance <30ml/min.
    6. Liver cirrhosis, transaminases >3x of normal limits or bilirubin >2.0 unless due to Gilbert disease.

      5. HIV positive.

      6. Uncontrolled diabetes mellitus, or any other illness that in the opinion of the investigators would jeopardize the ability of the patient to tolerate aggressive treatment.

      7. Prior history of malignancy except localized basal cell or squamous skin cancer. Other malignancies for which the patient is judged to be cured by local surgical therapy, such as (but not limited to) head and neck cancer, or stage I or II breast cancer will be considered on an individual basis.

      8. 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.

      9. Psychiatric illness or mental deficiency making compliance with treatment or informed consent impossible.

      10. Inability to give informed consent.

      11. Major hematological abnormalities such as platelet count less than 100,000/ul or ANC less than 1000/ul.

      12. Failure to collect at least 2.0 x 106 CD34+ cells by apheresis and, if necessary, bone marrow harvest is a contraindication to treatment, i.e., receiving the conditioning regimen.

  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00278629

Contacts
Contact: Dzemila Spahovic, MD 312-908-0059 d-spahovic@northwestern.edu

Locations
United States, Illinois
Northwestern University, Feinberg School of Medicine Recruiting
Chicago, Illinois, United States, 60611
Principal Investigator: Richard Burt, MD            
Sub-Investigator: Kathleen Quigley, R.N.;MBA            
Sub-Investigator: Kimberly Yaung, R.N.            
Principal Investigator: Robert Sufit Sufit, MD            
Sponsors and Collaborators
Northwestern University
Investigators
Principal Investigator: Richard Burt, MD Northwestern University
  More Information

Responsible Party: Northwestern University ( Robert Craig, MD )
Study ID Numbers: NU FDA CIDP.AUTO2003
Study First Received: January 16, 2006
Last Updated: November 6, 2008
ClinicalTrials.gov Identifier: NCT00278629  
Health Authority: United States: Food and Drug Administration

Study placed in the following topic categories:
Polyradiculoneuropathy, Chronic Inflammatory Demyelinating
Autoimmune Diseases
Demyelinating Diseases
Neuromuscular Diseases
Polyradiculoneuropathy
Peripheral Nervous System Diseases
Polyneuropathies
Demyelinating diseases
Cyclophosphamide
Chronic inflammatory demyelinating polyneuropathy
Autoimmune Diseases of the Nervous System

Additional relevant MeSH terms:
Immune System Diseases
Nervous System Diseases

ClinicalTrials.gov processed this record on January 16, 2009