Hematopoietic Stem Cell Transplantation in Chronic Inflammatory Demyelinating Polyneuropathy

This study is currently recruiting participants. (see Contacts and Locations)
Verified September 2014 by Northwestern University
Sponsor:
Information provided by (Responsible Party):
Richard Burt, MD, Northwestern University
ClinicalTrials.gov Identifier:
NCT00278629
First received: January 16, 2006
Last updated: September 23, 2014
Last verified: September 2014
  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
Biological: hematopoietic stem cell transplantation
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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

Resource links provided by NLM:


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: 50
Study Start Date: March 2005
Estimated Study Completion Date: December 2016
Estimated Primary Completion Date: December 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Hematopoietic stem cell transplantation
Autologous hematopoietic stem cell transplantation will be performed after conditioning regimen
Biological: hematopoietic stem cell transplantation
Autologous hematopoietic stem cell transplantation

  Show Detailed Description

  Eligibility

Ages Eligible for Study:   18 Years to 65 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion criteria:

  • EMG definite CIDP according to the EFNS / EPS criteria (section 3.2.4c) AND
  • Clinically typical CIDP (section 3.2.4 b) AND
  • 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 either IVIG or plasmapheresis
  • 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 video fluoroscopic examination.

      OR

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

      AND

    4. If patients are on IVIG or plasmapheresis, neurologic condition is documented to deteriorate (for example, new, increase fingertip parasthesias, 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 variants of CIDP will be included if they have auto- antibodies e.g. Anti GM1 or Anti-MAG.

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.
  • Diptheria, drug, or toxin exposure likely to be cause of neuropathy
  • Multifocal motor neuropathy
  • Presence of sphincter disturbance
  • 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. 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 or myocardial infarction within the last 6 months
    4. DLCO <40% or FEV1/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.
  • 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 ANC less than 1000/ul.
  • 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
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.

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

Contacts
Contact: Dzemila Spahovic, MD 312-695-4960 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         
Sub-Investigator: Jeff Allen, MD         
Sponsors and Collaborators
Northwestern University
Investigators
Principal Investigator: Richard Burt, MD Northwestern University
  More Information

No publications provided

Responsible Party: Richard Burt, MD, MD, Northwestern University
ClinicalTrials.gov Identifier: NCT00278629     History of Changes
Other Study ID Numbers: NU FDA CIDP.AUTO2003
Study First Received: January 16, 2006
Last Updated: September 23, 2014
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Polyneuropathies
Polyradiculoneuropathy, Chronic Inflammatory Demyelinating
Autoimmune Diseases
Autoimmune Diseases of the Nervous System
Demyelinating Diseases
Immune System Diseases
Nervous System Diseases
Neuromuscular Diseases
Peripheral Nervous System Diseases
Polyradiculoneuropathy

ClinicalTrials.gov processed this record on October 23, 2014