Hematopoietic Stem Cell Transplantation in Chronic Inflammatory Demyelinating Polyneuropathy

Completed · Phase 2

Conditions studied: Chronic Inflammatory Demyelinating Polyneuropathy

In brief

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.

Key facts

Study ID

NCT00278629

Run by

Northwestern University

People needed

80

Starts

2005-02-21

Expected to finish

2019-11-04

Last updated by the study team

2020-07-23

Who can join

Age: 18 and older, up to 65. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Definite CIDP according to the European Federation of Neurological Societies/Peripheral Nerve Society (EFNS/PNS) criteria
• AND
• Clinically typical or atypical CIDP
• 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/or either intravenous immunoglobulin (IVIg) or plasmapheresis or cytoxan or rituxan
• Failure to respond to therapy is defined by:
• 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
• Persistent dysphagia documented by either aspiration or insufficient clearing on videofluoroscopic examination.
• OR
• Persistent incapacitating sensory loss (e.g. gait ataxia, falls > 1/month)
• AND
• 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.

You may not qualify if…

• 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):
• Left ventricular ejection fraction (LVEF) <40% or deterioration of LVEF during exercise test on multigated acquisition scan (MUGA) or echocardiogram.
• Untreated life-threatening arrhythmia.
• Active ischemic heart disease or heart failure or myocardial infarction within the last 6 months
• Diffusing capacity of lung for carbon monoxide (DLCO) <40% or forced expiratory volume at one second (FEV1) / forced expiratory volume (FEV) < 50%
• Serum creatinine >2.0.
• 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.

Where it is running

• Northwestern University, Feinberg School of Medicine — Chicago, Illinois, United States

Full record on ClinicalTrials.gov

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