Hematopoietic Stem Cell Transplantation in Chronic Inflammatory Demyelinating Polyneuropathy

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT00278629
• Date of registration: 16/01/2006
• Prospective Registration: No
• Primary sponsor: Northwestern University
• Public title: Hematopoietic Stem Cell Transplantation in Chronic Inflammatory Demyelinating Polyneuropathy
• Scientific title: Non-myeloablative Autologous Hematopoietic Stem Cell Transplantation in Patients With Chronic Inflammatory Demyelinating Polyneuropathy: A Phase II Trial
• Date of first enrolment: February 21, 2005
• Target sample size: 80
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00278629
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Richard Burt, MD
• Affiliation: Northwestern University

Key inclusion & exclusion criteria

Inclusion criteria:

- 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:

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.

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 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.



Age minimum: 18 Years
Age maximum: 65 Years
Gender: All

Health Condition(s) or Problem(s) studied

Chronic Inflammatory Demyelinating Polyneuropathy

Intervention(s)

Biological: hematopoietic stem cell transplantation

Primary Outcome(s)

Number of Participants With Survival [Time Frame: up to 5 years]

Secondary Outcome(s)

Disease Improvement - Medication Free Remission [Time Frame: 6 months, 1, 2, 3, 4 and 5 years post transplant]

Disease Improvement - Ambulatory Assistance [Time Frame: pre transplant, 6 months, 1, 2, 3, 4 and 5 years post transplant]

Change in Disability and Strength Functional Scales [Time Frame: Pre Transplant, 6 months, 1, 2, 3, 4 and 5 years post transplant]

Secondary ID(s)

NU FDA CIDP.AUTO2003

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 23/07/2020
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00278629