Northwestern University CIDP Trial (Enrolling)

“Hematopoietic Stem Cell Support in Chronic Inflammatory Demyelinating Polyneuropathy”

This study is currently recruiting patients.
Verified by Northwestern University March 2007
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

MedlinePlus related topics: Peripheral Nerve Disorders
Genetics Home Reference related topics: Peripheral Nerve Disorders

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 Outcomes: Survival; Disease improvement; Time to disease progression
Expected Total Enrollment: 10

Study start: March 2005

Eligibility
Ages Eligible for Study: up to 65 Years, Genders Eligible for Study: Both
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.

Location and Contact Information
Please refer to this study by ClinicalTrials.gov identifier NCT00278629

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

United States, Illinois
Northwestern University, Feinberg School of Medicine, Chicago, Illinois, 60611, United States; Recruiting
Richard Burt, MD, Principal Investigator
Yu Oyama, MD, Sub-Investigator
Kathleen Quigley, R.N.;MBA, Sub-Investigator
Kimberly Yaung, R.N., Sub-Investigator

Study chairs or principal investigators

Richard Burt, MD, Principal Investigator, Northwestern University

More Information
Study ID Numbers: NU FDA CIDP.AUTO2003
Last Updated: March 2, 2007
Record first received: January 16, 2006
ClinicalTrials.gov Identifier: NCT00278629
Health Authority: United States: Food and Drug Administration
ClinicalTrials.gov processed this record on 2007-03-09