Northwestern University CIDP Trial (Enrolling)

    • Anonymous
      March 10, 2007 at 6:02 pm

      “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 Identifier: NCT00278629


      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
      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

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

      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.

      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 identifier NCT00278629

      Dzemila Spahovic, MD 312-908-0059

      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 Identifier: NCT00278629
      Health Authority: United States: Food and Drug Administration processed this record on 2007-03-09

    • Anonymous
      March 10, 2007 at 7:19 pm

      If the study started in 2005, wouldn’t we have some data about it now??


    • Anonymous
      March 10, 2007 at 10:54 pm

      I spoke with Kim Yaung before (about 7 months ago) about where things were and they were still looking for canadates for the trails. She is the one I asked about the stem cells from the baby teeth and if we should be presueing the effort to havvest stem cells from the kids baby teeth. She said the amount they get is too small….remember now?

      Hope things go well tommorrow, how did the liadacane cream work?


    • Anonymous
      March 15, 2007 at 2:46 pm

      I, too, checked on this a few months ago, and fell under the criteria needed.
      BUT, they said it was experimental, and they were not covering expenses.
      So, only the wealthy could afford something like this, since the health insurance companies will not cover something experimental.
      The last time I talked with them, they had two people waiting to see if their insurance would cover it…
      “don’t hold your breath…”

    • Anonymous
      March 16, 2007 at 1:45 am

      This is actually very similar to the protocol that I went through back in 2003. I had 9 months of heavy doses of cytoxan (cyclopohsphamide) infusions, which did arrest my CIDP. I haven’t needed a treatment of any kind since Sept of 2003. But I did not have any stem cells harvested, just the cytoxan infusions. After about 3 months, I could tell that the CIDP had quit progressing, & it took about 6-9 months to start see noticeable improvements. I am still left with reisduals, but at least now I can walk again & I got most of the use of my hands back…