Clinical Trial: Hematopoietic Stem Cell Therapy for Patients With Refractory Myasthenia Gravis

Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional

Official Title: Hematopoietic Stem Cell Therapy for Patients With Refractory Myasthenia Gravis

Brief Summary:

MG may be neonatal, congenital, or autoimmune. Neonatal MG arises from transplacental transfer of ACh receptor antibodies from a mother with autoimmune MG to the fetus. Neonatal MG resolves with post delivery clearance of maternal antibodies. Congenital MG results from a genetic defect in the ACh receptor. Patients with congenital MG do not have ACh receptor antibodies. Both neonatal and congenital MG are excluded from this study. Autoimmune MG, which is the most common form of MG, affects approximately 25,000 Americans. Like most autoimmune diseases, it is associated with particular HLA genotypes, has a female predominance, and environmental factors involved in breaking tolerance to the ACh receptor are unknown. Patients with refractory and severe autoimmune MG will be considered candidates for this study.

The purpose of this study is to assess the toxicity/feasibility (phase I) of autologous hematopoietic stem cell transplantation for refractory myasthenia gravis.


Detailed Summary:

There will be no randomization in this study. All subjects who are determined to be eligible for the study treatment will receive high dose cyclophosphamide and ATG followed by infusion of autologous peripheral blood stem cells. The procedures the subject will undergo are as follows:

  1. Physician visit to determine potential eligibility for the study. Subjects will be evaluated by a transplant physician and a neurologist. They will have a complete physical examination and will provide a full medical history at these visits. The study will be described in detail by the transplant physician and nurse and the consent form will be provided to be taken home to read.
  2. Insurance verification. Subjects who remain interested in pursuing the study treatment and who have refractory myasthenia gravis will proceed to the insurance verification phase. Third party payment or self-pay must be verified before subjects can proceed.
  3. Consent form. Prior to proceeding, the appropriate signatures will be obtained on the consent form. Subjects will be given an opportunity to ask further questions of the attending physician and transplant nurse prior to signing the consent form.
  4. Pre-transplant testing. To determine final eligibility for the study, subjects will undergo a series of tests/procedures. These include: CXR; electrocardiogram; MUGA or echocardiogram; pulmonary function test; CT scan of the sinuses; CT scan of the chest to r/o thymoma; dental examination; urinalysis; and blood testing to include CBC, chemistries, liver and kidney function tests, coagulation studies, viral studies and, for females, a pregnancy test. All pre-transplant testing is routine medical testing done to verify diagnosis and to insure adequate organ function and absence of viral illne
    Sponsor: Northwestern University

    Current Primary Outcome: Survival at 5 years; Osserman score; Quantitative Myasthenia gravis score (QMGS);Improvement is defined as a 50% improvement in QMGS; Deterioration is defined as a 25% deterioration in QMGS [ Time Frame: 5 years after transplant ]

    Original Primary Outcome:

    • Survival at 5 years
    • Osserman score
    • Quantitative Myasthenia gravis score (QMGS)
    • Improvement is defined as a 50% improvement in QMGS
    • Deterioration is defined as a 25% deterioration in QMGS


    Current Secondary Outcome:

    Original Secondary Outcome:

    Information By: Northwestern University

    Dates:
    Date Received: January 18, 2007
    Date Started: February 2002
    Date Completion:
    Last Updated: July 11, 2016
    Last Verified: July 2016