Hematopoietic Stem Cell Therapy for Patients With Refractory Myasthenia Gravis
Stopped early · Phase 1
Conditions studied: Myasthenia Gravis
In brief
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.
Key facts
- Study ID
- NCT00424489
- Run by
- Northwestern University
- People needed
- 9
- Starts
- 2002-02-01
- Expected to finish
- 2016-06-01
- Last updated by the study team
- 2018-08-31
Who can join
Age: 15 and older, up to 65. Sex: any. Healthy volunteers: not accepted.
You may not qualify if…
- Significant end organ damage such as:
- LVEF <40% or deterioration of LVEF during exercise test on MUGA or echocardiogram.
- Untreated life-threatening arrhythmia, active ischemic heart disease or heart failure.
- DLCO < 40% of predicted value.
- Serum creatinine > 2.5 mg/dl.
- Liver cirrhosis, transaminases >3x of normal limits, or bilirubin >2.0 unless due to Gilberts disease.
- HIV positive.
- 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.
- 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.
- Positive pregnancy test, inability or unable to pursue effective means of birth control, 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
- Congenital myasthenia gravis
- Neonatal myasthenia gravis
- Osserman grade 1 or 2
- Pure red cell aplasia
- Any patient on insulin
Where it is running
- Northwestern University, Feinberg School of Medicine — Chicago, Illinois, United States
Full record on ClinicalTrials.gov
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