Hematopoietic Stem Cell Therapy for Patients With Refractory Myasthenia Gravis
Status:
Terminated
Trial end date:
2016-06-01
Target enrollment:
Participant gender:
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.