Stem Cell Therapy for Patients With Multiple Sclerosis Failing Alternate Approved Therapy- A Randomized Study
Status:
Completed
Trial end date:
2019-08-30
Target enrollment:
Participant gender:
Summary
Multiple sclerosis (MS) is at onset an immune-mediated demyelinating disease. In most cases,
it starts as a relapsing-remitting disease with distinct attacks and no symptoms between
flares. Over years or decades, virtually all cases transition into a progressive disease in
which insidious and slow neurologic deterioration occurs with or without acute flares.
Relapsing-remitting disease is often responsive to immune suppressive or modulating
therapies, while immune based therapies are generally ineffective in patients with a
progressive clinical course. This clinical course and response to immune suppression, as well
as neuropathology and neuroimaging studies, suggest that disease progression is associated
with axonal atrophy. Disability correlates better with measures of axonal atrophy than immune
mediated demyelination. Therefore, immune based therapies, in order to be effective, need to
be started early in the disease course while MS is predominately an immune-mediated and
inflammatory disease. While current immune based therapies delay disability, no intervention
has been proven to prevent progressive disability. We propose, as a randomized study,
autologous unmanipulated PBSCT using a conditioning regimen of cyclophosphamide and rabbit
antithymocyte globulin (rATG) versus FDA approved standard of care (i.e. interferon,
glatiramer acetate, mitoxantrone, natalizumab, fingolimod, or tecfidera) in patients with
inflammatory (relapsing) MS despite treatment with alternate approved therapy.
Phase:
Phase 2
Details
Lead Sponsor:
Northwestern University Sheffield Teaching Hospitals NHS Foundation Trust
Collaborators:
Sheffield Teaching Hospitals NHS Foundation Trust University of Sao Paulo Uppsala University