NCT03342638

Maximizing Outcome of Multiple Sclerosis Transplantation

Maximizing Outcome of Multiple Sclerosis Transplantation: "MOST" Trial

• Status: Terminated
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 66 (actual)
• Sponsor: Northwestern University · Academic / Other
• Sex: All
• Age: 18 Years – 58 Years
• Healthy volunteers: Not accepted

Summary

Randomized study of autologous un-manipulated peripheral blood hematopoietic stem cell transplant (HSCT) comparing two regimens: (1) cyclophosphamide and rabbit anti-thymoglobulin (rATG) versus (2) cyclophosphamide, rATG, and Intravenous Immunoglobulin (IVIg).

Detailed description

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 predominantly an immune-mediated and inflammatory disease. While current immune based therapies delay disability, no intervention has been proven to prevent progressive disability.The investigators propose a randomized study of autologous unmanipulated peripheral blood hematopoietic stem cell transplant (HSCT) comparing two different conditioning regimens: (1) cyclophosphamide and rabbit anti-thymoglobulin (rATG) versus (2) cyclophosphamide, rATG, and Intravenous Immunoglobulin (IVIg).

Conditions

• Multiple Sclerosis, Relapsing-Remitting

Interventions

Type	Name	Description
DRUG	Cyclophosphamide	Potent immunosuppressive agent; an alkylating agent
DRUG	Mesna	Medication used to decrease the risk of hemorrhagic cystitis prophylaxis
DRUG	rATG	A predominantly lymphocyte-specific immunosuppressive agent which contains antibodies specific to the antigens commonly found on the surface of T cells
DRUG	Methylprednisolone	Steroid
DRUG	G-CSF	Granulocyte-colony stimulating factor; a glycoprotein that stimulates the bone marrow to produce granulocytes and stem cells and release them into the bloodstream
BIOLOGICAL	IVIg	Sterile, purified immunoglobulin G (IgG) products manufactured from pooled human plasma and typically contain more than 95% unmodified IgG, which has intact Fc-dependent effector functions and only trace amounts of immunoglobulin A (IgA) or immunoglobulin M (IgM).
BIOLOGICAL	Autologous Stem Cells	Infusion of participant's own stem cells

Timeline

• Start date: 2017-11-08
• Primary completion: 2019-07-23
• Completion: 2019-10-09
• First posted: 2017-11-17
• Last updated: 2021-01-11
• Results posted: 2021-01-11

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study