NCT03829566

Autologous Transplant To End NMO Spectrum Disorder

Autologous Hematopoietic Stem Cell Transplant for Neuromyelitis Optica Spectrum Disorder (NMOSD)

• Status: Withdrawn
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: Northwestern University · Academic / Other
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

This study is designed to treat your disease with an autologous stem cell transplant using a regimen of immune suppressant drugs and chemotherapy to reset your immune system and to determine if your disease will go into long-term remission.

Detailed description

The autologous stem cell transplant used in this research study is an investigational procedure that uses cyclophosphamide (chemotherapy), rabbit antithymocyte globulin (rATG) (a protein that kills the immune cells that are thought to be causing your disease), rituximab (a biologic drug that targets B cells of your immune system), and intravenous immunoglobulin (IVIg) (pooled IgG antibodies from plasma donors with immunomodulatory and anti-inflammatory effects), followed by return of your own previously collected blood stem cells (autologous stem cell transplant). One day of plasmapheresis will also be performed the day prior to admission for stem cell transplant to remove disease-causing antibodies. The ability of this experimental treatment to stop relapses and progression (worsening) of your NMOSD will be assessed.

Conditions

• Neuromyelitis Optica
• Devic's Disease
• NMO Spectrum Disorder

Interventions

Type	Name	Description
DRUG	Rituximab	Monoclonal antibody therapy used to treat certain autoimmune diseases and types of cancer
DRUG	Cyclophosphamide	A medication used as chemotherapy and to suppress the immune system
DRUG	Mesna	A medication used in those taking cyclophosphamide or ifosfamide to decrease the risk of bleeding from the bladder
DRUG	rATG	A rabbit polyclonal antibody to lymphocytes
DRUG	Methylprednisolone	A corticosteroid medication used to suppress the immune system and decrease inflammation
DRUG	G-CSF	A glycoprotein that stimulates the bone marrow to produce granulocytes and stem cells and release them into the bloodstream
BIOLOGICAL	IVIg	Pooled immunoglobulin (IgG) from thousands of plasma donors that has immunomodulatory and anti-inflammatory effects
BIOLOGICAL	Autologous Stem Cells	Infusion of patient's own stem cells

Timeline

• Start date: 2019-11-01
• Primary completion: 2025-01-01
• Completion: 2025-11-28
• First posted: 2019-02-04
• Last updated: 2019-11-20

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study