Autologous Hematopoietic Stem Cell Transplant in Neuromyelitis Optica — SCT-NMO  

Neuromyelitis Optica Clinical Trial

Official title: Autologous Hematopoietic Stem Cell Transplant in Patients With Neuromyelitis Optica

Status Terminated

Clinical Trial Summary

Neuromyelitis Optica (NMO) is a demyelinating and degenerative disorder of the CNS affecting vision and spinal cord function. This disease is rare compared to Multiple Sclerosis (MS), but it is devastating and often leads to accumulating disability with a 5 year-mortality of approximately 30%. Survivors are typically left with severe morbidity secondary to blindness, quadriparesis and respiratory failure. No agent has been found to be highly effective in halting disease activity. Based on recent outcomes of stem cell transplant trials and reports in autoimmune diseases including MS, and based on the mechanisms of NMO, we anticipate that stem cell transplantation may provide lasting disease stability for NMO patients. The hypothesis of the present trial is that autologous hematopoetic stem cell transplantation in patients with NMO will provide lasting benefit in relapse prevention. Specifically, we anticipate a 50% reduction in the proportion of patients experiencing relapse over a three year period. We will be following patients for a total of five years after transplantation.

Clinical Trial Description

Patients who are deemed eligible will be enrolled and undergo a two stage transplant process followed by neurological assessments every 6 months for the following 5 years assessing EDSS, visual metrics, MRI, AQP-4 antibodies, MSFC and SF36. ;

Related Conditions & MeSH terms

• Neuromyelitis Optica

• NCT number: NCT01339455
• Study type: Interventional
• Source: University of Calgary
• Status: Terminated
• Phase: Phase 1/Phase 2
• Start date: March 2011
• Completion date: March 2017