NCT01339455

Autologous Hematopoietic Stem Cell Transplant in Neuromyelitis Optica

Autologous Hematopoietic Stem Cell Transplant in Patients With Neuromyelitis Optica

• Status: Terminated
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 3 (actual)
• Sponsor: University of Calgary · Academic / Other
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

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.

Detailed 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.

Conditions

• Neuromyelitis Optica

Interventions

Type	Name	Description
PROCEDURE	AHSCT	AHSCT Procedure: 1. Mobilization and Harvesting: * Cyclophosphamide * Rituximab * GSCF * Dexamethasone * Apheresis 2. Conditioning and Infusion (3-4 weeks after Mobilization and Harvesting): * Cyclophosphamide * MESNA * Rabbit ATG * Rituximab * Methylprednisolone * Stem Cell infusion * GSCF

Timeline

• Start date: 2011-03-01
• Primary completion: 2017-03-01
• Completion: 2017-03-01
• First posted: 2011-04-20
• Last updated: 2018-05-02

Locations

1 site across 1 country: Canada