Clinical Trial: Autologous Hematopoietic Stem Cell Transplant in Neuromyelitis Optica
Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional
Official Title: Autologous Hematopoietic Stem Cell Transplant in Patients With Neuromyelitis Optica
Brief 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 Summary: 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.
Sponsor: University of Calgary
Current Primary Outcome: Proportion relapse-free at three years [ Time Frame: 3 years ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Proportion relapse-free at five years [ Time Frame: 5 years ]The proportion of surviving patients relapse-free at year five
- Relapse count [ Time Frame: Annually over 5 years ]Number of NMO relapse events
- Disability progression [ Time Frame: Over 5 years ]Time to progression of EDSS by one step
- Retinal nerve fiber layer (RFNL) status [ Time Frame: 5 years ]Change in RNFL by optical coherence tomography over trial
- 25 foot timed walk test [ Time Frame: 5 years ]Change in 25 ft timed walk test over trial
- PASAT [ Time Frame: Annually over 5 years ]Annual and change from baseline to end of trial in Paced Auditory Serial Addition Test to assess cognitive function.
- Hospitalization [ Time Frame: Over 5 years ]Number of hospitalizations, days in hospital over trial period
- Overall survival [ Time Frame: Over 5 years ]Survival over trial period
- Time to next relapse [ Time Frame: Over 5 years ]Time to next relapse after transplant
Original Secondary Outcome: Same as current
Information By: University of Calgary
Dates:
Date Received: April 18, 2011
Date Started: March 2011
Date Completion: December 2020
Last Updated: December 30, 2015
Last Verified: December 2015