Autologous Hematopoietic Stem Cell Transplant in Neuromyelitis Optica (SCT-NMO)
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.
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Autologous Hematopoietic Stem Cell Transplant in Patients With Neuromyelitis Optica|
- Proportion relapse-free at three years [ Time Frame: 3 years ] [ Designated as safety issue: No ]The proportion of surviving patients who are relapse-free at three years after transplant
- Proportion relapse-free at five years [ Time Frame: 5 years ] [ Designated as safety issue: No ]The proportion of surviving patients relapse-free at year five
- Relapse count [ Time Frame: Annually over 5 years ] [ Designated as safety issue: No ]Number of NMO relapse events
- Disability progression [ Time Frame: Over 5 years ] [ Designated as safety issue: No ]Time to progression of EDSS by one step
- Retinal nerve fiber layer (RFNL) status [ Time Frame: 5 years ] [ Designated as safety issue: No ]Change in RNFL by optical coherence tomography over trial
- 25 foot timed walk test [ Time Frame: 5 years ] [ Designated as safety issue: No ]Change in 25 ft timed walk test over trial
- PASAT [ Time Frame: Annually over 5 years ] [ Designated as safety issue: No ]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 ] [ Designated as safety issue: Yes ]Number of hospitalizations, days in hospital over trial period
- Overall survival [ Time Frame: Over 5 years ] [ Designated as safety issue: Yes ]Survival over trial period
- Time to next relapse [ Time Frame: Over 5 years ] [ Designated as safety issue: No ]Time to next relapse after transplant
|Study Start Date:||March 2011|
|Estimated Study Completion Date:||December 2020|
|Estimated Primary Completion Date:||March 2020 (Final data collection date for primary outcome measure)|
All patients undergo autologous hematopoietic stem cell transplantation in a two stage process.
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01339455
|Contact: Jodie M Burton, MD,MSc,FRCPC||403 firstname.lastname@example.org|
|Foothills Medical Centre, University of Calgary||Recruiting|
|Calgary, Alberta, Canada, T2N 2T9|
|Principal Investigator:||Jodie M Burton, MD,MSc,FRCPC||Department of Clinical Neurosciences, Hotchkiss Brain Institute, University of Calgary|
|Principal Investigator:||Jan Storek, MD,PhD||Department of Medicine, University of Calgary|