Devic's Disease Clinical Trial
Official title:
Autologous Mesenchymal Stem Cells for the Treatment of Progressive and Refractory Neuromyelitis Optica Spectrum Disorders: an Open-label Phase 2a Proof-of-concept Study
Verified date | October 2018 |
Source | Tianjin Medical University General Hospital |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
Neuromyelitis optica (NMO) is a demyelinating and degenerative disorder of the central nervous system affecting vision and brain and spinal cord function which 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 Multipotent mesenchymal stromal cells in autoimmune diseases including multiple sclerosis, and based on the mechanisms of neuromyelitis optica, the investigators anticipate that mesenchymal stem cells transplantation may provide lasting disease stability for neuromyelitis optica patients.
Status | Completed |
Enrollment | 15 |
Est. completion date | December 31, 2014 |
Est. primary completion date | December 31, 2014 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years to 80 Years |
Eligibility |
Inclusion Criteria: - Clinically definite neuromyelitis optica or neuromyelitis optica spectrum disorder - Age > 18 year - EDSS > 3 - Progression continued relapses or worsening MRI after at least a year of attempted therapy as evidenced by one or more of the following: - Increase of 1 EDSS point (if baseline EDSS<5.0) or 0.5 EDSS points (if baseline EDSS >5.5) - Moderate-severe relapses in past 18 months - Gadolinium enhancing lesions (double or triple dose Gd) - 1 new T2 lesion - Evidence of recent inflammatory disease, as evidenced by any one of the following: - 1 moderate-severe relapses in past 18 months - 1 Gd-enhancing lesions (single, double or triple dose Gd) - 1 new T2 lesion Exclusion Criteria: - Received Immune inhibitors immunomodulator during the three months before the trial - Significant cardiac, renal, or hepatic failure or any other disease that may affect the results of the study - Allergies - Pregnant or possibly pregnant - Cognitive decline to understand or sign the informed consent - Brain tumor, HIV (+) tumor marker (+), blood pressure (BP): 200 /110 mmHg - Judged not suitable by doctors |
Country | Name | City | State |
---|---|---|---|
China | Tianjin Medical University General Hospital | Tianjin | Tianjin |
Lead Sponsor | Collaborator |
---|---|
Tianjin Medical University General Hospital |
China,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | EDSS | Compare EDSS change before and one year after mesenchymal stem cells (MSC) infusion | change from baseline to one year | |
Secondary | Annual relapse rate | Compare annual relapse rate before and one year after MSC infusion | 1 year after infusion | |
Secondary | Lesion load | Compared lesion load before and one year after MSC infusion | 1 year after infusion | |
Secondary | Retinal nerve fiber layer (RNFL) | Compared RNFL before and one year after MSC infusion | 1 year after infusion | |
Secondary | Cognition | Compare cognition questionnaire scale before and one year after MSC infusion | 1 year after infusion | |
Secondary | Immunological assessments | Compare anti-aquaporin4-ab before and one year after MSC infusion. | 1 year after infusion | |
Secondary | Immunological assessments | Compare immune cell subpopulation before and one year after MSC infusion. | 1 year after infusion | |
Secondary | Immunological assessments | Compare cytokine kinetics before and one year after MSC infusion. | 1 year after infusion | |
Secondary | cerebral volume | Compare cerebral volume before and one year after MSC infusion | 1 year after infusion |
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