Autologous Mesenchymal Stem Cells for the Treatment of Neuromyelitis Optica Spectrum Disorders

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02249676
• Other study ID #: IRB2013-055-02
• Status: Completed
• Phase: Phase 2
• Start date: January 2013
• Est. completion date: December 31, 2014

Study information

• Verified date: October 2018
• Source: Tianjin Medical University General Hospital
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

Description:

Primary objective was to assess feasibility and safety; the investigators compared adverse events from up to 3months before treatment until up to 12 months after the infusion.

As a secondary objective, the investigators chose efficacy outcomes to assess the Expanded Disability Status (EDSS)、annual relapse rate (ARR) and time to next relapse after transplant.

Third objective anterior visual pathway and pyramidal tract as a model of wider disease. Masked endpoint analyses was used for electrophysiological and selected imaging outcomes.

Recruitment information / eligibility

• 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

Related Conditions & MeSH terms

• Devic Disease
• Devic Syndrome
• Devic's Disease
• Devic's Neuromyelitis Optica
• Devic's Syndrome
• Neuromyelitis Optica
• Syndrome

Intervention

Biological:
• Autologous mesenchymal stem cells
Autologous mesenchymal stem cells

Locations

Country	Name	City	State
China	Tianjin Medical University General Hospital	Tianjin	Tianjin

Sponsors (1)

Lead Sponsor	Collaborator
Tianjin Medical University General Hospital

Country where clinical trial is conducted

China, 

Outcome

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