Devic's SyndromePhase 2NCT02249676
Autologous Mesenchymal Stem Cells for the Treatment of Neuromyelitis Optica Spectrum Disorders
A Phase 2 study testing Autologous mesenchymal stem cells in people with Devic's Syndrome and Devic's Neuromyelitis Optica, run by Tianjin Medical University General Hospital at 1 site in China.
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%. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Devic's Syndrome, Devic's Neuromyelitis Optica, Devic Syndrome
- What is being tested?
- Autologous mesenchymal stem cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 15 participants
- Where
- 1 siteChina
- Study dates
- Jan 2013to 31 Dec 2014
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Show all 12Show fewer
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
China1 site
Tianjin Medical University General Hospital
Tianjin, Tianjin Municipality, 300052
About this study
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
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.
Read the detailed 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.
- Conditions
- Devic's Syndrome, Devic's Neuromyelitis Optica, Devic Syndrome, Devic's Disease, Devic Disease
- Being tested
- Autologous mesenchymal stem cells
- Keywords
- Autologous mesenchymal stem cells, neuromyelitis optica, treatment
- Medical subject headings
- Neuromyelitis Optica
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Autologous mesenchymal stem cellsBiological
Also called MSC
Autologous mesenchymal stem cells
| Study group | Role | What they receive |
|---|---|---|
| Autologous mesenchymal stem cells group | Experimental | Generated clinical-grade MSC 10 mg chlorpheniramine Po.;100 mg hydrocortisone iv.;10 mg metoclopramide im.;30 min before administration of the cells .
MSC a day-case 2·0×106 cells/kg i.v. 15min Infused normal saline 500 Ml over 4 h i.v. Receives: Biological: Autologous mesenchymal stem cells |
| Control group | Placebo comparator | Patients with progressive and refractory NMO treated with regular methods Receives: Biological: Autologous mesenchymal stem cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
EDSS
Compare EDSS change before and one year after mesenchymal stem cells (MSC) infusion
Measured: change from baseline to one year
Secondary outcomes (8)
Annual relapse rate
Compare annual relapse rate before and one year after MSC infusion
Measured: 1 year after infusion
Lesion load
Compared lesion load before and one year after MSC infusion
Measured: 1 year after infusion
Retinal nerve fiber layer (RNFL)
Compared RNFL before and one year after MSC infusion
Measured: 1 year after infusion
Cognition
Compare cognition questionnaire scale before and one year after MSC infusion
Measured: 1 year after infusion
Immunological assessments
Compare anti-aquaporin4-ab before and one year after MSC infusion.
Measured: 1 year after infusion
Immunological assessments
Compare immune cell subpopulation before and one year after MSC infusion.
Measured: 1 year after infusion
Immunological assessments
Compare cytokine kinetics before and one year after MSC infusion.
Measured: 1 year after infusion
cerebral volume
Compare cerebral volume before and one year after MSC infusion
Measured: 1 year after infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Sep 2014 (estimated)
- Study startJan 2013
- Primary completion31 Dec 2014
- Study completion31 Dec 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Devic's SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Tianjin Medical University General Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Fu-Dong Shi, Tianjin Medical University General Hospital
- Investigators
- Fu-Dong Shi, MD,PhD, Principal investigator, Tianjin Medical University General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02249676
- Sponsor's protocol ID
- IRB2013-055-02
- Status last verified
- Oct 2018
- First submitted
- 15 Sep 2014
- Last updated
- 17 Oct 2018
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 17 Oct 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02249676
- Acronym / sponsor ID
- IRB2013-055-02
- Conditions
- Devic's Syndrome, Devic's Neuromyelitis Optica, Devic Syndrome, Devic's Disease, Devic Disease
- Keywords
- Autologous mesenchymal stem cells, neuromyelitis optica, treatment
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Tianjin Medical University General HospitalUniversity, hospital or other
- Study start
- Jan 2013 (actual)
- Primary completion
- 31 Dec 2014 (actual)
- Study completion
- 31 Dec 2014 (actual)
- First posted
- 25 Sep 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Autologous mesenchymal stem cells group | Experimental | Biological: Autologous mesenchymal stem cellsGenerated clinical-grade MSC 10 mg chlorpheniramine Po.;100 mg hydrocortisone iv.;10 mg metoclopramide im.;30 min before administration of the cells . MSC a day-case 2·0×106 cells/kg i.v. 15min Infused normal saline 500 Ml over 4 h i.v. |
| Control group | Placebo comparator | Biological: Autologous mesenchymal stem cellsPatients with progressive and refractory NMO treated with regular methods |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- 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:
- Evidence of recent inflammatory disease, as evidenced by any one of the following:
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Autologous mesenchymal stem cellsAlso: MSCAutologous mesenchymal stem cells | Biological | Autologous mesenchymal stem cells group, Control group |
Primary outcome
EDSS
Time frame: change from baseline to one year
Compare EDSS change before and one year after mesenchymal stem cells (MSC) infusion
8 secondary outcomes
Annual relapse rate
Time frame: 1 year after infusion
Lesion load
Time frame: 1 year after infusion
Retinal nerve fiber layer (RNFL)
Time frame: 1 year after infusion
Cognition
Time frame: 1 year after infusion
Immunological assessments
Time frame: 1 year after infusion
Immunological assessments
Time frame: 1 year after infusion
Immunological assessments
Time frame: 1 year after infusion
cerebral volume
Time frame: 1 year after infusion
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Oct 2018.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Fu-Dong Shi, MD,PhD principal investigator
Tianjin Medical University General Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Autologous mesenchymal stem cells in people with Devic's Syndrome and Devic's Neuromyelitis Optica, run by Tianjin Medical University General Hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 17 Oct 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
EDSS (time frame: change from baseline to one year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Oct 2018; AzTrials last refreshed its copy on 8 Nov 2026.