Neuromyelitis Optica Spectrum DisordersPhase 4MONICANCT02809079
Mycophenolate Mofetil Treatment With Neuromyelitis Optica Spectrum Disorders in Chinese Patients
A Phase 4 study testing Mycophenolate mofetil and Prednisone in people with Neuromyelitis Optica Spectrum Disorders and Mycophenolate Mofetil, run by Third Affiliated Hospital, Sun Yat-Sen University.
Trial summary
Neuromyelitis optica (NMO) is an autoimmune inflammatory demyelinating disease of the central nervous system that leads to blindness and paralysis. Since disability accrues incrementally related to attacks, attack prevention with immunosuppressive therapy is the mainstay of preventing disability. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Neuromyelitis Optica Spectrum Disorders, Mycophenolate Mofetil, Efficacy and Safety
- What is being tested?
- Mycophenolate mofetil and Prednisone
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 100 participants
- Where
- No sites listed yet
- Study dates
- Jan 2016to Dec 2017
- Phase
- Phase 4
- 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 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Meet the 2006 Wingerchuk diagnostic criteria;
- NMO-immunoglobulin G seropositive;
- Between 18 to 65 years old;
- Relapse: more than 2 relapses in recent 2 years; more than 1 relapses in recent 1 years;
- Expanded disability status scale: expanded disability status scale≤7.0, and visual acuity ≥20 / 100 at least in one eye
- Understand the purpose and procedures of this study, and written informed consent is obtained.
Who cannot join
- Using immunosuppressive agents and other drugs affect evaluation, and drug withdrawal less than 3 months;
- Patients with any of the following diseases: transaminases elevation exceed 2 times of the normal upper limit; white blood cell <4 × 109 / L, Hemoglobin <80g / L, platelet <100 × 109 / L;
- With serious cardiovascular, liver, kidneys and other vital organs and blood, endocrine system diseases, cancer history;
- With immunodeficiency, uncontrolled infection and active gastrointestinal diseases (such as gastric ulcer, etc.);
- Pregnancy, breast-feeding women and male or female who plans to conceive recently;
- Allergy to mycophenolate mofetil and prednisone.
About this study
Neuromyelitis optica (NMO) is an autoimmune inflammatory demyelinating disease of the central nervous system that leads to blindness and paralysis. Since disability accrues incrementally related to attacks, attack prevention with immunosuppressive therapy is the mainstay of preventing disability. However, there is no standard immunosuppressive treatment strategy for NMO relapse prevention. In a previous study, the investigators provided evidence supporting the use of azathioprine plus a low dose corticosteroid as an effective strategy which is associated with a reduction in the risk of relapse in Chinese patients with NMO, but azathioprine has bone marrow suppression and other side effects. Mycophenolate mofetil (MMF) is a new immunosuppressant with rapid onset, fewer side effects and other advantages. In recent years, MMF has been used in different immune-related neurological diseases; some literature shown the possible efficacy of MMF in NMO treatment.
In this research, a multi-center (Third Affiliated Hospital of Sun Yat-sen University, Zhongshan Ophthalmic Centre of Sun Yat-sen University, Nangfang Hospital of Southern Medical University) study will carry out to evaluate the efficacy and safety of mycophenolate mofetil therapy in NMO spectrum disorders.
- Being tested
- Mycophenolate mofetil, Prednisone
- Medical subject headings
- Neuromyelitis Optica
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Mycophenolate mofetil 500mg Bid
PrednisoneDrug
prednisone 10mg Qd
| Study group | Role | What they receive |
|---|---|---|
| Mycophenolate mofetil plus prednisone | Experimental | Mycophenolate mofetil 500mg Bid and prednisone 10mg Qd Receives: Drug: Mycophenolate mofetil; Drug: Prednisone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annualized relapse rate day 360
Patients come back for follow-up visit on day 360 after staring treatment, and annualized relapse rate was evaluated.
Measured: day 360 after staring treatment
Secondary outcomes (5)
expanded disability status scale
Patients come back for follow-up visit on day 1, 14, 30, 90, 180, 270, 360 after staring treatment, and expanded disability status scale was evaluated.
Measured: day 1, 14, 30, 90, 180, 270, 360 after staring treatment
Hauser scale
Patients come back for follow-up visit on day 1, 14, 30, 90, 180, 270, 360 after staring treatment, and Hauser scale was evaluated.
Measured: day 1, 14, 30, 90, 180, 270, 360 after staring treatment
vision scale
Patients come back for follow-up visit on day 1, 14, 30, 90, 180, 270, 360 after staring treatment, and vision scale was evaluated.
Measured: day 1, 14, 30, 90, 180, 270, 360 after staring treatment
Lesions in brain and spinal cord
Patients come back for follow-up visit on day 1, 14, 30, 90, 180, 270, 360 after staring treatment, lesions in brain and spinal cord were evaluated by MRI.
Measured: day 1, 14, 30, 90, 180, 270, 360 after staring treatment
Annualized relapse rate
Patients come back for follow-up visit on day 1, 14, 30, 90, 180, 270 after staring treatment, and annualized relapse rate was evaluated.
Measured: day 1, 14, 30, 90, 180, 270 after staring treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jun 2016 (estimated)
- Study startJan 2016
- Primary completionDec 2017 (estimated)
- Study completionDec 2017 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Neuromyelitis Optica Spectrum DisordersStudies 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 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Third Affiliated Hospital, Sun Yat-Sen University
- Sponsor type
- University, hospital or other
- Collaborators
- Zhongshan Ophthalmic Center, Sun Yat-sen University, Southern Medical University, China
- Responsible party
- Principal investigator, Wei Qiu, Third Affiliated Hospital, Sun Yat-Sen University
- Investigators
- Wei Qiu, Medical PhD, Principal investigator, Third Affiliated Hospital, Sun Yat-Sen University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02809079
- Sponsor's protocol ID
- 2016017
- Acronym
- MONICA
- Status last verified
- Jun 2016
- First submitted
- 12 Jun 2016
- Last updated
- 22 Jun 2016
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- Yes: share by publications and meetings
Publications
Falcini F, Trapani S, Ricci L, Resti M, Simonini G, de Martino M. Sustained improvement of a girl affected with Devic's disease over 2 years of mycophenolate mofetil treatment. Rheumatology (Oxford). 2006 Jul;45(7):913-5. doi: 10.1093/rheumatology/kei263. Epub 2006 Apr 25. No abstract available.
PubMed 16638802Huh SY, Kim SH, Hyun JW, Joung AR, Park MS, Kim BJ, Kim HJ. Mycophenolate mofetil in the treatment of neuromyelitis optica spectrum disorder. JAMA Neurol. 2014 Nov;71(11):1372-8. doi: 10.1001/jamaneurol.2014.2057.
PubMed 25199960Jacob A, Matiello M, Weinshenker BG, Wingerchuk DM, Lucchinetti C, Shuster E, Carter J, Keegan BM, Kantarci OH, Pittock SJ. Treatment of neuromyelitis optica with mycophenolate mofetil: retrospective analysis of 24 patients. Arch Neurol. 2009 Sep;66(9):1128-33. doi: 10.1001/archneurol.2009.175.
PubMed 19752302Huang Q, Wang J, Zhou Y, Yang H, Wang Z, Yan Z, Long Y, Yin J, Feng H, Li C, Lu Z, Hu X, Qiu W. Low-Dose Mycophenolate Mofetil for Treatment of Neuromyelitis Optica Spectrum Disorders: A Prospective Multicenter Study in South China. Front Immunol. 2018 Sep 11;9:2066. doi: 10.3389/fimmu.2018.02066. eCollection 2018.
PubMed 30258442
Study at a glance
From the registry record, last updated by the sponsor on 22 Jun 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Mycophenolate Mofetil Treatment With Neuromyelitis Optica Spectrum Disorders in Chinese Patients
- ClinicalTrials.gov ID
- NCT02809079
- Acronym / sponsor ID
- MONICA / 2016017
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Third Affiliated Hospital, Sun Yat-Sen UniversityUniversity, hospital or other
- Collaborators
- Zhongshan Ophthalmic Center, Sun Yat-sen University, Southern Medical University, China
- Study start
- Jan 2016
- Primary completion
- Dec 2017 (estimated)
- Study completion
- Dec 2017 (estimated)
- First posted
- 22 Jun 2016 (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 an autoimmune inflammatory demyelinating disease of the central nervous system that leads to blindness and paralysis. Since disability accrues incrementally related to attacks, attack prevention with immunosuppressive therapy is the mainstay of preventing disability. However, there is no standard immunosuppressive treatment strategy for NMO relapse prevention. In a previous study, the investigators provided evidence supporting the use of azathioprine plus a low dose corticosteroid as an effective strategy which is associated with a reduction in the risk of relapse in Chinese patients with NMO, but azathioprine has bone marrow suppression and other side effects. Mycophenolate mofetil (MMF) is a new immunosuppressant with rapid onset, fewer side effects and other advantages. In recent years, MMF has been used in different immune-related neurological diseases; some literature shown the possible efficacy of MMF in NMO treatment.
In this research, a multi-center (Third Affiliated Hospital of Sun Yat-sen University, Zhongshan Ophthalmic Centre of Sun Yat-sen University, Nangfang Hospital of Southern Medical University) study will carry out to evaluate the efficacy and safety of mycophenolate mofetil therapy in NMO spectrum disorders.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Mycophenolate mofetil plus prednisone | Experimental | Drug: Mycophenolate mofetil, Drug: PrednisoneMycophenolate mofetil 500mg Bid and prednisone 10mg Qd |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Meet the 2006 Wingerchuk diagnostic criteria;
- NMO-immunoglobulin G seropositive;
- Between 18 to 65 years old;
- Relapse: more than 2 relapses in recent 2 years; more than 1 relapses in recent 1 years;
- Expanded disability status scale: expanded disability status scale≤7.0, and visual acuity ≥20 / 100 at least in one eye
Key exclusion criteria (6 listed)
- Using immunosuppressive agents and other drugs affect evaluation, and drug withdrawal less than 3 months;
- Patients with any of the following diseases: transaminases elevation exceed 2 times of the normal upper limit; white blood cell <4 × 109 / L, Hemoglobin <80g / L, platelet <100 × 109 / L;
- With serious cardiovascular, liver, kidneys and other vital organs and blood, endocrine system diseases, cancer history;
- With immunodeficiency, uncontrolled infection and active gastrointestinal diseases (such as gastric ulcer, etc.);
- Pregnancy, breast-feeding women and male or female who plans to conceive recently;
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 |
|---|---|---|
| Mycophenolate mofetilMycophenolate mofetil 500mg Bid | Drug | Mycophenolate mofetil plus prednisone |
| Prednisoneprednisone 10mg Qd | Drug | Mycophenolate mofetil plus prednisone |
Primary outcome
Annualized relapse rate day 360
Time frame: day 360 after staring treatment
Patients come back for follow-up visit on day 360 after staring treatment, and annualized relapse rate was evaluated.
5 secondary outcomes
expanded disability status scale
Time frame: day 1, 14, 30, 90, 180, 270, 360 after staring treatment
Hauser scale
Time frame: day 1, 14, 30, 90, 180, 270, 360 after staring treatment
vision scale
Time frame: day 1, 14, 30, 90, 180, 270, 360 after staring treatment
Lesions in brain and spinal cord
Time frame: day 1, 14, 30, 90, 180, 270, 360 after staring treatment
Annualized relapse rate
Time frame: day 1, 14, 30, 90, 180, 270 after staring treatment
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jun 2016.
- Results publication: PubMed 16638802Falcini F, Trapani S, Ricci L, Resti M, Simonini G, de Martino M. Sustained improvement of a girl affected with Devic's disease over 2 years of mycophenolate mofetil treatment. Rheumatology (Oxford). 2006 Jul;45(7):913-5. doi: 10.1093/rheumatology/kei263. Epub 2006 Apr 25. No abstract available.
- Results publication: PubMed 25199960Huh SY, Kim SH, Hyun JW, Joung AR, Park MS, Kim BJ, Kim HJ. Mycophenolate mofetil in the treatment of neuromyelitis optica spectrum disorder. JAMA Neurol. 2014 Nov;71(11):1372-8. doi: 10.1001/jamaneurol.2014.2057.
- Results publication: PubMed 19752302Jacob A, Matiello M, Weinshenker BG, Wingerchuk DM, Lucchinetti C, Shuster E, Carter J, Keegan BM, Kantarci OH, Pittock SJ. Treatment of neuromyelitis optica with mycophenolate mofetil: retrospective analysis of 24 patients. Arch Neurol. 2009 Sep;66(9):1128-33. doi: 10.1001/archneurol.2009.175.
- Derived from this study: PubMed 30258442Huang Q, Wang J, Zhou Y, Yang H, Wang Z, Yan Z, Long Y, Yin J, Feng H, Li C, Lu Z, Hu X, Qiu W. Low-Dose Mycophenolate Mofetil for Treatment of Neuromyelitis Optica Spectrum Disorders: A Prospective Multicenter Study in South China. Front Immunol. 2018 Sep 11;9:2066. doi: 10.3389/fimmu.2018.02066. eCollection 2018.
- Individual participant dataSharing planned: Yes. share by publications and meetings
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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.
Wei Qiu, Medical PhD principal investigator
Third Affiliated Hospital, Sun Yat-Sen University
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Mycophenolate mofetil and Prednisone in people with Neuromyelitis Optica Spectrum Disorders and Mycophenolate Mofetil, run by Third Affiliated Hospital, Sun Yat-Sen University.
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 6 inclusion and 6 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: status not confirmed, as of the sponsor's last update on 22 Jun 2016. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
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?
Annualized relapse rate day 360 (time frame: day 360 after staring treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02809079, which also lists 4 linked publications.
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 22 Jun 2016.