Clinical trial NCT02809079: Mycophenolate Mofetil Treatment With Neuromyelitis Optica Spectrum Disorders in Chinese Patients
Official title: Mycophenolate Mofetil Treatment With Neuromyelitis Optica Spectrum Disorders in Chinese Patients
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE4
Conditions: Neuromyelitis Optica Spectrum Disorders
Mycophenolate Mofetil
Efficacy and Safety
Lead sponsor: Third Affiliated Hospital, Sun Yat-Sen University
Sponsor class: OTHER
Collaborators: name: Zhongshan Ophthalmic Center, Sun Yat-sen University
name: Southern Medical University, China
First posted: 2016-06-22
Enrollment: 100
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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. 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.


Eligibility criteria: Inclusion Criteria:

* 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.

Exclusion Criteria:

* 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.

Primary outcomes:
measure: Annualized relapse rate day 360
timeFrame: day 360 after staring treatment
Secondary outcomes:
measure: expanded disability status scale
timeFrame: day 1, 14, 30, 90, 180, 270, 360 after staring treatment
measure: Hauser scale
timeFrame: day 1, 14, 30, 90, 180, 270, 360 after staring treatment
measure: vision scale
timeFrame: day 1, 14, 30, 90, 180, 270, 360 after staring treatment
measure: Lesions in brain and spinal cord
timeFrame: day 1, 14, 30, 90, 180, 270, 360 after staring treatment
measure: Annualized relapse rate
timeFrame: day 1, 14, 30, 90, 180, 270 after staring treatment
Other outcomes:
Locations:
Study officials:
affiliation: Third Affiliated Hospital, Sun Yat-Sen University
name: Wei Qiu, Medical PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.
citation: Huh 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.
citation: Jacob 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.
citation: Huang 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.