Mycophenolate Mofetil Treatment With Neuromyelitis Optica Spectrum Disorders in Chinese Patients

Phase 4

• Conditions: Neuromyelitis Optica Spectrum Disorders; Efficacy and Safety; Mycophenolate Mofetil
• Interventions: Drug: Mycophenolate mofetil; Drug: Prednisone

• Registration Number: NCT02809079
• Lead Sponsor: Third Affiliated Hospital, Sun Yat-Sen University

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2016-01
• Status Verified: 2016-06
• Study First Submitted: 2016-06-12
• Study First Submitted QC: 2016-06-19
• Last Update Submitted: 2016-06-19
• Study First Posted: 2016-06-22
• Last Update Posted: 2016-06-22
• Primary Completion: 2017-12
• Study Completion: 2017-12

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 100

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Mycophenolate mofetil plus prednisone	Mycophenolate mofetil	Mycophenolate mofetil 500mg Bid and prednisone 10mg Qd
Mycophenolate mofetil plus prednisone	Prednisone	Mycophenolate mofetil 500mg Bid and prednisone 10mg Qd

Primary Outcome Measures

Name	Time	Method
Annualized relapse rate day 360	day 360 after staring treatment	Patients come back for follow-up visit on day 360 after staring treatment, and annualized relapse rate was evaluated.

Secondary Outcome Measures

Name	Time	Method
expanded disability status scale	day 1, 14, 30, 90, 180, 270, 360 after staring treatment	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.
Hauser scale	day 1, 14, 30, 90, 180, 270, 360 after staring treatment	Patients come back for follow-up visit on day 1, 14, 30, 90, 180, 270, 360 after staring treatment, and Hauser scale was evaluated.
vision scale	day 1, 14, 30, 90, 180, 270, 360 after staring treatment	Patients come back for follow-up visit on day 1, 14, 30, 90, 180, 270, 360 after staring treatment, and vision scale was evaluated.
Lesions in brain and spinal cord	day 1, 14, 30, 90, 180, 270, 360 after staring treatment	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.
Annualized relapse rate	day 1, 14, 30, 90, 180, 270 after staring treatment	Patients come back for follow-up visit on day 1, 14, 30, 90, 180, 270 after staring treatment, and annualized relapse rate was evaluated.