Mycophenolate Mofetil Treatment With Neuromyelitis Optica Spectrum Disorders in Chinese Patients

NCT02809079 | Unknown Phase 4 DMC

• 1 other identifier: 2016017
• Study Type: interventional
• Target: 100
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Neuromyelitis Optica Spectrum Disorders; Mycophenolate Mofetil; Efficacy and Safety

Outcome Measures

Primary Outcomes (1)

• Annualized relapse rate day 360

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

  day 360 after staring treatment

Secondary Outcomes (5)

• expanded disability status scale

  day 1, 14, 30, 90, 180, 270, 360 after staring treatment

• Hauser scale

  day 1, 14, 30, 90, 180, 270, 360 after staring treatment

• vision scale

  day 1, 14, 30, 90, 180, 270, 360 after staring treatment

• Lesions in brain and spinal cord

  day 1, 14, 30, 90, 180, 270, 360 after staring treatment

• Annualized relapse rate

  day 1, 14, 30, 90, 180, 270 after staring treatment

Study Arms (1)

Mycophenolate mofetil plus prednisone

EXPERIMENTAL

Mycophenolate mofetil 500mg Bid and prednisone 10mg Qd

Drug: Mycophenolate mofetil; Drug: Prednisone

Interventions

Mycophenolate mofetil DRUG

Mycophenolate mofetil 500mg Bid

Mycophenolate mofetil plus prednisone

Prednisone DRUG

prednisone 10mg Qd

Mycophenolate mofetil plus prednisone

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Third Affiliated Hospital, Sun Yat-Sen University: lead
• Zhongshan Ophthalmic Center, Sun Yat-sen University: collaborator
• Southern Medical University, China: collaborator

Related Publications (4)

• 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.

  PMID: 16638802 RESULT

• 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.

  PMID: 25199960 RESULT

• 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.

  PMID: 19752302 RESULT

• 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.

  PMID: 30258442 DERIVED

MeSH Terms

Conditions

Neuromyelitis Optica

Interventions

Mycophenolic Acid; Prednisone

Condition Hierarchy (Ancestors)

Myelitis, Transverse; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Optic Neuritis; Optic Nerve Diseases; Cranial Nerve Diseases; Demyelinating Diseases; Eye Diseases; Autoimmune Diseases; Immune System Diseases

Intervention Hierarchy (Ancestors)

Caproates; Acids, Acyclic; Carboxylic Acids; Organic Chemicals; Fatty Acids; Lipids; Pregnadienediols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds

Study Officials

• Wei Qiu, Medical PhD

  Third Affiliated Hospital, Sun Yat-Sen University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Professor

Study Record Dates

• First Submitted: June 12, 2016
• First Posted: June 22, 2016
• Study Start: January 1, 2016
• Primary Completion: December 1, 2017
• Study Completion: December 1, 2017
• Last Updated: June 22, 2016

Record last verified: 2016-06

Data Sharing

• IPD Sharing: Will share