Mycophenolate Mofetil Treatment With Neuromyelitis Optica Spectrum Disorders in Chinese Patients

Efficacy and Safety Clinical Trial

— MONICA  

Official title:

Mycophenolate Mofetil Treatment With Neuromyelitis Optica Spectrum Disorders in Chinese Patients

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT02809079
• Other study ID #: 2016017
• Status: Enrolling by invitation
• Phase: Phase 4
• First received: June 12, 2016
• Last updated: June 19, 2016
• Start date: January 2016
• Est. completion date: December 2017

Study information

• Verified date: June 2016
• Source: Third Affiliated Hospital, Sun Yat-Sen University
• Contact: n/a
• Is FDA regulated: No
• Health authority: China: Ethics Committee of the Third Affiliated Hospital of Sun Yat-sen University
• Study type: Interventional

Clinical 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. 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.

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 100
• Est. completion date: December 2017
• Est. primary completion date: December 2017
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 65 Years

Eligibility

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

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Efficacy and Safety
• Mycophenolate Mofetil
• Neuromyelitis Optica
• Neuromyelitis Optica Spectrum Disorders

Intervention

Drug:
• Mycophenolate mofetil
Mycophenolate mofetil 500mg Bid
• Prednisone
prednisone 10mg Qd

Locations

Country	Name	City	State
n/a

Sponsors (3)

Lead Sponsor	Collaborator
Third Affiliated Hospital, Sun Yat-Sen University	Nangfang Hospital, Southern Medical University, Zhongshan Ophthalmic Centre, Sun Yat-sen University

References & Publications (3)

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. Epub 2006 Apr 25. — View 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. — View 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):11 — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Yes
Secondary	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.	day 1, 14, 30, 90, 180, 270, 360 after staring treatment	Yes
Secondary	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.	day 1, 14, 30, 90, 180, 270, 360 after staring treatment	Yes
Secondary	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.	day 1, 14, 30, 90, 180, 270, 360 after staring treatment	Yes
Secondary	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.	day 1, 14, 30, 90, 180, 270, 360 after staring treatment	Yes
Secondary	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.	day 1, 14, 30, 90, 180, 270 after staring treatment	Yes