## Protocol Section
### Identification Module
**NCT ID:** NCT02809079

**Acronym:** MONICA

**Brief Title:** Mycophenolate Mofetil Treatment With Neuromyelitis Optica Spectrum Disorders in Chinese Patients

**Official Title:** Mycophenolate Mofetil Treatment With Neuromyelitis Optica Spectrum Disorders in Chinese Patients

#### Organization Study ID Info
**ID:** 2016017
#### Organization
**Class:** OTHER
**Full Name:** Third Affiliated Hospital, Sun Yat-Sen University
### Status Module
#### Completion Date
**Date:** 2017-12

**Type:** ESTIMATED

#### Expanded Access Info
**Last Known Status:** ENROLLING_BY_INVITATION
#### Last Update Post Date
**Date:** 2016-06-22

**Type:** ESTIMATED

**Last Update Submit Date:** 2016-06-19

**Overall Status:** UNKNOWN

#### Primary Completion Date
**Date:** 2017-12

**Type:** ESTIMATED

#### Start Date
**Date:** 2016-01

**Status Verified Date:** 2016-06

#### Study First Post Date
**Date:** 2016-06-22
**Type:** ESTIMATED
**Study First Submit Date:** 2016-06-12
**Study First Submit QC Date:** 2016-06-19
### Sponsor Collaborators Module

#### Collaborators

**Class:** OTHER

**Name:** Zhongshan Ophthalmic Center, Sun Yat-sen University

**Class:** OTHER

**Name:** Southern Medical University, China

#### Lead Sponsor

**Class:** OTHER

**Name:** Third Affiliated Hospital, Sun Yat-Sen University

#### Responsible Party

**Investigator Affiliation:** Third Affiliated Hospital, Sun Yat-Sen University

**Investigator Full Name:** Wei Qiu

**Investigator Title:** Associate Professor

**Type:** PRINCIPAL_INVESTIGATOR

### Oversight Module

**Oversight Has DMC:** True

### Description Module

**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 Module

**Conditions:**

- Neuromyelitis Optica Spectrum Disorders

- Mycophenolate Mofetil

- Efficacy and Safety

### Design Module

#### Design Info

**Allocation:** NA

**Intervention Model:** SINGLE_GROUP

##### Masking Info

**Masking:** NONE

**Primary Purpose:** TREATMENT

#### Enrollment Info

**Count:** 100

**Type:** ESTIMATED

**Phases:**

- PHASE4

**Study Type:** INTERVENTIONAL

### Arms Interventions Module

#### Arm Group 1

**Description:** Mycophenolate mofetil 500mg Bid and prednisone 10mg Qd

**Intervention Names:**

- Drug: Mycophenolate mofetil

- Drug: Prednisone

**Label:** Mycophenolate mofetil plus prednisone

**Type:** EXPERIMENTAL

### Interventions

#### Intervention 1

**Arm Group Labels:**

- Mycophenolate mofetil plus prednisone

**Description:** Mycophenolate mofetil 500mg Bid

**Name:** Mycophenolate mofetil

**Type:** DRUG

#### Intervention 2

**Arm Group Labels:**

- Mycophenolate mofetil plus prednisone

**Description:** prednisone 10mg Qd

**Name:** Prednisone

**Type:** DRUG

### Outcomes Module

#### Primary Outcomes

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

**Measure:** Annualized relapse rate day 360

**Time Frame:** day 360 after staring treatment

#### Secondary Outcomes

**Description:** 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.

**Measure:** expanded disability status scale

**Time Frame:** day 1, 14, 30, 90, 180, 270, 360 after staring treatment

**Description:** Patients come back for follow-up visit on day 1, 14, 30, 90, 180, 270, 360 after staring treatment, and Hauser scale was evaluated.

**Measure:** Hauser scale

**Time Frame:** day 1, 14, 30, 90, 180, 270, 360 after staring treatment

**Description:** Patients come back for follow-up visit on day 1, 14, 30, 90, 180, 270, 360 after staring treatment, and vision scale was evaluated.

**Measure:** vision scale

**Time Frame:** day 1, 14, 30, 90, 180, 270, 360 after staring treatment

**Description:** 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.

**Measure:** Lesions in brain and spinal cord

**Time Frame:** day 1, 14, 30, 90, 180, 270, 360 after staring treatment

**Description:** Patients come back for follow-up visit on day 1, 14, 30, 90, 180, 270 after staring treatment, and annualized relapse rate was evaluated.

**Measure:** Annualized relapse rate

**Time Frame:** day 1, 14, 30, 90, 180, 270 after staring treatment

### Eligibility Module

**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.

**Maximum Age:** 65 Years

**Minimum Age:** 18 Years

**Sex:** ALL

**Standard Ages:**

- ADULT

- OLDER_ADULT

### Contacts Locations Module

#### Overall Officials

**Official 1:**

**Affiliation:** Third Affiliated Hospital, Sun Yat-Sen University

**Name:** Wei Qiu, Medical PhD

**Role:** PRINCIPAL_INVESTIGATOR

### IPD Sharing Statement Module

**Description:** share by publications and meetings

**IPD Sharing:** YES

### References Module

#### 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.

**PMID:** 16638802

**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.

**PMID:** 25199960

**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.

**PMID:** 19752302

**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.

**PMID:** 30258442

## Derived Section

### Condition Browse Module - Ancestors

- ID: D000009188

- Term: Myelitis, Transverse

- ID: D000020278

- Term: Demyelinating Autoimmune Diseases, CNS

- ID: D000020274

- Term: Autoimmune Diseases of the Nervous System

- ID: D000009422

- Term: Nervous System Diseases

- ID: D000009902

- Term: Optic Neuritis

- ID: D000009901

- Term: Optic Nerve Diseases

- ID: D000003389

- Term: Cranial Nerve Diseases

- ID: D000003711

- Term: Demyelinating Diseases

- ID: D000005128

- Term: Eye Diseases

- ID: D000001327

- Term: Autoimmune Diseases

- ID: D000007154

- Term: Immune System Diseases

### Condition Browse Module - Browse Branches

- Abbrev: BC10

- Name: Nervous System Diseases

- Abbrev: BC11

- Name: Eye Diseases

- Abbrev: BC20

- Name: Immune System Diseases

- Abbrev: All

- Name: All Conditions

- Abbrev: BC01

- Name: Infections

- Abbrev: BC04

- Name: Neoplasms

- Abbrev: Rare

- Name: Rare Diseases

### Condition Browse Module - Browse Leaves

- ID: M12414

- Name: Neuromyelitis Optica

- Relevance: HIGH

- As Found: Neuromyelitis Optica

- ID: M12142

- Name: Myelitis

- Relevance: LOW

- As Found: Unknown

- ID: M12143

- Name: Myelitis, Transverse

- Relevance: LOW

- As Found: Unknown

- ID: M4629

- Name: Autoimmune Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M22098

- Name: Demyelinating Autoimmune Diseases, CNS

- Relevance: LOW

- As Found: Unknown

- ID: M22094

- Name: Autoimmune Diseases of the Nervous System

- Relevance: LOW

- As Found: Unknown

- ID: M12387

- Name: Neuritis

- Relevance: LOW

- As Found: Unknown

- ID: M12833

- Name: Optic Neuritis

- Relevance: LOW

- As Found: Unknown

- ID: M12832

- Name: Optic Nerve Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M6605

- Name: Cranial Nerve Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M6909

- Name: Demyelinating Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M8271

- Name: Eye Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M10200

- Name: Immune System Diseases

- Relevance: LOW

- As Found: Unknown

- ID: T4103

- Name: Neuromyelitis Optica Spectrum Disorder

- Relevance: HIGH

- As Found: Neuromyelitis Optica Spectrum Disorder

- ID: T3988

- Name: Myelitis

- Relevance: LOW

- As Found: Unknown

- ID: T4263

- Name: Optic Neuritis

- Relevance: LOW

- As Found: Unknown

### Condition Browse Module - Meshes

- ID: D000009471

- Term: Neuromyelitis Optica

### Intervention Browse Module - Ancestors

- ID: D000000893

- Term: Anti-Inflammatory Agents

- ID: D000005938

- Term: Glucocorticoids

- ID: D000006728

- Term: Hormones

- ID: D000006730

- Term: Hormones, Hormone Substitutes, and Hormone Antagonists

- ID: D000045505

- Term: Physiological Effects of Drugs

- ID: D000018931

- Term: Antineoplastic Agents, Hormonal

- ID: D000000970

- Term: Antineoplastic Agents

- ID: D000000903

- Term: Antibiotics, Antineoplastic

- ID: D000000904

- Term: Antibiotics, Antitubercular

- ID: D000000995

- Term: Antitubercular Agents

- ID: D000000900

- Term: Anti-Bacterial Agents

- ID: D000000890

- Term: Anti-Infective Agents

- ID: D000004791

- Term: Enzyme Inhibitors

- ID: D000045504

- Term: Molecular Mechanisms of Pharmacological Action

### Intervention Browse Module - Browse Branches

- Abbrev: Infl

- Name: Anti-Inflammatory Agents

- Abbrev: ANeo

- Name: Antineoplastic Agents

- Abbrev: All

- Name: All Drugs and Chemicals

- Abbrev: Infe

- Name: Anti-Infective Agents

### Intervention Browse Module - Browse Leaves

- ID: M14121

- Name: Prednisone

- Relevance: HIGH

- As Found: Min

- ID: M12128

- Name: Mycophenolic Acid

- Relevance: HIGH

- As Found: Mindfulness

- ID: M4217

- Name: Anti-Inflammatory Agents

- Relevance: LOW

- As Found: Unknown

- ID: M9047

- Name: Glucocorticoids

- Relevance: LOW

- As Found: Unknown

- ID: M9789

- Name: Hormones

- Relevance: LOW

- As Found: Unknown

- ID: M9788

- Name: Hormone Antagonists

- Relevance: LOW

- As Found: Unknown

- ID: M20966

- Name: Antineoplastic Agents, Hormonal

- Relevance: LOW

- As Found: Unknown

- ID: M4222

- Name: Anti-Bacterial Agents

- Relevance: LOW

- As Found: Unknown

- ID: M4224

- Name: Antibiotics, Antitubercular

- Relevance: LOW

- As Found: Unknown

- ID: M4311

- Name: Antitubercular Agents

- Relevance: LOW

- As Found: Unknown

- ID: M4214

- Name: Anti-Infective Agents

- Relevance: LOW

- As Found: Unknown

- ID: M7951

- Name: Enzyme Inhibitors

- Relevance: LOW

- As Found: Unknown

### Intervention Browse Module - Meshes

- ID: D000009173

- Term: Mycophenolic Acid

- ID: D000011241

- Term: Prednisone

### Misc Info Module

- Version Holder: 2024-05-24
