Efficacy and Safety of Tocilizumab in the Treatment of Generalized Myasthenia Gravis

NCT05067348 | Recruiting Phase 2 DMC

• 1 other identifier: V4.0, 20220410
• Study Type: interventional
• Target: 64
• Locations: 1 country
• Sites: 6

Brief Summary

Randomized, double-blind, placebo-controlled, parallel-group study with optional open-label extension.

Conditions

Myasthenia Gravis, Generalized

Keywords

Generalized Myasthenia Gravis; Tocilizumab

Outcome Measures

Primary Outcomes (1)

• Change in Quantitative Myasthenia Gravis (QMG) scores.

  16 weeks

Secondary Outcomes (7)

• Proportion of subjects with both (1) ≥ 3-point improvement in QMG and (2) lasts ≥4 weeks

  16 weeks

• Proportion of subjects with both (1) ≥ 2-point improvement in MG-ADL and (2) lasts ≥4 weeks

  16 weeks

• Change from baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) Profile score.

  16 weeks

• Change in Myasthenia Gravis Composite (MGC) score

  16 weeks

• Change in Myasthenia Gravis Quality of Life-15, revised (MGQOL-15r) score.

  16 weeks

Study Arms (2)

Tocilizumab

EXPERIMENTAL

Participants will receive tocilizumab administered intravenously (IV) on weeks 1,5,9 and 13 of the randomized controlled period.

Drug: Tocilizumab Injectable Product

Placebo

PLACEBO COMPARATOR

Participants will receive placebo administered intravenously (IV) on weeks 1,5,9 and 13 of the randomized controlled period.

Drug: Tocilizumab Injectable Product

Interventions

Tocilizumab Injectable Product DRUG

Participants will receive IV tocilizumab

Placebo; Tocilizumab

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis of MG with anti-AChRantibody.
• MGFA Clinical Classification Class II, III, or IV.
• MG-ADL score of 5 or greater at screening and at randomization with \> 50% of this score attributed to non-ocular items.
• QMG score of 11 or greater.
• Subjects must be on:
• Cholinesterase inhibitor, with no dose increase within 4 weeks prior to randomization;
• Corticosteroids, with no dose increase within 4 weeks prior to randomization; or/and c. non-steroidal IST (including azathioprine, mycophenolate mofetil, tacrolimus, cyclosporine A), with continuous use for at least 6 months prior to randomization and no dose increase within 4 months prior to randomization.

You may not qualify if:

• Participants had clinically relevant active infections (such as sepsis, pneumonia, or abscess) or severe infections (resulting in hospitalization or requiring antibiotic treatment) in the 4 weeks before randomization;
• Those with a history of high-risk tuberculosis infection, acquired tuberculosis infection, and chronic hepatitis;
• Human immunodeficiency virus (HIV) infection;
• Thymomas that have received thymectomy or planned thymectomy during RCP within 6 months before randomization, or require chemotherapy and/or radiotherapy at any time;
• Received rituximab treatment in the past 6 months before randomization;
• Received tocilizumab or eculizumab treatment within 3 months before randomization;
• Received IVIG or plasma exchange within 4 weeks before randomization;
• Unresected thymoma.
• History of other tumor diseases.

Sponsors & Collaborators

• Tang-Du Hospital: lead

Study Sites (6)

Beijing Tiantan Hospital, Capital Medical University

Beijing, Beijing Municipality, 100050, China

NOT YET RECRUITING

Xiangya Hospital Central South University

Changsha, Hunan, 410008, China

NOT YET RECRUITING

Tangdu Hospital, The Fourth Military Medical University

Xi'an, Shaanxi, 710038, China

RECRUITING

Huashan Hospital

Shanghai, Shanghai Municipality, 200040, China

NOT YET RECRUITING

West China Hospital of Sichuan University

Chengdu, Sichuan, 610041, China

NOT YET RECRUITING

Tianjin medical university general hospital

Tianjin, Tianjin Municipality, 300052, China

NOT YET RECRUITING

MeSH Terms

Conditions

Myasthenia Gravis

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Study Officials

• Ting Chang, MD,PHD

  The Second Affiliated Hospital of Air Force Medical University

  STUDY DIRECTOR

Central Study Contacts

Ting Chang, MD,PHD

CONTACT

+86-29-84778845; changting1981@163.com

Zhe Ruan

CONTACT

+86-29-84778845; ruanzhe573291596@126.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 23, 2021
• First Posted: October 5, 2021
• Study Start: July 21, 2022
• Primary Completion: December 31, 2025
• Study Completion: March 30, 2026
• Last Updated: May 1, 2025

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will not share

Locations

CN (6)