Efficacy and Safety of Low-dose Rituximab in the Treatment of Refractory Myasthenia Gravis

NCT05332587 | Unknown Phase 3

• 1 other identifier: [2020]414-1
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 1

Brief Summary

This study is designed to evaluate the effectiveness and safety of low-dose rituximab in patients with refractory myasthenia gravis. The traditional treatment of MG is immunosuppressive therapy, usually beginning with corticosteroids. However, up to 70% of treated patients show an incomplete response, including 10 - 30% who are unresponsive. Corticosteroids and other immunosuppressive therapies presented also many side effects. The investigators propose to evaluate in a pilot, open, prospective, single central study, the interest of rituximab (RTX) in the treatment of patients with refractory MG. Fifty patients with refractory MG will be included in the study and divided into two stages: 14 patients in the first stage were followed up after using RTX according to the study protocol. The study will move into the second stage on if the number of effective cases is greater than 3, otherwise, the study will be discontinued (based on Simon's Optimal Two-stage Design). The remaining 36 patients were enrolled in the second stage. In the first and second stages, the treatment plan and follow-up plan were consistent. The therapeutical schema is rituximab 100-200 mg/week for a total of 2-4 times, followed by adequate organ function, laboratory parameters and assessment of MG after each injection and end of follow up for 24 weeks.

Conditions

Refractory Myasthenia Gravis; Rituximab

Outcome Measures

Primary Outcomes (1)

• Time to reach MM-1, the proportion of drug remission.

  Mild clinical Manifestations（MM-1）was the primary efficacy endpoint observed. Record the time the patient appears from the time of medication to the time of MM-1，and calculate the percentage of Pharmacologic Remission (PR). Criteria for PR: The patient has had no symptoms or signs of MG and continues to take some form of therapy for MG. There is no weakness of any muscle on careful examination by someone skilled in the evaluation of neuromuscular disease. Isolated weakness of eyelid closure is accepted. Patients taking cholinesterase inhibitors are excluded from this category because their use suggests the presence of weakness.

  27 weeks

Secondary Outcomes (1)

• The changes of the disease severity

  27 weeks

Study Arms (1)

Rituximab Treated

EXPERIMENTAL

rituximab

Drug: Rituximab

Interventions

Rituximab DRUG

The therapeutical schema is rituximab 100-200 mg/week for a total of 2-4 times

Rituximab Treated

Eligibility Criteria

• Age: Up to 80 Years
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Aged 80 and under;
• Myasthenia gravis:
• Patients who are diagnosed as generalized or ocular myasthenia gravis
• Patients must have disease refractory to treatment: the condition did not improve (QMG score decreased by less than 3 points after treatment) or even deteriorated after treatment with sufficient prednisone or other immunosuppressive agents.
• Patients sign informed consent forms

You may not qualify if:

• Over the age of 80
• Patients with serious complications such as infection
• Patients with active TB (during the screening period, a chest X-ray or chest CT would be performed unless the patient can provide chest X-ray or chest CT reports in the last month); or patients with active HBV, HBV DNA\> 200
• Patients suffering from cardiomyopathy, acute coronary events, or severe arrhythmia.
• Patients who were allergic to rituximab
• Pregnant or suckling period woman
• Patients accompanied with mental disorders and have difficult to communication
• Patients with a significant abnormality in white blood cells, hemoglobin, and platelet count.

Sponsors & Collaborators

• First Affiliated Hospital, Sun Yat-Sen University: lead

Study Sites (1)

First Affiliated Hospital, Sun Yat-Sen University

Guangzhou, 510080, China

RECRUITING

MeSH Terms

Conditions

Myasthenia Gravis

Interventions

Rituximab

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

Intervention Hierarchy (Ancestors)

Antibodies, Monoclonal, Murine-Derived; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director of Neurology Department

Study Record Dates

• First Submitted: December 4, 2021
• First Posted: April 18, 2022
• Study Start: August 1, 2020
• Primary Completion: July 1, 2022
• Study Completion: July 1, 2022
• Last Updated: May 2, 2022

Record last verified: 2022-04

Locations

CN (1)