Skip to main content
OpenTrials
Completed

NCT Number: NCT04431895

Safety And Efficacy Of Tofacitinib In Patients With Refractory Myasthenia Gravis: A Pilot Study

This is a 6 month study that will evaluate the efficacy of 5mg tofacitinib tablet taken twice a day in patients with refractory myasthenia gravis. Twenty adult participants will receive a four month treatment of tofacitinib followed by elution for one month.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

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

Age range

18 year–70 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Early Phase 1

Primary location

Huashan Hospital

Shanghai, 200040, China

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • fluctuating muscle weakness and fatigability classified as the Myasthenia Gravis Foundation of America (MGFA) clinical classification Type IIa-IVa;
  • more than 10% amplitude decrement in low frequency repetitive nerve stimulation, and less than 10% amplitude increment in high frequency repetitive nerve stimulation;
  • understanding and assigning the informed consent form, and having a good compliance with treatment.

meet all of the criteria in above as well as at least one of the below:

  • refractory: insufficient response to full dose- and course- steroids treatment (0.75-1mg per kg per day for at least 3-6 months), failure to respond adequately to tacrolimus (no less than 3mg per day) for 3 months;
  • severe or intolerable adverse effects from conventional immunosuppressive therapy.

Exclusion criteria

  • positive for muscle-specific kinase (MuSK) antibodies;
  • a tumor history except for thymoma;
  • a history of Type B and Type C hepatitis;
  • a history of tuberculosis or T-SPOT.TB tset positivity;
  • hepatic, renal and cardial insufficiency (baseline: ALT/AST>50U/L;BNP>200pg/ml）;
  • severe allergy or infection, or chronic or recurrent infection;
  • pregnancy;
  • hyperlipidemia;
  • participating other clinical trials

Treatment and study plan

Tofacitinib 5 MG

Drug

tofacitinib 5 mg tablet administered twice a day for 6 months

Primary outcomes

  1. Change From Baseline in Quantitative Myasthenia Gravis (QMG) Scores at Month 6

    Time frame: Baseline, Month 6

    A quantitative MG scoring system (QMG Score) is essential in the objective evaluation of therapy for MG.This scoring system is based on quantitative testing of sentinel muscle groups. Total QMG score range from 0 (extreme disease severity) to 39 (none), higher score indicated less disease severity.

Secondary outcomes

  1. Change From Baseline in Myasthenia Gravis-related Activities of Daily Living (MG-ADL) Scores at Month 6

    Time frame: Baseline, Month 6

    The myasthenia gravis activities of daily living (MG-ADL) profile is an eight-item patient-reported scale developed to assess MG symptoms and their effects on daily activities.Total MG-ADL score range from 0 (none) to 24 (extreme disease conditions), higher score indicated more disease severity.

  2. Change From Baseline in Myasthenia Gravis Composite (MGC) Scores at Month 6

    Time frame: Baseline, Month 6

    The MGC score consists of test items that measure symptoms and signs of MG, with weighted response options. Total MGC score range from 0 (none) to 50 (extreme muscle weakness), higher score indicated more disease severity.

  3. Change From Baseline in Myasthenia Gravis-Quality of Life Questionnaire-15 item (MG-QOL15) Scores at Month 6

    Time frame: Baseline, Month 6

    The MG-QOL15 is helpful in informing the clinician about the patient's perception of the extent of and dissatisfaction with MG-related dysfunction. Total MG-QOL15 score range from 0 (none) to 60 (extreme disease severity), higher score indicated more disease severity.

  4. Change From Baseline in Doses of Steroids at Month 6

    Time frame: Baseline, Month 6

    Steroids is recommended as the first-line treatment for myasthenia gravis. Here we followed the combined doseage of steroids while administrating tofacitinib for enrolled participants.

Other outcomes

  1. Change From Baseline in Serum Titers of anti-acetylcholine receptor antibodies at Week 16

    Time frame: Baseline, Week 16

    Anti-acetylcholine receptor antibodies are the major pathological autoantibodies of MG. For anti-AChR antibodies-positive candidates, we followed the antibodies titers at Week 16.

  2. Change From Baseline in Inflammatory Cytokines at Week 12

    Time frame: Baseline, Week 12

    MG is an autoimmune disease manifested by impairment at the neuromuscular junction featured by weakness and fatigability. We compared the inflammatory cytokines (IL-6, IL-10, IL-17A, and IL-23) at Baseline and Week 12

Sponsors and collaborators

Lead sponsor

Huashan Hospital

Other

Registry information

Official study title

A Prospective,Single-center,Single-Arm,Single-Blind Pilot Study To Investigate The Efficacy Of Tofacitinib In Patients With Refractory Myasthenia Gravis

Important dates

Study start
2020
Primary completion
2024
Study completion
2024
First posted
Jun 16, 2020
Registry last updated
Sep 16, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.