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

NCT ID: NCT04431895

Last Updated: 2025-09-16

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: EARLY_PHASE1
• Total Enrollment: 19 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-06-11
• Study Completion Date: 2024-03-31

Brief Summary

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.

Conditions

Myasthenia Gravis, Generalized

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

tofacitinib 5mg twice a day

Group Type: EXPERIMENTAL

Tofacitinib 5 MG

Intervention Type: DRUG

tofacitinib 5 mg tablet administered twice a day for 6 months

Interventions

Tofacitinib 5 MG

tofacitinib 5 mg tablet administered twice a day for 6 months

Intervention Type: DRUG

Eligibility Criteria

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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 70 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Huashan Hospital

OTHER

Sponsor Role: lead

Responsible Party

Chongbo Zhao

Professor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Locations

Huashan Hospital

Shanghai, , China

Countries

China

Other Identifiers

KY2020-019

Identifier Type: -

Identifier Source: org_study_id