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

• Status: Recruiting
• Trial end date: 2022-02-09
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Early Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Huashan Hospital
• Treatments: Tofacitinib

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