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

Fudan University

Status and phase

Enrolling

Early Phase 1

Conditions

Myasthenia Gravis, Generalized

Treatments

Drug: Tofacitinib 5 MG

Study type

Interventional

Funder types

Other

Identifiers

NCT04431895

KY2020-019

Details and patient eligibility

About

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.

Enrollment

20 estimated patients

Sex

All

Ages

18 to 60 years old

Volunteers

No Healthy Volunteers

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