Safety And Efficacy Of Tofacitinib In Patients With Refractory Myasthenia Gravis: A Pilot Study (NCT04431895) | Clinical Trial Compass
CompletedEarly Phase 1
Safety And Efficacy Of Tofacitinib In Patients With Refractory Myasthenia Gravis: A Pilot Study
China19 participantsStarted 2020-06-11
Plain-language 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.
Who can participate
Age range18 Years – 70 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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
What they're measuring
1
Change From Baseline in Quantitative Myasthenia Gravis (QMG) Scores at Month 6