Clinical trial NCT04431895: Safety And Efficacy Of Tofacitinib In Patients With Refractory Myasthenia Gravis: A Pilot Study
Official title: A Prospective,Single-center,Single-Arm,Single-Blind Pilot Study To Investigate The Efficacy Of Tofacitinib In Patients With Refractory Myasthenia Gravis
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: EARLY_PHASE1
Conditions: Myasthenia Gravis, Generalized
Keywords: myasthenia gravis
tofacitinib
Lead sponsor: Huashan Hospital
Sponsor class: OTHER
First posted: 2020-06-16
Enrollment: 20
Sex: ALL
Minimum age (years): 18
Maximum age (years): 60
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


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

Primary outcomes:
measure: Change From Baseline in Quantitative Myasthenia Gravis (QMG) Scores at Month 6
timeFrame: Baseline, Month 6
Secondary outcomes:
measure: Change From Baseline in Myasthenia Gravis-related Activities of Daily Living (MG-ADL) Scores at Month 6
timeFrame: Baseline, Month 6
measure: Change From Baseline in Manual Muscle Testing (MMT) Scores at Month 6
timeFrame: Baseline, Month 6
measure: Change From Baseline in Myasthenia Gravis-Quality of Life Questionnaire-15 item (MG-QOL15) Scores at Month 6
timeFrame: Baseline, Month 6
measure: Change From Baseline in Doses of Steroids at Month 6
timeFrame: Baseline, Month 6
Other outcomes:
measure: Change From Baseline in Serum Titers of anti-acetylcholine receptor antibodies at Month 6
timeFrame: Baseline, Month 6
measure: Change From Baseline in Inflammatory Cytokines at Month 6
timeFrame: Baseline, Month 6
Locations:
city: Shanghai
country: China
facility: Huashan Hospital
Study officials:
References: