NCT04431895CompletedEarly phase 1
Safety And Efficacy Of Tofacitinib In Patients With Refractory Myasthenia Gravis: A Pilot Study
A Prospective,Single-center,Single-Arm,Single-Blind Pilot Study To Investigate The Efficacy Of Tofacitinib In Patients With Refractory Myasthenia Gravis
Source: ClinicalTrials.gov · Last updated 16 Sept 2025
Brief summary (registry, in English)
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.
- Status (registry)
- Completed
- Phase (registry)
- Early phase 1
- Study type
- Interventional
- Enrollment
- 19 (actual)
- First submitted
- 12 Jun 2020
- Start date
- 11 Jun 2020
- Primary completion date
- 21 Mar 2024
- Lead sponsor
- Huashan Hospital
- Diseases
- Myasthenia gravis
- NCT number
- NCT04431895
What you commit to
- Study type: Interventional · Early phase 1
- Participants enrolled: 19
- From 11 Jun 2020 to the primary completion date, 21 Mar 2024
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- positive for muscle-specific kinase (MuSK) antibodies;
Other illness
- a history of Type B and Type C hepatitis;
- a history of tuberculosis or T-SPOT.TB tset positivity;
- severe allergy or infection, or chronic or recurrent infection;
Lab values
- hepatic, renal and cardial insufficiency (baseline: ALT/AST\>50U/L;BNP\>200pg/ml）;
Other
- a tumor history except for thymoma;
- pregnancy;
- hyperlipidemia;
- participating other clinical trials
Who the study is looking for
- 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.
Age in the registry: 18 years to 70 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Huashan Hospital Coordinator of this site? Confirm its status | Shanghai | China | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.