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Could a common arthritis drug ease tough myasthenia gravis?

NCT ID NCT04431895

Disease control Sponsor: Huashan Hospital Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 26, 2026 · Last updated Jun 26, 2026

Summary

This pilot study tested the drug tofacitinib in 19 adults with refractory myasthenia gravis, a condition where standard treatments fail. Participants took a 5 mg tablet twice daily for 4 months, followed by a 1-month washout. The study measured changes in muscle strength and daily living activities. As an early-phase, single-arm trial, it provides initial safety and efficacy data but cannot prove the drug works.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
tofacitinib (a JAK inhibitor, taken as a 5 mg tablet twice daily)
What this could lead to
If it works, this could offer a new oral treatment option for people with myasthenia gravis that does not respond to standard therapies.
What could go wrong
This is a very early, small pilot study with only 19 participants and no placebo group. Results may not apply to all patients, and tofacitinib has known risks like infections and blood clots.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Early phase 1

The earliest testing in people: a first look at safety, in a very small group.

Participants

19 people

The number who actually took part.

Started

Jun 2020

Finished

Mar 2024

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 70 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

myasthenia gravis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Myasthenia gravis, generalized

Contacts and locations

Locations

  • Huashan Hospital

    Shanghai, 200040, China

More trials for these conditions

Other studies related to the condition(s) this trial covers.