Myasthenia Gravis, GeneralizedEarly Phase 1NCT04431895
Safety And Efficacy Of Tofacitinib In Patients With Refractory Myasthenia Gravis: A Pilot Study
A Early Phase 1 study testing Tofacitinib 5 MG in people with Myasthenia Gravis, Generalized, run by Huashan Hospital at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Myasthenia Gravis, Generalized
- What is being tested?
- Tofacitinib 5 MG
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 19 participants
- Where
- 1 siteChina
- Study dates
- 11 Jun 2020to 31 Mar 2024
- Phase
- Early Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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;
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- pregnancy;
- hyperlipidemia;
- participating other clinical trials
Trial locations and contacts
1 site in 1 country.
About this 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
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.
- Conditions
- Myasthenia Gravis, Generalized
- Being tested
- Tofacitinib 5 MG
- Keywords
- myasthenia gravis, tofacitinib
- Medical subject headings
- Myasthenia Gravis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Tofacitinib 5 MGDrug
tofacitinib 5 mg tablet administered twice a day for 6 months
| Study group | Role | What they receive |
|---|---|---|
| tofacitinib 5mg twice a day | Experimental | Not described Receives: Drug: Tofacitinib 5 MG |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Quantitative Myasthenia Gravis (QMG) Scores at Month 6
A quantitative MG scoring system (QMG Score) is essential in the objective evaluation of therapy for MG.This scoring system is based on quantitative testing of sentinel muscle groups. Total QMG score range from 0 (extreme disease severity) to 39 (none), higher score indicated less disease severity.
Measured: Baseline, Month 6
Secondary outcomes (4)
Change From Baseline in Myasthenia Gravis-related Activities of Daily Living (MG-ADL) Scores at Month 6
The myasthenia gravis activities of daily living (MG-ADL) profile is an eight-item patient-reported scale developed to assess MG symptoms and their effects on daily activities.Total MG-ADL score range from 0 (none) to 24 (extreme disease conditions), higher score indicated more disease severity.
Measured: Baseline, Month 6
Change From Baseline in Myasthenia Gravis Composite (MGC) Scores at Month 6
The MGC score consists of test items that measure symptoms and signs of MG, with weighted response options. Total MGC score range from 0 (none) to 50 (extreme muscle weakness), higher score indicated more disease severity.
Measured: Baseline, Month 6
Change From Baseline in Myasthenia Gravis-Quality of Life Questionnaire-15 item (MG-QOL15) Scores at Month 6
The MG-QOL15 is helpful in informing the clinician about the patient's perception of the extent of and dissatisfaction with MG-related dysfunction. Total MG-QOL15 score range from 0 (none) to 60 (extreme disease severity), higher score indicated more disease severity.
Measured: Baseline, Month 6
Change From Baseline in Doses of Steroids at Month 6
Steroids is recommended as the first-line treatment for myasthenia gravis. Here we followed the combined doseage of steroids while administrating tofacitinib for enrolled participants.
Measured: Baseline, Month 6
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Jun 2020
- Study start11 Jun 2020
- Primary completion21 Mar 2024
- Study completion31 Mar 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myasthenia Gravis, GeneralizedStudies of the same condition, open and under way.
- More trials run by Huashan HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 19 participants (actual)
Sponsor and investigators
- Lead sponsor
- Huashan Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Chongbo Zhao, Huashan Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04431895
- Sponsor's protocol ID
- KY2020-019
- Status last verified
- Sep 2022
- First submitted
- 12 Jun 2020
- Last updated
- 16 Sep 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Individual participant data (IPD) are not available to other researchers.
Study at a glance
From the registry record, last updated by the sponsor on 16 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective,Single-center,Single-Arm,Single-Blind Pilot Study To Investigate The Efficacy Of Tofacitinib In Patients With Refractory Myasthenia Gravis
- ClinicalTrials.gov ID
- NCT04431895
- Acronym / sponsor ID
- KY2020-019
- Conditions
- Myasthenia Gravis, Generalized
- Keywords
- myasthenia gravis, tofacitinib
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 19 participants (actual)
- Lead sponsor
- Huashan HospitalUniversity, hospital or other
- Study start
- 11 Jun 2020 (actual)
- Primary completion
- 21 Mar 2024 (actual)
- Study completion
- 31 Mar 2024 (actual)
- First posted
- 16 Jun 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| tofacitinib 5mg twice a day | Experimental | Drug: Tofacitinib 5 MG |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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.
- 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.
Key exclusion criteria (9 listed)
- 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）;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Tofacitinib 5 MGtofacitinib 5 mg tablet administered twice a day for 6 months | Drug | tofacitinib 5mg twice a day |
Primary outcome
Change From Baseline in Quantitative Myasthenia Gravis (QMG) Scores at Month 6
Time frame: Baseline, Month 6
A quantitative MG scoring system (QMG Score) is essential in the objective evaluation of therapy for MG.This scoring system is based on quantitative testing of sentinel muscle groups. Total QMG score range from 0 (extreme disease severity) to 39 (none), higher score indicated less disease severity.
4 secondary outcomes
Change From Baseline in Myasthenia Gravis-related Activities of Daily Living (MG-ADL) Scores at Month 6
Time frame: Baseline, Month 6
Change From Baseline in Myasthenia Gravis Composite (MGC) Scores at Month 6
Time frame: Baseline, Month 6
Change From Baseline in Myasthenia Gravis-Quality of Life Questionnaire-15 item (MG-QOL15) Scores at Month 6
Time frame: Baseline, Month 6
Change From Baseline in Doses of Steroids at Month 6
Time frame: Baseline, Month 6
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Sep 2025.
- Individual participant dataSharing planned: No. Individual participant data (IPD) are not available to other researchers.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Tofacitinib 5 MG in people with Myasthenia Gravis, Generalized, run by Huashan Hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 16 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change From Baseline in Quantitative Myasthenia Gravis (QMG) Scores at Month 6 (time frame: Baseline, Month 6). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Sep 2025.