Clinical trial · Interventional
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
NCT04431895CI-TRIAL-00095195completedEarly Phase 1ClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Myasthenia Gravis, Generalized | — | UNRESOLVED | — |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Tofacitinib 5 MG | Drug | Tofacitinib | ALIAS |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- tofacitinib 5mg twice a day
- interventionNames
- Drug: Tofacitinib 5 MG
Primary outcomes (1)
- measure
- Change From Baseline in Quantitative Myasthenia Gravis (QMG) Scores at Month 6
- timeFrame
- Baseline, Month 6
- description
- 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.
Secondary outcomes (4)
- measure
- Change From Baseline in Myasthenia Gravis-related Activities of Daily Living (MG-ADL) Scores at Month 6
- timeFrame
- Baseline, Month 6
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
- Maximum age
- 70 Years
Show eligibility criteria text
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
References
Publications (0)
Data not yet available
No reference posted for this study.