Safety And Efficacy Of Tofacitinib In Patients With Refractory Myasthenia Gravis: A Pilot Study

NCT04431895 | Completed Early Phase 1 DMC

• 1 other identifier: KY2020-019
• Study Type: interventional
• Target: 19
• Locations: 1 country
• Sites: 1

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.

Conditions

Myasthenia Gravis, Generalized

Keywords

myasthenia gravis; tofacitinib

Outcome Measures

Primary Outcomes (1)

• 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.

  Baseline, Month 6

Secondary Outcomes (4)

• Change From Baseline in Myasthenia Gravis-related Activities of Daily Living (MG-ADL) Scores at Month 6

  Baseline, Month 6

• Change From Baseline in Myasthenia Gravis Composite (MGC) Scores at Month 6

  Baseline, Month 6

• Change From Baseline in Myasthenia Gravis-Quality of Life Questionnaire-15 item (MG-QOL15) Scores at Month 6

  Baseline, Month 6

• Change From Baseline in Doses of Steroids at Month 6

  Baseline, Month 6

Other Outcomes (2)

• Change From Baseline in Serum Titers of anti-acetylcholine receptor antibodies at Week 16

  Baseline, Week 16

• Change From Baseline in Inflammatory Cytokines at Week 12

  Baseline, Week 12

Study Arms (1)

tofacitinib 5mg twice a day

EXPERIMENTAL

Drug: Tofacitinib 5 MG

Interventions

Tofacitinib 5 MG DRUG

tofacitinib 5 mg tablet administered twice a day for 6 months

tofacitinib 5mg twice a day

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Huashan Hospital: lead

Study Sites (1)

Huashan Hospital

Shanghai, 200040, China

Location

MeSH Terms

Conditions

Myasthenia Gravis

Interventions

tofacitinib

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: June 12, 2020
• First Posted: June 16, 2020
• Study Start: June 11, 2020
• Primary Completion: March 21, 2024
• Study Completion: March 31, 2024
• Last Updated: September 16, 2025

Record last verified: 2022-09

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)