Efficacy and safety of methylprednisolone pulse therapy for ocular myasthenia gravis: a pilot study

JRCT ID: jRCTs051200137

Registered date:17/02/2021

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: ocular myasthenia gravis
• Date of first enrollment: 17/02/2021
• Target sample size: 10
• Study type: Interventional
• Intervention(s): Methylprednisolone 1 g daily is administered intravenously for 3 consecutive days for 2 to 3 courses, and then 5 mg of prednisolone daily is orally administered until 16 weeks.

Outcome(s)

• Primary Outcome: Ocular MG-ADL change 4 weeks after the start of IVMP

Secondary Outcome

MM achievement rate at 4 to 16 weeks
Ocular-QMG scores at baseline and at 4 and 8 weeks, 12 weeks, 16 weeks
Ocular MG-ADL scale at baseline and at 4 and 8 weeks, 12 weeks, 16 weeks
MG-QOL15 at baseline and at 4 and 16 weeks
Adverse event

Key inclusion & exclusion criteria

• Age minimum: >= 20age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

(1) Diagnosed of OMG without thymoma with any of the symptoms of ocular motility disorder, ptosis, or facial muscle weakness showing easy fatigue
(2) One or more positive findings of either A or B:
A. Pathogenic autoantibodies (1 or 2)
1 Anti-acetylcholine receptor (AChR) antibody
2 Anti-muscle-specific receptor tyrosine kinase (MuSK) antibody
B. Neuromuscular junction disorders (one or more of the following)
1 Eyelid fatigue test
2 Ice pack test
3 Edrophonium test
4 Repeated stimulation test
5 Increased jitter in single-fiber EMG
(3) An ocular MG-ADL scale 1 or higher, and ocular symptoms that interfere with daily activities
(4) Treating the maximum dose of AChE-I or difficulty increasing the dose of AChE-I due to adverse events
(5) Aged 20 years or older
(6) Capable of providing informed consent and complying with study procedures

Exclude criteria

(1) Disease duration 10 years or more after onset
(2) Treatment with systemic corticosteroids, immunosuppressive drugs, immunoglobulins or plasmapheresis within 90 days of enrollment.
(3) Patients with thymoma (post-resection patients are acceptable)
(4) Poorly controlled diabetes (HbA1c 8.0% or more)
(5) Poorly controlled glaucoma
(6) Poorly controlled hypertension (systolic blood pressure 180 mmHg or higher, or diastolic blood pressure 100 mmHg or higher)
(7) prior steroid intolerance
(8) Obesity (BMI over 40 kg/m2)
(9) A history of osteoporotic fracture
(10) HBs antigen positive or HBV-DNA amount detection sensitivity or higher, or HCV antibody positive
(11) Tuberculosis infection within 2 years, or interferon gamma release assay (T-spot. TB or QFT) positive
(12) Active thyroid disease, liver or renal failure, poorly controlled cardiac or blood disease, active peptic ulcer or infection, or any other illness that would make it unsafe for the patient to participate in the trial
(13) Pregnant or lactating
(14) Receipt of another study within 30 days prior to enrollment

Related Information

• Primary Sponsor: Koh Jinsoo
• Source(s) of Monetary Support: Japan Blood Products Organization