NCT04768465

Tacrolimus Combined With Low-dose Prednisone for Treatment of Myasthenia Gravis

Effectiveness and Safety of Tacrolimus Combined With Low-dose Prednisone for Treatment of Myasthenia Gravis: A Real-world Study

• Status: Unknown
• Phase: —
• Study type: Observational
• Enrollment: 160 (estimated)
• Sponsor: Da, Yuwei, M.D. · Individual
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This study is designed to evaluate the effectiveness and safety of tacrolimus combined with low-dose prednisone in the management of myasthenia gravis patients, compared to tacrolimus as initial immune monotherapy.

Detailed description

This is a single center, observational real-world study recruiting myasthenia gravis patients from Neurology Departments of Xuanwu Hospital, aiming to compare effectiveness and safety of 2 different inmunotherapy for MG. The study plans to recruit 160 MG participants and divides into 2 treatment groups according to physician's judgment and preferences of patients, one is combined immunotherapy group in which tacrolimus added with low-dose prednisone (0.25mg/kg/d), and the other is tacrolimus monotherapy group. Both groups can be treated with pyridostigmine to relieve symptoms. Patients are followed up at 1, 3 and 6 month after treatment initiation to assess the efficacy of both regimen. The primary outcome is the change of MG-ADL scores. Also, liver and renal functions are tested to monitor any side effects. Patients' clinical records are uploaded to an online database.

Conditions

• Myasthenia Gravis

Interventions

Type	Name	Description
DRUG	Pyridostigmine, Prednisone, Tacrolimus	Dose of tacrolimus should be initiated based on CYP3A5\*3 polymorphism and adjusted to achieve blood trough concentration of 4.8-10.0 ng/ml. Prednisone is administrated with an initial dose of 0.25mg/kg/d and started to tamper with the achievement of MMS or the presence of any intolerable side effects. The rate of tampering is considered by the physician, usually no more than 5mg/month. If the participants failed to maintain MMS, dose of prednisone should be increased 5mg/week to 0.25mg/kg/d and maintained until MMS reached again. After MMS sustained for 1 month, prednisone dose would be tapered again with 2.5mg/month. Calcium and potassium supplements and gastric mucosa protectors could be addressed to avoid any adverse effects of prednisone. Treatment regimens are determined based on the physician's judgment and preferences of the patients. This study was observational and do not change the clinical course of patients.
DRUG	Pyridostigmine, Tacrolimus	Dose of tacrolimus should be initiated based on CYP3A5\*3 polymorphism and adjusted to achieve blood trough concentration of 4.8-10.0 ng/ml. Treatment regimens are determined based on the physician's judgment and preferences of the patients. This study was observational and do not change the clinical course of patients.

Timeline

• Start date: 2021-01-01
• Primary completion: 2023-12-31
• Completion: 2024-10-31
• First posted: 2021-02-24
• Last updated: 2021-02-24

Locations

1 site across 1 country: China

Regulatory

• FDA-regulated drug study