Clinical trial NCT04768465: Tacrolimus Combined With Low-dose Prednisone for Treatment of Myasthenia Gravis
Official title: Effectiveness and Safety of Tacrolimus Combined With Low-dose Prednisone for Treatment of Myasthenia Gravis: A Real-world Study
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Myasthenia Gravis
Keywords: myasthenia gravis
treatment
tacrolimus
low-dose prednisone
Lead sponsor: Da, Yuwei, M.D.
Sponsor class: INDIV
First posted: 2021-02-24
Enrollment: 160
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with Myasthenia Gravis

Brief 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.

Eligibility criteria: Inclusion Criteria:

* Age ≥18
* Clinical Diagnosis of MG is confirmed based on typical clinical features of fluctuating muscle weakness, with at least 1 of the following supporting evidence:

  1. positive clinical response to acetylcholinesterase inhibitor
  2. positive AchR-Ab or MuSK-Ab testing
  3. decrement \>10% in repetitive nerve stimulations study (RNS) or increased jitter on single-fibre electromyography (SFEMG)
* MGFA clinical classification: I - IV
* Baseline MG-ADL ≥ 3
* Disease course from onset to enrollment ≤ 12 months
* Cooperation to followup
* Written informed consent

Exclusion Criteria:

* Initiation of immunosuppressant for MG prior to screening, including Prednisone, Methylprednisolone, Azathioprine, Methotrexate, Cyclosporine A, Mycophenolate Mofetil, Tacrolimus and Cyclophosphamide
* Treatment of immunosuppressant for other concomitant disease 6 months prior to recruitment
* Rapid immunosuppressive treatments like Intravenous immunoglobulin or plasma exchange 1 month prior to recruitment
* Thymectomy within 3 months prior to Screening
* Concomitant chronic degenerative, psychiatric, or neurologic disorder that can cause weakness or fatigue
* Consciousness, dementia or schizophrenia
* Pregnancy or lactation, unwillingness to avoid pregnancy
* Uncontrolled hypertension or diabetes, Liver or kidney dysfunction, Cataract, Severe osteoporosis, Femoral head necrosis; Hyperkalemia, HIV, Acute or chronic infection
* Other conditions that would preclude participation

Primary outcomes:
measure: Change of MG-specific Activities of Daily Living scale (MG-ADL) from Baseline
timeFrame: Baseline, 1 month, 3 months, 6 months
Secondary outcomes:
measure: Time to achievement of minimal manifestations (MMS) or better
timeFrame: From Baseline to 6 months
measure: Time to achievement of Patient-Acceptable Symptom States
timeFrame: From Baseline to 6 months
measure: Change of Quantitative Myasthenia Gravis (QMG) Scores from Baseline
timeFrame: Baseline, 1 month, 3 months, 6 months
measure: Change of Myasthenia Gravis Quantity-of-Life Scale (MG-QoL15) from Baseline
timeFrame: Baseline, 1 month, 3 months, 6 months
measure: Changes of MG-ADL subscores from baseline
timeFrame: Baseline, 1 month, 3 months, 6 months
measure: Serum IL-2 level
timeFrame: Baseline, 1 month, 3 months, 6 months
measure: Treatment Failure
timeFrame: Baseline to 6 months
measure: Withdrawal
timeFrame: Baseline to 6 months
Other outcomes:
Locations:
city: Beijing
country: China
facility: Yuwei Da
state: Beijing
Study officials:
affiliation: Xuanwu Hospital, Beijing
name: Yuwei Da, M.D.
role: STUDY_CHAIR
References: