Clinical trial NCT06723548: Telitacicept and Low-dose Steroids in Refractory Myasthenia Gravis
Official title: The Efficacy and Safety of Telitacicept Combined With Low-dose Steroids in Patients With Refractory Myasthenia Gravis
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE4
Conditions: Myasthenia Gravis
Autoimmune Diseases
Lead sponsor: First Affiliated Hospital of Wenzhou Medical University
Sponsor class: OTHER
First posted: 2024-12-09
Enrollment: 30
Sex: ALL
Minimum age (years): 18
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This study is designed to explore the efficacy and safety of Telitacicept combined with low-dose steroids for the treatment of refractory MG, and to investigate related biomarkers such as immunoglobulins, BlyS/APRIL, and AChR-Ab titers, in order to clarify whether Telitacicept can rapidly and effectively help achieve MG treatment goals and assist in steroid reduction.


Eligibility criteria: Inclusion Criteria:

* Age between 18-85 years, both genders included；
* Meet the diagnostic criteria of the 2020 Chinese MG guidelines, with positive serological testing for AChR-Ab;
* Clinical classification of Type I to Type IVa according to the MGFA;
* Meet the criteria for refractory MG in the 2022 Japanese MG guidelines: poor response to standard treatment, intolerance to standard treatment drugs due to adverse reactions, frequent relapses/exacerbations after reduction of standard treatment drugs, frequent need for rescue treatment due to disease fluctuations, frequent myasthenic crises, and comorbidities that limit the use of standard treatment;
* Patients with unstable symptoms (MG-ADL score ≥6 or QMG ≥8) despite treatment with standard therapeutic regimens before enrollment, defined as follows:

  1. Patients on monotherapy with corticosteroids: a corticosteroid dose ≤60mg/d, and a stable dose for at least 1 month before enrollment;
  2. Patients on combination therapy with corticosteroids and other immunosuppressants: a corticosteroid dose ≤60mg/d, and a stable dose for at least 1 month before enrollment, while other immunosuppressants such as azathioprine, methotrexate, tacrolimus, and mycophenolate mofetil have been stable for 6 months prior to the study start and will remain stable during the study period;
* Patients must provide written informed consent.

Exclusion Criteria:

* Patients with active infections, such as herpes zoster, HIV, active pulmonary tuberculosis, or active hepatitis;
* Patients with thymic tumors or those who have undergone thymectomy within the past 6 months;
* Patients with coexisting malignant tumors;
* Patients with severe hepatic or renal insufficiency;
* Patients who have received intravenous immunoglobulin or undergone plasmapheresis within the last 2 months;
* Patients who have received any live vaccines within the last 3 months or plan to receive any vaccines during the study period;
* Women who are currently pregnant or breastfeeding, and patients who plan to conceive during the trial period;
* Patients with allergies to human-derived biological products;
* Patients who have participated in any clinical trial within the last 28 days or within 5 half-lives of the study medication (whichever is longer);
* Any other patients deemed unsuitable for enrollment by the investigator (e.g., severe psychiatric disorders).

Primary outcomes:
measure: The change in patients' ADL scores
timeFrame: from baseline to week 52
measure: The change in patients' QMG scores
timeFrame: from baseline to week 52
Secondary outcomes:
measure: MGFA-PIS
timeFrame: from baseline to week 52
measure: the average daily corticosteroid usage
timeFrame: from baseline to week 24；from baseline to week 52
measure: the usage of traditional non-steroidal immunosuppressants
timeFrame: from baseline at weeks 24 and 52
measure: Number of relapses
timeFrame: week 52
measure: AUDTC
timeFrame: week 52
measure: Proportion of patients with a corticosteroid dose ≤10mg/d
timeFrame: at weeks 24 and 52
measure: Proportion of patients achieving MMS with a corticosteroid dose ≤ 5mg/d
timeFrame: week 24
measure: MG QoL15r scores
timeFrame: from baseline at weeks 24 and 52
measure: safety（Incidence, severity, and outcome of adverse events）
timeFrame: 52 weeks
measure: relevant biomarkers
timeFrame: from baseline at week 52
Other outcomes:
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