Telitacicept and Low-dose Steroids in Refractory Myasthenia Gravis

Официальное название

The Efficacy and Safety of Telitacicept Combined With Low-dose Steroids in Patients With Refractory Myasthenia Gravis

Обзор

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.

Вмешательства

• Препарат Telitacicept
  Patients with MG who fulfill the inclusion criteria will receive Telitacicept 240mg weekly as an adjunct to their medication. Upon reaching MMS, significant symptom improvement, or a QMG score reduction of at least 6 points, Telitacicept is reduced to biweekly doses. Pyridostigmine and NSISTs are tapered based on patient response. Following this, Prednisone is tapered, starting with rapid reduction early and slowing later. If a patient on 60mg Prednisone daily achieves treatment goals within 6-8

Первичные конечные точки

• The change in patients' ADL scores [Срок оценки: from baseline to week 52]
• The change in patients' QMG scores [Срок оценки: from baseline to week 52]

Вторичные конечные точки (10)

• MGFA-PIS [Срок оценки: from baseline to week 52]
• the average daily corticosteroid usage [Срок оценки: from baseline to week 24；from baseline to week 52]
• the usage of traditional non-steroidal immunosuppressants [Срок оценки: from baseline at weeks 24 and 52]
• Number of relapses [Срок оценки: week 52]
• AUDTC [Срок оценки: week 52]
• Proportion of patients with a corticosteroid dose ≤10mg/d [Срок оценки: at weeks 24 and 52]
• Proportion of patients achieving MMS with a corticosteroid dose ≤ 5mg/d [Срок оценки: week 24]
• MG QoL15r scores [Срок оценки: from baseline at weeks 24 and 52]
• safety（Incidence, severity, and outcome of adverse events） [Срок оценки: 52 weeks]
• relevant biomarkers [Срок оценки: from baseline at week 52]

Критерии участия

Критерии включения

• 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:

• Patients on monotherapy with corticosteroids: a corticosteroid dose ≤60mg/d, and a stable dose for at least 1 month before enrollment;
• 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.

Критерии исключения

• 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).

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Список центров уточняется — проверьте первичный протокол.

Идентификаторы

NCT: NCT06723548 · 2024-ps-195

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗