Telitacicept and Low-dose Steroids in Refractory Myasthenia Gravis

NCT ID: NCT06723548

Last Updated: 2024-12-09

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: PHASE4
• Total Enrollment: 30 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-12-31
• Study Completion Date: 2026-12-31

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.

Conditions

Myasthenia Gravis; Autoimmune Diseases

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Patients with refractory MG treated with Telitacicept combined with low-dose steroids

This is an open-label, single-arm exploratory study of Telitacicept (240mg weekly, then every two weeks after achieving MMS or QMG reduction of ≥ 6 points) combined with a gradual reduction of steroids and other immunosuppressants. When the steroid dose is reduced to 5mg/day or 10mg/every other day, Telitacicept can be reduced to 160mg, administered subcutaneously every two weeks.

Group Type: EXPERIMENTAL

Telitacicept

Intervention Type: DRUG

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 weeks of Telitacicept initiation, the tapering sequence is 60mg every other day for 4 weeks, then 30mg daily for 2-4 weeks, and so on, until reaching 5mg daily or 10mg every other day. At this point, Telitacicept may be reduced to 160mg biweekly.

Interventions

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 weeks of Telitacicept initiation, the tapering sequence is 60mg every other day for 4 weeks, then 30mg daily for 2-4 weeks, and so on, until reaching 5mg daily or 10mg every other day. At this point, Telitacicept may be reduced to 160mg biweekly.

Intervention Type: DRUG

Other Intervention Names

Pyridostigmine Bromide; NSISTs; prednisone

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).

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 85 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

First Affiliated Hospital of Wenzhou Medical University

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Central Contacts

Jia Li, Master's Degree

Role: CONTACT

Phone: 19016577562

Email: lijia@wzhospital.cn

Other Identifiers

2024-ps-195

Identifier Type: -

Identifier Source: org_study_id