Telitacicept and Low-dose Steroids in Refractory Myasthenia Gravis

NCT06723548 | Not Yet Recruiting Phase 4

• 1 other identifier: 2024-ps-195
• Study Type: interventional
• Target: 30
• Locations: 0 countries
• Sites: N/A

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

Outcome Measures

Primary Outcomes (2)

• The change in patients' ADL scores

  The variation in ADL scores at 52 weeks compared to baseline in patients. Total MG -ADL scores range from 0 (normal) to 24 (severe).

  from baseline to week 52

• The change in patients' QMG scores

  The variation in QMG scores at 52 weeks compared to baseline in patients. Total QMG scores range from 0 (none) to 39 (severe).

  from baseline to week 52

Secondary Outcomes (10)

• MGFA-PIS

  from baseline to week 52

• the average daily corticosteroid usage

  from baseline to week 24；from baseline to week 52

• the usage of traditional non-steroidal immunosuppressants

  from baseline at weeks 24 and 52

• Number of relapses

  week 52

• AUDTC

  week 52

Study Arms (1)

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

EXPERIMENTAL

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.

Drug: Telitacicept

Interventions

Telitacicept 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.

Also known as: Pyridostigmine Bromide, NSISTs, prednisone

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

Eligibility Criteria

• Age: 18 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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).

Sponsors & Collaborators

• First Affiliated Hospital of Wenzhou Medical University: lead

MeSH Terms

Conditions

Myasthenia Gravis; Autoimmune Diseases

Interventions

telitacicept; Pyridostigmine Bromide; Prednisone

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Immune System Diseases

Intervention Hierarchy (Ancestors)

Pyridinium Compounds; Pyridines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Pregnadienediols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds

Central Study Contacts

Jia Li, Master's Degree

CONTACT

19016577562; lijia@wzhospital.cn

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 15, 2024
• First Posted: December 9, 2024
• Study Start: December 1, 2024
• Primary Completion: December 1, 2025
• Study Completion (Estimated): December 1, 2026
• Last Updated: December 9, 2024

Record last verified: 2024-11

Data Sharing

• IPD Sharing: Will not share