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NCT Number: NCT06723548

Telitacicept and Low-dose Steroids in Refractory Myasthenia Gravis

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.

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Key information

Conditions

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

Age range

18 year–85 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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:
  • 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.

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).

Treatment and study plan

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.

Other names: Pyridostigmine Bromide, NSISTs, prednisone

Primary outcomes

  1. The change in patients' ADL scores

    Time frame: from baseline to week 52

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

  2. The change in patients' QMG scores

    Time frame: from baseline to week 52

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

Secondary outcomes

  1. MGFA-PIS

    Time frame: from baseline to week 52

    Change in MGFA-PIS grading from baseline to week 52

  2. the average daily corticosteroid usage

    Time frame: from baseline to week 24；from baseline to week 52

    Change in the average daily corticosteroid usage from baseline to week 24 during follow-up；Change in the average daily corticosteroid usage from baseline to week 52 during follow-up

  3. the usage of traditional non-steroidal immunosuppressants

    Time frame: from baseline at weeks 24 and 52

    Change in the usage of traditional non-steroidal immunosuppressants from baseline to week 24 during follow-up; Change in the usage of traditional non-steroidal immunosuppressants from baseline to week 52 during follow-up. Medication usage logs or medical record reviews to document the changes in the use of traditional non-steroidal immunosuppressants (such as Azathioprine, Mycophenolate Mofetil, Tacrolimus, Methotrexate, Cyclosporine, etc.) from baseline to week 52. The change in the use of traditional non-steroidal immunosuppressants will be assessed by comparing the medication usage at baseline and at weeks 24 and 52. This includes details on the type of medication, dosage, frequency, and any adjustments or discontinuations.

  4. Number of relapses

    Time frame: week 52

    Number of relapses in both groups by the end of treatment (clinical relapse is defined as an increase in QMG score of ≥3 points);

  5. AUDTC

    Time frame: week 52

    Area under the corticosteroid dose-time curve (AUDTC) at week 52

  6. Proportion of patients with a corticosteroid dose ≤10mg/d

    Time frame: at weeks 24 and 52

    Proportion of patients with a corticosteroid dose ≤10mg/d at weeks 24 and 52 during follow-up;

  7. Proportion of patients achieving MMS with a corticosteroid dose ≤ 5mg/d

    Time frame: week 24

    Proportion of patients achieving MMS with a corticosteroid dose ≤5mg/d at week 24;

  8. MG QoL15r scores

    Time frame: from baseline at weeks 24 and 52

    Change in MG QoL15r scores from baseline at weeks 24 and 52 during follow-up

  9. safety（Incidence, severity, and outcome of adverse events）

    Time frame: 52 weeks

    Incidence, severity, and outcome of adverse events

  10. relevant biomarkers

    Time frame: from baseline at week 52

    relevant biomarkers, such as immunoglobulins,lymphocyte subpopulation changes, BlyS/APRIL, and AChR-Ab titers.

Study contacts

Contact information is provided by the study sponsor or research team.

Jia Li, Master's Degree

CONTACT

[email protected]

19016577562

Sponsors and collaborators

Lead sponsor

First Affiliated Hospital of Wenzhou Medical University

Other

Registry information

Official study title

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

Important dates

Study start
2024
Primary completion
2025
Study completion
2026
First posted
Dec 9, 2024
Registry last updated
Dec 9, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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