Efficacy and Safety of Efgartigimod Sequential Therapy with Telitacicept in Generalized Myasthenia Gravis

NCT06827587 | Not Yet Recruiting DMC

• 1 other identifier: KY2024-298
• Study Type: interventional
• Target: 60
• Locations: 0 countries
• Sites: N/A

Brief Summary

Title: The Efficacy, Safety, and PK/PD of Efgartigimod Followed by Telitacicept in Generalized Myasthenia Gravis: Protocol of a Randomized Controlled Trial Objective: This study aims to evaluate the efficacy, safety, and pharmacokinetics/pharmacodynamics (PK/PD) of efgartigimod followed by telitacicept in patients with generalized myasthenia gravis (gMG). Specifically, the trial seeks to determine the optimal treatment strategy for gMG by assessing the effect of sequential biologic therapy on disease management. Study Design: This is a multi-center, open-label, randomized controlled trial involving 60 acetylcholine receptor antibody-positive gMG patients.

Conditions

Myasthenia Gravis; Autoimmune Diseases; Neuromuscular Diseases

Outcome Measures

Primary Outcomes (1)

• Change from baseline in QMG score

  Change in QMG score from baseline to Weeks 30. Total QMG scores range from 0 (none) to 39 (severe).

  30 weeks after randomization

Secondary Outcomes (8)

• Change from baseline in MG-ADL score

  Weeks 4, 8, 12, 18, 24, and 30 after randomization.

• Proportion of subjects with minimal manifestation status (MMS)

  4, 8, 12, 18, 24, and 30 weeks after randomization.

• Proportion of subjects with a ≥2-point reduction in MG-ADL score from baseline

  4, 8, 12, 18, 24, and 30 weeks after randomization.

• Proportion of subjects with a ≥3-point reduction in QMG score from baseline

  4, 8, 12, 18, 24, and 30 weeks after randomization.

• Changes in the doses of steroids and other immunosuppressants

  24 and 30 weeks after randomization.

Other Outcomes (5)

• Plasma concentration of telitacicept in the two sequential treatment groups

  30 minutes before the first dose of telitacicept, and 6 hours, 24 hours, 48 hours, 72 hours, Week 1, Week 4, Week 8, Week 12, and Week 24 after the first dose.

• Serum immunoglobulins

  At baseline, after one cycle (4 weeks) of efgartigimod treatment, before the first dose of telitacicept, and at Week 1, Week 4, and every subsequent 4-week interval after the first dose of telitacicept

• B-cell subpopulation count and BLyS/APRIL measurement

  Before the first dose of telitacicept, at Week 4, at Week 12, and at Week 24.

Study Arms (3)

Efgartigimod + Telitacicept (1-week interval) group

EXPERIMENTAL

Starting from Week 0, participants will receive efgartigimod (10 mg/kg) for induction treatment by intravenous infusion over 1 hour, administered once a week for 4 consecutive weeks. After a 1-week interval, at Week 5, participants will receive telitacicept (240 mg) for maintenance treatment, administered once a week by subcutaneous injection for a total of 25 weeks. Follow-up will continue until Week 30.

Drug: Efgartigimod + Telitacicept (1-week interval) group

Efgartigimod + Telitacicept (2-week interval) group

EXPERIMENTAL

Starting from Week 0, participants will receive efgartigimod (10 mg/kg) for induction treatment by intravenous infusion over 1 hour, administered once a week for 4 consecutive weeks. After a 2-week interval, at Week 6, participants will receive telitacicept (240 mg) for maintenance treatment, administered once a week by subcutaneous injection for a total of 24 weeks. Follow-up will continue until Week 30.

Drug: Efgartigimod + Telitacicept (2-week interval) group

Telitacicept-only group

ACTIVE COMPARATOR

Participants will not receive efgartigimod for induction treatment. Starting from Week 0, participants will receive telitacicept (240 mg) for maintenance treatment, administered once a week by subcutaneous injection for a total of 30 weeks. Follow-up will continue until Week 30.

Drug: Telitacicept-only group

Interventions

Efgartigimod + Telitacicept (1-week interval) group DRUG

Drug 1: Efgartigimod Generic Name: Efgartigimod Formulation: Injection (Intravenous) Dose: 10 mg/kg Frequency: Once a week Route of Administration: Intravenous infusion Duration: 4 weeks (Week 0 to Week 4) Drug 2: Telitacicept Generic Name: Telitacicept Formulation: Injection (Subcutaneous) Dose: 240 mg Frequency: Once a week Route of Administration: Subcutaneous injection Duration: 25 weeks (Week 5 to Week 29)

Also known as: E+1w+T

Efgartigimod + Telitacicept (1-week interval) group

Efgartigimod + Telitacicept (2-week interval) group DRUG

Drug 1: Efgartigimod Generic Name: Efgartigimod Formulation: Injection (Intravenous) Dose: 10 mg/kg Frequency: Once a week Route of Administration: Intravenous infusion Duration: 4 weeks (Week 0 to Week 4) Drug 2: Telitacicept Generic Name: Telitacicept Formulation: Injection (Subcutaneous) Dose: 240 mg Frequency: Once a week Route of Administration: Subcutaneous injection Duration: 24 weeks (Week 6 to Week 29)

Also known as: E+2w+T

Efgartigimod + Telitacicept (2-week interval) group

Telitacicept-only group DRUG

Drug: Telitacicept Generic Name: Telitacicept Formulation: Injection (Subcutaneous) Dose: 240 mg Frequency: Once a week Route of Administration: Subcutaneous injection Duration: 30 weeks (Week 0 to Week 29)

Also known as: T-only

Telitacicept-only group

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• The patient voluntarily signs the informed consent form.
• Age ≥18 and ≤80 years, regardless of gender.
• Meets the diagnostic criteria for Myasthenia Gravis (MG) as per the 2020 China MG Diagnosis and Treatment Guidelines, with serological evidence of positive AChR-Ab.
• MG Clinical Classification according to the Myasthenia Gravis Foundation of America (MGFA) Staging System: Grade II-IV.
• Patients with fluctuating MG symptoms before enrollment, as indicated by MG-ADL score ≥6 or Quantitative Myasthenia Gravis score (QMG) ≥8, maintained for more than 24 hours.

You may not qualify if:

• Coexisting active autoimmune diseases, such as Systemic Lupus Erythematosus (SLE), Rheumatoid Arthritis, Sjögren's Syndrome, etc.
• Patients with active infections, such as Herpes Zoster, HIV, active Tuberculosis, active Hepatitis.
• Patients with thymoma who have undergone surgery within the past 6 months.
• Patients with malignancies other than thymoma.
• Patients with severe liver or renal dysfunction, defined as: Liver function: ALT or AST \> 3 ×ULN (upper limit of normal). Renal function: Glomerular filtration rate (GFR) \< 30 mL/min/1.73m².
• IgG ≤ 400 mg/dL.
• Patients who have used biologic agents before enrollment and are within 5 half-lives of the drug, such as those who have used Telitacicept within the past 2 months, Efgartigimod within the past 1 month, or Rituximab within the past 6 months.
• Patients who have received intravenous immunoglobulin (IVIg) or undergone plasmapheresis within the past 2 months before enrollment.
• Patients who have received any live vaccines within 3 months before the study or plan to receive any vaccines during the study.
• Pregnant or breastfeeding women, or those planning to conceive during the trial.
• Patients with allergies to human-derived biologics.
• Patients who have participated in any clinical trial within the 28 days before enrollment or are within 5 half-lives of the investigational drug used in a prior clinical trial.
• Other patients deemed unsuitable for enrollment by the investigator (e.g., severe mental disorders).

Sponsors & Collaborators

• First Affiliated Hospital of Wenzhou Medical University: lead
• Zhejiang Provincial People's Hospital: collaborator
• Sir Run Run Shaw Hospital: collaborator
• Second Affiliated Hospital, School of Medicine, Zhejiang University: collaborator
• Ningbo Medical Center Lihuili Hospital: collaborator

Related Publications (1)

• Li J, Zhang Y, Deng Y, Li W, Wang Y, Qi F, Zhang Q, Wan B, Li X, Weng Y, Fang Z, Zhang Y, Qu X, Pan S, Yang S, Zhang X. The efficacy, safety, and pharmacokinetics/pharmacodynamics of telitacicept following efgartigimod in generalized myasthenia gravis: protocol of a randomized controlled trial. Front Immunol. 2025 Oct 22;16:1604786. doi: 10.3389/fimmu.2025.1604786. eCollection 2025.

  PMID: 41200184 DERIVED

MeSH Terms

Conditions

Myasthenia Gravis; Autoimmune Diseases; Neuromuscular Diseases

Interventions

telitacicept; Population Groups

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; Immune System Diseases

Intervention Hierarchy (Ancestors)

Demography; Population Characteristics

Study Officials

• Xu Zhang, Bachelor

  First Affiliated Hospital of Wenzhou Medical University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Xu Zhang, Bachelor

CONTACT

0086-13587688130; drzhangxu@live.cn

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 23, 2025
• First Posted: February 14, 2025
• Study Start: February 1, 2025
• Primary Completion: February 1, 2026
• Study Completion (Estimated): December 1, 2026
• Last Updated: February 14, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will not share