Telitacicept for Myasthenia Gravis

Recruiting at 12 trial locations

Overseen By RemeGen

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: RemeGen Co., Ltd.

Disqualifiers: Autoimmune disease, Infection, Thymoma, Immunodeficiency, Malignancy, Cardiovascular, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 24 weeks

180 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

How is the drug Telitacicept different from other treatments for myasthenia gravis?

Telitacicept is unique because it targets specific immune system components involved in autoimmune diseases, potentially offering a novel approach compared to existing treatments for myasthenia gravis, which often focus on general immune suppression or symptom management. ^{1 2 3 4 5}

What is the purpose of this trial?

The purpose of this study is to evaluate the efficacy and safety of telitacicept in the treatment of generalized myasthenia gravis.

Eligibility Criteria

This trial is for adults over 18 with generalized myasthenia gravis, a muscle weakness condition. Participants must have certain antibodies and scores indicating moderate to severe symptoms but not solely ocular-related. They should have a confirmed diagnosis fitting specific MGFA clinical classifications.

Inclusion Criteria

My tests show positive for antibodies against AChR or MuSK.

MG-ADL score ≥6 points at screening and baseline with ocular-related score <50% of the total score

QMG score ≥11 points at screening and baseline

Exclusion Criteria

I am currently dealing with an infection.

I had thymoma within the last 5 years or a thymectomy within the last 6 months.

Patients having current or history of primary immunodeficiency

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive telitacicept or placebo in a double-blind manner to evaluate efficacy and safety in treating generalized myasthenia gravis

24 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Participants may opt into continuation of treatment with telitacicept long-term

Treatment Details

Interventions

• Telitacicept

Trial Overview The study is testing Telitacicept's effectiveness and safety in treating generalized myasthenia gravis compared to a placebo. Patients will be randomly assigned to receive either the actual medication or an inactive substance.

Participant Groups

Group I: Telitacicept Experimental Treatment 1 Intervention

Telitacicept

Group II: Placebo Placebo Group 1 Intervention

Placebo

Telitacicept is already approved in China for the following indications:

Approved in China as Tai'ai for:

• Systemic lupus erythematosus (SLE)

Who Is Running the Clinical Trial?

RemeGen Co., Ltd.

Lead Sponsor

Trials

84

Recruited

11,100+

Dr. Jianmin Fang

RemeGen Co., Ltd.

Chief Executive Officer since 2008

PhD in Molecular Biology

Dr. Ruyi He

RemeGen Co., Ltd.

Chief Medical Officer since 2023

MD

Findings from Research

A new sum score has been developed to define treatment-refractory generalized myasthenia gravis (TRgMG), which includes criteria such as disease severity and inefficacy of previous therapies, making it easier for clinicians to make treatment decisions.

This scoring system, created through a consensus of 12 experts and based on a thorough literature review, aims to clarify when eculizumab should be used and can also serve as a basis for inclusion criteria in future clinical studies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A Sum Score to Define Therapy-Refractory Myasthenia Gravis: A German Consensus. Schroeter, M., Berger, B., Blaes, F., et al.[2022]

This systematic review aims to evaluate the effectiveness and safety of acupuncture as an adjuvant therapy for myasthenia gravis (MG) by analyzing randomized controlled trials (RCTs) from multiple databases, ensuring a comprehensive assessment.

The review will provide high-quality evidence regarding acupuncture's role in treating MG, with a focus on minimizing bias and ensuring rigorous trial quality assessment through established methods.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Acupuncture for patients with myasthenia gravis: A systematic review protocol. Shi, S., Ji, X., Wang, Y., et al.[2022]

The FDA has recently approved new therapies, including complement and Fc receptor inhibitors, for treating generalized myasthenia gravis, highlighting advancements in treatment options for this condition.

There is ongoing uncertainty regarding which patients will benefit most from these expensive therapies, prompting discussions on the need for better clinical trial readiness and biomarker development to guide future research.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The best and worst of times in therapy development for myasthenia gravis. Benatar, M., Cutter, G., Kaminski, HJ.[2023]

Observational comparative effectiveness research (CER) offers a promising alternative to randomized controlled trials (RCTs) for evaluating treatments in myasthenia gravis (MG), addressing challenges like disease rarity and variability by using real-life clinical data from routine care.

Recent advancements, including the creation of common data elements for MG and a standardized web-based database, enhance the feasibility of conducting multisite CER studies, allowing for better comparisons of therapies and improved understanding of treatment outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Comparative effectiveness clinical trials to advance treatment of myasthenia gravis. Guptill, JT., Raja, S., Sanders, DB., et al.[2019]

The virtual Myasthenia Gravis Impairment Index (vMGII) effectively correlates with other patient-reported outcomes, demonstrating its utility in assessing disease status during telephone consultations for 214 myasthenia gravis patients.

A vMGII score of 11.5 or higher reliably predicts a positive response on the Patient-Acceptable Symptom State (vPASS), with a sensitivity of 78.7% and specificity of 81.4%, indicating its effectiveness in monitoring disease progression even without in-person evaluations.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Telephone consultation for myasthenia gravis care during the COVID-19 pandemic: Assessment of a novel virtual myasthenia gravis index. Menon, D., Alnajjar, S., Barnett, C., et al.[2023]

References

1. United States pubmed.ncbi.nlm.nih.gov

A Sum Score to Define Therapy-Refractory Myasthenia Gravis: A German Consensus. [2022]

2. United States pubmed.ncbi.nlm.nih.gov

Acupuncture for patients with myasthenia gravis: A systematic review protocol. [2022]

3. United States pubmed.ncbi.nlm.nih.gov

The best and worst of times in therapy development for myasthenia gravis. [2023]

4. United States pubmed.ncbi.nlm.nih.gov

Comparative effectiveness clinical trials to advance treatment of myasthenia gravis. [2019]

5. United States pubmed.ncbi.nlm.nih.gov

Telephone consultation for myasthenia gravis care during the COVID-19 pandemic: Assessment of a novel virtual myasthenia gravis index. [2023]