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Myasthenia GravisPhase 2NCT06221501

Perioperative Efgartigimod for Thymoma and Myasthenia Gravis

A Phase 2 study testing Efgartigimod Alfa in people with Myasthenia Gravis, run by Shanghai Zhongshan Hospital.

Not yet recruiting

Opening soon. The study is approved but enrolment has not started yet. Sites may already take enquiries.

Official record
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Trial summary

It is evidenced that efgartigimod can rapidly and significantly improve the symptoms of myasthenia gravis. The global multicenter clinical trials hace confirmed that efgartigimod is safe and well tolerated. Few case reports showed that perioperative efgartigimod combined with thymectomy was safe and feasible. Read more about this study

Who is it for?
Ages 18 to 75 years
Conditions the study is for
Myasthenia Gravis
What is being tested?
Efgartigimod Alfa
Type of treatment
Injection or infusion
How many people
30 participants
Where
No sites listed yet
Study dates
1 Feb 2024to 30 Jun 2027
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Adult patients aged ≥ 18 years and ≤ 75 years with expected survival time > 12 months;
  • Patients with systemic Myasthenia gravis;
  • AChR Antibody positive;
  • MGFA Type II- IV;
  • Clinical diagnosis of Neoplasm of thymus by enhanced chest CT (clinical stage: Masaoka-Koga Stage I-IVa);
  • Patients with American Society of Anesthesiologists (ASA) classification of 1-2;
  • Subjects have no dysfunction of major organs; blood routine, lung, liver, kidney function and cardiac function are basically normal; laboratory test indicators must meet the following requirements: Blood: White cells > 4.0 x 109/L, absolute count of neutrophils (ANC) ≥ 2.0 x 109/L, Thrombocyte count > 100 x 109/L, Hemoglobin > 90 g/L; Lung function: FEV1 ≥ 1.2 L, FEV1% ≥ 50% and DLCO ≥ 50%. Note: FEV1: Forced vital capacity measured value (liter). FEV1%:% of Forced vital capacity observed/predicted. DLCO%: measured/predicted value% of diffusion capacity of the CO in one breath; Liver function: Serum bilirubin less than 1.5 times the maximum normal value; ALT and AST less than 1.5 times the maximum normal value; Renal function: blood creatinine (SCr) ≤ 120 µmol/L, creatinine clearance (CCr) ≥ 60 ml/min;
  • Understand the study and sign the informed consent form.

Who cannot join

  • Patients whose imaging investigation suggests that the tumor has already had hematogenous metastasis (clinical stage: Masaoka-Koga IVb);
  • Patients with ocular muscular myasthenia gravis (OMG);
  • Patients who have received median sternotomy ;
  • Documented history of congestive cardiac failure; poorly controlled drug therapy on Anginal pain; electrocardiogram (ECG) documented transmural myocardial infarction; poorly controlled hypertensive; clinically significant heart valve disorders; or high-risk uncontrolled arrhythmia;
  • Patients with a loss of more than 5 Kg within the past month; severe uncontrolled systemic intercurrent illness such as active Infection or poorly controlled Diabetes mellitus; patients with combined Hemorrhagic disorder and Haemorrhagic diathesis; patients with abnormal coagulation function, having haemorrhagic diathesis or receiving thrombolysis or anticoagulant therapy; and patients with grade II-IV myelosuppression.
  • Serum pregnancy test positive or lactating females, as well as males and females of childbearing potential who are unwilling to take adequate contraception measures during treatment;
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  • History of organ transplant (including autologous bone marrow Transplant and peripheral Stem cell transplant);
  • Patients with peripheral nervous system disorders or significant history of Mental disorder and central nervous system disorders;
  • Concurrent participation in other clinical investigators.
  • Patients who cannot tolerate one-lung ventilation in the Surgery; patients with severe cardiac complications, cardiovascular compensatory dysfunction, cardiac pacemaker implantation;
  • Patients with acute Inflammation due to bacterial, viral or other pathogenic microorganism Infection; known Immunodeficiency virus (HIV), Hepatitis B virus (HBV) or Hepatitis C virus (HCV) active Infection or known HIV seropositivity;
  • Patients who previously underwent Thoracic operation for Tuberculous pleurisy, Mesothelioma, Pulmonary disorder, diaphragmatic disease, ipsilateral Atelectasis involving one lobe or more than one lobe;
  • Serum IgG level < 6 g/L;
  • Received biologic agents such as rituximab or eculizumab within 6 months; IV Immunoglobulins or Plasma exchange within 1 month;

About this study

Official title: Safety and Efficacy of Perioperative Efgartigimod Combined With Thymectomy for Thymoma and Myasthenia Gravis: A Prospective, Single-arm Study

It is evidenced that efgartigimod can rapidly and significantly improve the symptoms of myasthenia gravis. The global multicenter clinical trials hace confirmed that efgartigimod is safe and well tolerated. Few case reports showed that perioperative efgartigimod combined with thymectomy was safe and feasible. However, there was no sufficient data on safety and efficacy of this regimen in the treatment for patients with myasthenia gravis and thymomas. Therefore, this trial aims to evaluate the efficacy and safety of perioperative efgartigimod and thymectomy for patients with myasthenia gravis and thymomas.

Being tested
Efgartigimod Alfa
Medical subject headings
Myasthenia Gravis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Efgartigimod AlfaDrug

    Efgartigimod will be administered intravenously on Day (D)1, D8, D15, and D22 at a dose of 10 mg/kg according to the patient's weight (maximum infusion dose of 1200 mg) over approximately 1 hour for a total of 4 doses. Thymectomy will be performed on Day 9.

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change of the activity of daily living (ADL) score at the fourth visit from the baseline visit (first visit)

    The value was calculated by the ADL score at the fourth visit minus the ADL score at the first visit (baseline).

    Measured: Baseline and 3 weeks after the first visit.

Secondary outcomes (8)

  1. Change of the ADL score at the second visit from the baseline visit (first visit)

    The value was calculated by the ADL score at the second visit minus the ADL score at the first visit (baseline).

    Measured: Baseline and 1 day before surgery (always at the 1 week after the first visit).

  2. Change of quantitative myasthenia gravis (QMG) score at the second visit from the baseline visit (first visit)

    The value was calculated by the QMG score at the second visit minus the QMG score at the first visit (baseline).

    Measured: Baseline and 1 day before surgery (always at the 1 week after the first visit).

  3. Change of myasthenia gravis composite (MGC) score at the second visit from the baseline visit (first visit)

    The value was calculated by the MGC score at the second visit minus the MGC score at the first visit (baseline).

    Measured: Baseline and 1 day before surgery (always at the 1 week after the first visit).

  4. Change of QMG score at the fourth visit from the baseline visit (first visit)

    The value was calculated by the QMG score at the fourth visit minus the QMG score at the first visit (baseline).

    Measured: Baseline and 3 weeks after the first visit.

  5. Change of MGC score at the fourth visit from the baseline visit (first visit)

    The value was calculated by the MGC score at the fourth visit minus the MGC score at the first visit (baseline).

    Measured: Baseline and 3 weeks after the first visit.

  6. Change of lgG-AChR level at the fourth visit from the baseline visit (first visit)

    The value (percent) was calculated by the lgG-AChR level at the fourth visit minus the lgG-AChR level score at the first visit (baseline).

    Measured: Baseline and 3 weeks after the first visit.

  7. The rate of postoperative myasthenia crisis

    Measured: From the day when trial begin to the day when the follow-up is ended. Up to 48 weeks.

  8. Postoperative hospital stay

    Measured: From the day when trial begin to the day when the follow-up is ended. Up to 48 weeks.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted24 Jan 2024
  2. Study start1 Feb 2024 (estimated)
  3. Primary completion30 Dec 2026 (estimated)
  4. Study completion30 Jun 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
30 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06221501
Sponsor's protocol ID
2023-374R
Status last verified
Jan 2024
First submitted
27 Dec 2023
Last updated
24 Jan 2024
Results posted
No
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • Wang S, Zhu M, Dong J, Zhang Y, Luo S, Jiang J, Cheng Z, Li Z, Yang W, Yu Y, Liu Z, Fan J, Xu X, Liu P, Zhang Z, Can F, Liang F, Jiang X, Tan L, Ding J. Perioperative Safety and Efficacy of Efgartigimod for Thymoma-Associated Myasthenia Gravis: A Prospective, Multicenter, Phase II Clinical Trial. J Thorac Oncol. 2025 Aug;20(8):1120-1130. doi: 10.1016/j.jtho.2025.04.014. Epub 2025 May 2.

    PubMed 40320172

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Efgartigimod Alfa in people with Myasthenia Gravis, run by Shanghai Zhongshan Hospital.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 8 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: opening soon, as of the sponsor's last update on 24 Jan 2024. Status can change between updates, so confirm with the study contact before making plans.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change of the activity of daily living (ADL) score at the fourth visit from the baseline visit (first visit) (time frame: Baseline and 3 weeks after the first visit.). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06221501, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Jan 2024.

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