Perioperative Efgartigimod for Thymoma and Myasthenia Gravis
Safety and Efficacy of Perioperative Efgartigimod Combined With Thymectomy for Thymoma and Myasthenia Gravis: A Prospective, Single-arm Study
Study Overview
Status
Status
Conditions
Conditions
Intervention / Treatment
Intervention / Treatment
Study Type
Study Type
Enrollment (Estimated)
Enrollment
Phase
Phase
- Phase 2
Participation Criteria
Eligibility Criteria
Eligibility Criteria
Ages Eligible for Study
- Adult
- Older Adult
Accepts Healthy Volunteers
Description
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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;
- 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;
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
What is the study measuring?
Primary Outcome Measures
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
|---|---|---|
|
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.
|
The value was calculated by the ADL score at the fourth visit minus the ADL score at the first visit (baseline).
|
Baseline and 3 weeks after the first visit.
|
Secondary Outcome Measures
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
|---|---|---|
|
Change of the ADL score at the second visit from the baseline visit (first visit)
Time Frame: Baseline and 1 day before surgery (always at the 1 week after the first visit).
|
The value was calculated by the ADL score at the second visit minus the ADL score at the first visit (baseline).
|
Baseline and 1 day before surgery (always at the 1 week after the first visit).
|
|
Change of quantitative myasthenia gravis (QMG) score at the second visit from the baseline visit (first visit)
Time Frame: Baseline and 1 day before surgery (always at the 1 week after the first visit).
|
The value was calculated by the QMG score at the second visit minus the QMG score at the first visit (baseline).
|
Baseline and 1 day before surgery (always at the 1 week after the first visit).
|
|
Change of myasthenia gravis composite (MGC) score at the second visit from the baseline visit (first visit)
Time Frame: Baseline and 1 day before surgery (always at the 1 week after the first visit).
|
The value was calculated by the MGC score at the second visit minus the MGC score at the first visit (baseline).
|
Baseline and 1 day before surgery (always at the 1 week after the first visit).
|
|
Change of QMG score at the fourth visit from the baseline visit (first visit)
Time Frame: Baseline and 3 weeks after the first visit.
|
The value was calculated by the QMG score at the fourth visit minus the QMG score at the first visit (baseline).
|
Baseline and 3 weeks after the first visit.
|
|
Change of MGC score at the fourth visit from the baseline visit (first visit)
Time Frame: Baseline and 3 weeks after the first visit.
|
The value was calculated by the MGC score at the fourth visit minus the MGC score at the first visit (baseline).
|
Baseline and 3 weeks after the first visit.
|
|
Change of lgG-AChR level at the fourth visit from the baseline visit (first visit)
Time Frame: Baseline and 3 weeks after the 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).
|
Baseline and 3 weeks after the first visit.
|
|
The rate of postoperative myasthenia crisis
Time Frame: From the day when trial begin to the day when the follow-up is ended. Up to 48 weeks.
|
From the day when trial begin to the day when the follow-up is ended. Up to 48 weeks.
|
|
|
Postoperative hospital stay
Time Frame: From the day when trial begin to the day when the follow-up is ended. Up to 48 weeks.
|
From the day when trial begin to the day when the follow-up is ended. Up to 48 weeks.
|
Collaborators and Investigators
Sponsor
Sponsor
Study record dates
Study Major Dates
Study Start (Estimated)
Study Start
Primary Completion (Estimated)
Primary Completion
Study Completion (Estimated)
Study Completion
Study Registration Dates
First Submitted
First Submitted
First Submitted That Met QC Criteria
First Submitted That Met QC Criteria
First Posted (Actual)
First Posted
Study Record Updates
Last Update Posted (Actual)
Last Update Posted
Last Update Submitted That Met QC Criteria
Last Update Submitted That Met QC Criteria
Last Verified
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Pathologic Processes
- Nervous System Diseases
- Immune System Diseases
- Neoplasms by Histologic Type
- Neoplasms
- Lymphatic Diseases
- Autoimmune Diseases of the Nervous System
- Autoimmune Diseases
- Neoplasms by Site
- Neurologic Manifestations
- Musculoskeletal Diseases
- Thoracic Neoplasms
- Muscular Diseases
- Neuromuscular Diseases
- Neurodegenerative Diseases
- Neuromuscular Manifestations
- Nervous System Neoplasms
- Neoplasms, Complex and Mixed
- Paraneoplastic Syndromes, Nervous System
- Paraneoplastic Syndromes
- Neuromuscular Junction Diseases
- Thymus Neoplasms
- Muscle Weakness
- Thymoma
- Myasthenia Gravis
Other Study ID Numbers
Other Study ID Numbers
- 2023-374R
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
product manufactured in and exported from the U.S.
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.