Lambert-Eaton Myasthenic Syndrome

Researchers are studying adult patients with generalized myasthenia gravis (gMG) who are positive for acetylcholine receptor (AChR) antibodies. The study aims to gather real-world evidence from Italian clinical practice on how efgartigimod alfa affects clinical outcomes and patient-reported experiences in those treated for gMG. This observational study will follow patients over a total duration of up to 23 months. The study involves collecting primary data at a baseline visit and follow-up visits at 6 and 12 months after baseline. Researchers will also gather secondary data from medical charts covering the 12 months before baseline, including details on MG-related therapies, exacerbations, hospitalizations, and the patients' medical history. The treatment with efgartigimod alfa is decided independently by the treating physician as part of routine care, not influenced by study participation. Participants will be monitored through clinical assessments and questionnaires measuring changes in MG-Activities of Daily Living (MG-ADL) scores and the percentage achieving minimal symptom expression (MSE) up to 12 months. Additional measures include quality of life, fatigue, anxiety and depression, sleep quality, and caregiver burden scores tracked over the same period. The study collects comprehensive data to understand disease experience and treatment impact, with safety and outcomes evaluated throughout the 23-month period.

Researchers are evaluating a specific oral corticosteroid (OCS) tapering schedule to reduce steroid use in adults with acetylcholine receptor positive generalized myasthenia gravis (gMG) who are treated with intravenous ravulizumab. This phase 4, multicenter, single-arm study aims to assess both the effectiveness and safety of gradually lowering OCS doses while managing gMG symptoms. Participants will follow a predefined oral corticosteroid tapering schedule during the study period until they complete the tapering process. They will continue receiving ravulizumab as part of their standard medical care, along with prednisone or prednisolone as needed. The treatment period lasts up to approximately 32 weeks, focusing on reducing or discontinuing OCS while monitoring disease status. Throughout the study, researchers will assess how many participants can reduce their daily OCS dose to 5 mg or less, or stop it entirely, without worsening gMG for at least four weeks. They will also track changes in quality of life, daily living activities, and glucocorticoid toxicity. Participants will undergo regular evaluations and safety monitoring during the study, which runs from March 2026 to August 2027.

Researchers are evaluating the safety and effectiveness of different drug regimens in people with myasthenia gravis, a condition affecting muscle strength. The study aims to find the best treatment options that reduce side effects and improve quality of life. It uses a master protocol to test multiple treatments in separate groups called intervention-specific appendixes (ISAs). The platform study includes a common screening period and ISA-specific screening, treatment, and safety follow-up periods. One ISA focuses on adding empasiprubart intravenous infusion to efgartigimod intravenous infusion for participants with generalized myasthenia gravis who have a partial response to efgartigimod. Each ISA has its own schedule and duration. Participants will undergo screening, receive assigned treatments, and be monitored for safety and treatment effects over up to approximately seven years, depending on the ISA. Researchers will assess safety, tolerability, and how well the treatments work for different myasthenia gravis subtypes during the study periods.

Researchers are evaluating the safety and therapeutic relevance of empasiprubart as an add-on therapy to efgartigimod in people with AChR-Ab seropositive generalized myasthenia gravis who have a partial clinical response to efgartigimod. This study is part of the ADAPT Forward platform, aiming to identify the best treatments to reduce side effects and improve quality of life in myasthenia gravis. It is an exploratory Phase 2a study sponsored by argenx. The study includes a screening period followed by a run-in period (part A) where participants receive intravenous efgartigimod. Eligible participants then enter an add-on period (part B) receiving both efgartigimod and empasiprubart intravenously. Those not eligible for part B continue to a safety follow-up period (part C) receiving only efgartigimod. The total study duration for each participant is up to approximately 54 weeks. Participants will be monitored for adverse events and serious adverse events up to 21 weeks, focusing on safety and tolerability. Researchers will also assess changes in Myasthenia Gravis Activities of Daily Living (MG-ADL) scores, Quantitative Myasthenia Gravis (QMG) scores, and other clinical measures over the study periods. Safety follow-up continues for participants not entering the add-on phase, ensuring ongoing evaluation throughout the study.

Researchers are evaluating how well the TetraGraph device measures muscle responses at two sites on the arm: the adductor pollicis (thumb) and the abductor digiti minimi (fifth digit). The study focuses on the timing of muscle response during the onset and recovery from neuromuscular blockade caused by rocuronium and reversed by sugammadex. This is important because residual muscle weakness after surgery can cause complications, and monitoring this accurately helps improve patient safety. In this observational study, patients undergoing elective surgeries receive neuromuscular blocking agents including rocuronium followed by reversal with sugammadex. Two TetraGraph devices are placed on different muscles to monitor electrical activity through electromyography (EMG). Measurements are taken frequently from the start of neuromuscular blockade until recovery, including every 20 seconds after sugammadex administration until the patient is extubated. Participants are monitored continuously during surgery with standard equipment and the TetraGraph devices, with recordings taken every 15 seconds during baseline and every 5 minutes during surgery. After surgery, assessments continue until muscle function recovers to a defined level. Researchers measure the rate of recovery from blockade and the incidence of residual muscle weakness one hour after extubation. The study aims to improve understanding of muscle site sensitivity and monitoring techniques in surgical patients.

Researchers are evaluating a new device that combines electromyography (EMG) and acceleromyography (AMG) signals to monitor neuromuscular block during surgery. This study aims to compare the new technology with the TOF Watch4 SX, a widely used AMG-based neuromuscular monitor, to assess its accuracy in measuring neuromuscular block and posttetanic count. The new device's reliability has been recognized in rehabilitation but has not yet been tested for neuromuscular monitoring in surgery. The study involves observing patients undergoing elective surgery requiring muscle relaxation. The two monitored methods are the existing TOF Watch4 SX AMG device and the new DELSYS4 Wireless surface EMG technology, which captures both EMG and AMG signals wirelessly. Researchers will compare the train-of-four (TOF) and posttetanic count (PTC) measurements obtained from these devices during the perioperative period. Participants will be assessed during surgery with neuromuscular monitoring devices. Data collected include TOF and PTC correlations between the two devices and analysis of signal sources to improve monitoring. The study is observational, with no treatment changes, and aims to validate this new monitoring method. Participants will be adults aged 18 to 90 undergoing elective surgery with muscle relaxation, with safety and monitoring overseen throughout the procedure.

Researchers are evaluating the use of two different methods to monitor recovery from deep neuromuscular blockade during surgery. This study focuses on comparing the traditional train-of-four ratio (cTOFR) and a modified train-of-four ratio (mTOFR) to see which better indicates safe extubation when reversing blockade with the drug sugammadex. The study is observational and addresses concerns about postoperative complications caused by residual neuromuscular block after anesthesia. Participants will undergo anesthesia involving rocuronium to induce muscle relaxation, followed by monitoring with the TOFscan device, which measures neuromuscular function. Sugammadex will be administered to reverse the blockade, and researchers will record classical and modified TOF ratios until full recovery is confirmed. The anesthesia process includes specific medication dosing, monitoring with multiple devices, and maintaining muscle relaxation at targeted levels during surgery. During the study, participants will be closely monitored with ECG, blood pressure, oxygen levels, brain activity, and acceleromyography. Neuromuscular function will be assessed repeatedly until reversal is complete. Data on drug use, TOF ratios, and recovery times will be collected. The primary outcome is the time measured up to 15 minutes, with a secondary outcome at 30 minutes. Participants will be observed until both TOF ratios reach 100%, ensuring complete neuromuscular recovery.

Researchers are investigating whether deep neuromuscular block (NMB) can improve recovery quality after laparoscopic hysterectomy compared to moderate NMB. This study focuses on women undergoing elective laparoscopic hysterectomy and evaluates surgical conditions, postoperative pain, and recovery quality. The trial is randomized and triple-blinded, aiming to clarify if deeper muscle relaxation during surgery benefits patients. Participants are divided into two groups: one receives low-pressure pneumoperitoneum (LPP) at 8-10 mmHg with deep NMB, and the other receives standard-pressure pneumoperitoneum (SPP) at 12-14 mmHg with moderate NMB. These interventions are applied during laparoscopic hysterectomy to assess their effect on surgical conditions and recovery. During the study, researchers will measure the quality of recovery one day after surgery using the QoR-15 score. They will also monitor postoperative pain, shoulder pain within 24 hours, nausea, vomiting, analgesic use, time to first flatus, and tracheal tube removal time. The study includes follow-up assessments to evaluate these outcomes and ensure participant safety throughout the process.

This research aims to evaluate how different levels of neuromuscular blockade affect postoperative pain in children aged over 2 months to under 7 years undergoing laparoscopic surgery with general anesthesia. The study focuses on understanding pain control in this young patient group during recovery after surgery. The trial is a prospective randomized controlled study led by Seoul National University Hospital. Participants will receive either a moderate or deep neuromuscular blockade during surgery. Moderate blockade is maintained by a continuous infusion of rocuronium at 0.2 mg/kg/hr to keep the Train-of-four count between 1 and 3, while deep blockade uses a higher infusion rate of 0.6 mg/kg/hr to achieve a Train-of-four count of 0. These two groups will be compared to assess differences in pain and recovery. Throughout the study, children will be closely monitored after surgery with assessments including the Analgesia Nociception Index 30 minutes after arrival in the recovery room, pain scales, emergence delirium scores, time to extubation, recovery room stay length, and medication use. Additional monitoring includes surgical and respiratory parameters during surgery, as well as any complications up to one month after discharge. The study will collect detailed data on pain and recovery outcomes over a 24-hour period following surgery.

Researchers are evaluating how sarcopenia and frailty influence the effects of rocuronium, a muscle relaxant used during general anesthesia, in older adults scheduled for elective surgery. This observational study focuses on patients aged 65 to 80 years and aims to understand how these conditions affect rocuronium's pharmacodynamics during surgical procedures. Participants are grouped based on whether they have frailty, sarcopenia, both conditions, or neither. All participants will undergo elective surgery with general anesthesia requiring endotracheal intubation, where rocuronium is used for neuromuscular blockade. Preoperative assessments of frailty and sarcopenia are conducted clinically and with ultrasound. Neuromuscular monitoring is planned throughout anesthesia to observe muscle response. During the study, researchers will measure the onset time of rocuronium, time to muscle recovery after sugammadex administration, and total rocuronium dose used. Participants will be monitored with quantitative neuromuscular tools during surgery. The study also observes recovery times related to muscle function during and after surgery. Participation lasts through the surgical procedure with assessments focused on anesthesia induction and recovery phases.