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 treatments in people with myasthenia gravis, a condition that affects muscle strength. This platform study uses a single master protocol to test multiple treatment regimens in separate groups called intervention-specific appendixes ISAs. The goal is to find the best treatments that reduce side effects and improve quality of life for patients with this disease. The study includes various treatment groups, such as those receiving intravenous infusions of efgartigimod, empasiprubart, or placebo, and subcutaneous administration of efgartigimod PH20 via pre-filled syringe. Each ISA has its own screening, treatment, and safety follow-up periods, which vary in length and design. Two ISAs are included one evaluating empasiprubart as add-on therapy to efgartigimod in people with partial responses, and another assessing empasiprubart monotherapy. Participants are involved in screening and treatment phases, with assessments of safety, tolerability, and effectiveness lasting up to about seven years depending on the ISA. Researchers will monitor clinical symptoms, side effects, and quality of life during and after treatment. The study aims to gather detailed information on how well these drug regimens work and their impact on patients living with myasthenia gravis.

This research aims to evaluate the safety and therapeutic importance of empasiprubart as an add-on treatment to efgartigimod in adults with AChR-Ab seropositive generalized myasthenia gravis who have a partial clinical response to efgartigimod. It is part of the ADAPT Forward platform study, which looks at the safety and effectiveness of different drugs to find the best ways to reduce side effects and improve quality of life for people with myasthenia gravis. Participants first complete screening and then enter a run-in period part A receiving efgartigimod intravenously. Those eligible move on to the add-on period part B, receiving both efgartigimod and empasiprubart intravenously. Participants not eligible for part B continue to a safety follow-up period part C where they receive efgartigimod only. The total study duration is approximately 54 weeks per participant. Throughout the study, participants undergo assessments including monitoring for adverse events, measuring changes in MG-ADL and QMG scores, and evaluating clinical responses. Researchers track safety and efficacy up to 21 weeks during parts A and B. Participants have scheduled visits for treatment and safety evaluations, with ongoing observation through the safety follow-up period to monitor treatment effects and side effects.

Researchers are evaluating empasiprubart IV monotherapy in adults with AChR-Ab seropositive generalized myasthenia gravis gMG as part of the ADAPT Forward platform study. This Phase 2a trial aims to assess the safety, tolerability, and effectiveness of empasiprubart compared to placebo to find the best treatment approach that reduces side effects and improves quality of life for people with this condition. Eligible participants will be randomly assigned to receive either empasiprubart intravenously or a placebo during the double-blinded treatment period lasting up to 12 weeks. After this, all participants will receive open-label subcutaneous efgartigimod PH20 via pre-filled syringe during the safety follow-up period. The total study duration for each participant is approximately 45 weeks. During the study, participants will be monitored for adverse events and serious adverse events. Researchers will assess changes in muscle strength and daily living activities using scales like MG-ADL and QMG scores at various timepoints. Participants will have regular visits during the treatment and follow-up periods for assessments, safety monitoring, and to track treatment effects over time.

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 devices 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 comparing two electromyography EMG devices, the TetraGraph and TwitchView, to monitor deep neuromuscular blockade during elective laparoscopic, robotic, or thoracic video-assisted surgeries. The study aims to evaluate how well each device detects spontaneous diaphragmatic movements and breath-initiation efforts, which indicate the depth of muscle relaxation during surgery. This intraoperative device comparison trial uses a randomized, double-masked design to assess monitoring performance under standard anesthesia care. Participants will have both devices placed on opposite hands, with the dominant hand device guiding neuromuscular blockade management. The devices measure muscle responses using Train-of-Four and post-tetanic count methods continuously during surgery. Standard anesthesia protocols, including rocuronium administration and sugammadex reversal, are maintained. Data collected include device outputs, neuromuscular blocking agent dosing, physiological parameters, and clinical observations. After surgery, providers will complete surveys to assess device usability and workflow integration. Participants undergo assessments during surgery, including neuromuscular function monitoring at various time points from baseline through emergence from anesthesia. Researchers will track diaphragmatic movements, breath efforts, intubation difficulty, vocal cord visibility and movement, recovery times, and usability scores. All data are gathered without altering standard patient care. The study runs from February 2026 to August 2028, with participant involvement limited to the surgery and immediate postoperative period.

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 mgkghr to keep the Train-of-four count between 1 and 3, while deep blockade uses a higher infusion rate of 0.6 mgkghr 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 the effects of whole-body electrical muscle stimulation WB-EMS exercise on neuromuscular and physical function in adults with neuromuscular diseases NMD such as amyotrophic lateral sclerosis, spinal muscular atrophy, and myasthenia gravis. People with NMD often face challenges with traditional exercise because of impaired voluntary muscle activation, which can lead to inactivity and worsening health. WB-EMS bypasses these limits by directly stimulating muscle contractions using electrical currents, potentially offering a new way to exercise safely. The intervention uses the Katalyst system, an FDA-cleared device applied off-label, which delivers electrical stimulation to major muscle groups during guided movements. Participants wear a special suit with electrodes connected to an impulse pack controlled via an iPad app. They complete 20-minute supervised exercise sessions 1-2 times per week for 4-8 weeks, following Level 1 strength training videos. Each exercise involves repetitions with stimulation on and off periods, and stimulation levels are adjusted based on participant responses and comfort. Participants will be monitored carefully throughout the study, with evaluations before and after the intervention including neural excitability using transcranial magnetic stimulation, motor unit behavior via electromyography and nerve conduction studies, and functional tests like walking, balance, and strength assessments. Patient-reported outcomes on fatigue, pain, and quality of life will also be collected. Safety monitoring is strict, and the study will provide preliminary data on the feasibility and impact of WB-EMS exercise for adults with NMD.