Clinical Research Directory

Investigating an mRNA CAR T-cell Therapy, Known as Descartes-08, as a Potential Approach to Treat Myasthenia Gravis

The AURORA Study is evaluating the safety, tolerability, and efficacy of an investigational mRNA CAR T-cell therapy known as Descartes-08 in adults with acetylcholine receptor autoantibody -positive generalized myasthenia gravis. Part 1 of the study will last around 6 months. For eligible participants, Part 2 will last around 8 months.

A Long-Term Follow-Up Study for Participants Previously Treated With KYV-101

The purpose of this long-term follow-up (LTFU) study is to collect delayed adverse events (AEs) and understand the persistence of KYV-101 (autologous CAR T cell product; gene-modified product), in participants who have been administered KYV-101 (gene-modified product; autologous CAR T cell product). This LTFU protocol will be open to any participant who received at least one infusion of KYV-101 in a previous Kyverna sponsored clinical trial or Investigator Initiated Trial (IIT).

Efgartigimod in IVIG Dependent Myasthenia Gravis Patients

This study is an open label, single center, prospective, 26 weeks study with descriptive analysis where IVIG is replaced by efgartigimod therapy. MG-ADL and MGQOL evaluations will occur weekly throughout the study to week 26.

The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.

The planned project is an intervention study to assess the risk of falling after adaptation of an assistive gait devices in patients with the following neuromuscular diseases: Inclusion body myositis, myotonic dystrophy, limb girdle and facioscapulohumeral muscular dystrophies, Pompe disease, Lambert-Eaton syndrome, myasthenia gravis, spinal muscular atrophy, Guillain-Barré syndrome, chronic inflammatory demyelinating polyradiculoneuropathy, Friedreich's ataxia and hereditary motor and sensory neuropathy. The primary aim is to assess the risk of falling after a suitable assistive gait device has been provided with an adaptation phase through training. The data should help to improve the provision of aids for patients with neuromuscular diseases. This should have a positive effect on the risk of falling and thus improve quality of life and reduce mortality and morbidity. To achieve these goals, a one-week intervention with training sessions on handling, balance and coordination as well as fall prevention will becarried out after the patient has been fitted with a suitable assistive gait device. The interventions will be embedded in the inpatient rehabilitation programme. The functional gait and balance tests 'Timed Up and Go', '10 metre walk test', '6-minute walk test' and 'Dynamic Gait Index' will be recorded additionally. The Falls Efficacy Scale International questionnaire will be utilised to evaluate the risk of falling, while the Quebec User Evaluation of Satisfaction with Assistive Technology questionnaire will be employed to ascertain satisfaction with the assistive devices. The study is scheduled to run for a period of 14 days, during which participants will undergo three functional walking and balance tests. As part of the inpatient rehabilitation programme, participants will undergo a week-long period of rehabilitation without assistive technology, followed by a subsequent week of rehabilitation with adapted assistive technology.

Neuromuscular Complications of MEK Inhibitors: a French Case Series and a Systematic Review of the Literature

MEK inhibitors (trametinib, cobimetinib, selumetinib or binimetinib) have been used since 2016 to treat metastatic melanoma, by targeting the MAPK pathway Neuromuscular complications (neuropathy, myasthenia or myositis) have been reported in patients treated with MEK inhibitors. With the growing use of these new oncology therapies, neurologists, oncologists and other clinicians are likely to be increasingly confronted with MEK inhibitor-induced neuropathy, myasthenia or myositis. Yet, so far, these complications have only been documented in a few single case reports. Our aim was to characterize the neuromuscular complications associated with MEK inhibitors used either alone or in combination.

Characteristics and Prognostic Outcomes of Diaphragmatic Ultrasound in Myasthenia Gravis

The goal of this observational study is to learn about the characteristics and prognostic outcomes of diaphragmatic ultrasound in myasthenia gravis. The main question it aims to answer is: Does diaphragmatic ultrasound predict prognosis of myasthenia gravis in six months? Participants diagnosed as myasthenia gravis will take the diaphragm ultrasound examinations.

Blinatumomab in Refractory Myasthenia Gravis (BLINA-MG)

The goal of this clinical trial is to evaluating the efficacy and safety of Blinatumomab in the treatment of refractory myasthenia gravis and exploring the immunological mechanisms of Blinatumomab therapy in refractory myasthenia gravis. The main questions it aims to answer are: Will Blinatumomab improve the symptoms of participants? What medical problems do participants have when using Blinatumomab? Participants will: Continuous intravenous infusion of Blinatumomab for 5 days, pause for one week, and continue continuous intravenous infusion for 5 days Visit the clinic once every 1 month for checkups and tests Keep a diary of their symptoms

Myasthenia Gravis Foundation of America Global MG Patient Registry

The goal of this observational study is to learn about the experiences of people living with Myasthenia Gravis (MG) in the United States. The main questions it aims to answer are: * How and when are people with MG diagnosed? * What are the most common symptoms associated with MG? * What treatments are being used to treat MG? * What are the impacts of MG on activities of daily living, employment and quality of life? * What are the experiences with exacerbation, hospitalization and healthcare access for people with MG? Participants will answer a survey to enroll in the study, and be invited to fill out an update survey twice a year.