Clinical Research Directory

Talquetamab in Patients With Refractory Generalized Myasthenia Gravis

Myasthenia Gravis (MG) is a chronic autoimmune disease mediated by pathogenic antibodies. Approximately 10%-15% of patients present with refractory status, defined as having an inadequate response to existing therapies or an inability to tolerate the side effects of the medication, highlighting an urgent need for the development of more targeted innovative therapies. Talquetamab is a bispecific antibody that targets G Protein-Coupled Receptor Class C Group 5 Member D (GPRC5D) and the Cluster of Differentiation 3 (CD3) molecule on the surface of T cells, thereby inducing T cells to precisely eliminate GPRC5D-positive cells. This study will conduct an exploratory case series to investigate the efficacy and safety of Talquetamab in refractory MG.

The Effectiveness of an ETP Programme in Myasthenia Gravis: a Proof-of-concept Study.

CONTEXTE. Myasthenia gravis is a rare neuromuscular junction disorder affecting one in five thousand people (ORPHA 589). It is a chronic condition that progresses in episodes. Its severity varies, ranging from the invisible disability of fatigue to respiratory distress requiring intensive care. Currently, 270 patients with myasthenia gravis are being treated at Grenoble University Hospital in the Reference Centre for Rare Neuromuscular Diseases. The impact of this disease on quality of life is significant, and medication alone is not sufficient. However, to date, there are virtually no psychosocial interventions for patients with myasthenia gravis and no studies evaluating their effectiveness. In this context, the Cognitive Behavioural Stress Management (CBSM) programme is one of the stress management programmes applied to chronic health conditions whose effectiveness has already been demonstrated, particularly in terms of treatment adherence, quality of life, patients' coping strategies in the face of a chronic illness, and the outcomes of medical treatments (Antoni, 2003; Antoni et al., 2002, 2006). PRIMARY OBJECTIVE. Evaluation of the adaptation of an existing patient education programme, 'Living Better with Myasthenia', following the inclusion of an eight-session CBSM-based stress management module for adult patients living with myasthenia. METHODOLOGY (brief). Adaptation of the CBSM programme to the specific characteristics of myasthenia gravis: 1. Focus groups: exploration of beliefs associated with stress in myasthenia gravis. 2. DELPHI group: validation of the adaptation of the ETP programme 'Living better with myasthenia gravis' implemented at CHUGA following the inclusion of a stress management module (CBSM). Assessment of the feasibility of the MY-EDUC programme among groups of patients with myasthenia gravis. PRIMARY OUTCOME MEASURE. Comparison of levels of anxiety, depression (HADS), perceived stress (PSS) and quality of life (SF-36) as self-reported by patients enrolled in the programme before and after the MY-EDUC intervention. RESEARCH PROCEDURE. 1.1. FOCUS GROUPS. Expert patients and partner patients. They will be contacted via the list maintained by the rare disease healthcare network. 'General public' patients. A call for participants will be displayed in the CHUGA waiting room and circulated by partner organisations to their members. The study will be presented to expert and partner patients, as well as to "walk-in" patients in the focus groups, using the contact details on the "rare disease healthcare network" lists and via posters. An information leaflet outlining the study and its objectives will be provided to them. 1.2. FEASIBILITY AND ACCEPTABILITY STUDY OF THE MY-EDUC PROGRAMME. 1.2.1. Participant inclusion. Patient eligibility. The assessment of patient eligibility will be carried out by a specialist doctor from the CHUGA Rare Disease Centre of Excellence during a routine consultation with the patient (routine care). Participant inclusion. Once patient eligibility has been verified, the investigator from the CHUGA Rare Disease Centre, or a person to whom they have delegated this task, will contact the patient to invite them to participate in the MY-EDUC study. They will provide a verbal explanation of the study and hand over an information sheet during a follow-up consultation with the patient as part of routine care. If the patient volunteers to participate, they will give their consent to participate. The patient's consent will be recorded in the medical record. 1.2.2. Baseline assessment (T0). In the week leading up to the first session of the MY-EDUC programme, participants will complete the baseline assessment online (using the LimeSurvey platform - secure servers at the University of Grenoble Alpes) with questionnaires presented in a randomised order. 1.2.3. Assessment during the intervention (T1). Based on repeated ecological measurements (Shiffman et al., 2008) via the study participants' mobile phones (two measurement points, before and after each session, randomly scheduled at least 36 hours apart, i.e. 16 measurements) using the free PielsSurvey software. 1.2.4. Final assessments (T2 and T3). Within one week of the final session of the MY-EDUC programme (T2: week 10 after T0), participants will complete a set of questionnaires electronically (using the LimeSurvey platform - secure servers at the University of Grenoble Alpes), with the validated questionnaires presented in a randomised order.

Clinical Study of EVM18001 in the Treatment of Refractory Autoimmune Diseases

A FIH, single arm, open-label, Investigator Initiated Trial (IIT) study to evaluate the safety and tolerability of EVM18001 in the treatment of active refractory autoimmune diseases (SLE, MG, and SSc), and determine the recommended dose for subsequent treatment. At the same time, the PK/PD characteristics of EVM18001 will be evaluated, preliminary efficacy will be observed, and related biomarkers and immunogenicity will be explored.

Ripertamab for the Treatment of Myasthenia Gravis

The goal of this clinical trial is to learn if ripertamab works to treat myasthenia gravis. It will also learn about the safety of ripertamab. The main questions it aims to answer are:Will ripertamab improve the symptoms of participants?What medical problems do participants have when using ripertamab?Researchers will compare ripertamab to a placebo (a look-alike substance that contains no drug) to see if ripertamab works to treat chronic inflammatory demyelinating polyneuropathy.Participants will:A single intravenous infusion of ripertamab.Visit the clinic for checkups and tests during W1, W2, W4, W8, W12. Keep a diary of their symptoms and the number of times they undergo rescue therapy.

CD19/BCMA-Targeted UCAR-T for Patients With Neurological Autoimmune Diseases

This single-arm, open-label investigator-initiated trial (IIT) evaluates the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of RD06-05 in patients with autoimmune neurological diseases, including Multiple Sclerosis (MS), Myasthenia Gravis (MG), Chronic Inflammatory Demyelinating Polyneuropathy (CIDP), Autoimmune Encephalitis (AE), and other B-cell-mediated neuroautoimmune disorders. In this study, the dose of CAR-T cells administered is 10×10⁶ CAR⁺T cells per kilogram of body weight. Investigators may decide whether to add other dose groups based on the subjects' safety data, pharmacokinetic (PK) data, pharmacodynamic (PD) data, and preliminary efficacy data. For each indication, 6 to 9 subjects will be enrolled, with a total of 24 to 36 subjects planned for enrollment in the entire study.