Clinical Research Directory

Efficacy and Safety of Ravulizumab in Chinese Adults Participants With Generalized Myasthenia Gravis (gMG)

The primary purpose of this study is to evaluate the efficacy, safety, pharmacokinetics, pharmacodynamics, and immunogenicity of ravulizumab in Chinese adult participants with Acetylcholine receptor (AChR) + Generalized Myasthenia Gravis (gMG).

A Study of Rozanolixizumab in Pediatric Study Participants With Moderate to Severe Generalized Myasthenia Gravis

The purpose of the study is to assess the safety and tolerability of subcutaneous (sc) administration of rozanolixizumab in pediatric participants aged ≥2 to \<18 years with generalized Myasthenia Gravis (gMG).

Study to Assess Safety, Efficacy, and Cellular Kinetics of YTB323 in Generalized Myasthenia Gravis

This is a phase I/II study to assess safety, efficacy, and cellular kinetics of YTB323 in participants with treatment-resistant generalized myasthenia gravis. YTB323 is a Biological CAR-T cell therapy.

Clinical Study of Cizutamig in Generalized Myasthenia Gravis (gMG)

The purpose of this study is to assess the safety, tolerability, PK, PD, immunogenicity, and preliminary clinical activity of Cizutamig in patients with Generalized Myasthenia Gravis.

Monitor the Evolution of Myasthenia Gravis Symptoms in Real-life in Patients With Anti-AChR and Anti-MUSK Generalised Myasthenia Gravis in Therapy With RYSTIGGO® (Rozanolixizumab)

Generalized Myasthenia Gravis (gMG) is a rare autoimmune disease (a disease in which the body attacks its own tissues) that causes muscle weakness and significant fatigue. Current treatments (corticosteroids, plasma exchange, intravenous immunoglobulin infusions) improve symptoms in many patients. However, many continue to suffer from fatigue and fatigability that are not well measured by standard tools. Moreover, these treatments can cause significant long-term side effects, reducing quality of life. New treatments such as Rozanolixizumab (ROZ) are now available. They act rapidly and are well tolerated, allowing better symptom control while reducing the risks associated with conventional treatments. To properly evaluate these new treatments, it is essential to understand patients' perspectives on their effectiveness. The scales used by physicians do not always capture all the symptoms experienced by patients, particularly fatigability. This is why a new tool has been developed: the MG symptoms PRO. This questionnaire allows patients to assess their own symptoms (fatigue, weakness of the eyes, mouth, breathing, muscle fatigability) in detail. This research aims to better understand the effectiveness of treatments from the patients' perspective in order to improve their care. The goal of the study is to evaluate the impact of Rozanolixizumab administration in real-world practice through the MG symptoms PRO questionnaire. This is an observational study, meaning that the medication is prescribed by the physician according to current regulations, and the study simply collects routine medical data during your follow-up, over a period of approximately 9 months.

A Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety and Tolerability of Inebilizumab in Children With Generalized Myasthenia Gravis (gMG)

The primary objectives of this study are to characterize the pharmacokinetics (PK) and pharmacodynamics (PD) of inebilizumab administered in pediatric participants with gMG, and to assess the safety and tolerability of inebilizumab administered in pediatric participants with gMG.

ADAPT Forward - Master Protocol of a Platform Study to Evaluate the Safety and Efficacy of Multiple Regimens in Participants With Myasthenia Gravis

ADAPT Forward is a platform study with the aim to look at how safe different drugs are and how well they work for people with myasthenia gravis. The goal is to find the best therapeutic approach to reduce patients' side effects and improve their quality of life.

ADAPT Forward 1 - ISA1 - a Study to Evaluate Empasiprubart IV as add-on Therapy to Efgartigimod IV in Participants With AChR-Ab Seropositive Generalized Myasthenia Gravis With a Partial Clinical Response to Efgartigimod

This study is part of the ADAPT Forward platform study (NCT07294170). ADAPT Forward is a platform study with the aim to look at how safe different drugs are and how well they work for people with myasthenia gravis. The goal is to find the best therapeutic approach to reduce patients' side effects and improve their quality of life. The aim of this ISA1 is to evaluate the safety and therapeutic relevance of empasiprubart as add-on therapy to efgartigimod in participants with AChR-Ab seropositive generalized myasthenia gravis. The ADAPT Forward master protocol is registered on https://clinicaltrials.gov/study/NCT07294170 More information can be found here: https://clinicaltrials.argenx.com/adaptforward1

A Study to Evaluate Rozanolixizumab in Study Participants With Generalized Myasthenia Gravis

The purpose of this study is to assess the safety, tolerability and efficacy of additional 6-week treatment cycles with rozanolixizumab in study participants with generalized myasthenia gravis (gMG).

A Study to Test Efficacy and Safety of Rozanolixizumab in Adult Patients With Generalized Myasthenia Gravis

The purpose of the MycarinGstudy is to demonstrate the clinical efficacy and to assess safety and tolerability of rozanolixizumab in patients with generalized myasthenia gravis (MG).

An Extension Study Evaluating 6-week Treatment Cycles of Rozanolixizumab in Pediatric Study Participants With Generalized Myasthenia Gravis

The purpose of the study is to assess the long term safety and tolerability of additional 6-week treatment cycles with rozanolixizumab in pediatric participants with generalized Myasthenia Gravis (gMG) aged ≥2 at the time of the Screening Visit of MG0006.

A Clinical Study of B007 in the Treatment of Generalized Myasthenia Gravis.

The purpose of this study is to evaluate the efficacy and safety of B007 in subjects with generalized myasthenia gravis.

Open-Label Extension of Zilucoplan in Subjects With Generalized Myasthenia Gravis

The RAISE-XT study is an open-label extension study to evaluate the long-term efficacy, safety, and tolerability of zilucoplan in subjects with gMG who have previously participated in a qualifying Ra Pharmaceuticals sponsored zilucoplan study.

Efficacy and Safety of a New Formulation of Oral Cladribine Compared With Placebo in Participants With Generalized Myasthenia Gravis (MyClad)

The purpose of this clinical study is to determine the efficacy and safety of a new oral cladribine formulation in participants with Generalized Myasthenia Gravis (gMG) in comparison to placebo. It will also investigate the sustained efficacy, the need for retreatment, and the long-term safety of oral cladribine in gMG. An additional component is included to characterize the Pharmacokinetics (PK) of the new cladribine formulation in gMG participants. This study is divided into 3 periods: the double-blind placebo control (DBPC) pivotal period, and 2 extensions, the blinded extension (BE) and the retreatment (RT) period. Furthermore, in trial interviews will be conducted as a sub-study to MyClad with a sub-set of participants to gain an in depth understanding of the participant cladribine treatment and study experience.

A Phase III Study to Investigate Efficacy, Safety and Tolerability of Iptacopan Compared With Placebo in Participants Aged 18 to 85 Years With gMG.

The study is a randomized, double-blind, placebo-controlled, multicenter, Phase III study, to evaluate efficacy, safety and tolerability of iptacopan in patients with AChR+ gMG who are on stable SOC treatment. Participants who meet the eligibility criteria will be randomized in a ratio of 1:1, to receive either iptacopan or matching placebo, for 6 months (180 days) while continuing on a stable SOC treatment. The randomization will be stratified based on region.

Efficacy and Safety of HN2301 in Patients With Generalized Myasthenia Gravis (MG)

This is an open label, single arm study, to evaluate safety, tolerability and preliminary efficacy of HN2301 in patients with Generalized Myasthenia Gravis.

A Phase 3 Study to Evaluate the Efficacy and Safety of Efgartigimod IV in Patients With Acetylcholine Receptor Binding Antibody Seronegative Generalized Myasthenia Gravis

The primary purpose of this study is to measure the efficacy and safety of efgartigimod IV compared to placebo in participants with Acetylcholine Receptor Binding Antibody (AChR-Ab) seronegative Generalized Myasthenia Gravis (gMG). The study consists of a Part A where participants will be randomized to receive either efgartigimod IV or placebo IV and a Part B where participants completing part A will receive open-label efgartigimod IV. Participants will be in the study for up to (approximately) 3.5 years.

A Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis

The purpose of this study is to assess the pharmacokinetics, pharmacodynamics, safety, tolerability, immunogenicity and activity of zilucoplan (ZLP) in pediatric study participants with generalized myasthenia gravis (gMG).

An Open-label Extension Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis

The purpose of this study is to assess the long-term safety and tolerability of an additional 52 weeks of Zilucoplan treatment administered by subcutaneous injection once daily in pediatric study participants

Study to Assess the Efficacy and Safety of IMVT-1402 in Participants With Mild to Severe Generalized Myasthenia Gravis

The purpose of the study is to assess the efficacy, safety and tolerability of IMVT-1402 in adult participants with mild to severe generalized myasthenia gravis.

A Study to Investigate the Efficacy, Safety and Tolerability of Remibrutinib Versus Placebo in Adult Patients With Generalized Myasthenia Gravis

A study to evaluate the efficacy, safety and tolerability of Remibrutinib versus placebo in adult patients with Generalized Myasthenia Gravis who are on stable, standard-of-care (SOC) treatment.

KYSA-6: A Study of Anti-CD19 Chimeric Antigen Receptor T-Cell Therapy, in Patients With Generalized Myasthenia Gravis

A Study of the Anti-CD 19 Chimeric Antigen Receptor T Cell Therapy for Patients with Myasthenia Gravis

Universal STAR-T Cell Injection in Generalized Myasthenia Gravis

This is a Phase I, single-arm, open-label, dose-escalation and dose-expansion study. This is an exploratory clinical study of universal STAR-T cell injection in patients with refractory generalized myasthenia gravis (GMG). Approximately 10-24 participants aged 18-65 years (inclusive) with the condition are planned to be enrolled. The primary objective is to evaluate the safety, tolerability, preliminary efficacy, pharmacokinetic/pharmacodynamic (PK/PD) profile, and immunogenicity of universal STAR-T cell injection. The starting dose is 1.5E6 STAR+ T cells/kg, administered as a single intravenous infusion.Based on safety, PK results, and preliminary efficacy data obtained from the initial dose cohorts, a recommended dose will be selected for subsequent dose-expansion studies to further systematically evaluate the safety and efficacy of universal STAR-T cell injection. This study includes the screening period (from D-28 to D-6), the pre-clearance treatment and rest observation period (from D-5 to D-1), the cell infusion and main study endpoint observation period (from D0 to W12 after infusion), and the follow-up period (from W12 after infusion to W104). The study is being conducted at Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology.

A Study to Test How Safe Pozelimab and Cemdisiran Combination Therapy and Cemdisiran Alone Are and How Well They Work in Adult Patients With Generalized Myasthenia Gravis

This study is researching the experimental drugs called pozelimab and cemdisiran, and cemdisiran monotherapy. The study is focused on patients with generalized Myasthenia Gravis (gMG). Myasthenia gravis is a disease that causes weakness and fatigue in muscles in the body because the nerves and muscles are not communicating properly. The aim of the study is to see how effective pozelimab and cemdisiran are when used in combination and when pozelimab and cemdisiran are used alone for patients with gMG. The study is looking at several other research questions, including: * What side effects may happen from taking the study drugs * How the study drugs work inside the body * How much of the study drugs are in the blood at different times * Whether the body makes antibodies against pozelimab and cemdisiran (which could make the drugs less effective or could lead to side effects)