Treatment Trials

66 Clinical Trials for Various Conditions

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RECRUITING
A Study to Evaluate the Clinical Outcomes of Efgartigimod PH20 SC in Adults With New-onset Generalized Myasthenia Gravis (gMG)
Description

The main purpose of this study is to measure how well adults with new-onset gMG (which means they've had generalized disease signs and/or symptoms for less than 1 year) respond to treatment with efgartigimod PH20 SC. The study consists of a treatment period of 51 weeks. The study duration for each participant will be approximately 58 weeks.

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

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.

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

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.

RECRUITING
PK, PD, Safety, and Efficacy Study of Gefurulimab in Pediatric Patients With AChR+ Generalized Myasthenia Gravis
Description

The primary objective of this study is to assess the pharmacokinetics and pharmacodynamics of gefurulimab in pediatric participants with AChR+ gMG for the duration of the study.

COMPLETED
A Study to Evaluate the Safe and Effective Use of a Zilucoplan Auto-injector by Study Participants With Generalized Myasthenia Gravis
Description

The purpose of this study is to evaluate the effectiveness, safety and tolerability of zilucoplan auto-injector (ZLP-AI) self-administration.

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

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.

RECRUITING
A Study of Telitacicept for the Treatment of Generalized Myasthenia Gravis (RemeMG)
Description

The purpose of this study is to evaluate the efficacy and safety of telitacicept in the treatment of generalized myasthenia gravis.

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

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

RECRUITING
A Study of Efgartigimod PH20 SC in Children Between 2 and Less Than 18 Years of Age with Generalized Myasthenia Gravis
Description

The purpose of this study is to measure the pharmacokinetics (PK), pharmacodynamics (PD), safety, tolerability, and immunogenicity of efgartigimod PH20 SC in pediatric participants with gMG aged 2 to \<18 years. The primary goal is to confirm an appropriate dose of efgartigimod PH20 SC for pediatric patients using PK and PD results from this study. Participants will receive injections of efgartigimod PH20 SC and will be monitored for safety until the end of the study. At the end of the follow-up period, eligible participants may roll over to an open-label extension (OLE) study. The participants will be in the study for up to 14 weeks.

RECRUITING
RESET-MG: A Study to Evaluate the Safety and Efficacy of CABA-201 in Participants With Generalized Myasthenia Gravis
Description

RESET-MG: A Phase 1/2 Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Participants with Generalized Myasthenia Gravis

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

The primary purpose of this study is to measure the efficacy and safety of efgartigimod intravenously (IV) compared to placebo in participants with Acetylcholine Receptor Binding Antibody (AChR-Ab) seronegative Generalized Myasthenia Gravis (gMG). Other objectives are to assess long-term efficacy, safety, and tolerability of efgartigimod. Study will consist of: * Screening * Part A: participants will be randomized to receive either efgartigimod IV or placebo * Part B: participants completing part A will receive open-label efgartigimod IV

ACTIVE_NOT_RECRUITING
A Phase 2 Study to Evaluate DNTH103 in Adults With Generalized Myasthenia Gravis (MAGIC)
Description

The purpose of this Phase 2 study is to evaluate the safety, tolerability, pharmacometrics, and efficacy of DNTH103 in participants with generalized myasthenia gravis (gMG).

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

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

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

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).

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

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).

COMPLETED
A Phase 3, Open-label, Crossover Study to Evaluate Self-administration of Rozanolixizumab by Study Participants With Generalized Myasthenia Gravis (gMG)
Description

The purpose of this study is to evaluate the ability of study participants with generalized Myasthenia Gravis (gMG) to successfully self-administer rozanolixizumab after training in the self-administration technique using the syringe driver and manual push methods.

RECRUITING
Evaluation of Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of Ravulizumab Administered Intravenously in Pediatric Participants With Generalized Myasthenia Gravis (gMG)
Description

The primary purpose of this study is to characterize the pharmacokinetics and pharmacodynamics of treatment with ravulizumab intravenous infusion in pediatric participants with gMG.

ACTIVE_NOT_RECRUITING
Safety and Efficacy of ALXN1720 in Adults With Generalized Myasthenia Gravis
Description

The purpose of this study is to evaluate the safety and efficacy of ALXN1720 for the treatment of generalized MG (gMG) in adults with autoantibodies against acetylcholine receptor (AChR).

COMPLETED
An Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of Subcutaneous Zilucoplan in Participants With Generalized Myasthenia Gravis Who Were Previously Receiving Intravenous Complement Component 5 Inhibitors
Description

The purpose of the study is to evaluate the safety and tolerability of switching from intravenous (IV) complement component 5 (C5) inhibitors to subcutaneous (SC) Zilucoplan in study participants with generalized myasthenia gravis (gMG)

ACTIVE_NOT_RECRUITING
Phase 3 Study to Assess the Efficacy and Safety of Batoclimab as Induction and Maintenance Therapy in Adult Participants With Generalized Myasthenia Gravis
Description

The purpose of this 4-period study is to confirm the efficacy and safety of batoclimab in participants with gMG. In Period 1, participants will be randomized 1:1:1 to receive batoclimab 680 milligrams (mg) subcutaneously (SC) once a week (QW) or 340 mg SC QW or placebo. The primary efficacy endpoint will be assessed by change in the myasthenia gravis activities of daily living (MG- ADL) score in acetylcholine receptor antibody seropositive (AChRAb+) participants. In Period 2, participants previously treated with batoclimab will be re-randomized to stay on batoclimab (340 mg SC QW or 340 mg SC every two weeks) or receive placebo treatment. The secondary endpoint of maintenance of efficacy will be assessed by change in the MG- ADL score in AChRAb+ participants. Participants demonstrating a response to batoclimab during either Period 1 or 2 may enter the long-term extension (Period 3). Participants who complete Period 3 are eligible to participate in Period 4 (Optional Long-Term extension) according to their treatment assignment in Period 3.

ENROLLING_BY_INVITATION
Evaluating Long-term Safety of Efgartigimod Administered Intravenously and Efgartigimod PH20 Administered Subcutaneously in Children With Generalized Myasthenia Gravis
Description

The purpose of this trial is to evaluate the long-term safety of efgartigimod IV and efgartigimod PH20 SC administered to participants with gMG in the antecedent studies, ARGX-113-2006 and ARGX-113-2207, respectively.

RECRUITING
A Study of Nipocalimab in Children Aged 2 to Less Than 18 Years With Generalized Myasthenia Gravis
Description

The purpose of this study is to determine the effect of nipocalimab on total serum immunoglobulin G (IgG) in pediatric participants 2 to less than (\<) 18 years of age (globally) and 8 to \<18 years of age (for Unites Stated (US) sites only), the safety and tolerability of treatment with nipocalimab in children and adolescents and to evaluate the pharmacokinetics (PK) of nipocalimab in children and adolescents with generalized myasthenia gravis (gMG) who have an insufficient clinical response to ongoing, stable standard-of-care therapy.

TERMINATED
Study of ALXN2050 in Adult Participants With Generalized Myasthenia Gravis
Description

This study will evaluate the efficacy and safety of ALXN2050 (120 milligrams \[mg\], 180 mg) in participants with generalized myasthenia gravis (gMG). Safety will be monitored throughout the study.

TERMINATED
Efficacy and Safety of Tolebrutinib (SAR442168) Tablets in Adult Participants With Generalized Myasthenia Gravis
Description

This was a multicenter, randomized, double-blind, placebo-controlled, Phase 3 study to evaluate the efficacy and safety of tolebrutinib compared with placebo in adult participants aged 18 to 85 years old with moderate-to-severe generalized myasthenia gravis (gMG), who received Standard of Care (SoC). The double-blind (DB) treatment period of 26 weeks comprised of 7 site visits followed by a 2-year open label extension (OLE) period with quarterly visits. The efficacy of tolebrutinib versus placebo during the DB period was assessed by clinical evaluations, including scales based on physician examination or direct participant feedback i.e., patient reported outcomes (PROs). These evaluations continued during the OLE to measure long term efficacy and safety.

RECRUITING
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
Description

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 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)

WITHDRAWN
Study of ALXN1830 Administered Subcutaneously in Adults With Generalized Myasthenia Gravis
Description

This study will evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics (PD), immunogenicity, and efficacy of subcutaneous (SC) ALXN1830 in adults with generalized myasthenia gravis (gMG).

ACTIVE_NOT_RECRUITING
An Open-label Study to Investigate the Clinical Efficacy of Different Dosing Regimens of Efgartigimod IV in Patients With Generalized Myasthenia Gravis
Description

The purpose of this open-label study is to investigate the efficacy, safety, and tolerability of a continuous regimen of efgartigimod compared with a cyclic regimen in participants with Generalized Myasthenia Gravis (gMG). Study details include: The study duration will be up to 138 weeks (including screening and a safety follow-up of up to 9 weeks) * Part A (regimen comparison period) - 21 weeks * Part B (extension period) - up to 105 weeks The visit frequency, including virtual visits, will be weekly through Week 21 and every 5 weeks for the remainder of the study.

TERMINATED
A Study To Evaluate Efficacy, Safety, Pharmacokinetics, And Pharmacodynamics Of Satralizumab In Patients With Generalized Myasthenia Gravis
Description

This study will evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of satralizumab compared with placebo in participants with generalized myasthenia gravis (gMG).

RECRUITING
A Study of Nipocalimab Administered to Adults With Generalized Myasthenia Gravis
Description

The purpose of this study is to evaluate the efficacy and safety of nipocalimab compared to placebo in participants with generalized myasthenia gravis (gMG).

RECRUITING
Evaluating the Pharmacokinetics, Pharmacodynamics, and Safety of Efgartigimod Administered Intravenously in Children with Generalized Myasthenia Gravis
Description

The purpose of this trial is to investigate the PK, PD, safety, and activity of efgartigimod IV in children and adolescents aged from 2 to less than 18 years of age with gMG. Trial details include: * The maximum trial duration for each individual participant will be approximately 28 weeks * The treatment duration will be 8 weeks for the dose-confirmatory part (Part A) and 18 weeks for the treatment response-confirmatory part (Part B)