Clinical trials

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 ISA2 is to investigate the effects of empasiprubart in participants with AChR-Ab seropositive generalized myasthenia gravis (gMG). The ADAPT Forward master protocol is registered on https://clinicaltrials.gov/study/NCT07294170

This study aims to find out how much adimanebart gets into the blood circulation of healthy adults. Researchers will measure the amount of adimanebart in blood over time to learn how it moves through the body. The drug's safety will also be assessed. The participants will be assigned to one of 3 parts of the study: part 1 investigates the absolute bioavailability; part 2 investigates the effect of different body weights on adimanebart in blood after subcutaneous (SC) dosing; part 3 investigates formulation adaptations. Participants will be in the study for approximately up to 24 weeks.

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.

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

The main purpose of this study is to confirm the correct dose of efgartigimod IV for treating patients aged 12 to younger than 18 years with chronic immune thrombocytopenia (ITP). The study consists of a double-blinded treatment period (DBTP) in which the participants will be randomized in a 2:1 ratio to receive either efgartigimod IV or placebo IV. At the end of the treatment period (up to 24 weeks), all participants will receive efgartigimod IV during the first year open-label treatment period (OLTP1). At the end of the first OLTP1, participants may begin a second year (OLTP2). After the OLTP2, the participants will enter a follow-up period (approximately 8 weeks) while off study drug. The participants will be in the study for up to 138 weeks. More information can be found here: https://clinicaltrials.argenx.com/advancejunior

The main purpose of this study is to demonstrate the efficacy and safety of empasiprubart in adults with CIDP. The study consists of a part A where participants will either receive empasiprubart or placebo for 24 weeks (6 months). Following part A, participants will enter part B in which all participants will receive empasiprubart for 96 weeks (24 months). More information can be found here: https://clinicaltrials.argenx.com/emnergize

The main purpose of this study is to compare empasiprubart and IVIg for treating people with CIDP. This study consists of a Part A where participants will either receive empasiprubart and a placebo resembling IVIg, or IVIg and a placebo resembling empasiprubart for 24 weeks (6 months). Following Part A, participants will enter Part B in which all participants will receive empasiprubart for 96 weeks (24 months). More information can be found here: https://clinicaltrials.argenx.com/emvigorate

Participants will attend up to 4 study visits to collect clinical assessments. The assessments will evaluate participants' symptoms and quality of life to understand disease activity in patients with CMS due to mutations in DOK7, MUSK, AGRN, or LRP4. More information can be found here: https://clinicaltrials.argenx.com/cms

The main purpose of the study is to determine the correct dose of empasiprubart in adolescent participants. It also aims to evaluate if empasiprubart may work and how safe it is for the use in children living with CIDP. The study consists of an open label treatment phase where participants will receive empasiprubart for up to 27 months approximately. After the final dose of empasiprubart, participants will enter a safety follow-up period for up to 14 months approximately. The overall study duration for each participant is up to 43 months. More information can be found here: clinicaltrials.argenx.com/emlight

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.

This study aims to see how the body reacts to empasiprubart, administered using an autoinjector (AI). The study will also look at other effects of empasiprubart, how it works in the body, and if it is safe. The study consists of 2 parts: parts A and B. In part A, eligible participants will be randomized to receive empasiprubart SC AI via abdomen, empasiprubart SC AI via thigh, or empasiprubart IV (intravenously). In part B, eligible participants will be randomized to receive empasiprubart SC AI via abdomen or empasiprubart IV. Participants from part A will be in the study for approximately up to 37 weeks . Participants from part B will be in the study for up to approximately 43 weeks.

The main purpose of the study is to evaluate empasiprubart use in adults living with Multifocal Motor Neuropathy (MMN). The researchers will evaluate the effect of empasiprubart on the thickness of the nerves, muscle strength, movement, and on the potential improvement of grip strength of the participants in handling objects. The study consists of an open-label treatment period where all participants will receive empasiprubart intravenously (IV). After the last dose of empasiprubart participants will enter a safety follow-up period for up to 15 months. The total study duration for each participant will be approximately 29 months. More information can be found here: clinicaltrials.argenx.com/empasound

This study aims to find the correct dose of ARGX-119 for children with SMA. The study will also look at how safe the study drug is, how well it works, how it moves through the body, and how the immune system responds to it. The study consists of a double-blinded treatment period (DBTP) where participants will either receive ARGX-119 IV or placebo IV, in addition to disease-modifying therapy (DMT) for 24 weeks. Participants who complete the DBTP will enter the open-label active-treatment extension period (ATEP) during which all participants will receive ARGX-119 IV up to 100 weeks (approximately 2 years).

The main purpose of this study is to look at how efgartigimod affects thyroid function in adults with Graves' Disease (GD). The study will also check whether efgartigimod is safe and well tolerated. It will look at how efgartigimod is distributed and eliminated in the body, how it changes antibody levels, and how the immune system responds to it. The study consists of a part A double-blinded treatment period, a part B treatment/observation period and a part C open-label treatment/observation period. During the part A and part B treatment periods, participants will receive efgartigimod PH20 SC via Prefilled Syringe (PFS) or placebo. During the part C open-label treatment period, participants will receive efgartigimod PH20 SC PFS. Participation in the different parts of the study will depend on the participant's response to treatment. The total study duration for participants ranges from 63 to 135 weeks, depending on the response to treatment.

This study aims to generate real world evidence (RWE) from Italian clinical practice on the impact of efgartigimod alfa in gMG patients encompassing clinical outcomes and patient reported experiences. The study population will consist in adult patients with a documented diagnosis of gMG who are AChR-antibody positive and for whom the decision of treatment with efgartigimod alfa for gMG has been made independently of study participation as part of routine clinical care. The total study duration will be up to 23 months

The POLARIS study is designed to evaluate how well efgartigimod PH20 SC may work (called "efficacy") and how safe it is for people diagnosed with Autoimmune Encephalitis (AIE). The study consists of 4 parts: in part A participants will receive efgartigimod SC; in part B, participants will be randomized to receive either efgartigimod SC or placebo; in part C, participants who completed part B will receive efgartigimod SC; in part D, participants who completed part C will be observed after their last dose of efgartigimod SC. If AIE symptoms return, efgartigimod SC treatment may be restarted during this time. The maximum overall study duration for participants is up to 3 years. More information can be found in clinicaltrials.argenx.com/polaris

The main purpose of this study is to look at how efgartigimod affects thyroid function in adults with Graves' Disease (GD). The study will also check whether efgartigimod is safe and well tolerated. It will look at how efgartigimod is distributed and eliminated in the body, how it changes antibody levels, and how the immune system responds to it. The study consists of a part A double-blinded treatment period, a part B treatment/observation period and a part C open-label treatment/observation period. During the part A and part B treatment periods, participants will receive efgartigimod PH20 SC via Prefilled Syringe (PFS) or placebo. During the part C open-label treatment period, participants will receive efgartigimod PH20 SC PFS. Participation in the different parts of the study will depend on the participant's response to treatment. The total study duration for participants ranges from 63 to 135 weeks, depending on the response to treatment.

The main purpose of this study is to look at the effect (efficacy) and safety of efgartigimod IV in participants with primary immune thrombocytopenia (ITP). After an up to 2 weeks screening period, eligible participants will be randomized in a 2:1 ratio to receive either efgartigimod IV or placebo IV, respectively during the double-blinded treatment period (DBTP). At the end of the treatment period (up to 24 weeks), all participants will receive efgartigimod IV during the first 52-week open-label treatment period (OLTP1). At the end of the first OLTP1, participants may begin a second 52-week OLTP2. After the OLTP2, the participants will enter a follow-up period (approximately 8 weeks) while off study drug. The participants will be in the study for up to 138 weeks. More information can be found here: https://clinicaltrials.argenx.com/advancenext

The aims of this study are to assess the real-world effectiveness of efgartigimod in treating chronic inflammatory demyelinating polyradiculoneuropathy (CIDP), describe the "treatment journey" of participants with CIDP, and assess the utilization of health care services among adult participants with CIDP who initiate treatment with efgartigimod. As this is a noninterventional study, treatment choices and decisions will be left to the discretion of participants and their physicians, according to the standard of care. Each participant will be prospectively followed for up to 2 years from the date of initial administration of efgartigimod.

The main purpose of this study is to evaluate the effect and safety of efgartigimod PH20 SC compared to placebo in adults with systemic sclerosis. The study consists of a screening period, a treatment period of up to 48 weeks and a safety follow-up period. After the screening period, eligible participants will be randomized in a 2:1 ratio to receive either efgartigimod PH20 SC or placebo. The total study duration can be up to approximately 15 months. More information can be found on: https://clinicaltrials.argenx.com/esscape

The purpose of this study is to assess the safety and tolerability of ARGX-119 in adult participants with DOK7- Congenital Myasthenic Syndromes. The study will also assess how ARGX-119 is processed by the body (pharmacokinetics), how the immune system reacts to it (immunogenicity), and how it may improve the way patients feel and function. After the screening period, eligible participants will be randomized in a 4:1 ratio to receive intravenous infusions of ARGX-119 or placebo during the double-blinded treatment period. Participants will then enter the follow-up period. After the follow-up period, participants may enrol in the active-treatment period, where they will receive open-label ARGX-119. The full duration of the study is approximately 38 months.

The aim of the Pre-Approval Access program is to provide efgartigimod PH20 SC treatment to patients with CIDP who are ineligible to participate in a clinical study and have unmet medical need despite available treatment options. The program will continue until the patient discontinues treatment or until the reimbursement process for CIDP concludes, whichever comes first.

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.

This study aims to assess the safety of ARGX-124 in healthy adults. Another aim is to measure the amount of ARGX-124 in the blood over time to learn how it acts and moves in the body and how the immune system responds to it. Participants will remain in the study for up to approximately 23 weeks.

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)