argenx — Biomedical Watch V2

Traitements19programmes

Essais12liés

Publications5liées

SourceDBlocale

Traitements

19

Molécule	Indication / population	Phase	Objectif	Pays	Résultat
ARGX-113This is a Long-Term, Single-Arm, Open-Label, Multicenter Phase 3 follow-on trial of the ARGX-113-1704 study to evaluate the safety and tolerability of ARGX-113 in patients with gMG. Patients who have completed at least 1 cycle of treatment and at least 1 year of trial ARGX-113-1705 and have started Part B are eligible to enroll in the open-label trial ARGX-113-2002 to receive efgartigimod by SC administration.	Myasthénie	Phase 3	À vérifier	United States, Belgium, Canada, Czechia, Denmark, France, Georgia, Germany, …	À vérifier
Efgartigimod IVThe 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)	Myasthénie	Phase 3	À vérifier	United States, Belgium, Canada, China, Cyprus, Czechia, Denmark, Finland, …	À vérifier
Efgartigimod IV or Efgartigimod PH20 SCThe 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. Participants will receive efgartigimod IV or efgartigimod PH20 SC, using the dose administered in the antecedent studies. Participants who have not reached the age of 18 can remain in the study until efgartigimod becomes commercially available in the respective country or available through another continued access program for gMG. Participants who have reached the age of 18 can remain in the study for a maximum of 2 years, until efgartigimod becomes commercially available in the respective country or available through another continued access program for gMG, whichever comes first.	Myasthénie	Phase 2/3	À vérifier	United States, Belgium, Canada, France, Germany, Israel, Netherlands, Poland, …	À vérifier
Efgartigimod PH20 SCThe purpose of this study is to evaluate the efficacy and safety of efgartigimod PH20 SC given by a pre-filled syringe in adult patients with ocular myasthenia gravis. The study consists of a part A (approximately 7 weeks) and a part B (up to 2 years). In part A, half of the participants will receive efgartigimod PH20 SC and the other half will receive placebo. In part B, all participants will receive efgartigimod PH20 SC. The participants will be in the study for about up to 2 years and 12 weeks.	Myasthénie	Phase 2/3	À vérifier	United States, Australia, Belgium, Canada, Czechia, France, Germany, Israel, …	À vérifier
Efgartigimod PH20 SC PFSADAPT 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.	Myasthénie	À vérifier	À vérifier	United States, Belgium, Germany, Greece, Italy, Japan, Poland, Spain	À vérifier
Empasiprubart IVThis 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	Myasthénie	À vérifier	À vérifier	United States, Belgium, Germany, Greece, Italy, Japan, Poland, Spain	À vérifier
POEMA — A Study to Evaluate the Impact of Efgartigimod on Overall Disease Experience of People Suffering From Generalized Myasthenia Gravis (gMG) in ItalyThis 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	Myasthénie	À vérifier	À vérifier	Italy	À vérifier
Efgartigimod PH20 SCThe purpose of this study is to evaluate the efficacy and safety of efgartigimod PH20 SC given by a pre-filled syringe in adult patients with ocular myasthenia gravis. The study consists of a part A (approximately 7 weeks) and a part B (up to 2 years). In part A, half of the participants will receive efgartigimod PH20 SC and the other half will receive placebo. In part B, all participants will receive efgartigimod PH20 SC. The participants will be in the study for about up to 2 years and 12 weeks.	Myasthénie	Phase 3	À vérifier	United States, Australia, Austria, Belgium, Canada, China, Cyprus, Czechia, Denmark, Finland, France, Georgia, Germany, Greece, Italy, Japan, Netherlands, Poland, Portugal, Serbia, …	À vérifier
Efgartigimod IVThe 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)	Myasthénie	Phase 2	À vérifier	United States, Austria, Belgium, Canada, France, Georgia, Germany, Italy, Netherlands, Poland, Spain, United Kingdom	À vérifier
Efgartigimod IVThe 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)	Myasthénie	Phase 2	À vérifier	United States, Belgium, Germany, Greece, Italy, Japan, Poland, Spain	À vérifier
ARGX-113This is a Long-Term, Single-Arm, Open-Label, Multicenter Phase 3 follow-on trial of the ARGX-113-1704 study to evaluate the safety and tolerability of ARGX-113 in patients with gMG. Patients who have completed at least 1 cycle of treatment and at least 1 year of trial ARGX-113-1705 and have started Part B are eligible to enroll in the open-label trial ARGX-113-2002 to receive efgartigimod by SC administration.	Myasthénie	Phase 3	À vérifier	United States, Belgium, Canada, Czechia, Denmark, France, Georgia, Germany, Hungary, Italy, Japan, Netherlands, Poland, Russia, Serbia	À vérifier
Efgartigimod IV or Efgartigimod PH20 SCThe 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. Participants will receive efgartigimod IV or efgartigimod PH20 SC, using the dose administered in the antecedent studies. Participants who have not reached the age of 18 can remain in the study until efgartigimod becomes commercially available in the respective country or available through another continued access program for gMG. Participants who have reached the age of 18 can remain in the study for a maximum of 2 years, until efgartigimod becomes commercially available in the respective country or available through another continued access program for gMG, whichever comes first.	Myasthénie	Phase 2/3	À vérifier	United States, Belgium, Canada, France, Germany, Israel, Netherlands, Poland, Spain, United Kingdom	À vérifier
Efgartigimod IVThe 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)	Myasthénie	Phase 3	À vérifier	United States, Belgium, Canada, China, Cyprus, Czechia, Denmark, Finland, France, Georgia, Germany, Greece, Hungary, Netherlands, Norway, Poland, Portugal, Romania, Saudi Arabia, Serbia, …	À vérifier
Efgartigimod IVThe 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)	Myasthénie	À vérifier	À vérifier	United States, Belgium, Germany, Greece, Italy, Japan, Poland, Spain	À vérifier
Empasiprubart IVThis 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	Myasthénie	Phase 2	À vérifier	United States	À vérifier
Efgartigimod PH20 SCThe purpose of this study is to evaluate the efficacy and safety of efgartigimod PH20 SC given by a pre-filled syringe in adult patients with ocular myasthenia gravis. The study consists of a part A (approximately 7 weeks) and a part B (up to 2 years). In part A, half of the participants will receive efgartigimod PH20 SC and the other half will receive placebo. In part B, all participants will receive efgartigimod PH20 SC. The participants will be in the study for about up to 2 years and 12 weeks.	Myasthénie	Phase 2/3	À vérifier	United States, Australia, Belgium, Canada, Czechia, France, Germany, Israel, Italy, Netherlands, Poland, Spain, Switzerland, United Kingdom	À vérifier
Efgartigimod IVThe 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)	Myasthénie	Phase 3	À vérifier	United States, Austria, Belgium, Canada, France, Georgia, Germany, Italy, Netherlands, Poland, Spain	À vérifier
POEMA — A Study to Evaluate the Impact of Efgartigimod on Overall Disease Experience of People Suffering From Generalized Myasthenia Gravis (gMG) in ItalyThis 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	Myasthénie	À vérifier	À vérifier	Italy	À vérifier
Efgartigimod PH20 SCThe purpose of this study is to evaluate the efficacy and safety of efgartigimod PH20 SC given by a pre-filled syringe in adult patients with ocular myasthenia gravis. The study consists of a part A (approximately 7 weeks) and a part B (up to 2 years). In part A, half of the participants will receive efgartigimod PH20 SC and the other half will receive placebo. In part B, all participants will receive efgartigimod PH20 SC. The participants will be in the study for about up to 2 years and 12 weeks.	Myasthénie	Phase 4	À vérifier	United States	À vérifier

Essais cliniques

12

Molécule	Indication / population	Phase	NCT	Titre	Statut
Efgartigimod PH20 SC	Myasthénie	Phase 4	NCT06909214	ADAPT-EARLY — A Study to Evaluate the Clinical Outcomes of Efgartigimod PH20 SC in Adults With New-onset Generalized Myasthenia Gravis (gMG)	RECRUITING
POEMA — A Study to Evaluate the Impact of Efgartigimod on Overall Disease Experience of People Suffering From Generalized Myasthenia Gravis (gMG) in Italy	Myasthénie	À vérifier	NCT07595653	POEMA — A Study to Evaluate the Impact of Efgartigimod on Overall Disease Experience of People Suffering From Generalized Myasthenia Gravis (gMG) in Italy	RECRUITING
Efgartigimod IV	Myasthénie	Phase 3	NCT04980495	ADAPT NXT — An Open-label Study to Investigate the Clinical Efficacy of Different Dosing Regimens of Efgartigimod IV in Patients With Generalized Myasthenia Gravis	COMPLETED
Efgartigimod PH20 SC	Myasthénie	Phase 2/3	NCT06392386	ADAPT Jr SC — A Study of Efgartigimod PH20 SC in Children Between 2 and Less Than 18 Years of Age With Generalized Myasthenia Gravis	RECRUITING
Empasiprubart IV	Myasthénie	Phase 2	NCT07673627	ADAPT Forward2 — ADAPT Forward 2 - ISA2 - a Study to Evaluate the Safety, Tolerability and Efficacy of Empasiprubart IV Monotherapy in Participants With AChR-Ab Seropositive Generalized Myasthenia Gravis	RECRUITING
Efgartigimod IV	Myasthénie	À vérifier	NCT07294170	ADAPT Forward - Master Protocol of a Platform Study to Evaluate the Safety and Efficacy of Multiple Regimens in Participants With Myasthenia Gravis	RECRUITING
Efgartigimod IV	Myasthénie	Phase 3	NCT06298552	ADAPT SERON — A Phase 3 Study to Evaluate the Efficacy and Safety of Efgartigimod IV in Patients With Acetylcholine Receptor Binding Antibody Seronegative Generalized Myasthenia Gravis	ACTIVE_NOT_RECRUITING
Efgartigimod IV or Efgartigimod PH20 SC	Myasthénie	Phase 2/3	NCT05374590	ADAPT Jr + — Evaluating Long-term Safety of Efgartigimod Administered Intravenously and Efgartigimod PH20 Administered Subcutaneously in Children With Generalized Myasthenia Gravis	ENROLLING_BY_INVITATION
ARGX-113	Myasthénie	Phase 3	NCT03770403	ADAPT+ — A Safety and Tolerability Study of ARGX-113 in Patients With Myasthenia Gravis Who Have Generalized Muscle Weakness.	COMPLETED
Efgartigimod IV	Myasthénie	Phase 2	NCT07284420	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	RECRUITING
Efgartigimod IV	Myasthénie	Phase 2	NCT04833894	ADAPT Jr — Evaluating the Pharmacokinetics, Pharmacodynamics, and Safety of Efgartigimod Administered Intravenously in Children With Generalized Myasthenia Gravis	RECRUITING
Efgartigimod PH20 SC	Myasthénie	Phase 3	NCT06558279	ADAPT oculus — A Study to Assess Efficacy and Safety of Efgartigimod PH20 SC in Adults With Ocular Myasthenia Gravis	ACTIVE_NOT_RECRUITING

Publications

5

Molécule	Indication / population	Titre	Journal	Date
Empasiprubart IV		Randomized phase I trial outcomes show safe and sustainable inhibition of classical and lectin complement pathways by empasiprubart.	Nature communications
Efgartigimod IV		Povetacicept (ALPN-303; TACI vTD-Fc), an enhanced, potent dual inhibitor of BAFF and APRIL, ameliorates experimental autoimmune myasthenia gravis in C57BL/6N mice.	Frontiers in immunology
Efgartigimod IV		Efgartigimod in Sjögren's disease: a phase 2, randomised, placebo-controlled, parallel-group, double-blinded, proof-of-concept study (RHO).	Annals of the rheumatic diseases
Efgartigimod PH20 SC		Efficacy and Safety of Subcutaneous Efgartigimod PH20 in Adults With Primary Immune Thrombocytopenia (ADVANCE SC): A Multicenter, Randomized, Double-Blinded, Placebo-Controlled, Phase 3 Trial.	American journal of hematology
Efgartigimod PH20 SC		Real-world experience with switching from intravenous immunoglobulin to subcutaneous efgartigimod PH20 in patients with chronic inflammatory demyelinating polyradiculoneuropathy: A case series.	Journal of neuroimmunology