{"id":968,"date":"2017-03-24T14:30:34","date_gmt":"2017-03-24T14:30:34","guid":{"rendered":"https:\/\/www.institut-myologie.org\/imotion\/?page_id=968&#038;lang=en"},"modified":"2023-04-04T13:48:14","modified_gmt":"2023-04-04T11:48:14","slug":"current-trials-motion","status":"publish","type":"page","link":"https:\/\/www.institut-myologie.org\/imotion\/current-trials-motion\/?lang=en","title":{"rendered":"Current trials at I-Motion"},"content":{"rendered":"<p>The number of ongoing clinical trials in which the I-Motion Pediatric platform is involved is only increasing.<\/p>\n<p>I-Motion performs various roles in the clinical trials in which it is involved. When I-Motion is a \u00ab\u00a0promoter\u00a0\u00bb, it coordinates the trials from A to Z. Thus, I-Motion develops its own clinical studies through the trials promotion by the Institute of Myology. When it is an \u00ab\u00a0investigator\u00a0\u00bb, it organizes the inclusion and follow-up of patients in clinical trials conducted by industrialists, private laboratories (Genethon, for example) or even public organizations.<\/p>\n<div class=\"entry__gallery packery row large-12\"><div class=\"col-4 entry__gallery__column\"><div class=\"entry__image column\"><div class=\"background-container\" style=\"background-image:url(https:\/\/www.institut-myologie.org\/imotion\/wp-content\/uploads\/2021\/12\/AFMTLT_201104_IMOTION_Consult_Enf_DMD_CH_037-modif.png)\"><\/div><\/div><\/div><div class=\"col-4 entry__gallery__column\"><div class=\"entry__image column\"><div class=\"background-container\" style=\"background-image:url(https:\/\/www.institut-myologie.org\/imotion\/wp-content\/uploads\/2021\/12\/AFMTLT_201027_IMOTION_Consult_Enf_DMD_CH_001-modif-1.png)\"><\/div><\/div><\/div><div class=\"col-4 entry__gallery__column\"><div class=\"entry__image column\"><div class=\"background-container\" style=\"background-image:url(https:\/\/www.institut-myologie.org\/imotion\/wp-content\/uploads\/2021\/12\/AFMTLT_201104_IMOTION_Consult_Enf_DMD_CH_040-modif.png)\"><\/div><\/div><\/div><\/div>\n<p>Today, clinical trials in progress or under development being conducted at I-Motion concern:<\/p>\n<div class=\"has-custom-menu has_submenu\"><\/div>\n<h2>Spinal Muscular Atrophy<\/h2>\n<ul>\n<li><strong>IONIS-CS11-SHINE<\/strong>: An Open-label extension study for patients with SMA who previously participated in investigational studies of ISIS 396443<br \/>. contact: Andreea Seferian<br \/>. more on <a href=\"https:\/\/www.institut-myologie.org\/en\/recherche-clinique\/essais-en-cours\/spinal-muscular-atrophies\/\" target=\"_blank\" rel=\"noopener noreferrer\">Institute of Myology website<\/a><\/li>\n<li><strong>SUNFISH<\/strong>: A two-part seamless, multi-center randomized, placebo-controlled, doubleblind study to investigate the safety, tolerabilty, pharmacokinetics, pharmacodynamics and efficacy of RO7034067 in type 2 and 3 SMA patients<br \/>. contact: Andreea Seferian<br \/>. more on <a href=\"https:\/\/www.institut-myologie.org\/en\/recherche-clinique\/essais-en-cours\/spinal-muscular-atrophies\/\" target=\"_blank\" rel=\"noopener noreferrer\">Institute of Myology website<\/a><\/li>\n<li><strong>AVXS-101-LT-002<\/strong>:\u00a0A Long-term Follow-up Study of Patients in the Clinical Trials for SMA Receiving AVXS-101<br \/>. contact: Andreea Seferian<br \/>. more on <a href=\"https:\/\/www.institut-myologie.org\/en\/recherche-clinique\/essais-en-cours\/spinal-muscular-atrophies\/\" target=\"_blank\" rel=\"noopener noreferrer\">Institute of Myology website<\/a><\/li>\n<li><strong>Nusi-MFM<\/strong>: Responsiveness and validity study of MFM-32 in SMA patients treated with Nusinersen<br \/>. contact : Andreea Seferian<br \/>.\u00a0<span style=\"color: initial;\">more on <\/span><a href=\"https:\/\/www.institut-myologie.org\/en\/recherche-clinique\/essais-en-cours\/spinal-muscular-atrophies\/\" target=\"_blank\" rel=\"noopener noreferrer\">Institute of Myology website<\/a><\/li>\n<li><strong>MFM Play<\/strong>: Development and validation study of a Motor Function Measure digitalized playful completion modules:<i> MFM-Play study<br \/><\/i>. contact : Marta Gomez<br \/>.\u00a0<span style=\"color: initial;\">more on <\/span><a href=\"https:\/\/www.institut-myologie.org\/en\/recherche-clinique\/essais-en-cours\/spinal-muscular-atrophies\/\" target=\"_blank\" rel=\"noopener noreferrer\">Institute of Myology website<\/a><i><br \/><\/i><\/li>\n<li><strong>REALITY<\/strong>: <span style=\"font-size: revert;\">A randomized crossover pragmatic study to evaluate virtual reality for the mitigation <\/span><span style=\"font-size: revert;\"><span style=\"font-size: revert;\">of anxiety during intrathecal administration in patients with spinal muscular atrophy<br \/>. contact: <\/span><\/span><span style=\"font-size: revert;\">Marie-Christine Nougues<br \/><\/span><span style=\"font-size: revert;\">. more on<\/span> <a style=\"font-size: revert;\" href=\"https:\/\/www.institut-myologie.org\/en\/recherche-clinique\/essais-en-cours\/spinal-muscular-atrophies\/\" target=\"_blank\" rel=\"noopener noreferrer\">Institute of Myology website<\/a><\/li>\n<li>\u00a0<strong>SAPPHIRE<\/strong>: <span style=\"font-size: revert;\">Phase 3, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Apitegromab (SRK-015) in Patients with Later-Onset Spinal Muscular Atrophy Receiving Background Nusinersen or Risdiplam Therapy<br \/><\/span>. contact: Andreea Seferian<br \/>.\u00a0<span style=\"color: initial;\">more on <\/span><a href=\"https:\/\/www.institut-myologie.org\/en\/recherche-clinique\/essais-en-cours\/spinal-muscular-atrophies\/\" target=\"_blank\" rel=\"noopener noreferrer\">Institute of Myology website<\/a><\/li>\n<li><span style=\"font-size: revert;\"><span style=\"font-size: revert;\"><strong>RESILIENT<\/strong>: <\/span><\/span><span style=\"font-size: revert;\">A Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Ambulatory and Non-Ambulatory Participants with Spinal Muscular Atrophy with Open-Label Extension<br \/>. contact: Marina Colella<br \/>. more on <a href=\"https:\/\/www.institut-myologie.org\/en\/recherche-clinique\/essais-en-cours\/spinal-muscular-atrophies\/\" target=\"_blank\" rel=\"noopener noreferrer\">Institute of Myology website<\/a><\/span><\/li>\n<\/ul>\n<h2>Duchenne Muscular Dystrophy<\/h2>\n<ul>\n<li><strong>PTC-PASS<\/strong>: Long-Term Observational Study of Translarna safety and effectiveness in routine care<br \/>. contact: Silvana De Lucia<br \/>. more on <a href=\"https:\/\/www.institut-myologie.org\/en\/recherche-clinique\/essais-en-cours\/duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noopener noreferrer\">Institute of Myology website<\/a><\/li>\n<li><strong>SRP-4045-302<\/strong>:\u00a0Long-term, Open-label Extension Study for Patients With DMD Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen<br \/>. contact: Andreea Seferian<br \/>. more on <a href=\"https:\/\/www.institut-myologie.org\/en\/recherche-clinique\/essais-en-cours\/duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noopener noreferrer\">Institute of Myology website<\/a><\/li>\n<li><strong>Essence<\/strong>: A Double-blind, Placebo-Controlled, Multicenter Study With and Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With DMD<br \/>. contact: Andreea Seferian<br \/>. more on\u00a0<a href=\"https:\/\/www.institut-myologie.org\/en\/recherche-clinique\/essais-en-cours\/duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noopener noreferrer\">Institute of Myology website<\/a><\/li>\n<li><strong>GNT-014-MDYF<\/strong>: Natural History of Duchenne Muscular Dystrophy \u2013 A Prospective, Interventional, Baseline Study In Young Male Subjects Aged From 4 to 6 Years<br \/>. contact: Silvana De Lucia<br \/>. more on\u00a0<a href=\"https:\/\/www.institut-myologie.org\/en\/recherche-clinique\/essais-en-cours\/duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noopener noreferrer\">Institute of Myology website<\/a><\/li>\n<li><strong>GNT-016-MDYF<\/strong>:\u00a0Microdystrophin (GNT0004) Gene Therapy Clinical Trial in DMD<br \/>. contact: Silvana De Lucia<br \/>. more on\u00a0<a href=\"https:\/\/www.institut-myologie.org\/en\/recherche-clinique\/essais-en-cours\/duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noopener noreferrer\">Institute of Myology website<\/a><\/li>\n<li><strong>Italfarmaco 51<\/strong>:\u00a0Open label, long-term safety, tolerability, and efficacy study of Givinostat in all DMD patients who have been previously treated in one of the Givinostat studies<br \/>. contact: Odile Boespflug-Tanguy<br \/>. more on <a href=\"https:\/\/www.institut-myologie.org\/en\/recherche-clinique\/essais-en-cours\/duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noopener noreferrer\">Institute of Myology website<\/a><\/li>\n<li><strong>MIS51ON<\/strong>:\u00a0A Randomized, Double-Blind, Dose Finding and\u00a0Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with DMD\u00a0With Deletion Mutations Amenable to Exon\u00a051 Skipping<br \/>. contact: Andreea Seferian<br \/>. more on\u00a0<a href=\"https:\/\/www.institut-myologie.org\/en\/recherche-clinique\/essais-en-cours\/duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noopener noreferrer\">Institute of Myology website<\/a><\/li>\n<li><strong>FGCL-3019-093<\/strong>: A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in Subjects with Non-ambulatory DMD<br \/>. contact: Andreea Seferian<br \/>. more on\u00a0<a href=\"https:\/\/www.institut-myologie.org\/en\/recherche-clinique\/essais-en-cours\/duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noopener noreferrer\">Institute of Myology website<\/a><\/li>\n<li><strong>FGCL-3019-094<\/strong>:\u00a0A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in ambulatory subjects with DMD<br \/>. contact: Andreea Seferian<br \/>. more on\u00a0<a href=\"https:\/\/www.institut-myologie.org\/en\/recherche-clinique\/essais-en-cours\/duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noopener noreferrer\">Institute of Myology website<\/a><\/li>\n<\/ul>\n<h2>Myotonic Dystrophies and Non Dystrophic Myotonies<\/h2>\n<ul>\n<li><strong>PIP4 (MEX-NM-301)<\/strong>:\u00a0An Open-label, non-Comparative Study to Evaluate the Steady-State Pharmacokinetics, Safety, and Efficacy of Mexiletine in Adolescents and Children with Myotonic Disorders<br \/>. contact:\u00a0Arnaud Isapof<br \/>. more on\u00a0<a href=\"https:\/\/www.institut-myologie.org\/en\/recherche-clinique\/essais-en-cours\/myotonic-dystrophies\/\" target=\"_blank\" rel=\"noopener noreferrer\">Institute of Myology website<\/a><\/li>\n<li><strong>PIP7 (MEX-NM-303)<\/strong>:\u00a0Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Mexiletine in Paediatric Patients with Myotonic Disorders Who Have Completed MEX-NM-301 Study<br \/>. contact:\u00a0Arnaud Isapof<br \/>. more on\u00a0<a href=\"https:\/\/www.institut-myologie.org\/en\/recherche-clinique\/essais-en-cours\/myotonic-dystrophies\/\" target=\"_blank\" rel=\"noopener noreferrer\">Institute of Myology website<\/a><\/li>\n<\/ul>\n<h2>Myotubular Myopathy and\u00a0other Centronuclear Myopathies<\/h2>\n<ul>\n<li><strong>ASPIRO<\/strong>: A Phase 1\/2, Randomized, Open-Label, Ascending-Dose, Delayed-Treatment Concurrent Control Clinical Study to Evaluate the Safety and Preliminary Efficacy of AT132, an AAV8-Delivered Gene Therapy in X-Linked Myotubular Myopathy (XLMTM) Patients<br \/>. contact: Andreea Seferian<br \/>. more on <a href=\"https:\/\/www.institut-myologie.org\/en\/recherche-clinique\/essais-en-cours\/myotubular-myopathy\/\" target=\"_blank\" rel=\"noopener noreferrer\">Institute of Myology website<\/a><\/li>\n<\/ul>\n<h2>GM1 Gangliosidosis<\/h2>\n<ul>\n<li><strong>P1-GM-101<\/strong>:\u00a0An open-label adaptive design study of intracisternal administration of adeno-associated viral vector serotype rh.10 carrying the human \u03b2-galactosidase cDNA for the treatment of GM1 gangliosidosis<br \/>. contact: B\u00e9n\u00e9dicte H\u00e9ron<br \/>. more on <a href=\"https:\/\/www.institut-myologie.org\/en\/recherche-clinique\/essais-en-cours\/gm1-gangliosidosis\/\" target=\"_blank\" rel=\"noopener noreferrer\">Institute of Myology website<\/a><\/li>\n<\/ul>\n<h2>AADC Deficiency<\/h2>\n<ul>\n<li><strong>PTC-AADC-MA-406<\/strong>: An Two Part, International, Real-World, Observational Registry Of <span style=\"font-size: revert;\">Participants Diagnosed With Aromatic L-Amino Acid Decarboxylase Deficiency (AADC) <\/span><span style=\"font-size: revert;\">With or Without Treatment With Eladocagene Exuparvovec<br \/><\/span>. contact: Claudia Ravelli<br \/>. more on <a href=\"https:\/\/www.institut-myologie.org\/en\/recherche-clinique\/essais-en-cours\/aadc-deficiency\/\" target=\"_blank\" rel=\"noopener noreferrer\">Institute of Myology website<\/a><\/li>\n<\/ul>\n\n\n<p><\/p>\n","protected":false},"excerpt":{"rendered":"<p>The number of ongoing clinical trials in which the I-Motion Pediatric platform is involved is only increasing. I-Motion performs various roles in the clinical trials in which it is involved. When I-Motion is a \u00ab\u00a0promoter\u00a0\u00bb, it coordinates the trials from A to Z. Thus, I-Motion develops its own clinical studies through the trials promotion by &hellip; <a href=\"https:\/\/www.institut-myologie.org\/imotion\/current-trials-motion\/?lang=en\">Continued<\/a><\/p>\n","protected":false},"author":3,"featured_media":0,"parent":0,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"_acf_changed":false,"footnotes":""},"class_list":["post-968","page","type-page","status-publish","hentry"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.1.1 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Current trials at I-Motion - I-Motion<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/www.institut-myologie.org\/imotion\/current-trials-motion\/?lang=en\" \/>\n<meta property=\"og:locale\" content=\"fr_FR\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Current trials at I-Motion - I-Motion\" \/>\n<meta property=\"og:description\" content=\"The number of ongoing clinical trials in which the I-Motion Pediatric platform is involved is only increasing. 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