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{"id":5275,"date":"2021-05-03T20:05:12","date_gmt":"2021-05-04T01:05:12","guid":{"rendered":"https:\/\/cru.mcgill.ca\/?page_id=5275"},"modified":"2025-02-06T17:52:03","modified_gmt":"2025-02-06T21:52:03","slug":"nm-2","status":"publish","type":"page","link":"https:\/\/cru.mcgill.ca\/fr\/nm-2\/","title":{"rendered":"Essais cliniques sur la Neuromusculaire"},"content":{"rendered":"

[et_pb_section fb_built=”1″ fullwidth=”on” _builder_version=”4.9.10″ _module_preset=”default” background_enable_color=”off” width=”100%” max_width=”100%” min_height=”77px” height=”100px” max_height=”100px” custom_margin=”||||false|false” custom_padding=”20px||||false|false” global_module=”4368″ saved_tabs=”all” width__hover_enabled=”on|desktop”][et_pb_fullwidth_menu menu_id=”25″ menu_style=”centered” fullwidth_menu=”off” dropdown_menu_bg_color=”#353535″ dropdown_menu_line_color=”rgba(0,0,0,0)” dropdown_menu_text_color=”#ffffff” mobile_menu_bg_color=”rgba(0,0,0,0.67)” _builder_version=”4.9.10″ menu_font=”Fabriga||||||||” menu_text_color=”#ffffff” menu_font_size=”15px” menu_line_height=”0em” background_color=”#1e1e1e” background_size=”initial” background_position=”top_left” background_repeat=”repeat” background_layout=”dark” width=”100%” module_alignment=”left” height=”59px” custom_margin=”-12px||||false|false” custom_margin_last_edited=”off|desktop” custom_padding=”0px||0px||false|false”][\/et_pb_fullwidth_menu][\/et_pb_section][et_pb_section fb_built=”1″ fullwidth=”on” _builder_version=”4.9.10″ max_width=”100%” module_alignment=”center” custom_margin=”-80px||||false|false” custom_padding=”0px||||false|false” box_shadow_style=”preset1″ box_shadow_vertical=”6px” box_shadow_blur=”37px” box_shadow_spread=”8px” global_module=”3061″ saved_tabs=”all”][et_pb_fullwidth_menu menu_id=”25″ menu_style=”centered” fullwidth_menu=”off” dropdown_menu_bg_color=”#353535″ dropdown_menu_line_color=”rgba(0,0,0,0)” dropdown_menu_text_color=”#ffffff” mobile_menu_bg_color=”rgba(0,0,0,0.67)” _builder_version=”4.9.10″ menu_font=”Fabriga||||||||” menu_text_color=”#ffffff” menu_font_size=”15px” menu_line_height=”0em” background_color=”#1e1e1e” background_size=”initial” background_position=”top_left” background_repeat=”repeat” background_layout=”dark” width=”100%” module_alignment=”left” height=”52px” custom_margin=”-12px||||false|false” custom_margin_last_edited=”off|desktop” custom_padding=”0px||0px||false|false”][\/et_pb_fullwidth_menu][\/et_pb_section][et_pb_section fb_built=”1″ fullwidth=”on” disabled_on=”on|off|off” _builder_version=”4.8.1″ _module_preset=”default” custom_margin=”||-24px||false|false” custom_padding=”||||false|false”][et_pb_fullwidth_header _builder_version=”4.9.4″ _module_preset=”default” title_level=”h2″ title_font=”Arial|700|||||||” title_text_color=”#ff6720″ content_text_color=”#470a68″ subhead_font=”Arial|700|||||||” subhead_text_color=”#470a68″ subhead_font_size=”32px” background_color=”rgba(0,0,0,0)” background_image=”https:\/\/cru.mcgill.ca\/wp-content\/uploads\/NEUROMUSCULAR_H140_NEURO_logo_horiz_RGB_BIL_fr.png” background_size=”initial” background_position=”center_left” min_height=”140px” custom_margin=”||35px|||”][\/et_pb_fullwidth_header][\/et_pb_section][et_pb_section fb_built=”1″ _builder_version=”4.8.1″ _module_preset=”default”][et_pb_row column_structure=”3_5,1_5,1_5″ _builder_version=”4.8.1″ _module_preset=”default” custom_padding=”0px|||||”][et_pb_column type=”3_5″ _builder_version=”4.8.1″ _module_preset=”default”][et_pb_text admin_label=”Current Recruiting” _builder_version=”4.9.4″ text_font=”Montserrat|300|||||||” text_text_color=”#677a92″ text_line_height=”1.8em” header_font=”Fabriga Bold|700|||||||” header_font_size=”32px” header_line_height=”1.3em” header_2_font=”||||||||” header_3_font=”Arial||||||||” min_height=”48px” custom_margin=”||8px||false|false” custom_margin_tablet=”0px|50px||50px” custom_margin_phone=”|20px||” custom_margin_last_edited=”off|desktop” custom_padding=”20px||||false|false” text_line_height_tablet=”1.8em” text_line_height_phone=”1.8em” text_line_height_last_edited=”on|phone” header_font_size_tablet=”” header_font_size_phone=”36px” header_font_size_last_edited=”on|phone” header_2_font_size_phone=”30px” header_2_font_size_last_edited=”off|desktop” locked=”off”]<\/p>\n

\u00c9TUDES EN RECRUTEMENT<\/h1>\n

[\/et_pb_text][et_pb_text _builder_version=”4.9.10″ _module_preset=”default” text_font=”Fabriga Bold||||||||” text_line_height=”1.2em” background_color=”#ff6720″ text_orientation=”center” custom_margin=”20px||||false|false” custom_padding=”10px|10px|10px|10px|false|false” link_option_url=”mailto:nm.neurocru@mcgill.ca” border_radii=”on|12px|12px|12px|12px” background__hover_enabled=”on|hover” background_color__hover=”rgba(255,103,32,0.42)” background_enable_color__hover=”on”]<\/p>\n

Pour plus d’infos,\u00a0 contactez<\/span> \u00a0nm.neurocru@mcgill.ca.<\/a><\/span><\/b><\/h4>\n

[\/et_pb_text][et_pb_accordion db_closeable=”on” db_initial_state=”all_closed” open_toggle_text_color=”#2cccd3″ icon_color=”#2cccd3″ admin_label=”CURRENT ACCORDIAN” _builder_version=”4.9.10″ _module_preset=”default” toggle_text_color=”#2cccd3″ toggle_font=”Fabriga Medium||||||||” closed_toggle_line_height=”1.1em” body_font=”Arial||||||||” border_radii=”on|20px|20px|20px|20px” border_width_all=”2px”][et_pb_accordion_item title=”1821-FSH-301 Losmapimod ( FSHD)” open=”on” use_icon_font_size=”on” icon_font_size=”32px” _builder_version=”4.9.10″ _module_preset=”default” body_font=”Fabriga||||||||” custom_margin=”||8px||false|false” toggle_font=”|700|||||||” toggle_font_size=”28px” closed_toggle_font_size=”20px” closed_toggle_line_height=”1em” open_use_icon_font_size=”on” open_icon_font_size=”32px”]<\/p>\n

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\u00c9valuer l’innocuit\u00e9 et l’efficacit\u00e9 du losmapimod chez les individus atteints de dystrophie musculaire facio-scapulo-hum\u00e9rale (FSH)<\/span><\/a><\/p>\n

Cette \u00e9tude \u00e9valuera l’innocuit\u00e9 et l’efficacit\u00e9 du losmapimod pour traiter des individus atteints de dystrophie musculaire facio-scapulo-hum\u00e9rale (FSH) pour 48 semaines.\u00a0\u00a0<\/span><\/p>\n<\/div>\n

Plus d\u2019information<\/a><\/p>\n

[\/et_pb_accordion_item][et_pb_accordion_item title=”CRU SCIG (Myasth\u00e9nie Grave)” use_icon_font_size=”on” icon_font_size=”32px” _builder_version=”4.9.10″ _module_preset=”default” body_font=”Fabriga||||||||” custom_margin=”||8px||false|false” global_colors_info=”{}” toggle_font=”|700|||||||” toggle_font_size=”28px” closed_toggle_font_size=”20px” closed_toggle_line_height=”1em” open_use_icon_font_size=”on” open_icon_font_size=”32px” open=”off”]<\/p>\n

Passage de l’immunoglobuline intraveineuse \u00e0 l’immunoglobuline sous-cutan\u00e9e chez les patients atteints de myasth\u00e9nie grave (MG) et de polyneuropathie d\u00e9my\u00e9linisante inflammatoire chronique (PDIC)\u00a0: une \u00e9tude pilote prospective \u00e0 un seul bras, en ouvert, pour d\u00e9terminer l’efficacit\u00e9, la satisfaction du traitement, l’utilisation des ressources de soins de sant\u00e9 et la tol\u00e9rabilit\u00e9<\/p>\n

Le but de cette \u00e9tude \u00e9tudiera l’efficacit\u00e9, la satisfaction du traitement, l’utilisation des ressources de soins de sant\u00e9 et la tol\u00e9rabilit\u00e9 des SCIg chez les patients MG et CIDP pr\u00e9c\u00e9demment trait\u00e9s avec des IgIV. . Les donn\u00e9es recueillies au cours de l’\u00e9tude pourraient \u00e9galement \u00eatre utiles aux professionnels de la sant\u00e9 et aux chercheurs \u00e0 l’avenir.<\/p>\n

Plus d’infos<\/span><\/a><\/span><\/p>\n

[\/et_pb_accordion_item][et_pb_accordion_item title=”GALILEO Vertex VX-670 (Dystrophie myotonique)” use_icon_font_size=”on” icon_font_size=”32px” _builder_version=”4.9.10″ _module_preset=”default” body_font=”Fabriga||||||||” custom_margin=”||8px||false|false” global_colors_info=”{}” toggle_font=”|700|||||||” toggle_font_size=”28px” closed_toggle_font_size=”20px” closed_toggle_line_height=”1em” open_use_icon_font_size=”on” open_icon_font_size=”32px” open=”off”]<\/p>\n

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Une \u00e9tude de phase 1\/\u200b2 sur le VX-670 chez des participants adultes atteints de\u00a0dystrophie\u00a0myotonique\u00a01\u00a0(DM1) <\/span><\/strong><\/a><\/p>\n

Le but de l’\u00e9tude est d’\u00e9valuer l’innocuit\u00e9, la tol\u00e9rabilit\u00e9, la pharmacocin\u00e9tique et la pharmacodynamique du VX-670 \u00e0 diff\u00e9rentes doses uniques et multiples chez les participants atteints de DM1. <\/span><\/p>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n

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Plus d’info<\/span><\/a><\/span><\/p>\n

[\/et_pb_accordion_item][et_pb_accordion_item title=”LUMINESCE Satralizumab (Myasth\u00e9nie grave g\u00e9n\u00e9ralis\u00e9e)” use_icon_font_size=”on” icon_font_size=”32px” _builder_version=”4.9.10″ _module_preset=”default” body_font=”Fabriga||||||||” custom_margin=”||8px||false|false” global_colors_info=”{}” toggle_font=”|700|||||||” toggle_font_size=”28px” closed_toggle_font_size=”20px” closed_toggle_line_height=”1em” open_use_icon_font_size=”on” open_icon_font_size=”32px” open=”off”]<\/p>\n

Une \u00e9tude multicentrique de phase III, randomis\u00e9e, en double aveugle, contr\u00f4l\u00e9e par placebo\u00a0pour\u00a0\u00e9valuer l’efficacit\u00e9, l’innocuit\u00e9, la pharmacocin\u00e9tique et la pharmacodynamique du\u00a0satralizumab\u00a0chez les patients atteints de myasth\u00e9nie grave g\u00e9n\u00e9ralis\u00e9e<\/p>\n

Le but de cette \u00e9tude est\u00a0de\u00a0comparer les effets, bons ou mauvais, des injections sous-cutan\u00e9es (sous la peau) de\u00a0satralizumab\u00a0en association avec le traitement actuel (th\u00e9rapie de fond) chez les patients atteints de\u00a0gMG.<\/p>\n

Cette \u00e9tude collectera \u00e9galement davantage de donn\u00e9es sur l’innocuit\u00e9 et l’efficacit\u00e9 \u00e0 long terme du\u00a0satralizumab\u00a0et continuera \u00e0 vous fournir un traitement par satralizumab\u00a0pendant une p\u00e9riode de prolongation en ouvert.<\/p>\n

Pour cette \u00e9tude,\u00a0240\u00a0participants masculins et f\u00e9minins,\u00a0dont 20\u00a0adolescents,\u00a0seront recrut\u00e9s dans\u00a0120\u00a0sites. Les\u00a0autres\u00a0participants seront \u00e2g\u00e9s de\u00a018\u00a0ans et plus. L’Institut neurologique de Montr\u00e9al-H\u00f4pital ne recrutera que des patients adultes \u00e2g\u00e9s de 18 ans et plus.<\/p>\n

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Plus d’infos<\/span><\/a><\/span><\/p>\n

[\/et_pb_accordion_item][et_pb_accordion_item title=”MARINA (Dystrophie Myotonique)” use_icon_font_size=”on” icon_font_size=”32px” _builder_version=”4.9.10″ _module_preset=”default” body_font=”Fabriga||||||||” custom_margin=”||8px||false|false” global_colors_info=”{}” toggle_font=”|700|||||||” toggle_font_size=”28px” closed_toggle_font_size=”20px” closed_toggle_line_height=”1em” open_use_icon_font_size=”on” open_icon_font_size=”32px” open=”off”]<\/p>\n

AOC 1001-CS1 est une \u00e9tude de phase 1\/2 randomis\u00e9e, en double aveugle, contr\u00f4l\u00e9e par placebo, visant \u00e0 \u00e9valuer l'innocuit\u00e9, la tol\u00e9rabilit\u00e9, la pharmacocin\u00e9tique et la pharmacodynamique de doses uniques et multiples d'AOC 1001 administr\u00e9es par voie intraveineuse dans la dystrophie myotonique adulte de type 1 (DM1). ) patients (MARINA).\n\nLa partie A est une conception \u00e0 dose unique avec 1 cohorte (niveau de dose). Dans la partie A, la dur\u00e9e du patient est de 6 mois car la p\u00e9riode de traitement est de 1 jour suivi d'une p\u00e9riode de suivi de 6 mois.\n\nLa partie B est une conception \u00e0 doses multiples croissantes avec 2 cohortes (niveaux de dose). Dans la partie B, la dur\u00e9e du patient est de 6 mois car la p\u00e9riode de traitement est de 3 mois suivi d'une p\u00e9riode de suivi de 3 mois.<\/span><\/pre>\n
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Plus d’infos<\/span><\/a><\/span><\/p>\n
[\/et_pb_accordion_item][et_pb_accordion_item title=”MOM Nipocalimab (Myasth\u00e9nie grave g\u00e9n\u00e9ralis\u00e9e)” use_icon_font_size=”on” icon_font_size=”32px” _builder_version=”4.9.10″ _module_preset=”default” body_font=”Fabriga||||||||” custom_margin=”||8px||false|false” toggle_font=”|700|||||||” toggle_font_size=”28px” closed_toggle_font_size=”20px” closed_toggle_line_height=”1em” open_use_icon_font_size=”on” open_icon_font_size=”32px” open=”off”]<\/p>\n
Une \u00e9tude sur le nipocalimab administr\u00e9 \u00e0 des adultes atteints de myasth\u00e9nie grave g\u00e9n\u00e9ralis\u00e9e<\/strong><\/p>\n
La myasth\u00e9nie grave (MG) est une maladie neuromusculaire rare, h\u00e9t\u00e9rog\u00e8ne, caract\u00e9ris\u00e9e par une faiblesse musculaire fluctuante et fatigable. La MG est caus\u00e9e par des auto-anticorps pathog\u00e8nes qui alt\u00e8rent la transmission cholinergique dans la membrane postsynaptique au niveau de la jonction neuromusculaire et alt\u00e8rent ou emp\u00eachent la contraction musculaire.<\/p>\n
Le nipocalimab (\u00e9galement appel\u00e9 JNJ-80202135 ou M281) est un anticorps monoclonal (mAb) d’immunoglobuline (Ig)G1 aglycosyl\u00e9 enti\u00e8rement humain con\u00e7u pour se lier, saturer et bloquer s\u00e9lectivement le site de liaison IgG sur le r\u00e9cepteur Fc n\u00e9onatal endog\u00e8ne (FcRn) .<\/p>\n
Cette \u00e9tude consistera en une phase de d\u00e9pistage (jusqu’\u00e0 4 semaines), une phase de traitement (une phase de 24 semaines en double aveugle contr\u00f4l\u00e9e par placebo et une phase d’extension en ouvert [OLE] [jusqu’\u00e0 2 ans]) et un suivi – visite de s\u00e9curit\u00e9 (jusqu’\u00e0 8 semaines apr\u00e8s la derni\u00e8re perfusion de l’intervention \u00e0 l’\u00e9tude). Les \u00e9valuations d’efficacit\u00e9 comprendront des \u00e9valuations telles que le score Myasthenia Gravis – Activities of Daily Living (MG-ADL).<\/p>\n
La dur\u00e9e totale de l’\u00e9tude sera jusqu’\u00e0 4 ans et 8 mois.<\/p>\n
Plus d’infos<\/span><\/a><\/span><\/p>\n
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[\/et_pb_accordion_item][\/et_pb_accordion][\/et_pb_column][et_pb_column type=”1_5″ _builder_version=”4.8.1″ _module_preset=”default”][et_pb_text admin_label=”ALS TEAM” _builder_version=”4.9.10″ _module_preset=”default” custom_margin=”100px||||false|false” custom_padding=”10px||0px||false|false”]<\/p>\n
L’\u00e9quipe<\/h2>\n
[\/et_pb_text][et_pb_team_member name=”Dr Oliver Blanchard” position=”Chercheur principal” image_url=”https:\/\/cru.mcgill.ca\/wp-content\/uploads\/olivier-blanchard-2022-1rz2.jpg” admin_label=”Luciano” _builder_version=”4.9.10″ _module_preset=”default” body_line_height=”1.1em” position_line_height=”1.1em” border_radii_image=”on|12px|12px|12px|12px”][\/et_pb_team_member][et_pb_team_member name=”Dr Rami Massie” position=”Chercheur principal” image_url=”https:\/\/cru.mcgill.ca\/wp-content\/uploads\/Ramie-Massie-2023-DSC03717-sq.jpg” admin_label=”Luciano” _builder_version=”4.9.10″ _module_preset=”default” body_line_height=”1.1em” position_line_height=”1.1em” border_radii_image=”on|12px|12px|12px|12px”][\/et_pb_team_member][\/et_pb_column][et_pb_column type=”1_5″ _builder_version=”4.8.1″ _module_preset=”default”][et_pb_text disabled_on=”off|off|off” _builder_version=”4.9.10″ _module_preset=”default” text_font=”Fabriga Bold||||||||” text_line_height=”1.2em” background_color=”#FFFFFF” text_orientation=”center” custom_margin=”65px||0px||false|false” custom_padding=”10px|10px|10px|10px|false|false” link_option_url=”mailto:nm.neurocru@mcgill.ca” border_radii=”on|12px|12px|12px|12px” background__hover_enabled=”on|hover” background_color__hover=”rgba(255,103,32,0.42)” background_enable_color__hover=”on”]<\/p>\n
Pour plus d’informations, veuillez contacter<\/span>\u00a0<\/b><\/p>\n
[\/et_pb_text][et_pb_team_member name=”Dre Erin O\u2019Ferrall” position=”Chercheuse principale” image_url=”https:\/\/cru.mcgill.ca\/wp-content\/uploads\/Erin-OFarrell-71-rzsq.jpg” admin_label=”Christina” _builder_version=”4.9.10″ _module_preset=”default” body_line_height=”1.1em” position_line_height=”1.1em” custom_margin=”30px||||false|false” border_radii_image=”on|12px|12px|12px|12px”][\/et_pb_team_member][et_pb_team_member name=”Dre Angela Genge” position=”Chercheuse principale” image_url=”https:\/\/cru.mcgill.ca\/wp-content\/uploads\/210804-152758-0318-sq.jpg” admin_label=”Christina” _builder_version=”4.9.10″ _module_preset=”default” body_line_height=”1.1em” position_line_height=”1.1em” border_radii_image=”on|12px|12px|12px|12px”][\/et_pb_team_member][\/et_pb_column][\/et_pb_row][et_pb_row column_structure=”3_5,1_5,1_5″ _builder_version=”4.8.1″ _module_preset=”default” custom_padding=”0px|||||”][et_pb_column type=”3_5″ _builder_version=”4.8.1″ _module_preset=”default”][et_pb_text admin_label=”Upcoming” _builder_version=”4.9.4″ text_font=”Fabriga|300|||||||” text_text_color=”#677a92″ text_font_size=”18px” text_line_height=”1.8em” header_font=”Fabriga Bold||||||||” header_font_size=”32px” header_line_height=”1.3em” header_2_font=”||||||||” header_3_font=”Arial||||||||” min_height=”48px” custom_margin=”||8px||false|false” custom_margin_tablet=”0px|50px||50px” custom_margin_phone=”|20px||” custom_margin_last_edited=”off|desktop” custom_padding=”||||false|false” text_line_height_tablet=”1.8em” text_line_height_phone=”1.8em” text_line_height_last_edited=”on|phone” header_font_size_tablet=”” header_font_size_phone=”36px” header_font_size_last_edited=”on|phone” header_2_font_size_phone=”30px” header_2_font_size_last_edited=”off|desktop” locked=”off”]<\/p>\n
\u00c9TUDES \u00c0 VENIR<\/h1>\n
[\/et_pb_text][et_pb_accordion db_closeable=”on” db_initial_state=”all_closed” open_toggle_text_color=”#490e68″ icon_color=”#490e68″ disabled_on=”off|off|off” admin_label=”UPCOMING ACCORDIAN” _builder_version=”4.9.10″ _module_preset=”default” toggle_text_color=”#490e68″ toggle_font=”Fabriga Medium||||||||” body_font=”Arial||||||||” border_radii=”on|20px|20px|20px|20px” border_width_all=”2px”][et_pb_accordion_item title=”ARROWHEAD ARO-DM1 (Dystrophie myotonique)” open=”on” use_icon_font_size=”on” icon_font_size=”32px” _builder_version=”4.9.10″ _module_preset=”default” body_font=”Fabriga||||||||” custom_margin=”||8px||false|false” global_colors_info=”{}” toggle_level=”h3″ toggle_font=”|700|||||||” toggle_font_size=”23px” closed_toggle_font_size=”20px” closed_toggle_line_height=”1em” icon_color__hover_enabled=”off|desktop” open_icon_color__hover_enabled=”off|desktop” open_use_icon_font_size=”on” open_icon_font_size=”32px”]<\/p>\n
Une \u00e9tude de phase 1\/2a \u00e0 dose croissante pour \u00e9valuer l’innocuit\u00e9, la tol\u00e9rance, la pharmacocin\u00e9tique et la pharmacodynamique de l’ARO-DM1 chez des sujets atteints de dystrophie myotonique de type 1 \u00e2g\u00e9s de entre 18 et 65 ans<\/strong><\/a><\/p>\n
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Il s’agit d’une \u00e9tude de phase 1\/2a \u00e0 double insu, contr\u00f4l\u00e9e par placebo et \u00e0 dose croissante visant \u00e0 \u00e9valuer l’innocuit\u00e9, la tol\u00e9rabilit\u00e9, la pharmacocin\u00e9tique (PK) et la pharmacodynamique (PD) de doses uniques et multiples croissantes d’ARO-DM1 par rapport au placebo chez les hommes et les femmes atteints de dystrophie myotonique de type 1 (DM1).<\/p>\n
 <\/p>\n
More information<\/span><\/a><\/span><\/strong><\/p>\n<\/div>\n
[\/et_pb_accordion_item][\/et_pb_accordion][\/et_pb_column][et_pb_column type=”1_5″ _builder_version=”4.8.1″ _module_preset=”default”][\/et_pb_column][et_pb_column type=”1_5″ _builder_version=”4.8.1″ _module_preset=”default”][\/et_pb_column][\/et_pb_row][et_pb_row column_structure=”3_5,1_5,1_5″ _builder_version=”4.8.1″ _module_preset=”default” custom_padding=”0px|||||”][et_pb_column type=”3_5″ _builder_version=”4.8.1″ _module_preset=”default”][et_pb_text admin_label=”Active Not Recruiting” _builder_version=”4.9.1″ text_font=”Montserrat|300|||||||” text_text_color=”#677a92″ text_line_height=”1.8em” header_font=”Fabriga Bold|700|||||||” header_font_size=”32px” header_line_height=”1.3em” header_2_font=”||||||||” header_3_font=”Arial||||||||” min_height=”48px” custom_margin=”||8px||false|false” custom_margin_tablet=”0px|50px||50px” custom_margin_phone=”|20px||” custom_margin_last_edited=”off|desktop” custom_padding=”|||” text_line_height_tablet=”1.8em” text_line_height_phone=”1.8em” text_line_height_last_edited=”on|phone” header_font_size_tablet=”” header_font_size_phone=”36px” header_font_size_last_edited=”on|phone” header_2_font_size_phone=”30px” header_2_font_size_last_edited=”off|desktop” locked=”off”]<\/p>\n
\u00c9TUDES ACTIVES (NE RECRUTENT PLUS)<\/h1>\n
[\/et_pb_text][et_pb_accordion db_closeable=”on” db_initial_state=”all_closed” icon_color=”#ff6720″ use_icon_font_size=”on” icon_font_size=”32px” admin_label=”ACTIVE ACCORDIAN” _builder_version=”4.9.10″ _module_preset=”default” toggle_font=”Fabriga Medium||||||||” body_font=”Arial||||||||” border_radii=”on|20px|20px|20px|20px” border_width_all=”2px”][et_pb_accordion_item title=”FULCRUM (FSHD)” open=”on” open_toggle_text_color=”#ff6720″ icon_color=”#ff6720″ use_icon_font_size=”on” icon_font_size=”32px” _builder_version=”4.9.10″ _module_preset=”default” body_font=”Fabriga||||||||” custom_margin=”||8px||false|false” toggle_text_color=”#ff6720″ toggle_font=”|700|||||||” toggle_font_size=”28px” closed_toggle_font_size=”20px” closed_toggle_line_height=”1em” open_icon_color=”#ff6720″ open_use_icon_font_size=”on” open_icon_font_size=”32px”]<\/p>\n
\u00c9tude de phase\u202f2 \u00e0 r\u00e9partition al\u00e9atoire, \u00e0 double insu, contr\u00f4l\u00e9e par placebo, d\u2019une dur\u00e9e de 48\u202fsemaines, \u00e0 groupes parall\u00e8les visant \u00e0 \u00e9valuer l\u2019efficacit\u00e9 et l\u2019innocuit\u00e9 du losmapimod comme traitement de sujets atteints de myopathie facio-scapulo-hum\u00e9rale (FSH) avec p\u00e9riode de prolongation ouverte (PO)<\/strong><\/a><\/p>\n
L\u2019objectif de la p\u00e9riode de traitement contr\u00f4l\u00e9 par placebo de 48\u202fsemaines de cette \u00e9tude est de d\u00e9terminer si le losmapimod peut ralentir ou arr\u00eater l\u2019\u00e9volution de la maladie chez des personnes atteintes de DFSH en emp\u00eachant ou en diminuant l\u2019expression du DUX4 dans le muscle squelettique et d\u2019autres organes touch\u00e9s. Cette \u00e9tude \u00e9valuera aussi comment vous vous sentez lorsque vous prenez le m\u00e9dicament \u00e0 l\u2019\u00e9tude et \u00e9valuera comment, selon vous, le m\u00e9dicament \u00e0 l\u2019\u00e9tude pourrait influer sur la progression de votre maladie.<\/p>\nPlus d’information<\/span><\/a>\n
[\/et_pb_accordion_item][et_pb_accordion_item title=”HBS (Dystrophie myotonique)” open_toggle_text_color=”#ff6720″ icon_color=”#ff6720″ use_icon_font_size=”on” icon_font_size=”32px” _builder_version=”4.9.10″ _module_preset=”default” body_font=”Fabriga||||||||” custom_margin=”||8px||false|false” toggle_text_color=”#ff6720″ toggle_font=”|700|||||||” toggle_font_size=”28px” closed_toggle_font_size=”20px” closed_toggle_line_height=”1em” open_icon_color=”#ff6720″ open_use_icon_font_size=”on” open_icon_font_size=”32px” open=”off”]<\/p>\n
<\/strong><\/a>Innocuit\u00e9 et efficacit\u00e9 du pitolisant sur la somnolence diurne excessive et d’autres sympt\u00f4mes non musculaires chez les patients atteints de dystrophie myotonique de type 1<\/h4>\n
L’objectif principal de cette \u00e9tude est d’\u00e9valuer l’innocuit\u00e9 et l’efficacit\u00e9 du pitolisant par rapport au placebo dans le traitement de la somnolence diurne excessive (EDS) chez les patients atteints de dystrophie myotonique de type 1 \u00e2g\u00e9s de 18 \u00e0 65 ans.<\/p>\n
Les objectifs secondaires de cette \u00e9tude sont d’\u00e9valuer l’impact du pitolisant sur la fatigue, la fonction cognitive et le fardeau de la maladie ainsi que d’\u00e9valuer l’innocuit\u00e9 et l’efficacit\u00e9 \u00e0 long terme du pitolisant chez les patients atteints de dystrophie myotonique de type 1 \u00e2g\u00e9s de 18 \u00e0 65 ans.<\/p>\n
Plus d’information<\/span><\/a><\/span><\/p>\n
[\/et_pb_accordion_item][et_pb_accordion_item title=”HELIOS ALNYLAM (hATTR Amyloidosis)” open_toggle_text_color=”#ff6720″ icon_color=”#ff6720″ use_icon_font_size=”on” icon_font_size=”32px” _builder_version=”4.9.10″ _module_preset=”default” body_font=”Fabriga||||||||” custom_margin=”||8px||false|false” toggle_text_color=”#ff6720″ toggle_font=”|700|||||||” toggle_font_size=”28px” closed_toggle_font_size=”20px” closed_toggle_line_height=”1em” open_icon_color=”#ff6720″ open_use_icon_font_size=”on” open_icon_font_size=”32px” open=”off”]<\/p>\n
A Phase 3 Global, Randomized, Open-label Study to Evaluate the Efficacy and Safety of ALN-TTRSC02 in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)<\/strong><\/a><\/p>\n
The purpose of this study is to evaluate the efficacy and safety of vutrisiran (ALN-TTRSC02) in patients with hereditary transthyretin amyloidosis (hATTR amyloidosis). Participants will receive vutrisiran or the reference comparator patisiran during the Treatment Period. The Treatment Period is followed by a Treatment Extension Period during which all participants in the patisiran group will switch to vutrisiran. This study will use the placebo arm of the APOLLO study (NCT01960348) as an external comparator for the primary and most other efficacy endpoints.<\/p>\n
Plus d’information<\/span><\/a><\/p>\n
[\/et_pb_accordion_item][et_pb_accordion_item title=”PREMIER (Charcot-Marie-Tooth)” open_toggle_text_color=”#ff6720″ icon_color=”#ff6720″ use_icon_font_size=”on” icon_font_size=”32px” _builder_version=”4.9.10″ _module_preset=”default” body_font=”Fabriga||||||||” custom_margin=”||8px||false|false” toggle_text_color=”#ff6720″ toggle_font=”|700|||||||” toggle_font_size=”28px” closed_toggle_font_size=”20px” closed_toggle_line_height=”1em” open_icon_color=”#ff6720″ open_use_icon_font_size=”on” open_icon_font_size=”32px” open=”off”]A multicenter, prospective, randomized, placebo-controlled, double-blind, parallel-group clinical tri<\/strong>A Multi-center, Randomized, Double-blind, Placebo controlled Phase III Study to Assess the Efficacy, Safety, and Tolerability of PXT3003 in Charcot-Marie-Tooth Type 1A<\/strong><\/span><\/a><\/p>\n
The purpose\u00a0of this study is\u00a0to investigate how effective PXT3003 is when compared to a placebo on the disease progression and symptoms of CMT1A.\u00a0PXT3003 consists of a combination of very low doses of 3 currently approved pharmaceutical\u00a0ingredients: (RS)-baclofen, naltrexone HCl, and D-sorbitol, which are thought to act in different\u00a0ways to\u00a0treat\u00a0CMT1A.\u00a0<\/span><\/p>\n
The placebo used in this study looks exactly like\u00a0PXT3003\u00a0but it does not contain any active medication. Placebos are sometimes called sugar pills, dummy treatments, or shams. We are using a placebo to compare with\u00a0PXT3003, and to ensure that the changes you report in your health, good or bad, are not only due to chance.\u00a0 In this information and consent form, the use of \u201cstudy drug\u201d refers either to\u00a0PXT3003\u00a0or to the placebo.\u00a0<\/span><\/p>\n
The study also aims to assess the safety and tolerability (whether side effects of a medication can be handled by patients) of PXT3003.\u00a0<\/span><\/p>\n
For this study,\u00a0350\u00a0male and female participants will be recruited at\u00a050\u00a0sites\u00a0globally. The participants will be aged\u00a0between\u00a016 to 65\u00a0years.\u00a0Only adults 18 years and older will be recruited at the Montreal Neurological Institute-Hospital.\u00a0<\/span><\/p>\n
More information<\/span><\/a><\/p>\n
[\/et_pb_accordion_item][et_pb_accordion_item title=”RAVULIZUMAB (Myasth\u00e9nie Grave)” open_toggle_text_color=”#ff6720″ icon_color=”#ff6720″ use_icon_font_size=”on” icon_font_size=”32px” _builder_version=”4.9.10″ _module_preset=”default” body_font=”Fabriga||||||||” custom_margin=”||8px||false|false” toggle_text_color=”#ff6720″ toggle_font=”|700|||||||” toggle_font_size=”28px” closed_toggle_font_size=”20px” closed_toggle_line_height=”1em” open_icon_color=”#ff6720″ open_use_icon_font_size=”on” open_icon_font_size=”32px” open=”off”]<\/p>\n
<\/strong><\/a>A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Ravulizumab in Complement-Inhibitor-Na\u00efve Adult Patients With Generalized Myasthenia Gravis<\/strong><\/a><\/p>\n
From\u00a0clinicaltrials.gov<\/a>:<\/p>\n
Inclusion criteria<\/p>\n
    \n
  1. Diagnosed with Myasthenia Gravis at least 6 months (180 days) prior to the date of the Screening Visit as confirmed by specific criteria.<\/li>\n
  2. Myasthenia Gravis Foundation of America Clinical Classification Class II to IV at screening.<\/li>\n
  3. MG-ADL profile must be \u2265 6 at screening and randomization (Day 1).<\/li>\n
  4. Vaccinated against meningococcal infections within 3 years prior to, or at the time of, initiating study drug to reduce the risk of meningococcal infection (N meningitidis).<\/li>\n<\/ol>\n
    More information<\/span><\/a><\/p>\n
    [\/et_pb_accordion_item][et_pb_accordion_item title=”RAISE” open_toggle_text_color=”#ff6720″ icon_color=”#ff6720″ use_icon_font_size=”on” icon_font_size=”32px” _builder_version=”4.9.10″ _module_preset=”default” body_font=”Fabriga||||||||” custom_margin=”||8px||false|false” toggle_text_color=”#ff6720″ toggle_font=”|700|||||||” toggle_font_size=”28px” closed_toggle_font_size=”20px” closed_toggle_line_height=”1em” open_icon_color=”#ff6720″ open_use_icon_font_size=”on” open_icon_font_size=”32px” open=”off”]<\/p>\n
    <\/strong><\/a><\/p>\n
    \u00c9tude de phase\u202f3, multicentrique, \u00e0 r\u00e9partition al\u00e9atoire, \u00e0 double insu et contr\u00f4l\u00e9e par placebo visant \u00e0 confirmer l\u2019innocuit\u00e9, la tol\u00e9rabilit\u00e9 et l\u2019efficacit\u00e9 du zilucoplan chez des sujets atteints de myasth\u00e9nie grave g\u00e9n\u00e9ralis\u00e9e<\/strong><\/a><\/p>\n
    Le but de cette \u00e9tude est de tester l\u2019efficacit\u00e9 du zilucoplan et son innocuit\u00e9 chez les patients atteints de MGg.<\/p>\nPlus d’information<\/span><\/a>\n
    [\/et_pb_accordion_item][et_pb_accordion_item title=”SPINRAZA ” open_toggle_text_color=”#ff6720″ icon_color=”#ff6720″ use_icon_font_size=”on” icon_font_size=”32px” _builder_version=”4.9.10″ _module_preset=”default” body_font=”Fabriga||||||||” custom_margin=”||8px||false|false” toggle_text_color=”#ff6720″ toggle_font=”|700|||||||” toggle_font_size=”28px” closed_toggle_font_size=”20px” closed_toggle_line_height=”1em” open_icon_color=”#ff6720″ open_use_icon_font_size=”on” open_icon_font_size=”32px” open=”off”]<\/p>\n
    \u00c9tude observationnelle, multicentrique, prospective \u00e9valuant l\u2019innocuit\u00e9 (s\u00fbret\u00e9), la tol\u00e9rabilit\u00e9 et l\u2019efficacit\u00e9 potentielle du SPINRAZA chez les patients adultes atteints d\u2019amyotrophie spinale<\/strong><\/a><\/p>\n
    De clinicaltrials.gov<\/a>. Malheureusement, les informations ne sont disponibles qu\u2019en anglais. Pour plus d\u2019informations en fran\u00e7ais, veuillez contacter Xin Di Dong.<\/a><\/p>\n
    This is a longitudinal, observational study of adult patients with genetically confirmed chromosome 5q SMA to examine the safety, tolerability, and effectiveness of SPINRAZA\u00ae (nusinersen) for up to 30 months.<\/p>\n
    This is a prospective, longitudinal, multi-center, observational study designed to evaluate the safety, tolerability, and effectiveness of SPINRAZA\u00ae (nusinersen) in ambulatory and non-ambulatory adult patients with SMA. Subjects with SMA II\/III that are 18 years to 70 years of age who are planning to initiate treatment with SPINRAZA\u00ae (nusinersen) as part of their clinical care plan will be enrolled in this study. This study does not provide SPINRAZA\u00ae (nusinersen) or cover costs associated with standard clinical care.These patients will be treated by their respective physicians according to standard clinical practice. Study visits including standardized assessments of strength and function will occur at baseline, day 15 after treatment initiation, day 30, day 70, and then 4-month intervals through month 30.<\/p>\nPlus d’information<\/span><\/a>\n
    [\/et_pb_accordion_item][\/et_pb_accordion][\/et_pb_column][et_pb_column type=”1_5″ _builder_version=”4.8.1″ _module_preset=”default”][et_pb_team_member name=”Dre Daria Trojan” position=”Chercheuse principal” image_url=”https:\/\/cru.mcgill.ca\/wp-content\/uploads\/Daria-Trojan-sq2.png” admin_label=”Luciano” _builder_version=”4.9.10″ _module_preset=”default” body_line_height=”1.1em” position_line_height=”1.1em” border_radii_image=”on|12px|12px|12px|12px”][\/et_pb_team_member][et_pb_team_member name=”Vanessa Bertone” position=”Chef d’\u00e9quipe, SLA + neuromusculaire” image_url=”https:\/\/cru.mcgill.ca\/wp-content\/uploads\/CRU-Vanessa-Bertone-DSC02469-rzsqthm-1.jpg” admin_label=”Luciano” _builder_version=”4.9.10″ _module_preset=”default” body_line_height=”1.1em” position_line_height=”1.1em” hover_enabled=”0″ border_radii_image=”on|12px|12px|12px|12px” sticky_enabled=”0″][\/et_pb_team_member][et_pb_team_member name=”Julia Chiappini” position=”Coordinatrice de recherche clinique” image_url=”https:\/\/cru.mcgill.ca\/wp-content\/uploads\/CRU-Julia-Chiappini-2025-PXL_20250206_194854589-sq3.jpg” admin_label=”Luciano” _builder_version=”4.9.10″ _module_preset=”default” body_line_height=”1.1em” position_line_height=”1.1em” hover_enabled=”0″ border_radii_image=”on|12px|12px|12px|12px” title_text=”CRU Julia Chiappini 2025 PXL_20250206_194854589 sq3″ sticky_enabled=”0″][\/et_pb_team_member][\/et_pb_column][et_pb_column type=”1_5″ _builder_version=”4.8.1″ _module_preset=”default”][et_pb_team_member name=”Dr Bernard Brais” position=”Chercheur principal” image_url=”https:\/\/cru.mcgill.ca\/wp-content\/uploads\/neuro_0785_cropped-rz.jpg” admin_label=”Christina” _builder_version=”4.9.10″ _module_preset=”default” body_line_height=”1.1em” position_line_height=”1.1em” border_radii_image=”on|12px|12px|12px|12px”][\/et_pb_team_member][et_pb_team_member name=”Marie-Lise B\u00e9land” position=”Coordinatrice de recherche clinique” image_url=”https:\/\/cru.mcgill.ca\/wp-content\/uploads\/Marie-Lise-Beland-2024-sq-1.jpg” admin_label=”Christina” _builder_version=”4.9.10″ _module_preset=”default” body_line_height=”1.1em” position_line_height=”1.1em” border_radii_image=”on|12px|12px|12px|12px”][\/et_pb_team_member][\/et_pb_column][\/et_pb_row][\/et_pb_section]<\/p>\n","protected":false},"excerpt":{"rendered":"
    \u00c0 propos L’\u00e9quipe Investigateurs Principaux \u00c9tudes en recrutement Autisme AVC D\u00e9mence \u00c9pilepsie Migraine Neuro-Chirurgie Neuromusculaire Neuro-oncologie Parkinson Scl\u00e9rose en plaques SLA Soins intensifs Patients Professionnels Nouvelles Carri\u00e8res Nous joindre EN \u00c0 propos L’\u00e9quipe Investigateurs Principaux \u00c9tudes en recrutement Autisme AVC D\u00e9mence \u00c9pilepsie Migraine Neuro-Chirurgie Neuromusculaire Neuro-oncologie Parkinson Scl\u00e9rose en plaques SLA Soins intensifs Patients Professionnels […]<\/p>\n","protected":false},"author":12,"featured_media":0,"parent":0,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"_et_pb_use_builder":"on","_et_pb_old_content":"","_et_gb_content_width":"","_monsterinsights_skip_tracking":false,"_monsterinsights_sitenote_active":false,"_monsterinsights_sitenote_note":"","_monsterinsights_sitenote_category":0},"yoast_head":"\nEssais cliniques sur la Neuromusculaire - Clinical Research Unit<\/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:\/\/cru.mcgill.ca\/fr\/nm-2\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Essais cliniques sur la Neuromusculaire - Clinical Research Unit\" \/>\n<meta property=\"og:description\" content=\"\u00c0 propos L’\u00e9quipe Investigateurs Principaux \u00c9tudes en recrutement Autisme AVC D\u00e9mence \u00c9pilepsie Migraine Neuro-Chirurgie Neuromusculaire Neuro-oncologie Parkinson Scl\u00e9rose en plaques SLA Soins intensifs Patients Professionnels Nouvelles Carri\u00e8res Nous joindre EN \u00c0 propos L’\u00e9quipe Investigateurs Principaux \u00c9tudes en recrutement Autisme AVC D\u00e9mence \u00c9pilepsie Migraine Neuro-Chirurgie Neuromusculaire Neuro-oncologie Parkinson Scl\u00e9rose en plaques SLA Soins intensifs Patients Professionnels […]\" \/>\n<meta property=\"og:url\" content=\"https:\/\/cru.mcgill.ca\/fr\/nm-2\/\" \/>\n<meta property=\"og:site_name\" content=\"Clinical Research Unit\" \/>\n<meta property=\"article:publisher\" content=\"https:\/\/www.facebook.com\/cru.mcgill\/\" \/>\n<meta property=\"article:modified_time\" content=\"2025-02-06T21:52:03+00:00\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:label1\" content=\"Est. reading time\" \/>\n\t<meta name=\"twitter:data1\" content=\"12 minutes\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\/\/schema.org\",\"@graph\":[{\"@type\":\"Organization\",\"@id\":\"https:\/\/cru.mcgill.ca\/#organization\",\"name\":\"Clinical Research Unit\",\"url\":\"https:\/\/cru.mcgill.ca\/\",\"sameAs\":[\"https:\/\/www.facebook.com\/cru.mcgill\/\"],\"logo\":{\"@type\":\"ImageObject\",\"inLanguage\":\"en-CA\",\"@id\":\"https:\/\/cru.mcgill.ca\/#\/schema\/logo\/image\/\",\"url\":\"https:\/\/cru.mcgill.ca\/wp-content\/uploads\/CRUlogo.png\",\"contentUrl\":\"https:\/\/cru.mcgill.ca\/wp-content\/uploads\/CRUlogo.png\",\"width\":268,\"height\":150,\"caption\":\"Clinical Research Unit\"},\"image\":{\"@id\":\"https:\/\/cru.mcgill.ca\/#\/schema\/logo\/image\/\"}},{\"@type\":\"WebSite\",\"@id\":\"https:\/\/cru.mcgill.ca\/#website\",\"url\":\"https:\/\/cru.mcgill.ca\/\",\"name\":\"Clinical Research Unit\",\"description\":\"CRU at the Neuro (Montreal Neurological Institute-Hospital)\",\"publisher\":{\"@id\":\"https:\/\/cru.mcgill.ca\/#organization\"},\"potentialAction\":[{\"@type\":\"SearchAction\",\"target\":{\"@type\":\"EntryPoint\",\"urlTemplate\":\"https:\/\/cru.mcgill.ca\/?s={search_term_string}\"},\"query-input\":\"required name=search_term_string\"}],\"inLanguage\":\"en-CA\"},{\"@type\":\"WebPage\",\"@id\":\"https:\/\/cru.mcgill.ca\/fr\/nm-2\/\",\"url\":\"https:\/\/cru.mcgill.ca\/fr\/nm-2\/\",\"name\":\"Essais cliniques sur la Neuromusculaire - 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