This database addresses the need for
- Accurate and up-to-date information: our database provides reliable details about recruiting and not yet recruiting neuromuscular clinical trials, including clinical trial site locations and contact information.
- Collaboration: our database enables investigators to identify other sites conducting the same trial at different locations, fostering collaboration to address challenges and share best practices.
- Streamlined patient referral: our database can be used as a tool to facilitate patient referral to appropriate clinical trials and/or clinical sites.
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| Disease/Condition | Title | Sponsor and Treatment | Pediatric/Adult | Age range (in years unless indicated) | Site | Location | Recruitment status | Principal Investigator | Contact |
|---|---|---|---|---|---|---|---|---|---|
| IIM | A Study to Investigate the Safety and Preliminary Efficacy of ALLO-329, an Allogeneic CAR T-cell Therapy, in Adults With Autoimmune Disease (RESOLUTION) | Allogene Therapeutics - ALLO-329 | Adult | 18 to 69 | Hôpital Maisonneuve-Rosemont | Montreal, QC | Recruiting | Nicolas Richard | Olivier Cormier |
| CDM | Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy (REACH CDM X) | AMO Pharma - Tideglusib | Adult; Pediatric | 6 to 45 | Children's Hospital London Health Sciences Centre (LHSC) | London, ON | Enrolling by invitation | Craig Campbell | Rhiannon Hicks |
| CDM | Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy (REACH CDM X) | AMO Pharma Limited - Tideglusib | Pediatric | 6 to 18 | Children's Hospital of Eastern Ontario (CHEO) | Ottawa, ON | Recruiting | Hanns Lochmüller | Prachi Inamdar |
| SBMA | Phase 3, 52-Week, Randomized, Double-Blind, Placebo-Controlled Parallel Group and 52-Week Open-Label Extension study in Ambulatory Participants with Spinal and Bulbar Muscular Atrophy (or SBMA) | AnnJi Pharmaceuticals - AJ201 | Adult | University of Saskatchewan | Saskatoon, SK | Not yet recruiting | Kerri Schellenberg | Joanne Boyer | |
| CMS | A Natural History Study in Participants With Congenital Myasthenic Syndromes (CMS) Due to Mutations in DOK7, MUSK, AGRN, or LRP4 | argenx | Adult | 2 and older | Ottawa Hospital Research Institute (OHRI) | Ottawa, ON | Recruiting | Hanns Lochmüller | Mahsa Haghighi |
| CMS | A phase 3, Multicenter, Randomized, Double-Blinded, Placebo-Controlled, Parallel-Arm, single-Crossover Study With an Active-Treatment Extension to Evaluate the Efficacy and Safety of Intravenous Adimanebart in Adult and Pediatric Participants With DOK7-, MUSK-, AGRN-, or LRP4-CMS | argenx - ARGX-119 (Adimanebart) | Adult | 18 and older | Ottawa Hospital Research Institute (OHRI) | Ottawa, ON | Not yet recruiting | Hanns Lochmüller | Claudia Wyld |
| CMS | A phase 3, Multicenter, Randomized, Double-Blinded, Placebo-Controlled, Parallel-Arm, single-Crossover Study With an Active-Treatment Extension to Evaluate the Efficacy and Safety of Intravenous Adimanebart in Adult and Pediatric Participants With DOK7-, MUSK-, AGRN-, or LRP4-CMS | argenx - ARGX-119 (Adimanebart) | Adult | 18 and older | Santé Québec Saguenay-Lac-Saint-Jean – Universitaire | Saguenay, QC | Not yet recruiting | Jean-Denis Brisson | Valerie Harvey |
| gMG | Evaluating the Pharmacokinetics, Pharmacodynamics, and Safety of Efgartigimod Administered Intravenously in Children With Generalized Myasthenia Gravis (ADAPT Jr) | argenx - Efgartigimod | Pediatric | 2 to 18 | British Columbia Children's Hospital | Vancouver, BC | Not yet recruiting | Kathryn Selby | Ritu Ratan |
| CIDP | A Study to Assess Efficacy and Safety of Empasiprubart Versus IVIg in Adults With CIDP (emvigorate) | argenx - Empasiprubart | Adult | 18 and older | Heritage Medical Research Clinic (HMRC) | Calgary, AB | Recruiting | Theodore Mobach | Cora Dawn Carr |
| CIDP | A Study to Assess Efficacy and Safety of Empasiprubart Versus IVIg in Adults With CIDP (emvigorate) | argenx - Empasiprubart | Adult | 18 and older | St. Paul's Neurology Associates | Vancouver, BC | Recruiting | Katherine Beadon | Melika Nouri |
| CIDP | A Study to Assess the Efficacy and Safety of Empasiprubart in Adults With CIDP (emnergize) | argenx - Empasiprubart | Adult | 18 and older | St. Paul's Neurology Associates | Vancouver, BC | Not yet recruiting | Katherine Beadon | Melika Nouri |
| CIDP | A Study to Assess Efficacy and Safety of Empasiprubart Versus IVIg in Adults With CIDP (emvigorate) | argenx - Empasiprubart | Adult | 18 and older | GP Research Inc. | Montreal, QC | Recruiting | Maxime Bérubé | Vincent Etten |
| DM1 | Study of ATX-01 in Participants With DM1 (ArthemiR) | Arthrex Biotech - ATX-01 | Adult | 18 to 64 | Santé Québec Saguenay-Lac-Saint-Jean – Universitaire | Saguenay, QC | Recruiting | Jean-Denis Brisson | Valerie Harvey |
| XLMTM | A Study to Check Liver Health in Boys With XLMTM, a Serious Genetic Muscle Condition (EXCEL) | Astellas Pharma | Pediatric | 0 to 17 | The Hospital for Sick Children (SickKids) | Toronto, ON | Recruiting | Ashish Deshwar | Terry Ho |
| XLMTM | Study of ASP2957 in Male Participants With X-linked Myotubular Myopathy Who Need Ventilators | Astellas Pharma - ASP2957 | Pediatric | up to 36 months | The Hospital for Sick Children (SickKids) | Toronto, ON | Recruiting | Hernan Gonorazky | Alireza Tavakoli |
| ATTR | Non-interventional Study of Patients With Transthyretin (ATTR) Amyloidosis (MaesTTRo) | Astra Zeneca | Adult | 18 and older | Rimouski Regional Hospital* | Rimouski, QC | Not yet recruiting | ||
| ATTR | Non-interventional Study of Patients With Transthyretin (ATTR) Amyloidosis (MaesTTRo) | Astra Zeneca | Adult | 18 and older | London Health Sciences Centre* | London, ON | Recruiting | ||
| ATTR | Non-interventional Study of Patients With Transthyretin (ATTR) Amyloidosis (MaesTTRo) | Astra Zeneca | Adult | 18 and older | Maritime Neurology* | Halifax, NS | Recruiting | ||
| ATTR | Non-interventional Study of Patients With Transthyretin (ATTR) Amyloidosis (MaesTTRo) | Astra Zeneca | Adult | 18 and older | North York General Hospital* | Toronto, ON | Recruiting | ||
| ATTR | Non-interventional Study of Patients With Transthyretin (ATTR) Amyloidosis (MaesTTRo) | Astra Zeneca | Adult | 18 and older | Centre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM) | Montreal, QC | Not yet recruiting | Geneviève Matte | Amélie Bujold and Richard Miallot |
| ATTR | Non-interventional Study of Patients With Transthyretin (ATTR) Amyloidosis (MaesTTRo) | Astra Zeneca | Adult | 18 and older | Vancouver General Hospital | Vancouver, BC | Recruiting | Michelle Mezei | Deborah Kraus |
| IIM | A Study to Investigate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AZD5492 in Adult Participants With Systemic Lupus Erythematosus or Idiopathic Inflammatory Myopathies or Rheumatoid Arthritis. (TITAN) | Astra Zeneca - AZD5492 | Adult | 18 to 70 | McMaster University* | Hamilton, ON | Not yet recruiting | ||
| IIM | A Study to Investigate the Efficacy and Safety of Anifrolumab Administered as Subcutaneous Injection and Added to Standard of Care Compared With Placebo Added to Standard of Care in Adult Participants With Idiopathic Inflammatory Myopathies (Polymyositis and Dermatomyositis) (JASMINE) | Astra Zeneca - Anifrolumab | Adult | 18 to 75 | CaRe Clinics* | Calgary, AB | Recruiting | ||
| IIM | A Study to Investigate the Efficacy and Safety of Anifrolumab Administered as Subcutaneous Injection and Added to Standard of Care Compared With Placebo Added to Standard of Care in Adult Participants With Idiopathic Inflammatory Myopathies (Polymyositis and Dermatomyositis) (JASMINE) | Astra Zeneca - Anifrolumab | Adult | 18 to 75 | Jewish General Hospital | Montreal, QC | Recruiting | Marie Hudson | Melanie Banina |
| IIM | A Study to Investigate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AZD5492 in Adult Participants With Systemic Lupus Erythematosus or Idiopathic Inflammatory Myopathies or Rheumatoid Arthritis. (TITAN) | Astra Zeneca - AZD5492 | Adult | 18 to 70 | CHU Sherbrooke* | Sherbrooke, QC | Recruiting | ||
| FSHD | A Study to Evaluate Del-brax (Also Referred to as AOC 1020) in Participants With FSHD (FORTITUDE-3) | Avidity Biosciences - AOC-1020 (Del-brax) | Adult | 18 to 70 | Heritage Medical Research Clinic (HMRC) | Calgary, AB | Recruiting | Lawrence Korngut | Carissa Wong |
| FSHD | A Study to Evaluate Del-brax (Also Referred to as AOC 1020) in Participants With FSHD (FORTITUDE-3) | Avidity Biosciences - Del-brax (AOC 1020) | Adult | 16 to 70 | University of Alberta* | Edmonton, AB | Recruiting | ||
| DMD | A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping (SAFARI44) | Avidity Biosciences - Del-zota | Pediatric | 7 to 16 | Children's Hospital London Health Sciences Centre (LHSC) | London, ON | Recruiting | Craig Campbell | Rhiannon Hicks |
| SMA | A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety in Participants Aged 15 to 60 Years With Spinal Muscular Atrophy (SMA) Who Are Either New to SMA Treatment or Were Previously Treated With Risdiplam (SOLAR) | Biogen - Salanersen (BIIB115) | Adult; Pediatric | 15 to 60 | Stan Cassidy Centre for Rehabilitation | Fredericton, NB | Not yet recruiting | Colleen O'Connell | Shane McCullum |
| IIM | A Study to Understand How Nerandomilast Works in People With Active Idiopathic Inflammatory Myopathies (VERANDA™-IIM) | Boehringer Ingelheim - Nerandomilast | Adult | 18 and older | Vancouver General Hospital* | Vancouver, BC | Not yet recruiting | ||
| IIM | A Study to Understand How Nerandomilast Works in People With Active Idiopathic Inflammatory Myopathies (VERANDA™-IIM) | Boehringer Ingelheim - Nerandomilast | Adult | 18 and older | DIEX Recherche* | Sherbrooke, QC | Not yet recruiting | ||
| IIM | A Study to Understand How Nerandomilast Works in People With Active Idiopathic Inflammatory Myopathies (VERANDA™-IIM) | Boehringer Ingelheim - Nerandomilast | Adult | 18 and older | South Health Campus* | Calgary, AB | Not yet recruiting | ||
| ATTR | Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant | BridgeBio Pharma - Acoramidis | Adult | 18 to 75 | St. Michael's Hospital* | Toronto, ON | Recruiting | ||
| ATTR | Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant | BridgeBio Pharma - Acoramidis | Adult | 18 to 75 | St. Paul's Hospital* | Vancouver, BC | Recruiting | ||
| ATTR | Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant | BridgeBio Pharma - Acoramidis | Adult | 18 to 75 | University of Calgary* | Calgary, AB | Recruiting | ||
| DM/IMNM | Phase 1b Trial of RAY121 in Immunological Diseases (RAINBOW Trial) | Chugai Pharmaceutical - RAY121 | Adult | 18 to 85 | Centre de rheumatologie de l'Est du Québec* | Rimouski, QC | Recruiting | ||
| DM/IMNM | Phase 1b Trial of RAY121 in Immunological Diseases (RAINBOW Trial) | Chugai Pharmaceutical - RAY121 | Adult | 18 to 85 | DIEX Recherche* | Sherbrooke, QC | Recruiting | ||
| DM/IMNM | Phase 1b Trial of RAY121 in Immunological Diseases (RAINBOW Trial) | Chugai Pharmaceutical - RAY121 | Adult | 18 to 85 | University of Alberta* | Edmonton, AB | Recruiting | ||
| DM/IMNM | Phase 1b Trial of RAY121 in Immunological Diseases (RAINBOW Trial) | Chugai Pharmaceutical - RAY121 | Adult | 18 to 85 | McGill University - Montreal Neurological Institute/Hospital (MNI) | Montreal, QC | Recruiting | Erin O'Ferrall | NM research team |
| gMG | Efgartigimod in IVIG Dependent Myasthenia Gravis Patients | Clinique Neuro-Outaouais - Efgartigimod | Adult | 18 to 80 | Clinique Neuro-Outaouais | Gatineau, QC | Recruiting | François Henri Jacques | Ibrahim Sangare |
| DM1 | A Phase 1 Open-Label Trial to Assess the Safety and Pharmacokinetics of Multiple Ascending Doses of DT-818 | Design Therapeutics - DT-818 | Adult | 18 to 65 | Biopharma Services | Toronto, ON | Recruiting | Bruno Francoeur | Sukriti Malla |
| DM1 | Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1 | Dyne Therapeutics - DYNE101 | Adult | 18 to 65 | Santé Québec Saguenay-Lac-Saint-Jean – Universitaire | Saguenay, QC | Not yet recruiting | Jean-Denis Brisson | Valerie Harvey |
| gMG | Efficacy and Safety of a New Formulation of Oral Cladribine Compared With Placebo in Participants with Generalized Myasthenia Gravis (MyClad) | EMD Serono - Oral Cladribine | Adult | 18 and older | Toronto General Hospital* | Toronto, ON | Not yet recruiting | Lubna Daniyal (416-340-3898) | |
| gMG | Efficacy and Safety of a New Formulation of Oral Cladribine Compared With Placebo in Participants with Generalized Myasthenia Gravis (MyClad) | EMD Serono - Oral Cladribine | Adult | 18 and older | Burnaby Hospital | Burnaby, BC | Recruiting | Marketa Van den Elzen | Tatstsyana Kadach |
| FA | Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study (UNIFAI) | Friedreich's Ataxia Research Alliance | Adult; Pediatric | all ages | Centre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM) | Montreal, QC | Recruiting | Antoine Duquette | Martine Comeau |
| DMD | Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy (ULYSSES) | Italfarmaco - Givinostat | Pediatric | 9 to 17 | Children's Hospital of Eastern Ontario (CHEO) | Ottawa, ON | Recruiting | Hugh McMillan | Haifa Kourdi |
| DMD | Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy (ULYSSES) | Italfarmaco - Givinostat | Pediatric | 9 to 17 | Holland Bloorview Kids Rehabilitation Hospital | Toronto, ON | Recruiting | Laura McAdam | Olivia Mastracci |
| DMD | Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy (ULYSSES) | Italfarmaco - Givinostat | Pediatric | 9 to 17 | Children's Hospital London Health Sciences Centre (LHSC) | London, ON | Recruiting | Craig Campbell | Rhiannon Hicks |
| DMD | Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy (ULYSSES) | Italfarmaco - Givinostat | Pediatric | 9 to 17 | British Columbia Children's Hospital | Vancouver, BC | Recruiting | Kathryn Selby | Ritu Ratan |
| gMG | A Study of Nipocalimab Administered to Adults With Generalized Myasthenia Gravis | Johnson&Johnson Innovative Medicine - Nipocalimab | Adult | 18 and older | Toronto General Hospital* | Toronto, ON | Recruiting | ||
| gMG | A Study of Nipocalimab Administered to Adults With Generalized Myasthenia Gravis | Johnson&Johnson Innovative Medicine - Nipocalimab | Adult | 18 and older | Ottawa Hospital Research Institute (OHRI) | Ottawa, ON | Recruiting | Ariel Breiner | Isabel Horton |
| CIDP | Efficacy and Safety Study of Nipocalimab for Adults With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) | Johnson&Johnson Innovative Medicine - Nipocalimab | Adult | 18 and older | Ottawa Hospital Research Institute (OHRI) | Ottawa, ON | Recruiting | Ariel Breiner | Isabel Horton |
| SBMA | Understanding the prevalence, clinical features, and genetics of spinal-bulbar muscular atrophy (SBMA) in people of Indigenous descent | Kennedy Disease Association | Adult | 18 and older | Heritage Medical Research Clinic (HMRC) | Calgary, AB | Recruiting | Gerald Pfeffer | |
| SBMA | Understanding the prevalence, clinical features, and genetics of spinal-bulbar muscular atrophy (SBMA) in people of Indigenous descent | Kennedy Disease Association | Adult | 18 and older | University of Alberta* | Edmonton, AB | Recruiting | Wendy Johnston | |
| SBMA | Understanding the prevalence, clinical features, and genetics of spinal-bulbar muscular atrophy (SBMA) in people of Indigenous descent | Kennedy Disease Association | Adult | 18 and older | University of Saskatchewan | Saskatoon, SK | Recruiting | Kerri Schellenberg | Joanne Boyer |
| gMG | Study of KITE-363 in Participants With Autoimmune Neurologic Diseases That Did Not Respond to Usual Treatment (HERMOSA-2) | Kite Pharma - KITE-363 | Adult | 18 to 75 | Hôpital Maisonneuve-Rosemont | Montreal, QC | Recruiting | Nicole Khairallah | Olivier Cormier |
| CIDP | Study of KITE-363 in Participants With Autoimmune Neurologic Diseases That Did Not Respond to Usual Treatment (HERMOSA-2) | Kite Pharma - KITE-363 | Adult | 18 to 75 | Hôpital Maisonneuve-Rosemont | Montreal, QC | Recruiting | Nicole Khairallah | Olivier Cormier |
| gMG | Study of KITE-363 in Participants With Autoimmune Neurologic Diseases That Did Not Respond to Usual Treatment (HERMOSA-2) | Kite Pharma - KITE-363 | Adult | 18 to 75 | Jewish General Hospital* | Montreal, QC | Recruiting | ||
| CIDP | Study of KITE-363 in Participants With Autoimmune Neurologic Diseases That Did Not Respond to Usual Treatment (HERMOSA-2) | Kite Pharma - KITE-363 | Adult | 18 to 75 | Jewish General Hospital* | Montreal, QC | Recruiting | ||
| gMG | Study of KITE-363 in Participants With Autoimmune Neurologic Diseases That Did Not Respond to Usual Treatment (HERMOSA-2) | Kite Pharma - KITE-363 | Adult | 18 to 75 | Ottawa Hospital, General Campus | Ottawa, ON | Recruiting | Michael Kennah | Aimee Lemay |
| CIDP | Study of KITE-363 in Participants With Autoimmune Neurologic Diseases That Did Not Respond to Usual Treatment (HERMOSA-2) | Kite Pharma - KITE-363 | Adult | 18 to 75 | Ottawa Hospital, General Campus | Ottawa, ON | Recruiting | Michael Kennah | Aimee Lemay |
| IIM | A Study of KITE-363 in Participants With Refractory Autoimmune Diseases | Kite Pharma - KITE-363 | Adult | 18 and older | Ottawa Hospital, General Campus | Ottawa, ON | Recruiting | Michael Kennah | Danielle Tardiff |
| IIM | A Study of KITE-363 in Participants With Refractory Autoimmune Diseases | Kite Pharma - KITE-363 | Adult | 18 and older | Jewish General Hospital | Montreal, QC | Recruiting | Marie Hudson | Melanie Banina |
| FA | Clinical Course Of Disease In Participants With FA-CM | Lexeo Therapeutics | Adult; Pediatric | 6 and older | Centre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM) | Montreal, QC | Recruiting | Antoine Duquette | Célia Parinot |
| ARSACS | A randomized, placebo-controlled, double-blind, phase 2 trial of fampridine in patients with Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay (ARSACS) | McGill University - Fampridine | Adult | 18 and older | Santé Québec Saguenay-Lac-Saint-Jean – Universitaire | Saguenay, QC | Not yet recruiting | Jean-Denis Brisson | Valerie Harvey |
| SMA | A Registry-Based Parallel Group Randomized-Controlled Trial of an Upper Limb Exergaming Intervention for Children and Adolescents with Spinal Muscular Atrophy, with an Optional Open-Label Extension | McGill University Health Centre | Pediatric | 8 to 18 | The Hospital for Sick Children (SickKids) | Toronto, ON | Recruiting | Hernan Gonorazky | Ana Stosic |
| SMA | A Registry-Based Parallel Group Randomized-Controlled Trial of an Upper Limb Exergaming Intervention for Children and Adolescents with Spinal Muscular Atrophy, with an Optional Open-Label Extension | McGill University Health Centre | Pediatric | 8 to 18 | McGill University Health Centre - Glen Site | Montreal, QC | Recruiting | Maryam Oskoui | Mbaye Ndiaye |
| SMA | A Registry-Based Parallel Group Randomized-Controlled Trial of an Upper Limb Exergaming Intervention for Children and Adolescents with Spinal Muscular Atrophy, with an Optional Open-Label Extension | McGill University Health Centre | Pediatric | 8 to 18 | British Columbia Children's Hospital | Vancouver, BC | Recruiting | Kathryn Selby | Nela Martic |
| SMA | A Registry-Based Parallel Group Randomized-Controlled Trial of an Upper Limb Exergaming Intervention for Children and Adolescents with Spinal Muscular Atrophy, with an Optional Open-Label Extension | McGill University Health Centre | Pediatric | 8 to 18 | IWK Health* | Halifax, NS | Recruiting | Jordan Sheriko | Sara Drisdelle |
| CMT | A First in Human Study to Assess the Safety, Tolerability, and Pharmacokinetics of EDK060 in Adults With CMT1A | Novartis - EDK060 | Adult | 18 to 60 | CHU Sherbrooke | Sherbrooke, QC | Recruiting | Marie-Fance Rioux | Caroline Cayer |
| CMT | A First in Human Study to Assess the Safety, Tolerability, and Pharmacokinetics of EDK060 in Adults With CMT1A | Novartis - EDK060 | Adult | 18 to 60 | Ottawa Hospital Research Institute (OHRI) | Ottawa, ON | Enrolling by invitation | Alberto Aleman | Kathryn Beaudry |
| CMT | A First in Human Study to Assess the Safety, Tolerability, and Pharmacokinetics of EDK060 in Adults With CMT1A | Novartis - EDK060 | Adult | 18 to 60 | McGill University - Montreal Neurological Institute/Hospital (MNI) | Montreal, QC | Recruiting | Rami Massie | NM research team |
| gMG | A Study to Investigate the Efficacy, Safety and Tolerability of Remibrutinib Versus Placebo in Adult Patients With Generalized Myasthenia Gravis (RELIEVE) | Novartis - Remibrutinib | Adult | 18 to 75 | University of Alberta* | Edmonton, AB | Recruiting | ||
| gMG | A Study to Investigate the Efficacy, Safety and Tolerability of Remibrutinib Versus Placebo in Adult Patients With Generalized Myasthenia Gravis (RELIEVE) | Novartis - Remibrutinib | Adult | 18 to 75 | Ottawa Hospital Research Institute (OHRI) | Ottawa, ON | Recruiting | Ariel Breiner | Isabel Horton |
| DMD | NS-050/NCNP-03 in Boys With DMD (Meteor50) | NS Pharma - NS-050/NCNP-03 | Pediatric | 4 to 15 | Alberta Children's Hospital | Calgary, AB | Not yet recruiting | Jean Mah | Julie Dao |
| DMD | NS-050/NCNP-03 in Boys With DMD (Meteor50) | NS Pharma - NS-050/NCNP-03 | Pediatric | 4 to 15 | Children's Hospital London Health Sciences Centre (LHSC) | London, ON | Recruiting | Craig Campbell | Rhiannon Hicks |
| DMD | NS-050/NCNP-03 in Boys With DMD (Meteor50) | NS Pharma - NS-050/NCNP-03 | Pediatric | 4 to 15 | British Columbia Children's Hospital | Vancouver, BC | Recruiting | Kathryn Selby | Ritu Ratan |
| DM1 | A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 (FREEDOM2-DM1) | PepGen - PGN-EDODM1 | Adult | 18 to 65 | Heritage Medical Research Clinic (HMRC) | Calgary, AB | Recruiting | Gerald Pfeffer | Janet Petrillo |
| DM1 | A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 (FREEDOM2-DM1) | PepGen - PGN-EDODM1 | Adult | 16 to 65 | McGill University - Montreal Neurological Institute/Hospital (MNI) | Montreal, QC | Recruiting | Erin O'Ferrall | NM research team |
| DM1 | A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 (FREEDOM2-DM1) | PepGen - PGN-EDODM1 | Adult | 18 to 65 | Santé Québec Saguenay-Lac-Saint-Jean – Universitaire | Saguenay, QC | Recruiting | Jean-Denis Brisson | Valerie Harvey |
| CIDP | A Study to Test the Efficacy and Safety of Riliprubart Against the Usual Treatment of Intravenous Immunoglobulin (IVIg) in People With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) (VITALIZE) | Sanofi - Riliprubart | Adult | 18 and older | London Health Sciences Centre | London, ON | Enrolling by invitation | Anita Florendo-Cumbermack | |
| CIDP | A Study to Test the Efficacy and Safety of Riliprubart Against the Usual Treatment of Intravenous Immunoglobulin (IVIg) in People With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) (VITALIZE) | Sanofi - Riliprubart | Adult | 18 and older | CHU de Québec | Quebec City, QC | Recruiting | Annie Dionne | Alexandra Simard |
| CIDP | A Study to Test the Efficacy and Safety of Riliprubart Against the Usual Treatment of Intravenous Immunoglobulin (IVIg) in People With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) (VITALIZE) | Sanofi - Riliprubart | Adult | 18 and older | McGill University - Montreal Neurological Institute/Hospital (MNI) | Montreal, QC | Recruiting | Rami Massie | NM research team |
| DM1 | A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 50 Years of Age With Non-congenital Myotonic Dystrophy Type 1 (BrAAVe) | Sanofi - SAR446268 | Adult; Pediatric | 10 to 55 | McGill University - Montreal Neurological Institute/Hospital (MNI) | Montreal, QC | Recruiting | Erin O'Ferrall | NM research team |
| FSHD | Study of SRP-1001 in Adult and Adolescent Participants With Facioscapulohumeral Muscular Dystrophy Type 1 | Sarepta Therapeutics - SRP-1001 (ARO-DUX4) | Adult | 16 to 70 | University of Alberta* | Edmonton, AB | Recruiting | Cecile Phan | |
| FSHD | Study of SRP-1001 in Adult and Adolescent Participants With Facioscapulohumeral Muscular Dystrophy Type 1 | Sarepta Therapeutics - SRP-1001 (ARO-DUX4) | Adult | 18 to 70 | Heritage Medical Research Clinic (HMRC) | Calgary, AB | Recruiting | Lawrence Korngut | Carissa Wong |
| DM1 | Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy | Sarepta Therapeutics - SRP-1003 (ARO-DM1) | Adult | 18 to 65 | University of Alberta* | Edmonton, AB | Recruiting | ||
| DMD | A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE) | Solid Biosciences - SGT-003 | Pediatric | 7 to 11 | The Hospital for Sick Children (SickKids) | Toronto, ON | Recruiting | Hernan Gonorazky | Alireza Tavakoli |
| DMD | A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE) | Solid Biosciences - SGT-003 | Pediatric | 7 to 11 | Children's Hospital of Eastern Ontario (CHEO) | Ottawa, ON | Recruiting | Hugh McMillan | Emilie Hill-Smith |
| DMD | A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE) | Solid Biosciences - SGT-003 | Pediatric | 7 to 11 | Alberta Children's Hospital | Calgary, AB | Recruiting | Jean Mah | Israt Yasmeen |
| DMD | A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE) | Solid Biosciences - SGT-003 | Pediatric | 7 to 11 | British Columbia Children's Hospital | Vancouver, BC | Recruiting | Kathryn Selby | Nela Martic |
| CIDP | A Study of TAK-411 in Adults With Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) (CASCA) | Takeda - TAK-411 | Adult | 18 and older | Heritage Medical Research Clinic (HMRC) | Calgary, AB | Recruiting | Christopher White | Cora Dawn Carr |
| CIDP | A Study of TAK-411 in Adults With Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) (CASCA) | Takeda - TAK411 | Adult | 18 and older | Toronto General Hospital* | Toronto, ON | Not yet recruiting | ||
| CDM | Review of Patients with Congenital Myotonic Dystrophy type 1 | The Hospital for Sick Children (SickKids) | Pediatric | 0 to 18 | The Hospital for Sick Children (SickKids) | Toronto, ON | Recruiting | Hernan Gonorazky | Ana Stosic |
| SMA | Review of Treated patients with Spinal Muscular Atrophy | The Hospital for Sick Children (SickKids) | Pediatric | 0 to 18 | The Hospital for Sick Children (SickKids) | Toronto, ON | Recruiting | Hernan Gonorazky | Ana Stosic |
| DMD | The Expanded Duchenne Muscular Dystrophy (DMD) Natural History Study: Longitudinal Study of the Relationship Between Impairment, Activity Limitation, Participation and Quality of Life (eDNHS) | UC Davis / Sarepta | Pediatric | Alberta Children's Hospital | Calgary, AB | Recruiting | Jean Mah | Julie Dao | |
| oMG | A Study to Evaluate the Efficacy and Safety of Rozanolixizumab in Adult Participants With Ocular Myasthenia Gravis (MyVision) | UCB - Rozanolixizumab | Adult | 18 and older | GP Research Inc. | Montreal, QC | Recruiting | Angela Genge | Vincent Etten |
| FSHD | Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD) | University of Kansas Medical Center | Adult | Heritage Medical Research Clinic (HMRC) | Calgary, AB | Recruiting | Lawrence Korngut | Carissa Wong and Janet Petrillo | |
| FSHD | Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD) | University of Kansas Medical Center | Adult | All ages | Ottawa Hospital Research Institute (OHRI) | Ottawa, ON | Recruiting | Hanns Lochmüller | Jessica MacGregor |
| DM1 | A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1) (Galileo) | Vertex Pharmaceuticals - VX‑670 | Adult | 18 to 64 | Santé Québec Saguenay-Lac-Saint-Jean – Universitaire | Saguenay, QC | Recruiting | Jean-Denis Brisson | Valerie Harvey |
*Information from ClinicalTrials.gov