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 |
| ATTR | TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (TRITON-PN) | Alnylam Pharmaceuticals - Nucresiran | Adult | 18 to 85 | Vancouver General Hospital | Vancouver, BC | Recruiting | Michelle Mezei | Deborah Kraus |
| CDM | Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy (REACH CDM X) | AMO Pharma - Tideglusib | Adult and Pediatric | 6 to 45 | Children's Hospital London Health Sciences Centre (LHSC) | London, ON | Enrolling by invitation | Craig Campbell | Rhiannon Hicks |
| 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 Natural History Study in Participants With Congenital Myasthenic Syndromes (CMS) Due to Mutations in DOK7, MUSK, AGRN, or LRP4 | argenx | Pediatric | 2 and older | Children's Hospital of Eastern Ontario (CHEO) | Ottawa, ON | Recruiting | Hanns Lochmüller | Prachi Inamdar |
| 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 | Adult | 18 and older | CIUSSS du Saguenay-Lac-Saint-Jean | 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 17 | Alberta Children's Hospital | Calgary, AB | Recruiting | Jean Mah | Israt Yasmeen |
| gMG | Evaluating Long-term Safety of Efgartigimod Administered Intravenously and Efgartigimod PH20 Administered Subcutaneously in Children With Generalized Myasthenia Gravis (ADAPT Jr +) | argenx - Efgartigimod | Pediatric | 2 to 18 | Children's Hospital of Eastern Ontario (CHEO) | Ottawa, ON | Recruiting | Hugh McMillan | Prachi Inamdar |
| 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 |
| gMG | Evaluating Long-term Safety of Efgartigimod Administered Intravenously and Efgartigimod PH20 Administered Subcutaneously in Children With Generalized Myasthenia Gravis (ADAPT Jr +) | argenx - Efgartigimod | Pediatric | 2 to 18 | British Columbia Children's Hospital | Vancouver, BC | 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 | Not yet 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 | Not yet recruiting | Maxime Bérubé | Vincent Etten |
| FSHD | Study of ARO-DUX4 in Adult Patients with Fascioscapulohumeral Muscular Dystrophy Type 1 | Arrowhead Pharmaceuticals - ARO-DUX4 | Adult | 16 to 70 | University of Alberta* | Edmonton, AB | Recruiting | Cecile Phan | |
| FSHD | Study of ARO-DUX4 in Adult and Adolescent Patients With Facioscapulohumeral Muscular Dystrophy Type 1 | Arrowhead Pharmaceuticals - ARO-DUX4 | Adult and Pediatric | 16 to 70 | Heritage Medical Research Clinic (HMRC) | Calgary, AB | Recruiting | Lawrence Korngut | Carissa Wong |
| FSHD | Study of ARO-DUX4 in Adult and Adolescent Patients With Facioscapulohumeral Muscular Dystrophy Type 1 | Arrowhead Pharmaceuticals - ARO-DUX4 | Adult | 18 to 70 | McGill University Health Centre - CIM | Montreal, QC | Not yet recruiting | Angela Genge | Maria Gobbo |
| DM1 | Study of ATX-01 in Participants With DM1 (ArthemiR) | Arthrex Biotech - ATX-01 | Adult | 18 to 64 | CIUSSS du Saguenay-Lac-Saint-Jean | 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 | Not yet 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 | 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 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 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 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 and Pediatric | 16 to 70 | Ottawa Hospital Research Institute (OHRI) | Ottawa, ON | Recruiting | Hanns Lochmüller | Jessica MacGregor |
| 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 and Pediatric | 16 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 - AOC-1020 (Del-brax) | Adult and Pediatric | 16 to 70 | University of Alberta* | Edmonton, AB | Recruiting | ||
| FA | A Study to Learn More About the Effects and Long-Term Safety of Omaveloxolone (BIIB141) in Children and Teens With Friedreich's Ataxia (BRAVE) | Biogen - Omaveloxolone | Pediatric | 2 to 15 | CHU de Québec | Quebec City, QC | Recruiting | Nicolas Chrestian | Davy Eng |
| 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 | ||
| 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 | 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 | St. Michael's Hospital* | Toronto, ON | Recruiting | ||
| gMG | Investigating an mRNA CAR T-cell Therapy, Known as Descartes-08, as a Potential Approach to Treat Myasthenia Gravis | Cartesian Therapeutics - Descartes-08 | Adult | 18 and older | Toronto General Hospital* | Toronto, ON | 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 | CIUSSS du Saguenay-Lac-Saint-Jean | 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 and 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 |
| 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 |
| 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 |
| FA | Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia | Lexeo Therapeutics - LX2006 (AAVrh.10hFXN) | Pediatric | 18 to 50 | Centre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM) | Montreal, QC | Recruiting | Antoine Duquette | Martine Comeau |
| 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 |
| 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 | London Health Sciences Centre | London/ON | Enrolling by invitation | ||
| 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 | Recruiting | 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 | CHU de Québec | Quebec City, QC | Recruiting | Annie Dionne | Alexandra Simard |
| 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 |
| DMD | NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD) | NS Pharma - NS-089/NCNP-02 | Pediatric | 4 to 14 | Alberta Children's Hospital | Calgary, AB | Not yet recruiting | Jean Mah | Julie Dao |
| DMD | NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD) | NS Pharma - NS-089/NCNP-02 | Pediatric | 4 to 14 | 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 and Pediatric | 16 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 and Pediatric | 16 to 65 | Ottawa Hospital Research Institute (OHRI) | Ottawa, ON | Recruiting | Hanns Lochmüller | Jessica MacGregor |
| DM1 | A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 (FREEDOM2-DM1) | PepGen - PGN-EDODM1 | Adult and Pediatric | 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 | CIUSSS du Saguenay-Lac-Saint-Jean | Saguenay, QC | Recruiting | Jean-Denis Brisson | Valerie Harvey |
| DM1 | An Open-Label Extension Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 (FREEDOM-OLE) | PepGen - PGN-EDODM1 | Adult | 18 to 65 | CIUSSS du Saguenay-Lac-Saint-Jean | Saguenay, QC | Recruiting | Jean-Denis Brisson | Valerie Harvey |
| CIDP | A Study to Test the Effects and Safety of Riliprubart in People With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) for Which the Usual Treatments do Not Work (MOBILIZE) | 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 | 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 and Pediatric | 10 to 55 | McGill University - Montreal Neurological Institute/Hospital (MNI) | Montreal, QC | Recruiting | Erin O'Ferrall | NM research team |
| DMD | Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients (BASECAMP) | Satellos Bioscience - SAT-3247 | Pediatric | 7 to 9 | 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 | 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 | 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 - TAK-411 | Adult | 18 and older | Toronto General Hospital* | Toronto, ON | Not yet recruiting | ||
| DM1 | 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 | Magdalena Lovaglio |
| 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 | Magdalena Lovaglio |
| 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 |
| gMG | Efficacy, Safety and Tolerability of Efgartigimod in Patients with Seronegative Generalized Myasthenia Gravis: an Open-Label Study | University Health Network, Toronto - Efgartigimod IV | Adult | 18 and older | Toronto General Hospital* | Toronto, ON | Recruiting | Eduardo Ng | |
| FSHD | Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD) | University of Kansas Medical Center | Adult and Pediatric | 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 and Pediatric | 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 | Altasciences Montreal* | Montreal, QC | Recruiting | ||
| 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 | CHU de Québec | Quebec City, QC | Recruiting | Annie Dionne | Alexandra Simard |
| 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 | Ottawa Hospital Research Institute (OHRI) | Ottawa, ON | Recruiting | Hanns Lochmüller | Kathryn Beaudry |
| DM1 | A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type I | Vertex Pharmaceuticals - VX-670 | Adult | 18 and older | McGill University - Montreal Neurological Institute/Hospital (MNI) | Montreal, QC | Enrolling by Invitation | Angela Genge | NM research team |
| 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 | CIUSSS du Saguenay-Lac-Saint-Jean | Saguenay, QC | Recruiting | Jean-Denis Brisson | Valerie Harvey |
| gMG | A Study of Telitacicept for the Treatment of Generalized Myasthenia Gravis (UPSTREAM MG) | Vor Biopharma - Telitacicept | Adult | 18 and older | Toronto General Hospital* | Toronto, ON | Not yet recruiting |
*Information from ClinicalTrials.gov