Canadian Neuromuscular Clinical Trial Database

The Canadian Neuromuscular Clinical Trial Database has been created as an initiative of the NMD4C Clinical Trial Network in collaboration with Canadian neuromuscular clinical trial site teams to provide a comprehensive and reliable landscape of Canadian neuromuscular clinical trials. Monthly updates are provided by clinical research coordinators working at Canadian trial sites, ensuring the most current information is always available.

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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/ConditionTitleSponsor and TreatmentPediatric/AdultSiteLocationRecruitment statusPrincipal InvestigatorContact
DM1Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1 (FREEDOM-DM1)PepGen - PGN-EDODM1AdultHeritage Medical Research Clinic (HMRC)Calgary, ABRecruitingLawrence KorngutJanet Petrillo
DMDNS-050/?NCNP-03 in Boys With DMD (Meteor50)NS Pharma - NS-050/?NCNP-03PediatricAlberta's Children HospitalCalgary, ABNot yet recruitingJean MahIsrat Yasmeen
DMDA phase 2 study to assess the efficacy, safety, tolerability, and pharmacokinetics of NS-089/NCNP-02 (Brogidirsen) in boys with Duchenne muscular dystrophy (DMD)NS Pharma - NS-089/?NCNP-02-201PediatricAlberta's Children HospitalCalgary, ABNot yet recruitingJean MahIsrat Yasmeen
DMDThe Expanded Duchenne Muscular Dystrophy (DMD) Natural History Study: Longitudinal Study of the Relationship Between Impairment, Activity Limitation, Participation and Quality of Life (eDNHSUC Davis / SareptaPediatricAlberta's Children HospitalCalgary, ABRecruitingJean MahJulie Dao
FSHDMotor Outcomes to Validate Evaluations in FSHD (MOVE FSHD) (MOVE FSHD)University of Kansas Medical Center - NAAdultHeritage Medical Research Clinic (HMRC)Calgary, ABRecruitingLawrence KorngutCarissa Wong/Janet Petrillo
SMASafety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy (SYNAPSE-SMA)NMD Pharma A/S - NMD670AdultHeritage Medical Research Clinic (HMRC)Calgary, ABRecruitingLawrence KorngutJanet Petrillo
gMGA Study to Examine the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Adult Patients With Symptomatic Generalized Myasthenia Gravis (NIMBLE)Regeneron Pharmaceuticals - Pozelimab and Cemdisiran Combination TherapyAdultUniversity of Alberta*Edmonton, ABNot yet recruitingZaeem Siddiqi
gMGEfgartigimod in IVIG Dependent Myasthenia Gravis PatientsClinique Neuro-Outaouais - EfgartigimodAdultClinique Neuro-OutaouaisGatineau, QCRecruitingFrançois Henri JacquesVictorine Sikati Foko
CDMSafety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy (REACH CDM X)AMO Pharma Limited - TideglusibPediatricChildren's Hospital London Health Sciences Centre (LHSC)London, ONEnrolling by invitationCraig CampbellRhiannon Hicks
DMDEfficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy (ULYSSES)Italfarmaco - GivinostatPediatricChildren's Hospital London Health Sciences Centre (LHSC)London, ONRecruitingCraig CampbellRhiannon Hicks
DMDA phase 2 study to assess the efficacy, safety, tolerability, and pharmacokinetics of NS-089/NCNP-02 (Brogidirsen) in boys with Duchenne muscular dystrophy (DMD)NS Pharma - NS-089/?NCNP-02-201PediatricChildren's Hospital London Health Sciences Centre (LHSC)London, ONRecruitingCraig CampbellRhiannon Hicks
DMDNS-050/?NCNP-03 in Boys With DMD (Meteor50)NS Pharma - NS-050/?NCNP-03PediatricChildren's Hospital London Health Sciences Centre (LHSC)London, ONRecruitingCraig CampbellRhiannon Hicks
DMDA Phase 1/2 Open-label, Dose Escalation and Dose Expansion Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of Intravenous RGX-202 Gene Therapy in Males with Duchenne Muscular Dystrophy (DMD)REGENXBIO Inc. - RGX-202 (AAV8.Spc5-12.microdystrophin)PediatricChildren's Hospital London Health Sciences Centre (LHSC)London, ONRecruitingCraig CampbellRhiannon Hicks
CIDPA 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 - RiliprubartAdultMcGill University - Montreal Neurological Institute/Hospital (MNI)Montreal, QCRecruitingRami MassieMaria Gobbo
DM1A Phase 1/?2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1) (Galileo)Vertex - VX-670AdultMcGill University - Montreal Neurological Institute/Hospital (MNI)Montreal, QCRecruitingAngela GengeSabrina Yusuf
FAFriedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study (UNIFAI)Natural History StudyPediatricCentre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM)Montreal, QCRecruitingAntoine DuquetteMartine Comeau
FSHDA Phase 1/2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-DUX4 in Adult Patients with Facioscapulohumeral Muscular Dystrophy Type 1Arrowhead Pharmaceuticals ARO-DUX4AdultMcGill University - Montreal Neurological Institute/Hospital (MNI)Montreal, QCNot yet recruitingAngela GengeMaria Gobbo
FSHDMotor Outcomes to Validate Evaluations in FSHD (MOVE FSHD) (MOVE FSHD)University of Kansas Medical Center - NAAdultMcGill University - Montreal Neurological Institute/Hospital (MNI)Montreal, QCRecruitingErin O'FerrallJulia Chiappini
gMGSafety and Efficacy of ALXN1720 in Adults With Generalized Myasthenia GravisAlexion Pharmaceuticals - ALXN1720AdultCentre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM)Montreal, QCRecruitingStephan BotezNora Robert
IIMA Study to Investigate the Efficacy and Safety of Efgartigimod PH20 SC in Adult Participants With Active Idiopathic Inflammatory Myopathy. (ALKIVIA)Argenx - Efgartigimod PH20 SCAdultGenge Partners Inc.Montreal, QCRecruitingAngela GengeJulian Santorelli
MMNA multicenter Prospective Longitudinal study of Clinical Outcomes, Disease Course, Health related quality of life, and health care resource utilization in adult patients with multifocal motor neuropathy (MMN)Argenx-117-2202AdultCentre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM)Montreal, QCRecruitingGenevieve MatteNora Robert
SMASafety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy (SYNAPSE-SMA)NMD Pharma A/S - NMD670AdultCentre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM)Montreal, QCRecruitingGenevieve MatteNora Robert
SMASafety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy (SYNAPSE-SMA)NMD Pharma A/S - NMD670AdultGenge Partners Inc.Montreal, QCRecruitingErin O'FerrallVincent Etten/Julian Santorelli
SMAA 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 ExtensionResearch Institute- McGill University Helath Health CentrePediatricMcGill University Health Centre - Glen SiteMontreal, QCRecruitingMaryam OskouiMbaye Ndiaye
SMAA Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy (MANATEE)Hoffmann-La Roche - RO7204239 in Combination With Risdiplam (RO7034067)PediatricMcGill University Health Centre - Glen SiteMontreal, QCRecruitingMaryam OskouiMbaye Ndiaye
CDMSafety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy (REACH CDM X)AMO Pharma Limited - TideglusibPediatricChildren's Hospital of Eastern Ontario (CHEO)Ottawa, ONRecruitingHanns LochmüllerEmilie Hill-Smith
CIDPPhase 2/3, Multistage, Multicenter, Randomized, Double-Blind, Placebo-Controlled Parallel Group Withdrawal Study to Evaluate the Efficacy and Safety of Nipocalimab Administered to Adults with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)Janssen Research & Development, LLC - NipocalimabAdultOttawa Hospital Research Institute (OHRI)Ottawa, ONNot yet recruitingAriel BreinerIsabel Horton
CIDPPhase 2/3, Multistage, Multicenter, Randomized, Double-Blind, Placebo-Controlled Parallel Group Withdrawal Study to Evaluate the Efficacy and Safety of Nipocalimab Administered to Adults with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)Janssen Research & Development, LLC - NipocalimabAdultOttawa Hospital Research Institute (OHRI)Ottawa, ONNot yet recruitingAriel BreinerIsabel Horton
DM1Music Intervention for Brain-Heart Disease in Myotonic Dystrophy Type 1 (DM1)Hanns LochmüllerPediatricChildren's Hospital of Eastern Ontario (CHEO)Ottawa, ONRecruitingHanns LochmüllerTamara Burgess
DM1Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1 (FREEDOM-DM2)PepGen - PGN-EDODM1AdultOttawa Hospital Research Institute (OHRI)Ottawa, ONRecruitingHanns LochmüllerJessica MacGregor
DM1A Phase 1/?2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1) (Galileo)Vertex Pharmaceuticals - VX-670AdultOttawa Hospital Research Institute (OHRI)Ottawa, ONRecruitingHanns LochmüllerSydney Zakutney
DMDEfficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy (ULYSSES)Italfarmaco - GivinostatPediatricChildren's Hospital of Eastern Ontario (CHEO)Ottawa, ONRecruitingHugh McMillanEmilie Hill-Smith
DMDA Phase 1/2 Open-label, Dose Escalation and Dose Expansion Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of Intravenous RGX-202 Gene Therapy in Males with Duchenne Muscular Dystrophy (DMD)REGENXBIO Inc. - RGX-202 (AAV8.Spc5-12.microdystrophin)PediatricChildren's Hospital of Eastern Ontario (CHEO)Ottawa, ONRecruitingHugh McMillanEmilie Hill-Smith
FSHDMotor Outcomes to Validate Evaluations in FSHD (MOVE FSHD) (MOVE FSHD)University of Kansas Medical Center - NAAdultOttawa Hospital Research Institute (OHRI)Ottawa, ONRecruitingHanns LochmüllerJessica MacGregor
gMGA randomized, double-blind, placebo-controlled phase III study to evaluate the efficacy, safety, and tolerability of remibrutinib in patients with generalized Myasthenia Gravis, followed by an open-label extension phaseNovartis Research and Development- remibrutinibAdultOttawa Hospital Research Institute (OHRI)Ottawa, ONNot yet recruitingAriel BreinerIsabel Horton
NMDThe Canadian Neuromuscular Disease RegistryUniversity of CalgaryAdultOttawa Hospital Research Institute (OHRI)Ottawa, ONRecruitingHanns LochmüllerJessica MacGregor
oMGA Randomized, Double-Blinded, Placebo-Controlled, Phase 3, Parallel-Group Design Study Evaluating the Efficacy and Safety of Efgartigimod PH20 SC Administered by Prefilled Syringe in Adult Participants With Ocular Myasthenia GravisArgenx - Efgartigimod PH20 SCAdultOttawa Hospital Research Institute (OHRI)Ottawa, ONRecruitingAriel BreinerIsabel Horton
DM1A Phase 1/?2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1) (Galileo)Vertex - VX-670AdultCHU de QuébecQuebec City, QCRecruitingAnnie Dionne
DM1A Phase 1/2a Double-Blind, Placebo-controlled, Single- and Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Clinical Efficacy of Intravenous Administration of ATX-01 In Male and Female Participants aged 18 to 64 with Classic Myotonic Dystrophy Type 1 (DM1) Arthrex - ATX-01AdultCIUSSS du Saguenay-Lac-Saint-JeanSaguenay, QCRecruitingJean-Denis BrissonMarie Andree Regis/Valerie Harvey
DM1A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multiple-Ascending Dose Study of PGN-EDODM1 in Adult Participants with Myotonic Dystrophy Type 1 (FREEDOM2-DM1)PepGen - PGN-EDODM1AdultCIUSSS du Saguenay-Lac-Saint-JeanSaguenay, QCRecruitingJean-Denis BrissonMarie Andree Regis/Valerie Harvey
DM1A Phase 1/?2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1) (Galileo)Vertex Pharmaceuticals - VX?670AdultCIUSSS du Saguenay-Lac-Saint-JeanSaguenay, QCRecruitingJean-Denis BrissonMarie Andree Regis/Valerie Harvey
DM1Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1 (FREEDOM-DM1)PepGen - PGN-EDODM1AdultCIUSSS du Saguenay-Lac-Saint-JeanSaguenay, QCRecruitingJean-Denis BrissonMarie Andree Regis/Valerie Harvey
DMDEfficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy (ULYSSES)Italfarmaco - GivinostatPediatricHolland Bloorview Kids Rehabilitation HospitalToronto, ONRecruitingLaura McAdamChristina Ippolito
DMDA Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)Solid Biosciences Inc. - SGT-003PediatricThe Hospital for Sick Children (SickKids)Toronto, ONRecruitingHernan GonorazkyAna Stosic
gMGA Study to Examine the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Adult Patients With Symptomatic Generalized Myasthenia Gravis (NIMBLE)Regeneron Pharmaceuticals - Pozelimab and Cemdisiran Combination TherapyAdultToronto General Hospital*Toronto, ONNot yet recruitingVera Bril
gMGSafety and Efficacy of ALXN1720 in Adults With Generalized Myasthenia GravisAlexion Pharmaceuticals - ALXN1720AdultToronto General Hospital*Toronto, ONNot yet recruiting
gMGA Study of Nipocalimab Administered to Adults With Generalized Myasthenia GravisJanssen Research & Development, LLC - NipocalimabAdultToronto General Hospital*Toronto, ONRecruiting
gMGSubcutaneous Immunoglobulin for Myasthenia Gravis (MG_SCIG)University Health Network, Toronto - Subcutaneous ImmunoglobulinAdultToronto General Hospital*Toronto, ONRecruitingHans Katzberg
DMDA phase 2 study to assess the efficacy, safety, tolerability, and pharmacokinetics of NS-089/NCNP-02 (Brogidirsen) in boys with Duchenne muscular dystrophy (DMD)NS Pharma - NS-089/?NCNP-02-201PediatricBritish Columbia Children's HospitalVancouver, BCNot yet recruitingKathryn SelbyNela Martic
DMDNS-050/?NCNP-03 in Boys With DMD (Meteor50)NS Pharma - NS-050/?NCNP-03PediatricBritish Columbia Children's HospitalVancouver, BCNot yet recruitingKathryn SelbyNela Martic
DMDA Phase 1/2 Open-label, Dose Escalation and Dose Expansion Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of Intravenous RGX-202 Gene Therapy in Males with Duchenne Muscular Dystrophy (DMD)REGENXBIO Inc. - RGX-202 (AAV8.Spc5-12.microdystrophin)PediatricBritish Columbia Children's HospitalVancouver, BCRecruitingKathryn SelbyNela Martic
DMDEfficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy (ULYSSES)Italfarmaco - GivinostatPediatricBritish Columbia Children's HospitalVancouver, BCRecruitingKathryn SelbyStephanie Kwok
gMGOpen-label Uncontrolled Trial to Evaluate Pharmacokinetics, Pharmacodynamics, Safety, and Activity of Efgartigimod in Children From 2 to Less Than 18 Years of Age With Generalized Myasthenia Gravis Argenx - EfgartigimodPediatricBritish Columbia Children's HospitalVancouver, BCRecruitingKathryn SelbyDora Xiong

*Information from ClinicalTrials.gov