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/
Condition		TitleSponsor and TreatmentPed/
Adult		SiteRecruitment
Status		InvestigatorContact
ATTRMaesTTRo: A Non-interventional, Prospective, Multi-country Study Collecting Real-world Data on the Characteristics, Treatment Patterns, and Outcomes of Patients with Amyloid Transthyretin (ATTR) Amyloidosis (D8450R00003)AstraZeneca ABAdultVancouverNot yet recruitingMichelle MezeiDeborah Kraus
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)RecruitingHanns LochmüllerEmilie Hill-Smith
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)Enrolling by invitationCraig 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)Not yet recruitingRami MassieVanessa Bertone
CMSA Natural History Study in Participants With DOK7 Congenital Myasthenic Syndromes (CMS)Natural History StudyAdultOttawa Hospital Research Institute (OHRI)RecruitingHanns LochmüllerMahsa Haghighi
DM1A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1Avidity Biosciences, Inc. - AOC 1001AdultCIUSSS du Saguenay-Lac-Saint-JeanNot yet recruitingJean-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-JeanRecruitingJean-Denis BrissonMarie Andree Regis/Valerie Harvey
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-JeanRecruitingJean-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-JeanRecruitingJean-Denis BrissonMarie Andree Regis/Valerie Harvey
DM1A Phase 1/​2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1) (Galileo)Vertex - VX‑670AdultCIUSSS du Saguenay-Lac-Saint-JeanRecruitingJean-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-EDODM1AdultHeritage Medical Research Clinic (HMRC)Not yet recruitingLawrence KorngutJanet Petrillo
DM1A Phase 1/​2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1) (Galileo)Vertex - VX-670AdultOttawa Hospital Research Institute (OHRI)RecruitingHanns LochmüllerSydney Zakutney
DM1Global Study of Del-desiran for the Treatment of DM1 (HARBOR)Avidity Biosciences, Inc. - Delpacibart EtedesiranAdultOttawa Hospital Research Institute (OHRI)RecruitingHanns LochmüllerSydney Zakutney / Kathryn Beaudry
DM1A Phase 1/​2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1) (Galileo)Vertex - VX-670AdultCHU de QuébecRecruiting0
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)RecruitingAngela GengeSabrina Yusuf
DM1A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1Avidity Biosciences, Inc. - AOC 1001AdultMcGill University - Montreal Neurological Institute/Hospital (MNI)RecruitingErin O'FerrallMaryam Leclerc
DM1Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1 (FREEDOM-DM1)PepGen - PGN-EDODM1AdultOttawa Hospital Research Institute (OHRI)RecruitingHanns LochmüllerJessica MacGregor
DMDEfficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy (ULYSSES)Italfarmaco - GivinostatPediatricBritish Columbia Children's HospitalNot yet recruitingKathryn SelbyStephanie Kwok
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 HospitalNot yet recruitingKathryn SelbyNela Martic
DMDSafety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping (DELIVER)Dyne Therapeutics - DYNE-251PediatricBritish Columbia Children's HospitalRecruitingKathryn SelbyNela Martic
DMDA Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment (CONNECT1-EDO51)PepGen - PGN-EDO51PediatricChildren's Hospital Research Institute of Manitoba (CHRIM)RecruitingKristina JoyalJennie Pitura
DMDA Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment (CONNECT1-EDO51)PepGen - PGN-EDO51Pediatric and AdultCHU de QuébecRecruitingNicolas ChrestianDavy Eng
DMDA Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment (CONNECT1-EDO51)PepGen - PGN-EDO51PediatricChildren's Hospital of Eastern Ontario (CHEO)RecruitingHugh McMillanEmilie Hill-Smith
DMDSafety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping (DELIVER)Dyne Therapeutics - DYNE-251PediatricChildren's Hospital of Eastern Ontario (CHEO)RecruitingHugh McMillanEmilie Hill-Smith
DMDA Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment (CONNECT1-EDO51)PepGen - PGN-EDO51PediatricStan Cassidy Centre for RehabilitationRecruitingColleen O'ConnellShane McCullum
DMDA Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)Solid Biosciences Inc. - SGT-003PediatricThe Hospital for Sick Children (SickKids)RecruitingHernan GonorazkyAna Stosic
DMDEfficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy (ULYSSES)Italfarmaco - GivinostatPediatricHolland Bloorview Kids Rehabilitation HospitalRecruitingLaura McAdamChristina Ippolito
DMDEfficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy (ULYSSES)Italfarmaco - GivinostatPediatricChildren's Hospital of Eastern Ontario (CHEO)RecruitingHugh McMillanEmilie Hill-Smith
DMDA Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment (CONNECT1-EDO51)PepGen - PGN-EDO51PediatricBritish Columbia Children's HospitalNot yet recruitingKathryn SelbyNela Martic
DMDSafety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping (DELIVER)Dyne Therapeutics - DYNE-251PediatricChildren's Hospital London Health Sciences Centre (LHSC)RecruitingCraig 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)RecruitingCraig CampbellRhiannon Hicks
DMDA Phase 2, Open-Label, Multiple Ascending Dose Study of PGN-EDO51 With a Long-Term Extension in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (CONNECT1-EDO51)PepGen - PGN-EDO51PediatricThe Hospital for Sick Children (SickKids)RecruitingHernan GonorazkyAna Stosic
FAA Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich AtaxiaPTC Therapeutics - VatiquinoneAdultCentre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM)Not yet recruitingAntoine DuquetteMartine Comeau
FAFriedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study (UNIFAI)Natural History StudyPediatricCentre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM)RecruitingAntoine DuquetteMartine Comeau
FSHDPhase 1/​2 Study of AOC 1020 in Adults With Facioscapulohumeral Muscular Dystrophy (FSHD) (FORTITUDE)Avidity Biosciences, Inc. - AOC 1020AdultOttawa Hospital Research Institute (OHRI)RecruitingHanns LochmüllerJessica MacGregor
FSHDMotor Outcomes to Validate Evaluations in FSHD (MOVE FSHD) (MOVE FSHD)University of Kansas Medical Center - NAAdultHeritage Medical Research Clinic (HMRC)Not yet recruitingLawrence KorngutCarissa Wong/Janet Petrillo
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)Not yet recruitingAngela GengeVanessa Bertone
FSHDMotor Outcomes to Validate Evaluations in FSHD (MOVE FSHD) (MOVE FSHD)University of Kansas Medical Center - NAAdultOttawa Hospital Research Institute (OHRI)RecruitingHanns LochmüllerJessica MacGregor
FSHDStudy to Evaluate the Efficacy and Safety of Satralizumab in FSHD1 (REINFORCE)Centre Hospitalier Universitaire de Nice - SatralizumabAdultOttawa Hospital Research Institute (OHRI)RecruitingHanns LochmüllerJessica MacGregor
FSHDMotor Outcomes to Validate Evaluations in FSHD (MOVE FSHD) (MOVE FSHD)University of Kansas Medical Center - NAAdultMcGill University - Montreal Neurological Institute/Hospital (MNI)RecruitingErin 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)RecruitingStephan BotezNora Robert
gMGSubcutaneous Immunoglobulin for Myasthenia Gravis (MG_SCIG)University Health Network, Toronto - Subcutaneous ImmunoglobulinAdultToronto General Hospital*RecruitingHans Katzberg
gMGDescartes-08 CAR-T Cells in Generalized Myasthenia Gravis (MG)Cartesian Therapeutics - Descartes-08 CAR T-cellsAdultUniversity of Alberta*Recruiting0
gMGA Long-term, Single-Arm, Open-label, Multicenter, Follow-on Trial of ARGX-113-2006 to Evaluate Safety of Efgartigimod Administered Intravenously in Children With Generalized Myasthenia Gravis Argenx - EfgartigimodPediatricBritish Columbia Children's HospitalNot yet recruitingKathryn SelbyDora Xiong
gMGSafety and Efficacy of ALXN1720 in Adults With Generalized Myasthenia GravisAlexion Pharmaceuticals - ALXN1720AdultCHU de Québec*RecruitingAnnie DionAlexandra Simard
gMGSafety and Efficacy of ALXN1720 in Adults With Generalized Myasthenia GravisAlexion Pharmaceuticals - ALXN1720AdultGenge Partners Inc.RecruitingMaxime BérubéVincent Etten/Julian Santorelli
gMGA Study of Nipocalimab Administered to Adults With Generalized Myasthenia GravisJanssen Research & Development, LLC - NipocalimabAdultToronto General Hospital*Recruiting0
gMGSafety and Efficacy of ALXN1720 in Adults With Generalized Myasthenia GravisAlexion Pharmaceuticals - ALXN1720AdultToronto General Hospital*Not yet recruiting0
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*Not yet recruitingVera Bril
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 HospitalRecruitingKathryn SelbyDora Xiong
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*Not yet recruitingZaeem Siddiqi
gMGSafety and Efficacy of ALXN1720 in Adults With Generalized Myasthenia GravisAlexion Pharmaceuticals - ALXN1720AdultUniversity of Alberta*Not yet recruitingZaeem Siddiqi
gMGPhase 3 Study to Assess the Efficacy and Safety of Batoclimab as Induction and Maintenance Therapy in Adult Participants With Generalized Myasthenia GravisImmunovant Sciences GmbH - BatoclimabAdultUniversity of Alberta*RecruitingZaeem Siddiqi
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.RecruitingAngela GengeJulian Santorelli
IIMA Study to Investigate the Efficacy and Safety of Efgartigimod PH20 SC in Adult Participants With Active Idiopathic Inflammatory Myopathy. (ALKIVIA)Argenx - Efgartigimod PH20 SCAdultOttawa Hospital Research Institute (OHRI)RecruitingHanns LochmüllerJessica MacGregor
LGMDA Study of the Natural History of Participants With LGMD2E/​R4, LGMD2D/​R3, LGMD2C/​R5, and LGMD2A/​R1 ≥ 4 Years of Age, Who Are Managed in Routine Clinical PracticeSarepta Therapeutics-Natural History studyPediatricChildren's Hospital London Health Sciences Centre (LHSC)Recruiting0
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 NeuropathyArgenx-117-2202AdultMcGill University - Montreal Neurological Institute/Hospital (MNI)RecruitingRami MassieSabrina Yusuf
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 NeuropathyArgenx-117-2202AdultUniversity of SaskatchewanRecruitingKerri SchellenbergJoanne Boyer
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)RecruitingGenevieve MatteNora Robert
NMDThe Canadian Neuromuscular Disease RegistryUniversity of CalgaryAdultOttawa Hospital Research Institute (OHRI)RecruitingHanns LochmüllerJessica MacGregor
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)RecruitingGenevieve MatteNora Robert
SMALong-term follow-up of patients with spinal muscular atrophy Treated with OAV101 IT or OAV101 IV in Clinical TrialsNovartis Pharmaceuticals - OAV101PediatricChildren's Hospital of Eastern Ontario (CHEO)Not yet recruitingHugh McMillanLaura Thompson

*Information from ClinicalTrials.gov