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
CMDSafety 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
CMSA Natural History Study in Participants With DOK7 Congenital Myasthenic Syndromes (CMS)Natural History StudyAdultOttawa Hospital Research Institute (OHRI)RecruitingHanns LochmüllerMaria Liezl Vinci Duff
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, randomized, double-blind, placebo-controlled single and multiple dose escalation study evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 in adult subjects with myotonic dystrophy type 1Vertex - VX‑670AdultCIUSSS du Saguenay-Lac-Saint-JeanRecruitingJean-Denis BrissonMarie Andree Regis/Valerie Harvey
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-JeanNot yet recruitingJean-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) Arthex - ATX-01AdultCIUSSS du Saguenay-Lac-Saint-JeanNot yet recruitingJean-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-670AdultMcGill University - Montreal Neurological Institue/Hospital (MNI)RecruitingAngela GengeMaria Eugenia Gobbo
DM1Study of ARO-DM1 in Subjects With Type 1 Myotonic DystrophyArrowhead Pharmaceuticals - ARO-DM1AdultMcGill University - Montreal Neurological Institue/Hospital (MNI)Not yet recruitingAngela GengeVanessa
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 LochmüllerMaria Liezl Vinci Duff / Jessica MacGregor
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 LochmüllerJessica MacGregor
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 Hospital
Not 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 Hospital
Not yet recruitingKathryn SelbyNela Martic
DMDEfficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy (ULYSSES)Italfarmaco - GivinostatPediatricBritish Columbia Children's Hospital
Not 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 Hospital
Not yet recruitingKathryn SelbyNela Martic
DMDAmendment #3 for An Open-Label, Expanded Access Protocol for Boys with Duchenne Muscular Dystrophy who Have Completed the Long-Term Extension (VBP15-LTE), VBP15-004 or VBP15-006 Studies Santhera Pharmaceuticals - VamorolonePediatricBritish Columbia Children's Hospital
RecruitingKathryn 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 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
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-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
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
DMDEfficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy (ULYSSES)Italfarmaco - GivinostatPediatricHolland Bloorview Kids Rehabilitation HospitalNot yet recruitingLaura McAdamChristina Ippolito
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
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
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
FSHDMotor Outcomes to Validate Evaluations in FSHD (MOVE FSHD) (MOVE FSHD)University of Kansas Medical Center - NAAdultMcGill University - Montreal Neurological Institue/Hospital (MNI)Not yet recruitingErin O'FerrallSabrina
FSHDMotor Outcomes to Validate Evaluations in FSHD (MOVE FSHD) (MOVE FSHD)University of Kansas Medical Center - NAAdultOttawa Hospital Research Institute (OHRI)RecruitingHanns LochmüllerJessica MacGregor
FSHDPhase 1/​2 Study of AOC 1020 in Adults With Facioscapulohumeral Muscular Dystrophy (FSHD) (FORTITUDE)Avidity Biosciences, Inc. - AOC 1020AdultOttawa Hospital Research Institute (OHRI)Not yet 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
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 Hospital
Not yet recruitingKathryn SelbyDora Xiong
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 Hospital
RecruitingKathryn SelbyDora Xiong
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
gMGA Phase 2 Study to Evaluate DNTH103 in Adults With Generalized Myasthenia Gravis (MAGIC) (MAGIC)Dianthus Therapeutics - DNTH103AdultCentre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM)Not yet recruitingStephan BotezNora Robert
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 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
gMGDescartes-08 CAR-T Cells in Generalized Myasthenia Gravis (MG)Cartesian Therapeutics - Descartes-08 CAR T-cellsAdultUniversity of Alberta*Recruiting
gMGSubcutaneous Immunoglobulin for Myasthenia Gravis (MG_SCIG)University Health Network, Toronto - Subcutaneous ImmunoglobulinAdultToronto General Hospital*RecruitingHans Katzberg
gMGA Study of Nipocalimab Administered to Adults With Generalized Myasthenia GravisJanssen Research & Development, LLC - NipocalimabAdultToronto General Hospital*Recruiting
gMGSafety and Efficacy of ALXN1720 in Adults With Generalized Myasthenia GravisAlexion Pharmaceuticals - ALXN1720AdultToronto General Hospital*Not yet recruiting
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
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 LochmullerJessica MacGregor
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 SaskatchewanNot yet recruitingKerri SchellenbergJoanne Boyer clinical research nurse coordinator
NMDThe Canadian Neuromuscular Disease RegistryUniversity of CalgaryAdultOttawa Hospital Research Institute (OHRI)RecruitingHanns LochmullerJessica MacGregor
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)Pediatric and AdultBritish Columbia Children's Hospital
RecruitingKathryn SelbyNela Martic
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
SMAA Randomized, Blinded, Placebo-Controlled, Phase 1 Single
Ascending Dose Study in Healthy Adult Male Volunteers and an Open-Label Multiple
Ascending Dose Study in Pediatric SMA Participants Previously Treated with
Onasemnogene Abeparvovec (Zolgensma™) to Evaluate the Safety, Tolerability, and
Pharmacokinetics of BIIB115		Biogen - BIIB115PediatricChildren's Hospital of Eastern Ontario (CHEO)RecruitingHugh McMillanLaura Thompson
SMASafety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy (SYNAPSE-SMA)NMD Pharma A/S - NMD670AdultGenge Partners Inc.RecruitingErin O'FerrallVincent Etten/Julian Santorelli
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)RecruitingLawrence KorngutJanet Petrillo
SMAOutpatient Rehabilitation Intervention for Young Children With Spinal Muscular Atrophy (SMA) Treated With Genetic Based TherapyHolland Bloorview Kids Rehabilitation Hospital - NAPediatricHolland Bloorview Kids Rehabilitation HospitalRecruitingLaura McAdamChristina Ippolito
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 SiteRecruitingMaryam OskouiMbaye Ndiaye

*Information from ClinicalTrials.gov