Clinical Trial Database - The Neuromuscular Disease Network for Canada

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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Database Legend

Disease Name	Table Abbreviation
Amyloid Transthyretin Amyloidosis	ATTR
Charcot-Marie-Tooth Syndrome	CMT
Chronic Inflammatory Demyelinating Polyradiculoneuropathy	CIDP
Congenital Myasthenic Syndromes	CMS
Congenital Myotonic Dystrophy	CDM
Dermatomyositis	DM
Duchenne Muscular Dystrophy	DMD
Facioscapulohumeral Muscular Dystrophy	FSHD
Friedreich Ataxia	FA
Generalized Myasthenia Gravis	gMG
Idiopathic Inflammatory Myopathy	IIM
Limb-Girdle Muscular Dystrophy	LGMD
Multifocal Motor Neuropathy	MMN
Myotonic Dystrophy 1	DM1
Oculopharyngeal Muscular Dystrophy	OPMD
Spinal Muscular Atrophy	SMA
Sporadic Inclusion Body Myositis	sIBM
X-linked Myotubular Myopathy	XLMTM

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Disease/ Condition	Title	Sponsor and Treatment	Ped/ Adult	Site	Location	Recruitment Status	Investigator	Contact
ATTR	MaesTTRo: 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 AB	Adult	Vancouver	Vancouver, BC	Not yet recruiting	Michelle Mezei	Deborah Kraus
CDM	Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy (REACH CDM X)	AMO Pharma Limited - Tideglusib	Pediatric	Children's Hospital of Eastern Ontario (CHEO)	Ottawa, ON	Recruiting	Hanns Lochmüller	Emilie Hill-Smith
CDM	Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy (REACH CDM X)	AMO Pharma Limited - Tideglusib	Pediatric	Children's Hospital London Health Sciences Centre (LHSC)	London, ON	Enrolling by invitation	Craig Campbell	Rhiannon Hicks
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	McGill University - Montreal Neurological Institute/Hospital (MNI)	Montreal, QC	Not yet recruiting	Rami Massie	Maria Gobbo
DM1	A 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-01	Adult	CIUSSS du Saguenay-Lac-Saint-Jean	Saguenay, QC	Recruiting	Jean-Denis Brisson	Marie Andree Regis/Valerie Harvey
DM1	A 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-EDODM1	Adult	CIUSSS du Saguenay-Lac-Saint-Jean	Saguenay, QC	Recruiting	Jean-Denis Brisson	Marie Andree Regis/Valerie Harvey
DM1	A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1) (Galileo)	Vertex - VX-670	Adult	CHU de Québec	Quebec City, 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	CIUSSS du Saguenay-Lac-Saint-Jean	Saguenay, QC	Recruiting	Jean-Denis Brisson	Marie Andree Regis/Valerie Harvey
DM1	A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1) (Galileo)	Vertex - VX-670	Adult	McGill University - Montreal Neurological Institute/Hospital (MNI)	Montreal, QC	Recruiting	Angela Genge	Sabrina Yusuf
DM1	A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1) (Galileo)	Vertex Pharmaceuticals - VX-670	Adult	Ottawa Hospital Research Institute (OHRI)	Ottawa, ON	Recruiting	Hanns Lochmüller	Sydney Zakutney
DM1	Global Study of Del-desiran for the Treatment of DM1 (HARBOR)	Avidity Biosciences, Inc. - Delpacibart Etedesiran	Adult	Ottawa Hospital Research Institute (OHRI)	Ottawa, ON	Recruiting	Hanns Lochmüller	Sydney Zakutney / Kathryn Beaudry
DM1	Music Intervention for Brain-Heart Disease in Myotonic Dystrophy Type 1 (DM1)	Hanns Lochmüller	Pediatric	Children's Hospital of Eastern Ontario (CHEO)	Ottawa, ON	Recruiting	Hanns Lochmüller	Tamara Burgess
DM1	Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1 (FREEDOM-DM1)	PepGen - PGN-EDODM1	Adult	CIUSSS du Saguenay-Lac-Saint-Jean	Saguenay, QC	Recruiting	Jean-Denis Brisson	Marie Andree Regis/Valerie Harvey
DM1	Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1 (FREEDOM-DM1)	PepGen - PGN-EDODM1	Adult	Heritage Medical Research Clinic (HMRC)	Calgary, AB	Not yet recruiting	Lawrence Korngut	Janet Petrillo
DM1	Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1 (FREEDOM-DM1)	mo	Adult	Ottawa Hospital Research Institute (OHRI)	Ottawa, ON	Recruiting	Hanns Lochmüller	Jessica MacGregor
DMD	A 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)	Pediatric	British Columbia Children's Hospital	Vancouver, BC	Not yet recruiting	Kathryn Selby	Nela Martic
DMD	A 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)	Pediatric	Children's Hospital London Health Sciences Centre (LHSC)	London, ON	Not yet recruiting	Craig Campbell	Rhiannon Hicks
DMD	A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment (CONNECT1-EDO51)	PepGen - PGN-EDO51	Pediatric	The Hospital for Sick Children (SickKids)	Toronto, ON	Recruiting	Hernan Gonorazky	Ana Stosic
DMD	A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment (CONNECT1-EDO51)	PepGen - PGN-EDO51	Pediatric	Children's Hospital Research Institute of Manitoba (CHRIM)	Winnipeg, MB	Recruiting	Kristina Joyal	Jennie Pitura
DMD	A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment (CONNECT1-EDO51)	PepGen - PGN-EDO51	Pediatric	Stan Cassidy Centre for Rehabilitation	Fredericton, NB	Recruiting	Colleen O'Connell	Shane McCullum
DMD	A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)	Solid Biosciences Inc. - SGT-003	Pediatric	The Hospital for Sick Children (SickKids)	Toronto, ON	Recruiting	Hernan Gonorazky	Ana Stosic
DMD	Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy (ULYSSES)	Italfarmaco - Givinostat	Pediatric	Children's Hospital of Eastern Ontario (CHEO)	Ottawa, ON	Recruiting	Hugh McMillan	Emilie Hill-Smith
DMD	Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy (ULYSSES)	Italfarmaco - Givinostat	Pediatric	British Columbia Children's Hospital	Vancouver, BC	Recruiting	Kathryn Selby	Stephanie Kwok
DMD	Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy (ULYSSES)	Italfarmaco - Givinostat	Pediatric	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	Holland Bloorview Kids Rehabilitation Hospital	Toronto, ON	Recruiting	Laura McAdam	Christina Ippolito
DMD	Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping (DELIVER)	Dyne Therapeutics - DYNE-251	Pediatric	Children's Hospital London Health Sciences Centre (LHSC)	London, ON	Recruiting	Craig Campbell	Rhiannon Hicks
FA	A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia	PTC Therapeutics - Vatiquinone	Adult	Centre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM)	Montreal, QC	Not yet recruiting	Antoine Duquette	Martine Comeau
FA	Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study (UNIFAI)	Natural History Study	Pediatric	Centre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM)	Montreal, QC	Recruiting	Antoine Duquette	Martine Comeau
FSHD	A 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 1	Arrowhead Pharmaceuticals ARO-DUX4	Adult	McGill University - Montreal Neurological Institute/Hospital (MNI)	Montreal, QC	Not yet recruiting	Angela Genge	Maria Gobbo
FSHD	Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD) (MOVE FSHD)	University of Kansas Medical Center - NA	Adult	Heritage Medical Research Clinic (HMRC)	Calgary, AB	Recruiting	Lawrence Korngut	Carissa Wong/Janet Petrillo
FSHD	Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD) (MOVE FSHD)	University of Kansas Medical Center - NA	Adult	McGill University - Montreal Neurological Institute/Hospital (MNI)	Montreal, QC	Recruiting	Erin O'Ferrall	Julia Chiappini
FSHD	Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD) (MOVE FSHD)	University of Kansas Medical Center - NA	Adult	Ottawa Hospital Research Institute (OHRI)	Ottawa, ON	Recruiting	Hanns Lochmüller	Jessica MacGregor
gMG	A 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 - Efgartigimod	Pediatric	British Columbia Children's Hospital	Vancouver, BC	Not yet recruiting	Kathryn Selby	Dora Xiong
gMG	A Study of Nipocalimab Administered to Adults With Generalized Myasthenia Gravis	Janssen Research & Development, LLC - Nipocalimab	Adult	Toronto General Hospital*	Toronto, ON	Recruiting
gMG	A 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 Therapy	Adult	University of Alberta*	Edmonton, AB	Not yet recruiting	Zaeem Siddiqi
gMG	A 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 Therapy	Adult	Toronto General Hospital*	Toronto, ON	Not yet recruiting	Vera Bril
gMG	Descartes-08 CAR-T Cells in Generalized Myasthenia Gravis (MG)	Cartesian Therapeutics - Descartes-08 CAR T-cells	Adult	University of Alberta*	Edmonton, AB	Recruiting
gMG	Open-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 - Efgartigimod	Pediatric	British Columbia Children's Hospital	Vancouver, BC	Recruiting	Kathryn Selby	Dora Xiong
gMG	Phase 3 Study to Assess the Efficacy and Safety of Batoclimab as Induction and Maintenance Therapy in Adult Participants With Generalized Myasthenia Gravis	Immunovant Sciences GmbH - Batoclimab	Adult	University of Alberta*	Edmonton, AB	Recruiting	Zaeem Siddiqi
gMG	Safety and Efficacy of ALXN1720 in Adults With Generalized Myasthenia Gravis	Alexion Pharmaceuticals - ALXN1720	Adult	Centre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM)	Montreal, QC	Recruiting	Stephan Botez	Nora Robert
gMG	Safety and Efficacy of ALXN1720 in Adults With Generalized Myasthenia Gravis	Alexion Pharmaceuticals - ALXN1720	Adult	CHU de Québec*	Quebec City, QC	Recruiting	Annie Dion	Alexandra Simard
gMG	Safety and Efficacy of ALXN1720 in Adults With Generalized Myasthenia Gravis	Alexion Pharmaceuticals - ALXN1720	Adult	University of Alberta*	Edmonton, AB	Not yet recruiting	Zaeem Siddiqi
gMG	Safety and Efficacy of ALXN1720 in Adults With Generalized Myasthenia Gravis	Alexion Pharmaceuticals - ALXN1720	Adult	Toronto General Hospital*	Toronto, ON	Not yet recruiting
gMG	Subcutaneous Immunoglobulin for Myasthenia Gravis (MG_SCIG)	University Health Network, Toronto - Subcutaneous Immunoglobulin	Adult	Toronto General Hospital*	Toronto, ON	Recruiting	Hans Katzberg
IIM	A Study to Investigate the Efficacy and Safety of Efgartigimod PH20 SC in Adult Participants With Active Idiopathic Inflammatory Myopathy. (ALKIVIA)	Argenx - Efgartigimod PH20 SC	Adult	Genge Partners Inc.	Montreal, QC	Recruiting	Angela Genge	Julian Santorelli
IIM	A Study to Investigate the Efficacy and Safety of Efgartigimod PH20 SC in Adult Participants With Active Idiopathic Inflammatory Myopathy. (ALKIVIA)	Argenx - Efgartigimod PH20 SC	Adult	Ottawa Hospital Research Institute (OHRI)	Ottawa, ON	Recruiting	Hanns Lochmüller	Jessica MacGregor
MMN	A 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-117-2202	Adult	Centre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM)	Montreal, QC	Recruiting	Genevieve Matte	Nora Robert
MMN	A 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	Argenx-117-2202	Adult	McGill University - Montreal Neurological Institute/Hospital (MNI)	Montreal, QC	Recruiting	Rami Massie	Sabrina Yusuf
MMN	A 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	Argenx-117-2202	Adult	University of Saskatchewan	Saskatoon, SK	Recruiting	Kerri Schellenberg	Joanne Boyer
NMD	The Canadian Neuromuscular Disease Registry	University of Calgary	Adult	Ottawa Hospital Research Institute (OHRI)	Ottawa, ON	Recruiting	Hanns Lochmüller	Jessica MacGregor
oMG	A 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 Gravis	Argenx - Efgartigimod PH20 SC	Adult	Ottawa Hospital Research Institute (OHRI)	Ottawa, ON	Recruiting	Ariel Breiner	Sydney Zakutney
SMA	A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy (PUPFISH)	Hoffmann-La Roche - Risdiplam	Pediatric	Children's Hospital of Eastern Ontario (CHEO)	Ottawa, ON	Recruiting	Hugh McMillan	Ozge Aksel Kilicarslan
SMA	Long-term follow-up of patients with spinal muscular atrophy Treated with OAV101 IT or OAV101 IV in Clinical Trials	Novartis Pharmaceuticals - OAV101	Pediatric	Children's Hospital of Eastern Ontario (CHEO)	Ottawa, ON	Not yet recruiting	Hugh McMillan	Laura Thompson
SMA	Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy (SYNAPSE-SMA)	NMD Pharma A/S - NMD670	Adult	Centre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM)	Montreal, QC	Recruiting	Genevieve Matte	Nora Robert
SMA	Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy (SYNAPSE-SMA)	NMD Pharma A/S - NMD670	Adult	Genge Partners Inc.	Montreal, QC	Recruiting	Erin O'Ferrall	Vincent Etten/Julian Santorelli
SMA	Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy (SYNAPSE-SMA)	NMD Pharma A/S - NMD670	Adult	Heritage Medical Research Clinic (HMRC)	Calgary, AB	Recruiting	Lawrence Korngut	Janet Petrillo
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	Research Institute- McGill University Helath Health Centre	Pediatric	McGill University Health Centre - Glen Site	Montreal, QC	Recruiting	Maryam Oskoui	Mbaye Ndiaye
SMA	A 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	McGill University Health Centre - Glen Site	Montreal, QC	Recruiting	Maryam Oskoui	Mbaye Ndiaye

*Information from ClinicalTrials.gov

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