Find a Clinical Trial


Now more than ever there are clinical trials taking place to find better treatments and improve quality of life for people affected by neuromuscular disorders. And, Canadian clinician investigators are playing an active role in this research effort.

Listed below, grouped by disease type, are clinical studies with Canadian locations that are currently recruiting participants. To find more details, click on the hyperlinked title. If you have questions or want to learn more about eligibility, contact the sponsor or the investigator at the locations closest to you.

For a comprehensive global inventory of trials, visit – a searchable online database of clinical studies that are being conducted around the world.


Charcot Marie Tooth

Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients (PLEO-CMT)

Canadian Locations:

  • Vera Bril, MD, University Health Network / Toronto General Hospital
  • Jack Pumyriat, MD, University Hospital of Quebec

Sponsor: Pharnext SA Identifier: NCT02579759


Chronic Inflammatory Demyelinating
Polyneuropathy (CIDP)

Phase III Efficacy, Safety, and Tolerability Study of HYQVIA/HyQvia and GAMMAGARD LIQUID/KIOVIG in CIDP

Canadian Locations:

  • Montreal Neurological Institute Clinical Research Unit
  • Recherche Sepmus, Inc., Greenfield Park, Quebec

Sponsor: Baxalta US Inc. Identifier: NCT02549170



A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

Canadian Location: Alberta Children’s Hospital, Jean Mah

Sponsor: ReveraGen BioPharma, Inc.


  • University of Pittsburgh
  • National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
  • National Institute of Neurological Disorders and Stroke (NINDS)
  • Cooperative International Neuromuscular Research Group Identifier: NCT02760264


Study of Ataluren for Previously Treated Patients With nmDBMD in Europe, Israel, Australia, and Canada

This study is ongoing, but not recruiting participants.

Canadian Locations:

  • Alberta Children’s Hospital
  • British Columbia Children’s Hospital
  • Children’s Hospital of Western Ontario

Sponsor: PTC Therapeutics Identifier: NCT01557400


Whole Body Vibration Therapy in Boys with DMD

Leanne Ward, MD, Children’s Hospital of Eastern Ontario Identifier: NCT01954940M


Transplantation of Myoblasts to Duchenne Muscular Dystrophy

Canadian Locations: Identifier: NCT02196467


Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR-DMD)

The Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR DMD) study will compare three ways of giving corticosteroids to boys with Duchenne muscular dystrophy (DMD) to determine which of the three ways increases muscle strength the most, and which causes the fewest side effects. Using the results of this study, the investigators aim to provide patients and families with clearer information about the best way to take these drugs.

Canadian Locations:

  • Alberta Children’s Hospital, Calgary
    Contact: Natalia Rincon  403-955-3192
    Principal Investigator: Jean K. Mah, MD
  • University of British Columbia Children’s Hospital
    Contact: Nela Martic  604-875-2345 ext 6549
    Principal Investigator: Kathryn Selby, MD
  • Children’s Hospital London Health Sciences Centre
    Contact: Rhiannon Hicks  (519) 685-8441
    Rhiannon.Hicks@lhsc.on.caPrincipal Investigator: Craig Campbell, MD
  • Children’s Hospital of Eastern Ontario (CHEO)
    Contact: Angie Tuttle  613-737-7600 ext 4439
    Principal Investigator: Hugh McMillan, MD Identifier: NCT01603407



Study of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD)

Canadian Locations: Montreal Neurological Institute and Hospital

  • Alberta Children’s Hospital, Calgary, AB
  • Additional sites TBA

Contact : Xin Dong 514-398-5744.

Sponsor: Acceleron Pharma, Inc. Identifier Number: NCT02927080


A Multicenter Collaborative Study on the Clinical Features, Expression Profiling, and Quality of Life of Infantile Onset FSHD

Principal Investigator: Jean K Mah, MD, MS  Alberta Children’s Hospital

Sponsor: Cooperative International Neuromuscular Research Group
Zoë Sund (Project Manager) at 202-476-4110


  • FSH Society, Inc.
  • FSHD Global Research Foundation
  • Muscular Dystrophy Canada
  • aTyr Pharma, Inc. Identifier: NCT01437345

Dr. Jean Mah was named the 2012-2013 Researcher of the Year by Muscular Dystrophy Canada. Listen to Dr. Mah talk about the study’s objectives and participants as well as the passion she has for her work and patients.


Listen to Dr. Katie Manders, the first participant enrolled in Dr. Mah’s study of FSHD patients. Katie describes what was involved, why she volunteered, and why it’s important for others to participate as well. August 2014.



GNE Myopathy

Prospective, Longitudinal Study of the Natural History and Functional Status of Patients With Myotubular Myopathy (MTM) (MTM)

Canadian Location: Hospital for Sick Kids, Toronto  James Dowling, MD

Sponsor: Valerion Therapeutics, LLC


  • Institut de Myologie, France
  • Genethon Identifier: NCT02057705


A Clinical Assessment Study in X-Linked Myotubular Myopathy (XLMTM) Subjects (INCEPTUS)

Canadian Location:Hospital for Sick Kids, Toronto  James Dowling, MD

Sponsor: Audentes Therapeutics Identifier: NCT02704273


A Study to Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER) Tablets in GNE Myopathy (GNEM) (Also Known as Hereditary Inclusion Body Myopathy (HIBM)) Patients With Severe Ambulatory Impairment

Canadian Location: McMaster University

Sponsor: Ultragenyx Pharmaceutical Inc. Identifier: NCT02731690


GNE-Myopathy Disease Monitoring Program (GNEM-DMP): A Registry and Prospective Observational Natural History Study to Assess GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)

Canadian Location: McMaster University

Principal Investigator: Mark Tarnopolsky, MD

Sponsor: Ultragenyx Pharmaceutical Inc.

Collaborator: Newcastle University Identifier: NCT01784679


Myasthenia Gravis

Open Label Study of Subcutaneous Immunoglobulin (SCIg) in Myasthenia Gravis

Canadian Location: Toronto
Vera Bril, MD, University Health Network (UHN) – Toronto General Hospital

Sponsor: University of Kansas Medical Center Research Institute
Mazen Dimachkie, MD

Collaborator: CSL Behring Identifier: NCT02100969


Efficacy and Safety of IGIV-C in Corticosteroid Dependent Patients With Generalized Myasthenia Gravis

Canadian Locations:

  • London Health Sciences Centre University Hospital
    Contacts: Wilma Koopman and Denise Hulley
    Principal Investigator: Michael Nicolle
  • University Health Network (UHN) – Toronto General Hospital
    Contact: Eduardo Ng
    Principal Investigator: Vera Bril

Sponsor: Grifols Therapeutics Inc. Identifier: NCT02473965

A Pilot Trial To Assess The Feasibility And Efficacy Of SCIG In Patients With MG Exacerbation (SCIG-MG) (SCIG-MG)

Principal Investigator: Zaeem A Siddiqi, MD, PhD
University of Alberta
Edmonton, Alberta

Contact: Ashley K Mallon, LPN  780-407-6582

Collaborator: CSL Behring Identifier: NCT02774239

UCBMG0002 trial 

Canadian Location: Montreal Neurological Institute and Hospital

Contact: Victoria Eon: or 514-398-1688 Montreal Neurological Institute and Hospital Identifier: NCT03052751


Myositis Disorders

The Effect of Creatine Supplementation on Muscle Function in Childhood Myositis

Condition: Juvenile Dermatomyositis

Sponsor and location: Hospital for Sick Kids  Brian Feldman, MD Identifier: NCT02267005



A Study of CK-2127107 in Patients With Spinal Muscular Atrophy

Canadian Location: Montreal Neurological Institute and Hospital

Principal Investigator: Bernard Brais Angela Genge

Study coordinator: Victoria Eon, or 514-398-1688, Montreal Neurological Institute and Hospital

Study coordinator: Danny Kim DongHyun.Kim@MUHC.MCGILL.CA.

Sponsor: Cytokinetics

Collaborator: Astellas Pharma Global Development, Inc. Identifier: NCT02644668

Effect of Low-Dose Celecoxib on SMN2 in Patients With Spinal Muscular Atrophy (SMA)

This study is not yet open for participant recruitment (Sept. 2016).

Sponsor: Children’s Hospital of Eastern Ontario  Hugh McMillan, MD


  • Families of SMA Canada
  • Gwendolyn Strong Identifier: NCT02876094

Expanded Access Program (EAP) for Nusinersen in Participants With Infantile-onset (Consistent With Type 1) Spinal Muscular Atrophy (SMA)

*Expanded access is currently available for this treatment.

Canadian Locations:

  • Children’s Hospital at London Health Sciences Centre
  • Montreal Children’s Hospital
  • BC Children’s Hospital, Vancouver, BC

Sponsor: Biogen Identifier: NCT02865109


Late Onset Pompe Disease (LOPD) is recruiting newly diagnosed adult patients for the following trial

*Compares current standard of care treatment (myozyme) to a new formulation (neo-GAA).

Canadian Location: Montreal Neurological Institute and Hospital

Contact: Victoria Eon: or 514-398-1688 Montreal Neurological Institute and Hospital Identifier: NCT02782741


Questions? Contact us and we’ll help you find the answers. Muscular Dystrophy Canada is here to connect you with the information that you need. 1.800.687.2538

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