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.
Explore the Clinical Trial Database
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| Disease/Condition | Title | ClinicalTrials.gov ID | Sponsor and Treatment | Pediatric/Adult | Site | City, Province | Recruitment status | Principal Investigator | Contact name | Contact email | Information Link |
|---|---|---|---|---|---|---|---|---|---|---|---|
| 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) | NCT06465810 | 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) | NCT05004129 | AMO Pharma Limited - Tideglusib | Pediatric | Children's Hospital of Eastern Ontario (CHEO) | Ottawa, ON | Recruiting | Hanns Lochmüller | Emilie Hill-Smith | https://clinicaltrials.gov/study/NCT05004129?cond=Congenital%20muscular%20dystrophies&aggFilters=status:rec&rank=4 | |
| CDM | Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy (REACH CDM X) | NCT05004129 | AMO Pharma Limited - Tideglusib | Pediatric | Children's Hospital London Health Sciences Centre (LHSC) | London, ON | Enrolling by invitation | Craig Campbell | Rhiannon Hicks | https://clinicaltrials.gov/study/NCT05004129?cond=Congenital%20muscular%20dystrophies&aggFilters=status:rec&rank=4 | |
| 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) | NCT06290128 | Sanofi - Riliprubart | Adult | McGill University - Montreal Neurological Institute/Hospital (MNI) | Montreal, QC | Not yet recruiting | Rami Massie | Maria Gobbo | https://www.clinicaltrials.gov/study/NCT06290128?term=Riliprubart&rank=1 | |
| 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) | NCT06300307 | Arthrex - ATX-01 | Adult | CIUSSS du Saguenay-Lac-Saint-Jean | Saguenay, QC | Recruiting | Jean-Denis Brisson | Marie Andree Regis/Valerie Harvey | , | https://clinicaltrials.gov/study/NCT06300307?term=ATX-01&rank=1 |
| 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 | , | https://www.clinicaltrials.gov/study/NCT06667453?cond=DM1&aggFilters=status:not%20rec,studyType:int&page=1&rank=3 | |
| DM1 | A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1) (Galileo) | NCT06185765 | Vertex - VX-670 | Adult | CHU de Québec | Quebec City, QC | Recruiting | https://clinicaltrials.gov/study/NCT06185764 | |||
| DM1 | A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1) (Galileo) | NCT06185764 | Vertex Pharmaceuticals - VX‑670 | Adult | CIUSSS du Saguenay-Lac-Saint-Jean | Saguenay, QC | Recruiting | Jean-Denis Brisson | Marie Andree Regis/Valerie Harvey | , | https://clinicaltrials.gov/study/NCT06185764 |
| DM1 | A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1) (Galileo) | NCT06185764 | Vertex - VX-670 | Adult | McGill University - Montreal Neurological Institute/Hospital (MNI) | Montreal, QC | Recruiting | Angela Genge | Sabrina Yusuf | https://clinicaltrials.gov/study/NCT06185764?cond=Myotonic%20Dystrophy%20Type%201&aggFilters=status:rec,studyType:int&page=1&rank=3 | |
| DM1 | A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1) (Galileo) | NCT06185764 | Vertex Pharmaceuticals - VX-670 | Adult | Ottawa Hospital Research Institute (OHRI) | Ottawa, ON | Recruiting | Hanns Lochmüller | Sydney Zakutney | https://clinicaltrials.gov/study/NCT06185764 | |
| DM1 | Global Study of Del-desiran for the Treatment of DM1 (HARBOR) | NCT06411288 | Avidity Biosciences, Inc. - Delpacibart Etedesiran | Adult | Ottawa Hospital Research Institute (OHRI) | Ottawa, ON | Recruiting | Hanns Lochmüller | Sydney Zakutney / Kathryn Beaudry | ; | https://clinicaltrials.gov/study/NCT06411288 |
| DM1 | Music Intervention for Brain-Heart Disease in Myotonic Dystrophy Type 1 (DM1) | NCT06809049 | Hanns Lochmüller | Pediatric | Children's Hospital of Eastern Ontario (CHEO) | Ottawa, ON | Recruiting | Hanns Lochmüller | Tamara Burgess | https://clinicaltrials.gov/study/NCT06809049?id=NCT06809049&rank=1 | |
| DM1 | Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1 (FREEDOM-DM1) | NCT06204809 | PepGen - PGN-EDODM1 | Adult | CIUSSS du Saguenay-Lac-Saint-Jean | Saguenay, QC | Recruiting | Jean-Denis Brisson | Marie Andree Regis/Valerie Harvey | , | https://clinicaltrials.gov/study/NCT06204809?cond=NCT06204809&rank=1 |
| DM1 | Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1 (FREEDOM-DM1) | NCT06204809 | PepGen - PGN-EDODM1 | Adult | Heritage Medical Research Clinic (HMRC) | Calgary, AB | Not yet recruiting | Lawrence Korngut | Janet Petrillo | https://clinicaltrials.gov/study/NCT06204809?cond=NCT06204809&rank=1 | |
| DM1 | Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1 (FREEDOM-DM1) | NCT06204809 | mo | Adult | Ottawa Hospital Research Institute (OHRI) | Ottawa, ON | Recruiting | Hanns Lochmüller | Jessica MacGregor | https://clinicaltrials.gov/study/NCT06204809?cond=NCT06204809&rank=1 | |
| 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) | NCT05693142 | REGENXBIO Inc. - RGX-202 (AAV8.Spc5-12.microdystrophin) | Pediatric | British Columbia Children's Hospital | Vancouver, BC | Not yet recruiting | Kathryn Selby | Nela Martic | https://classic.clinicaltrials.gov/ct2/show/NCT05693142 | |
| 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) | NCT05693142 | 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 | https://clinicaltrials.gov/study/NCT05693142 | |
| DMD | A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment (CONNECT1-EDO51) | NCT06079736 | PepGen - PGN-EDO51 | Pediatric | The Hospital for Sick Children (SickKids) | Toronto, ON | Recruiting | Hernan Gonorazky | Ana Stosic | https://clinicaltrials.gov/study/NCT06079736?term=CONNECT1-EDO51&rank=1 | |
| DMD | A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment (CONNECT1-EDO51) | NCT06079736 | PepGen - PGN-EDO51 | Pediatric | Children's Hospital Research Institute of Manitoba (CHRIM) | Winnipeg, MB | Recruiting | Kristina Joyal | Jennie Pitura | https://clinicaltrials.gov/study/NCT06079736?term=CONNECT1-EDO51&rank=1 | |
| DMD | A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment (CONNECT1-EDO51) | NCT06079736 | PepGen - PGN-EDO51 | Pediatric | Stan Cassidy Centre for Rehabilitation | Fredericton, NB | Recruiting | Colleen O'Connell | Shane McCullum | https://clinicaltrials.gov/study/NCT06079736?term=CONNECT1-EDO51&rank=1 | |
| DMD | A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE) | NCT06138639 | Solid Biosciences Inc. - SGT-003 | Pediatric | The Hospital for Sick Children (SickKids) | Toronto, ON | Recruiting | Hernan Gonorazky | Ana Stosic | https://clinicaltrials.gov/study/NCT06138639 | |
| DMD | Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy (ULYSSES) | NCT05933059 | Italfarmaco - Givinostat | Pediatric | Children's Hospital of Eastern Ontario (CHEO) | Ottawa, ON | Recruiting | Hugh McMillan | Emilie Hill-Smith | https://clinicaltrials.gov/study/NCT05933057?locStr=Canada&country=Canada&cond=duchenne%20muscular%20dystrophy&aggFilters=status:rec&rank=1 | |
| DMD | Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy (ULYSSES) | NCT05933057 | Italfarmaco - Givinostat | Pediatric | British Columbia Children's Hospital | Vancouver, BC | Recruiting | Kathryn Selby | Stephanie Kwok | https://clinicaltrials.gov/study/NCT05933057?locStr=Canada&country=Canada&cond=duchenne%20muscular%20dystrophy&aggFilters=status:rec&rank=1 | |
| DMD | Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy (ULYSSES) | NCT05933058 | Italfarmaco - Givinostat | Pediatric | Children's Hospital London Health Sciences Centre (LHSC) | London, ON | Recruiting | Craig Campbell | Rhiannon Hicks | https://clinicaltrials.gov/study/NCT05933057?locStr=Canada&country=Canada&cond=duchenne%20muscular%20dystrophy&aggFilters=status:rec&rank=1 | |
| DMD | Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy (ULYSSES) | NCT05933060 | Italfarmaco - Givinostat | Pediatric | Holland Bloorview Kids Rehabilitation Hospital | Toronto, ON | Recruiting | Laura McAdam | Christina Ippolito | https://clinicaltrials.gov/study/NCT05933057?locStr=Canada&country=Canada&cond=duchenne%20muscular%20dystrophy&aggFilters=status:rec&rank=1 | |
| DMD | Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping (DELIVER) | NCT05524883 | Dyne Therapeutics - DYNE-251 | Pediatric | Children's Hospital London Health Sciences Centre (LHSC) | London, ON | Recruiting | Craig Campbell | Rhiannon Hicks | https://beta.clinicaltrials.gov/study/NCT05524883?distance=50&cond=NCT05524883&rank=1 | |
| FA | A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia | NCT05515536 | 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 | https://clinicaltrials.gov/study/NCT05515536?cond=Friedreich%20Ataxia&rank=1 | |
| FA | Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study (UNIFAI) | NCT06016946 | Natural History Study | Pediatric | Centre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM) | Montreal, QC | Recruiting | Antoine Duquette | Martine Comeau | https://clinicaltrials.gov/study/NCT06016946?cond=Friedreich%20Ataxia&rank=1 | |
| 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 | NCT06138743 | Arrowhead Pharmaceuticals ARO-DUX4 | Adult | McGill University - Montreal Neurological Institute/Hospital (MNI) | Montreal, QC | Not yet recruiting | Angela Genge | Maria Gobbo | https://www.clinicaltrials.gov/study/NCT06138743 | |
| FSHD | Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD) (MOVE FSHD) | NCT04635891 | University of Kansas Medical Center - NA | Adult | Heritage Medical Research Clinic (HMRC) | Calgary, AB | Recruiting | Lawrence Korngut | Carissa Wong/Janet Petrillo | , | https://clinicaltrials.gov/study/NCT04635891 |
| FSHD | Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD) (MOVE FSHD) | NCT04635891 | University of Kansas Medical Center - NA | Adult | McGill University - Montreal Neurological Institute/Hospital (MNI) | Montreal, QC | Recruiting | Erin O'Ferrall | Julia Chiappini | https://clinicaltrials.gov/study/NCT04635891?term=Motor%20Outcomes%20to%20Validate%20Evaluations%20in%20FSHD&rank=1 | |
| FSHD | Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD) (MOVE FSHD) | NCT04635891 | University of Kansas Medical Center - NA | Adult | Ottawa Hospital Research Institute (OHRI) | Ottawa, ON | Recruiting | Hanns Lochmüller | Jessica MacGregor | https://clinicaltrials.gov/study/NCT04635891?term=Motor%20Outcomes%20to%20Validate%20Evaluations%20in%20FSHD&rank=1 | |
| 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 | NCT05374590 | Argenx - Efgartigimod | Pediatric | British Columbia Children's Hospital | Vancouver, BC | Not yet recruiting | Kathryn Selby | Dora Xiong | https://clinicaltrials.gov/study/NCT05374590?term=argx-113-2006&rank=1 | |
| gMG | A Study of Nipocalimab Administered to Adults With Generalized Myasthenia Gravis | NCT04951622 | Janssen Research & Development, LLC - Nipocalimab | Adult | Toronto General Hospital* | Toronto, ON | Recruiting | https://clinicaltrials.gov/study/NCT04951622?cond=%20Myasthenia%20Gravis&aggFilters=status:rec,studyType:int&rank=1 | |||
| gMG | A Study to Examine the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Adult Patients With Symptomatic Generalized Myasthenia Gravis (NIMBLE) | NCT05070858 | Regeneron Pharmaceuticals - Pozelimab and Cemdisiran Combination Therapy | Adult | University of Alberta* | Edmonton, AB | Not yet recruiting | Zaeem Siddiqi | https://clinicaltrials.gov/study/NCT05070858?cond=%20Myasthenia%20Gravis&aggFilters=status:rec,studyType:int&page=2&rank=1 | ||
| gMG | A Study to Examine the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Adult Patients With Symptomatic Generalized Myasthenia Gravis (NIMBLE) | NCT05070858 | Regeneron Pharmaceuticals - Pozelimab and Cemdisiran Combination Therapy | Adult | Toronto General Hospital* | Toronto, ON | Not yet recruiting | Vera Bril | https://clinicaltrials.gov/study/NCT05070858?cond=%20Myasthenia%20Gravis&aggFilters=status:rec,studyType:int&page=2&rank=1 | ||
| gMG | Descartes-08 CAR-T Cells in Generalized Myasthenia Gravis (MG) | NCT04146051 | Cartesian Therapeutics - Descartes-08 CAR T-cells | Adult | University of Alberta* | Edmonton, AB | Recruiting | https://clinicaltrials.gov/study/NCT04146051 | |||
| 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 | NCT04833894 | Argenx - Efgartigimod | Pediatric | British Columbia Children's Hospital | Vancouver, BC | Recruiting | Kathryn Selby | Dora Xiong | https://classic.clinicaltrials.gov/ct2/show/NCT04833894 | |
| gMG | Phase 3 Study to Assess the Efficacy and Safety of Batoclimab as Induction and Maintenance Therapy in Adult Participants With Generalized Myasthenia Gravis | NCT05403541 | Immunovant Sciences GmbH - Batoclimab | Adult | University of Alberta* | Edmonton, AB | Recruiting | Zaeem Siddiqi | https://clinicaltrials.gov/study/NCT05403541?cond=%20Myasthenia%20Gravis&aggFilters=status:rec,studyType:int&page=2&rank=1 | ||
| gMG | Safety and Efficacy of ALXN1720 in Adults With Generalized Myasthenia Gravis | NCT05556096 | Alexion Pharmaceuticals - ALXN1720 | Adult | Centre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM) | Montreal, QC | Recruiting | Stephan Botez | Nora Robert | https://clinicaltrials.gov/study/NCT05556096?cond=%20Myasthenia%20Gravis&aggFilters=status:rec,studyType:int&page=2&rank=3 | |
| gMG | Safety and Efficacy of ALXN1720 in Adults With Generalized Myasthenia Gravis | NCT05556096 | Alexion Pharmaceuticals - ALXN1720 | Adult | CHU de Québec* | Quebec City, QC | Recruiting | Annie Dion | Alexandra Simard | https://clinicaltrials.gov/study/NCT05556096?cond=%20Myasthenia%20Gravis&aggFilters=status:rec,studyType:int&page=2&rank=4 | |
| gMG | Safety and Efficacy of ALXN1720 in Adults With Generalized Myasthenia Gravis | NCT05556096 | Alexion Pharmaceuticals - ALXN1720 | Adult | University of Alberta* | Edmonton, AB | Not yet recruiting | Zaeem Siddiqi | https://clinicaltrials.gov/study/NCT05556096?cond=%20Myasthenia%20Gravis&aggFilters=status:rec,studyType:int&page=2&rank=1 | ||
| gMG | Safety and Efficacy of ALXN1720 in Adults With Generalized Myasthenia Gravis | NCT05556096 | Alexion Pharmaceuticals - ALXN1720 | Adult | Toronto General Hospital* | Toronto, ON | Not yet recruiting | https://clinicaltrials.gov/study/NCT05556096?cond=%20Myasthenia%20Gravis&aggFilters=status:rec,studyType:int&page=2&rank=2 | |||
| gMG | Subcutaneous Immunoglobulin for Myasthenia Gravis (MG_SCIG) | NCT04728425 | University Health Network, Toronto - Subcutaneous Immunoglobulin | Adult | Toronto General Hospital* | Toronto, ON | Recruiting | Hans Katzberg | https://clinicaltrials.gov/study/NCT04728425?cond=%20Myasthenia%20Gravis&aggFilters=status:rec,studyType:int&rank=1 | ||
| IIM | A Study to Investigate the Efficacy and Safety of Efgartigimod PH20 SC in Adult Participants With Active Idiopathic Inflammatory Myopathy. (ALKIVIA) | NCT05523167 | Argenx - Efgartigimod PH20 SC | Adult | Genge Partners Inc. | Montreal, QC | Recruiting | Angela Genge | Julian Santorelli | https://clinicaltrials.gov/study/NCT05523167?cond=Immune-Mediated%20Necrotizing%20Myopathy&aggFilters=status:rec,studyType:int&rank=4 | |
| IIM | A Study to Investigate the Efficacy and Safety of Efgartigimod PH20 SC in Adult Participants With Active Idiopathic Inflammatory Myopathy. (ALKIVIA) | NCT05523167 | Argenx - Efgartigimod PH20 SC | Adult | Ottawa Hospital Research Institute (OHRI) | Ottawa, ON | Recruiting | Hanns Lochmüller | Jessica MacGregor | https://clinicaltrials.gov/study/NCT05523167?cond=Immune-Mediated%20Necrotizing%20Myopathy&aggFilters=status:rec,studyType:int&rank=4 | |
| 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) | NCT05988073 | Argenx-117-2202 | Adult | Centre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM) | Montreal, QC | Recruiting | Genevieve Matte | Nora Robert | https://clinicaltrials.gov/study/NCT05988073?term=NCT05988073&rank=1 | |
| 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 | NCT05988073 | Argenx-117-2202 | Adult | McGill University - Montreal Neurological Institute/Hospital (MNI) | Montreal, QC | Recruiting | Rami Massie | Sabrina Yusuf | https://clinicaltrials.gov/study/NCT05988073?term=NCT05988073&rank=1 | |
| 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 | NCT05988073 | Argenx-117-2202 | Adult | University of Saskatchewan | Saskatoon, SK | Recruiting | Kerri Schellenberg | Joanne Boyer | https://clinicaltrials.gov/study/NCT05988073?term=NCT05988073&rank=1 | |
| NMD | The Canadian Neuromuscular Disease Registry | University of Calgary | Adult | Ottawa Hospital Research Institute (OHRI) | Ottawa, ON | Recruiting | Hanns Lochmüller | Jessica MacGregor | https://cndr.org/ | ||
| 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 | NCT06436742 | 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) | NCT05808764 | Hoffmann-La Roche - Risdiplam | Pediatric | Children's Hospital of Eastern Ontario (CHEO) | Ottawa, ON | Recruiting | Hugh McMillan | Ozge Aksel Kilicarslan | https://clinicaltrials.gov/study/NCT05808764?a=1 | |
| SMA | Long-term follow-up of patients with spinal muscular atrophy Treated with OAV101 IT or OAV101 IV in Clinical Trials | NCT05335876 | Novartis Pharmaceuticals - OAV101 | Pediatric | Children's Hospital of Eastern Ontario (CHEO) | Ottawa, ON | Not yet recruiting | Hugh McMillan | Laura Thompson | https://clinicaltrials.gov/study/NCT05335876 | |
| SMA | Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy (SYNAPSE-SMA) | NCT05794139 | 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 | https://clinicaltrials.gov/study/NCT05794139 | |
| SMA | Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy (SYNAPSE-SMA) | NCT05794139 | NMD Pharma A/S - NMD670 | Adult | Genge Partners Inc. | Montreal, QC | Recruiting | Erin O'Ferrall | Vincent Etten/Julian Santorelli | , | https://clinicaltrials.gov/study/NCT05794139?cond=Spinal%20Muscular%20Atrophy&aggFilters=status:rec&page=2&rank=18 |
| SMA | Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy (SYNAPSE-SMA) | NCT05794139 | NMD Pharma A/S - NMD670 | Adult | Heritage Medical Research Clinic (HMRC) | Calgary, AB | Recruiting | Lawrence Korngut | Janet Petrillo | https://clinicaltrials.gov/study/NCT05794139?cond=Spinal%20Muscular%20Atrophy&aggFilters=status:rec&page=2&rank=18 | |
| 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 | NCT06396325 | Research Institute- McGill University Helath Health Centre | Pediatric | McGill University Health Centre - Glen Site | Montreal, QC | Recruiting | Maryam Oskoui | Mbaye Ndiaye | https://clinicaltrials.gov/study/NCT06396325?id=NCT06809049&limit=10&cond=NCT06396325&rank=1 | |
| SMA | A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy (MANATEE) | NCT05115110 | Hoffmann-La Roche - RO7204239 in Combination With Risdiplam (RO7034067) | Pediatric | McGill University Health Centre - Glen Site | Montreal, QC | Recruiting | Maryam Oskoui | Mbaye Ndiaye | https://clinicaltrials.gov/study/NCT05115110?cond=Spinal%20Muscular%20Atrophy&aggFilters=status:rec&page=4&rank=37 |
*Information from ClinicalTrials.gov