Clinical research
In clinical research, individuals volunteer to participate in studies that aim to uncover better ways to treat, prevent, diagnose and understand human disease. Our clinical research includes clinical trials, where a new treatment or intervention is tested; patient registries, which collect information about disease epidemiology and specific features; and natural history studies, which monitor patients in detail to discover more about how diseases progress. Trials are conducted at the Children’s Hospital of Eastern Ontario and The Ottawa Hospital for the following neuromuscular diseases: Amyotrophic Lateral Sclerosis, Charcot-Marie-Tooth, Congenital Myasthenic Syndromes, Duchenne Muscular Dystrophy, Myotonic Dystrophy, Facio-Scapulo-Humeral Muscular Dystrophy, Idiopathic Inflammatory Myopathy, Oculo-Pharyngeal Muscular Dystrophy, Myasthenia Gravis and Spinal Muscular Atrophy.
Dr Hanns Lochmüller is a highly experienced investigator who has led multiple academic and commercially sponsored clinical research studies in Europe and Canada and is now Principal Investigator for multiple adult and pediatric trials in Ottawa.
Pediatric Trials at CHEO
Dr Lochmüller and pediatric neurologist and neuromuscular specialist Dr Hugh McMillan work closely together on pediatric neuromuscular research at CHEO, further supported by pediatric neurologist Dr Amanda Yaworski. This collaborative approach allows the research coordinators to work as a joint team helping to run clinical studies for both doctors and provides further opportunities for pediatric neuromuscular patients to participate in clinical trials.
Adult Trials at The Ottawa Hospital
Dr Lochmüller sees adult neuromuscular patients in clinic at The Ottawa Hospital (TOH), working closely with Dr Ari Breiner and the team at the Ottawa Neuromuscular Centre to conduct neuromuscular research, further supported by neurologist Dr Alberto Aleman.
A list of currently active and past trials is provided below.
Contact Information
If you are affected by one of the neuromuscular diseases listed below and would be interested in participating in an Ottawa-based clinical trial, please contact coordinator Emilie Hill-Smith for children and Jessica MacGregor for adults.
If you are a pharmaceutical company interested in Ottawa as a participating centre for your study, please contact Dr Lochmüller directly.
For general inquiries about the Canadian neuromuscular clinical trial landscape from a patient perspective and advice about what it means to participate in clinical research, please contact Muscular Dystrophy Canada.
Investigators, pharmaceutical company representatives and other stakeholders interested in clinical trials in Canada are also encouraged to visit the Neuromuscular Disease Network for Canada website to see how they can support you.
Current Trials
| Trial Number | Trial Name | Sponsor | Disease | Recruitment Status | PI | Population |
|---|---|---|---|---|---|---|
| NCT06185764 | A Phase 1/2, Randomized, Double-blind, Placebo controlledSingle- and Multiple dose Escalation Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VX 670in Adult Subjects with Myotonic Dystrophy Type 1 | Vertex Pharmaceuticals Ltd | Myotonic Dystrophy Type 1 (DM1) | Active, Open for recruitment | Dr. Hanns Lochmüller | Adult |
| NCT06204809 | Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1 (FREEDOM-DM1) | PepGen Inc | Myotonic Dystrophy Type 1 (DM1) | Active, Open for recruitment | Dr. Hanns Lochmüller | Adult |
| NCT06078553 | A Natural History Study in Participants With DOK7 Congenital Myasthenic Syndromes (CMS) | Argenx | Congenital Myasthenic Syndromes (CMS) | Active, Open for recruitment | Dr. Hanns Lochmüller | Adult |
| NCT06079736 | A Study Of PGN-EDO51 In Participants With Duchene Muscular Dystrophy Amenable To Exon 51-Skipping Treatment (CONNECT1-EDO51) | PepGen Inc | Duchenne Muscular Dystrophy (DMD) | Active, Open for recruitment | Dr. Hugh McMillan | Pediatric |
| NCT05524883 | Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping (DELIVER) | Dyne | Duchenne Muscular Dystrophy (DMD) | Active, Open for recruitment | Dr. Hugh McMillan | Pediatric |
| NCT05523167 | A Study to Investigate the Efficacy and Safety of Efgartigimod PH20 SC in Adult Participants With Active Idiopathic Inflammatory Myopathy. (ALKIVIA) | Argenx | Idiopathic Inflammatory Myopathy | Active, Open for recruitment | Dr. Hanns Lochmüller | Adult |
| NCT04635891 | Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD) - Natural History Study | University of Kansas Medical Center | Facioscapulohumeral Muscular Dystrophy (FSHD) | Active, Open for recruitment | Dr. Hanns Lochmüller | Adult |
| NCT05185622 | A Phase II Open-Label, Multiple Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD) | ReveraGen/Santhera | Duchenne Muscular Dystrophy (DMD) | Active, Closed for recruitment | Dr. Hanns Lochmüller | Pediatric |
| NCT05747924 | Phase 1/2 Study of AOC 1020 in Adults With Facioscapulohumeral Muscular Dystrophy (FSHD) (FORTITUDE) | Avidity | Facioscapulohumeral Muscular Dystrophy (FSHD) | Active, Closed for recruitment | Dr. Hanns Lochmüller | Adult |
| NCT04281485 | A Phase 3, multicentre, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of PF-06939926 for the treatment of Duchenne Muscular Dystrophy. | Pfizer | Duchenne Muscular Dystrophy (DMD) | Active, Closed for recruitment | Dr. Hanns Lochmüller | Pediatric |
| NCT05397470 | A Phase 3 Global, Randomized, Double-Blind, Placebo-Controlled, 48-Week, Parallel-Group Study of the Efficacy and Safety of Losmapimod in Treating Subjects with Facioscapulohumeral Muscular Dystrophy (FSHD) | Fulcrum Therapeutics | Facioscapulohumeral Muscular Dystrophy (FSHD) | Active, Closed for recruitment | Dr. Hanns Lochmüller | Adult |
| NCT04003974 | A Phase 2, Randomized, Double-Blind, Placebo-Controlled, 48-Week, Parallel-Group Study of the Efficacy and Safety of Losmapimod in Treating Subjects with Facioscapulohumeral Muscular Dystrophy (FSHD) with Open-Label Extension (OLE) | Fulcrum Therapeutics | Facioscapulohumeral Muscular Dystrophy (FSHD) | Active, Closed for recruitment | Dr. Hanns Lochmüller | Adult |
| NCT04886518 | A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients with Myotonic Dystrophy Type 1, Followed by an Open-Label Extension. | Harmony Biosciences | Myotonic Dystrophy Type 1 (DM1) | Completed, Results pending | Dr. Hanns Lochmüller | Adult |
| NCT02500381 | A Double-Blind, Placebo-Controlled, Multi-Center Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy | Sarepta Therapeutics, Inc. | Duchenne Muscular Dystrophy (DMD) | Completed, Results pending | Dr. Hanns Lochmüller | Pediatric |
| NCT04762758 | A Multi-center, Randomized, Double-blind, Placebo-controlled Phase III Study to Assess the Efficacy, Safety, and Tolerability of PXT3003 in Charcot-Marie-Tooth Type 1A (CMT1A) | Pharnext | Charcot-Marie-Tooth Type 1A | Completed, Results pending | Dr. Hanns Lochmüller | Adult |
| NCT01247207 | An Open-Label, Safety Study for Ataluren (PTC124) Patients With Nonsense Mutation Dystrophinopathy | PTC Therapeutics | Duchenne Muscular Dystrophy (DMD) | Completed, Results pending | Dr. Hanns Lochmüller | Pediatric |
| NCT03692312 | A Randomized, Double-Blind Study to Evaluate the Efficacy and Safety of Tideglusib Versus Placebo for the Treatment of Children and Adolescents with Congenital Myotonic Dystrophy (REACH CDM) | AMO Pharma Limited | Congenital Myotonic Dystrophy | Completed, Results pending | Dr. Hanns Lochmüller | Pediatric |